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The Patient-Centered Outcomes Research

Institute: Methodology Committee


Resource Guide

Developed by the National Pharmaceutical Council


February 2011.

The National Pharmaceutical Council is a policy research organization dedicated to the


advancement of good evidence and science, and to fostering an environment in the United
States that supports medical innovation. Founded in 1953 and supported by the nation’s
major research-based pharmaceutical companies, NPC focuses on research development,
information dissemination, education and promotion of the critical issues of evidence,
innovation and the value of medicines for patients.

To order reprints, contact NPC at info@npcnow.org or 703-620-6390. This guide is


also available online at www.npcnow.org/methodsguide.
The Patient-Centered Outcomes Research
Institute: Methodology Committee
Resource Guide
Ta b l e o f C o nt e nt s

A Letter From the National Pharmaceutical Council...............................................4


About the PCORI Methodology Committee...............................................................5
Methodology Committee Member Biographies...........................................................7
Why Methods Are Important.............................................................................................42
Existing Methodological Standards and Good Practices.........................................45
Additional Resources...........................................................................................................47
Appendix.................................................................................................................................48
4

A Letter From the National Pharmaceutical Council

In March 2010, President Obama signed into law the Patient Protection and Affordable Care Act, broad
health reform legislation that established the Patient-Centered Outcomes Research Institute (PCORI).
Since that time, a number of important steps have been taken to implement PCORI, starting with
the September 2010 appointment of a Board of Governors and continuing with the January 2011
announcement of the members of the Methodology Committee. Among its many responsibilities, the
PCORI Board of Governors is tasked with setting the research agenda, while the Methodology Committee
members are tasked with the development of sound research methods and will have a large role in the
type of research that is undertaken to answer specific research questions.
As the Methodology Committee begins its work, it will focus first on the vitally important job of
developing and choosing the right research methods for conducting a comparative effectiveness study.
Done well, the resulting research will be sound, credible and able to inform treatment options. Even with
good research, however, the challenge remains in how to consider the rapidly evolving science related to
subpopulations or personalized medicine. As the science continues to evolve, we will need the flexibility
for methods to evolve as well, and it will be important for all stakeholders to remain engaged in those
efforts and share new methodologies with researchers.
To provide a better understanding of this new committee and its role, the National Pharmaceutical
Council has developed the PCORI Methodology Committee Resource Guide, which is a companion
booklet to the Patient-Centered Outcomes Research Institute Resource Guide (available online at
www.npcnow.org/pcoriguide). This new resource guide will assist you in understanding the most
common types of comparative effectiveness research (CER); introduce you to the members of the
Methodology Committee; and offer a variety of resources for additional information.
Both of these guides, along with the CER Daily Newsfeed, NPC’s source for the latest CER news and
activities, and Demystifying Comparative Effectiveness Research: A Case Study Learning Guide, which
aids in the basic understanding of the common types of CER, are available on NPC’s Web site at
www.npcnow.org. We encourage you to check out these resources to help you stay abreast of ongoing
CER developments.

Sincerely,

Robert W. Dubois, MD, PhD


Chief Science Officer
5

About the PCORI Methodology Committee


The Patient Protection and Affordable Care Act (PL 111-148) established the non-profit entity, the Patient-Centered
Outcomes Research Institute (PCORI), which is charged with the oversight of comparative effectiveness research (CER).
To assist PCORI with developing and improving “the scientific methods of comparative clinical research,” the law
established a standing methodology committee composed of 17 members. These members, including the directors of
the National Institutes of Health and the Agency for Healthcare Research and Quality (AHRQ) or their designees, “shall
be experts in their scientific field, such as health services research, clinical research, comparative clinical effectiveness
research, biostatistics, genomics, and research methodologies.” The methodology committee is appointed by the
Government Accountability Office Comptroller General.
Tasks
The purpose of this committee is to ensure that the comparative effectiveness research developed under PCORI’s
watch is of high quality, which is why this group is charged with the development of methodological standards for
research. The types of research include not only new or primary research such as randomized clinical trials, molecularly
informed trials, and observational studies, but also systematic reviews or synthesis of existing research, and any other
methodologies recommended by the methodology committee. The law states that, “such methodological standards
shall provide specific criteria for internal validity, generalizability, feasibility, and timeliness of research and for health
outcomes measures, risk adjustment, and other relevant aspects of research and assessment with respect to the design
of research.” In addition, the law allows for the flexibility to include new information, data and advances in technology
in ongoing research projects; calls for public input as methods are developed and updated; and ensures that patient
subpopulations are considered in different kinds of research.

Another important task for the committee is the development of a translation table, which would serve as a reference
tool to assist the PCORI Board of Governors in determining which research methods are best suited for a specific
research question. A translation table helps to lay out the framework for developing research by first looking at the
questions that decision makers need answered, and then determining what kinds of research can best address those
questions. This places the decision makers at the center of the development of CER, rather than as simply a recipient of
research results.
Reports and Deadlines
The committee has a short amount of time in which to complete its work—merely 18 months after establishment—so it
is permitted to rely on outside assistance. The methodology committee “may consult and contract with the Institute of
Medicine of the National Academies and academic, non-profit, or other private and governmental entities with relevant
expertise to carry out” its activities.

As the methodology committee moves forward with its tasks, it must submit reports to the PCORI Board of Governors.
The reports should include “recommendations for the Institute to adopt methodological standards developed and
updated by the methodology committee as well as other actions deemed necessary to comply with such
methodological standards.”
6

Ongoing Methodology Development


A number of public and private sector organizations, such as AHRQ, the National Institutes of Health, the Cochrane
Collaboration, the International Society for Pharmaceoconomics and Outcomes Research (ISPOR), the Center for Medical
Technology Policy (CMTP), and NPC, among others, are actively engaged in work to advance the methodology for
conducting comparative effectiveness research. This work includes developing new methods, garnering agreement on
good research practices, and exploring the types of questions facing researchers, providers, health systems and patients.

To date, there have been several initiatives resulting from those efforts, some of which were supported by NPC’s
funding and participation. Those include the GRACE Principles (Good ReseArch for Comparative Effectiveness), which
focus on observational studies; the PACE Initiative (Pragmatic Approaches to Comparative Effectiveness) for Bayesian
and pragmatic trials; and guidance documents developed by CMTP. AHRQ, as well as ISPOR, also has developed key
methodological documents. (See existing methodological standards and practices on page 47.) In the future, NPC will
continue to engage in thoughtful research and partnerships to help inform the advancement of CER.

While much work is ongoing, it will require the research community to engage in dialogue with the decision makers who
will need credible, reliable, and relevant CER in making treatment decisions. In the coming months, the members of the
methodology committee will be certain to play a key role in this ongoing discussion.

Source: Subtitle D–Patient-Centered Outcomes Research, SEC. 6301. PATIENT-CENTERED OUTCOMES RESEARCH, PL 111-148

Biographies of the Methodology Committee members follow:*

*Members of the Methodology Committee are widely published authors. Due to publication constraints, selected publications listed are
from the last two years or are publications highlighted on members’ webpages.
7

Naomi Aronson, PhD

Executive Director, Blue Cross and Blue Shield Association Technology


Evaluation Center

Biography: Naomi Aronson, PhD


Biography:
Naomi Aronson, PhD, is the executive director of the Blue Cross and Blue Shield Association Technology Evaluation Center (TEC).
Dr. Aronson has overseen TEC’s development as a nationally recognized technology assessment program and an Evidence-
based Practice Center (EPC) of the Agency for Healthcare Research and Quality (AHRQ). She has directed over 300 technology
assessments and 13 evidence reports for AHRQ. She represented the private sector on a US Agency for International Development
Team providing technical assistance to the Hungarian government on building evidence-based medicine capacity in the national
health insurance system. She was a member of the 2007 Ontario Health Technology Assessment Evaluation Review Team.
Dr. Aronson is a member of the Institute of Medicine Forum on Drug Discovery Translation and Development, the Institute of
Medicine Genomics Roundtable, the Steering Committee of the Chicago-Area DEcIDE Research Center, the National Business
Group on Health Committee on Evidence-Based Benefit Design, and a review committee co-chair for the International Society for
Pharmacoeconomics and Outcomes Research 14th Annual International Meeting. Previously, Dr. Aronson was a member of the
Northwestern University faculty, specializing in the sociology of science and medicine. She also was a post-doctoral fellow in the
Science, Technology and Society Program at the Massachusetts Institute of Technology and received research awards from the
National Science Foundation and the American Council of Learned Societies. Dr. Aronson’s academic research focused on how the
organization of scientific specialties in biomedical and clinical research affects the process of scientific discovery.

Selected Publications:
Samson DJ, Ratko TA, Rothenberg BM, Brown HM, Bonnell CJ, Ziegler KM, Aronson N. Comparative Effectiveness and Safety of
Radiotherapy Treatments for Head and Neck Cancer. Rockville, MD: US Agency for Healthcare Research and Quality;
May 2010.
Seidenfeld J, Samson DJ, Rothenberg BM, Bonnell CJ, Ziegler KM, Aronson N. HER2 testing to manage patients with breast
cancer or other solid tumors. Evid Rep Technol Assess. November 2008;(172):1-362.
Samson DJ, Seidenfeld J, Simon GR, Turrisi AT III, Bonnell C, Ziegler KM, Aronson N; American College of Chest Physicians.
Evidence for management of small cell lung cancer: ACCP evidence-based clinical practice guidelines. 2nd ed. Chest.
September 2007;132(3)(suppl):314S-323S.
Samson DJ, Grant MD, Ratko TA, Bonnell CJ, Ziegler KM, Aronson N. Treatment of primary and secondary osteoarthritis of the
knee. Evid Rep Technol Assess. September 2007;(157):1-157.
Rothenberg BM, Ziegler KM, Aronson N. Technology Evaluation Center assessment synopsis: full-field digital mammography.
J Am Coll Radiol. August 2006;3(8):586-588.
Seidenfeld J, Samson DJ, Bonnell CJ, Ziegler KM, Aronson N. Management of small cell lung cancer. Evid Rep Technol Assess.
July 2006;(143):1-154.
Seidenfeld J, Piper M, Bohlius J, Weingart O, Trelle S, Engert A, Skoetz N, Schwarzer G, Wilson J, Brunskill S, Hyde C, Bonnell C,
Ziegler KM, Aronson N. Comparative Effectiveness of Epoetin and Darbepoetin for Managing Anemia in Patients Undergoing
Cancer Treatment. Rockville, MD: US Agency for Healthcare Research and Quality; May 2006.
8

Tatsioni A, Zarin DA, Aronson N, Samson DJ, Flamm CR, Schmid C, Lau J. Challenges in systematic reviews of diagnostic
technologies. Ann Intern Med. June 21, 2005;142(12 Pt 2):1048-1055.
Flamm CR, Ziegler KM, Aronson N. Technology Evaluation Center assessment synopsis: use of magnetic resonance imaging to
avoid a biopsy in women with suspicious primary breast lesions. J Am Coll Radiol. June 2005;2(6):485-487.

Biography: Naomi Aronson, PhD


About the Blue Cross and Blue Shield Association Technology Evaluation Center:
Founded in 1985 by the Blue Cross and Blue Shield Association, the Technology Evaluation Center (TEC) pioneered the
development of scientific criteria for assessing medical technologies through comprehensive reviews of clinical evidence. TEC
operates as part of the Association’s Office of Clinical Affairs.
Since its inception, TEC has been recognized for leadership in producing evidence-based technology assessments. Each TEC
Assessment is a comprehensive evaluation of the clinical effectiveness and appropriateness of a given medical procedure, device
or drug. Averaging 20 to 25 assessments a year, TEC provides health care decision makers with timely, rigorous and credible
information on clinical effectiveness. TEC serves a wide range of clients in both the private and public sectors, including Kaiser
Permanente and the Centers for Medicare and Medicaid Services (CMS).

Additional Information:
● Blue Cross and Blue Shield Association Technology Evaluation Center. www.bcbs.com/blueresources/tec/
● Blue Cross and Blue Shield Association, Technology Evaluation Center (TEC). Agency for Healthcare Research and
Quality (AHRQ). www.ahrq.gov/clinic/epc/bcbsatec.htm

Sources:
Technology Evaluation Center staff. Blue Cross and Blue Shield Association Technology Evaluation Center Web site.
http://www.bcbs.com/blueresources/tec/tec-staff.html. Accessed January 30, 2011.
What is the Technology Evaluation Center? Blue Cross and Blue Shield Association Technology Evaluation Center Web site.
http://www.bcbs.com/blueres/tec/what-is-tec.html. Accessed January 30, 2011.
9

Ethan Basch, MD, MSc

Medical Oncologist and Health Services Researcher, Department of Medicine


and Department of Epidemiology, Memorial Sloan-Kettering Cancer Center

Biography: Ethan Basch, MD, MSc


Biography:
Ethan Basch, MD, MSc, is a practicing medical oncologist and health services researcher at Memorial Sloan-Kettering Cancer Center,
with appointments in the Department of Medicine and in the Department of Epidemiology and Biostatistics. His research focuses
on methods for using patient-reported data to evaluate the comparative effectiveness and safety of interventions. Dr. Basch is
chair of the Health Outcomes Committee of the Cancer and Leukemia Group B of the National Cancer Institute (NCI), chair of the
Clinical Practice Guidelines Committee of the American Society of Clinical Oncology (ASCO), and a member of ASCO’s Comparative
Effectiveness Task Force. He leads the NCI’s PRO-CTCAE initiative to develop a standard system for patient-reporting of safety data
in clinical trials. Dr. Basch received a BA from Brown University, an MD from Harvard Medical School, an MSc in epidemiology from
Harvard School of Public Health, and an MPhil in literature from Oxford University.

Selected Publications:
Basch E, Iasonos A, McDonough T, Barz A, Culkin A, Kris MG, Scher HI, Schrag D. Clinician versus patient self-reporting of
symptoms during cancer treatment: a paired analysis using the National Cancer Institute’s Common Terminology Criteria for
Adverse Events (CTCAE). Lancet Oncol. 2006;7(10):903-909.
Basch E, Artz D, Dulko D, Scher K, Sabbatini P, Hensley M, Mitra N, Speakman J, McCabe M, Schrag D. Patient online self-
reporting of toxicity symptoms during chemotherapy. J Clin Oncol. 2005;23(15):3552-3561.
Basch EM, Thaler HT, Shi W, Yakren S, Schrag D. Use of information resources by patients with cancer and their companions.
Cancer. 2004;100(11):2476-2483.
Danso M, Basch EM. Hematology and Oncology Pearls. New York, NY: Elsevier Science; 2005.
Basch EM, Ulbricht CE. Complementary, alternative, and integrative therapies in cancer care. In: Devita VT, Hellman S,
Rosenberg SA, eds. Cancer: Principles & Practice of Oncology. 7th ed. Philadelphia, PA: Lippincott Williams & Wilkins;
2004:chap 62.
Basch EM, Ulbricht CE, eds. Natural Standard Comprehensive Evidence-Based Guide. St. Louis, MO: Mosby; 2004.
Ulbricht CE, Basch EM, eds. Natural Standard Bottom-Line Guide. St Louis, MO: Mosby; 2004.
Ulbricht CE, Basch EM. Harvard Medical School Guide to CAM. Cambridge, MA: Harvard Health Publications; 2003.
Basch EM, Birnbaum, SL, Garza, C, Messersmith, WM, eds. The Massachusetts General Hospital (MGH) Primer of Outpatient
Medicine. Philadelphia, PA: Lippincott Williams & Wilkins; 2002.

About the Memorial Sloan-Kettering Cancer Center:


The world’s oldest and largest private cancer center, Memorial Sloan-Kettering Cancer Center (MSKCC) has devoted more than
a century to patient care as well as to innovative research, making significant contributions to new and better therapies for the
treatment of cancer.
Today, the Center has more than 11,000 employees including 768 Memorial Hospital attending staff and 140 Sloan-Kettering
Institute members. In 2009, more than 23,000 patients were admitted to Memorial Hospital, and Memorial Sloan-Kettering
accommodated 500,317 outpatient visits at its Manhattan and regional sites combined.
10

Additional Information:
● Memorial Sloan-Kettering Cancer Center. http://www.mskcc.org
● Twitter: http://twitter.com/sloan_kettering

Biography: Ethan Basch, MD, MSc


Sources:
Appointments announced to Methodology Committee of the Patient-Centered Outcomes Research Institute (PCORI) [press
release]. Washington, DC: US Government Accountability Office; January 21, 2011.
http://www.gao.gov/press/pcori_2011jan21.html. Accessed January 30, 2011.
Physician Biography: Ethan M. Basch, MD. Memorial Sloan-Kettering Cancer Center Web site.
http://www.mskcc.org/prg/prg/bios/901.cfm. Accessed January 30, 2011.
History and Overview. Memorial Sloan-Kettering Cancer Center Web site. http://www.mskcc.org/mskcc/html/511.cfm.
Accessed January 30, 2011.
11

Alfred O. Berg, MD, MPH

Professor, Department of Family Medicine, University of Washington

Biography: Alfred O. Berg, MD, MPH


Biography:
Alfred O. Berg, MD, MPH, is a professor in the Department of Family Medicine at the University of Washington. He is chair of the
Institute of Medicine’s Committee on Standards for Systematic Reviews of Clinical Effectiveness Research and chair of the Panel
on Evaluation of Genomic Applications in Practice and Prevention at the Centers for Disease Control and Prevention (CDC). He
has served on numerous national expert panels using evidence-based medicine to develop clinical guidelines, including serving
as chairman of the US Preventive Services Task Force. Dr. Berg also has served as chair of the CDC Sexually Transmitted Disease
Treatment Guidelines Panel, co-chair of the Agency for Healthcare Research and Quality’s otitis media panel, and as a member of
the American Medical Association/CDC panel that produced Guidelines for Adolescent Preventive Services. Dr. Berg received a BA
from Tabor College, an MD from Washington University School of Medicine, and an MPH in epidemiology from the University of
Washington School of Public Health and Community Medicine.

Selected Publications:
Berg AO. What do we get from participating in practice-based research networks? J Am Board Fam Med. July-August
2010;23(4):440-441.
Berg AO. The CDC’s EGAPP initiative: evaluating the clinical evidence for genetic tests. Am Fam Physician. December
2009;80(11):1218.
Berg AO, Baird MA, Botkin JR, Driscoll DA, Fishman PA, Guarino PD, Hiatt RA, Jarvik GP, Millon-Underwood S, Morgan TM,
Mulvihill JJ, Pollin TI, Schimmel SR, Stefanek ME, Vollmer WM, Williams JK. National Institutes of Health state-of-the-science
conference statement: family history and improving health: August 24-26, 2009. NIH Consens State Sci Statements. August
2009;26(1):1-19.
Berg AO, Baird MA, Botkin JR, Driscoll DA, Fishman PA, Guarino PD, Hiatt RA, Jarvik GP, Millon-Underwood S, Morgan TM,
Mulvihill JJ, Pollin TI, Schimmel SR, Stefanek ME, Vollmer WM, Williams JK. National Institutes of Health state-of-the-science
conference statement: family history and improving health. Ann Intern Med. December 2009;151(12):872-877.
Berg AO. The aftermath of efficacy. Ann Fam Med. January-February 2009;7(1):3-4.
Teutsch SM, Bradley LA, Palomaki GE, Haddow JE, Piper M, Calonge N, Dotson WD, Douglas MP, Berg AO; EGAPP Working
Group. The Evaluation of Genomic Applications in Practice and Prevention (EGAPP) Initiative: methods of the EGAPP working
group. Genet Med. January 2009;11(1):3-14.

About the Department of Family Medicine at the University of Washington:


The Department of Family Medicine at the University of Washington provides leadership for core curriculum in the School of
Medicine and is recognized for its research and academic programs in rural communities. With over 800 clinical faculty members
located throughout Washington, Wyoming, Alaska, Montana, and Idaho, and approximately 40 regular faculty located at the
university site in Seattle, the department is one of the largest of its kind in the country. The research section is one of the largest
family medicine research enterprises in the country.
12

Additional Information:
● University of Washington Department of Family Medicine. http://depts.washington.edu/fammed

Sources:

Biography: Alfred O. Berg, MD, MPH


Appointments announced to Methodology Committee of the Patient-Centered Outcomes Research Institute (PCORI) [press
release]. Washington, DC: US Government Accountability Office; January 21, 2011.
http://www.gao.gov/press/pcori_2011jan21.html. Accessed January 30, 2011.
University of Washington Department of Family Medicine Web site.
http://depts.washington.edu/fammed. Accessed January 30, 2011.
Detail view: Alfred O. Berg, MD, MPH. University of Washington Department of Family Medicine Web site.
http://depts.washington.edu/fammed/contacts/details.php?person=00041. Accessed January 30, 2011.
13

Carolyn M. Clancy, MD
Director, Agency for Healthcare Research and Quality (AHRQ)

Biography: Carolyn M. Clancy, MD


Biography:
Carolyn M. Clancy, MD, was appointed director of the Agency for Healthcare Research and Quality (AHRQ) on February 5, 2003,
and reappointed on October 9, 2009. Prior to her appointment, Dr. Clancy was director of AHRQ’s Center for Outcomes and
Effectiveness Research.
Dr. Clancy, a general internist and health services researcher, is a graduate of Boston College and the University of Massachusetts
Medical School. Following clinical training in internal medicine, Dr. Clancy was a Henry J. Kaiser Family Foundation fellow at the
University of Pennsylvania. Before joining AHRQ in 1990, she was also an assistant professor in the Department of Internal Medicine
at the Medical College of Virginia.
Dr. Clancy holds an academic appointment at the George Washington University School of Medicine (clinical associate professor,
Department of Medicine) and serves as senior associate editor for the journal Health Services Research. She serves on multiple
editorial boards, including Annals of Internal Medicine, Annals of Family Medicine, American Journal of Medical Quality, and
Medical Care Research and Review.
Dr. Clancy is a member of the Institute of Medicine and was elected a Master of the American College of Physicians in 2004. In
2009, she was awarded the William B. Graham Prize for Health Services Research. Dr. Clancy’s major research interests include
improving health care quality and patient safety and reducing disparities in care associated with patient race, ethnicity, gender,
income, and education. As director of AHRQ, she launched the first annual report to Congress on health care disparities and health
care quality.

Selected Publications:
Wu AW, Snyder C, Clancy CM, Steinwachs DM. Adding the patient perspective to comparative effectiveness research. Health Aff
(Millwood). October 2010;29(10):1863-1871.
Clancy C; The Patient-Centered Outcomes Research Institute. Shaping Convergent Strategies in CER. Washington, DC: US
Agency for Healthcare Research and Quality; June 24, 2010.
http://www.npcnow.org/App_Themes/Public/pdf/events/2010_events/June_24/Clancy_June_24.pdf. Accessed
January 31, 2011.
Clancy C, Collin FS. Patient-Centered Outcomes Research Institute: the intersection of science and health care. Sci Transl Med.
June 23, 2010;2(37):18.
Conway PH, Clancy C. Charting a path from comparative effectiveness funding to improved patient-centered health care. JAMA.
March 10, 2010;303(10):985-986.
Slutsky JR, Clancy CM. Patient-centered comparative effectiveness research: essential for high-quality care. Arch Intern Med.
March 8, 2010;170(5):403-404.
Clancy CM. The promise and future of comparative effectiveness research. J Nurs Care Qual. 2010; 25(1):1-4. PMID:20010250
Conway PH, Clancy C. Comparative-effectiveness research-implications of the Federal Coordinating Council’s report. N Engl J
Med. 2009;361(4):328-330.
14

Slutsky JR, Clancy CM. AHRQ’s Effective Health Care Program: why comparative effectiveness matters. Am J Med Qual.
2009;24(1):67-70.
Clancy C. Is the clinical advice you give your patients evidence-based? Medscape J Med. 2008;10(11):252.
Clancy CM. Interpreting the evidence: helping consumers understand medical information. Nurs Womens Health.

Biography: Carolyn M. Clancy, MD


2008;12(3):200-202.
Cohen SB, Clancy CM. ARHQ and data collection. Health Aff. 2008;27(2):586-587.
Coopey M, James MD, Lawrence W, Clancy CM. The challenge of comparative effectiveness: getting the right information to the
right people at the right time. J Nurs Care Qual. 2008;23(1):1-5.
Clancy CM. Getting to “smart” health care, comparative effectiveness research is a key component of, but tightly linked with,
health care delivery in the information age. Health Aff. November/December 2006;25(6):w589-w592.
Clancy CM, Slutsky JR. Advancing excellence in health care: getting to effectiveness. J Invest Med. 2005; 53(2):65-66.
Clancy CM, Cronin K. Evidence-based decision making: global evidence, local decisions, the challenge now is to further develop
and translate the worldwide evidence base for local application. Health Aff. January/February 2005;24(1):151-162.
Tunis SR, Stryer DB, Clancy CM. Practical clinical trials: increasing the value of clinical research for decision making in clinical
and health policy. JAMA. September 24, 2003;290(12):1624-1632.

About the Agency for Healthcare Research and Quality:


AHRQ is the lead federal agency charged with improving the quality, safety, efficiency, and effectiveness of health care for all
Americans. As one of 12 agencies within the Department of Health and Human Services, AHRQ supports health services research
that will improve the quality of health care and promote evidence-based decision making.

Additional Information:
● Agency for Healthcare Research and Quality. www.ahrq.gov
● Effective Healthcare Program, Agency for Healthcare Research and Quality. http://effectivehealthcare.ahrq.gov/
● Health Services Research Journal. www.hsr.org

Sources:
Biography: Carolyn M. Clancy, MD, director, AHRQ. Rockville, MD: Agency for Healthcare Research and Quality; April 2010.
http://www.ahrq.gov/about/clancybio.htm. Accessed January 30, 2011.
Agency for Healthcare Research and Quality (AHRQ) At A Glance. Rockville, MD: Agency for Healthcare Research and Quality;
May 2009. AHRQ Publication No. 09-P003. http://www.ahrq.gov/about/ataglance.htm.
15

David Flum, MD, MPH

 rofessor, Department of Surgery and Adjunct Professor, Department of Health


P
Services, University of Washington Schools of Medicine and Public Health;
Attending Physician, General Surgery, University of Washington Medical Center

Biography: David Flum, MD, MPH


Biography:
David Flum, MD, MPH, is a professor in the Department of Surgery and an adjunct professor in the Department of Health Services
at the University of Washington (UW) Schools of Medicine and Public Health. He is an attending physician in general surgery
at the University of Washington Medical Center. He is a co-founder and member of the Leadership Council of the UW Centers
for Comparative and Health Systems Effectiveness Alliance (CHASE) and directs UW’s Surgical Outcomes Research Center. Dr.
Flum’s investigations have compared interventional and non-interventional approaches for common clinical conditions such as
appendicitis, peripheral vascular disease, obesity, and multiple types of cancer. He received his BA and MD from the University of
Miami School of Medicine and his MPH from the University of Washington School of Public Health.

Selected Publications:
Massarweh NN, Park JO, Farjah F, Yeung RS, Symons RG, Vaughan TL, Baldwin LM, Flum DR. Trends in the utilization and
impact of radiofrequency ablation for hepatocellular carcinoma. J Am Coll Surg. April 2010;210(4):441-448.
Massarweh NN, Legner VJ, Symons RG, McCormick WC, Flum DR. Impact of advancing age on abdominal surgical outcomes.
Arch Surg. December 2009;144(12):1108-1114.
Farjah F, Wood DE, Varghese TK, Massarweh NN, Symons RG, Flum DR. Health care utilization among surgically treated
Medicare beneficiaries with lung cancer. Ann Thorac Surg. December 2009;88(6):1749-1756.
Flum DR. The cover. Spatial concept: expectations. JAMA. October 2009;302(14):1508.
Gosman GG, King WC, Schrope B, Steffen KJ, Strain GW, Courcoulas AP, Flum DR, Pender JR, Simhan HN. Reproductive health
of women electing bariatric surgery. Fertil Steril. September 2010;94(4):1426-1431.
McCulloch P, Altman DG, Campbell WB, Flum DR, Glasziou P, Marshall JC, Nicholl, J; for the Balliol Collaboration. No surgical
innovation without evaluation: the IDEAL recommendations. Lancet. September 2009;374(9695):1105-1112.

About the University of Washington Medicine:


University of Washington Medicine’s mission is to improve the health of the public by advancing medical knowledge, providing
outstanding primary and specialty care to the people of the region, and preparing tomorrow’s physicians, scientists and other health
professionals. UW Medicine owns or operates Harborview Medical Center, the University of Washington Medical Center, Northwest
Hospital and Medical Center, a network of seven UW Medicine Neighborhood Clinics that provide primary care, the UW School
of Medicine, the physician practice UW Physicians, and Airlift Northwest. In addition, UW Medicine shares in the ownership and
governance of Children’s University Medical Group and Seattle Cancer Care Alliance, a partnership composed of UW Medicine, Fred
Hutchinson Cancer Research Center and Seattle Children’s. UW Medicine faculty includes four Nobel Prize winners, 33 Institute of
Medicine members, 32 National Academy of Sciences members and 16 Howard Hughes Medical Institute investigators.

Additional Information:
● University of Washington Department of Surgery. http://depts.washington.edu/surgery
● UW Medicine. http://uwmedicine.washington.edu
16

Sources:
Appointments announced to Methodology Committee of the Patient-Centered Outcomes Research Institute (PCORI) [press
release]. Washington, DC: US Government Accountability Office; January 21, 2011.
http://www.gao.gov/press/pcori_2011jan21.html. Accessed January 30, 2011.

Biography: David Flum, MD, MPH


David R. Flum, M.D., M.P.H., F.A.C.S. University of Washington Department of Surgery Faculty Web site.
http://depts.washington.edu/surgery/faculty/flum.html. Accessed January 30, 2011.
About UW Medicine. UW Medicine, Seattle Web site. http://uwmedicine.washington.edu/global/about/Pages/default.aspx.
Accessed January 30, 2011.
17

Sherine Gabriel, MD, MSc

Methodology Committee Chair

Biography: Sherine Gabriel, MD, MSc


Professor of Medicine and of Epidemiology, and the William J. and Charles H.
Mayo Professor, Mayo Clinic

Biography:
Sherine Gabriel, MD, MSc, is a professor of medicine and of epidemiology and the William J. and Charles H. Mayo Professor at
Mayo Clinic. She has served as chair of the Department of Health Sciences Research at Mayo Clinic, which includes the divisions of
Epidemiology, Health Care Policy and Research, Biomedical Informatics, and Biostatistics. Dr. Gabriel also has served as vice-chair
of Mayo Clinic’s Research Committee and as a member of the Mayo Clinic Executive Board. She founded Mayo Clinic’s Center
for Patient Oriented Research as well as clinical research training programs that later transitioned into the Mayo Clinic’s Center for
Translational Sciences Activities, where she serves as co-principal investigator. She has been president of the American College
of Rheumatology (ACR) and is the founding chair of the ACR Quality Measures Committee. Dr. Gabriel received an MD, with
distinction, from the University of Saskatchewan and an MSc in clinical epidemiology and biostatistics from McMaster
University, Canada.

Selected Publications:
Crowson CS, Myasoedova E, Davis JM III, Roger VL, Karon BL, Borgeson D, Rodeheffer RJ, Therneau TM, Gabriel SE. B-type
natriuretic peptide is a poor screening tool for left ventricular diastolic dysfunction in rheumatoid arthritis patients without
clinical cardiovascular disease. Arthritis Care Res (Hoboken). January 2011.
Myasoedova E, Crowson CS, Kremers HM, Roger VL, Fitz-Gibbon PD, Therneau TM, Gabriel SE. Lipid paradox in rheumatoid
arthritis: the impact of serum lipid measures and systemic inflammation on the risk of cardiovascular disease. Ann Rheum Dis.
January 2011.
Crowson CS, Matteson EL, Myasoedova E, Michet CJ, Ernste FC, Warrington KJ, Davis JM III, Hunder GG, Therneau TM, Gabriel
SE. The lifetime risk of adult-onset rheumatoid arthritis and other inflammatory autoimmune rheumatic diseases. Arthritis
Rheum. December 2010.
Crowson CS, Myasoedova E, Davis JM III, Matteson EL, Roger VL, Therneau TM, Fitz-Gibbon P, Rodeheffer RJ, Gabriel SE.
Increased prevalence of metabolic syndrome associated with rheumatoid arthritis in patients without clinical cardiovascular
disease. J Rheumatol. January 2011;38(1):29-35.
Hoganson DD, Crowson CS, Warrington KJ, Gabriel SE, Matteson EL. Lack of association of high body mass index with risk for
developing polymyalgia rheumatica. Int J Rheum Dis. August 2010;13(3):e1-5.
Myasoedova E, Davis JM III, Crowson CS, Gabriel SE. Epidemiology of rheumatoid arthritis: rheumatoid arthritis and mortality.
Curr Rheumatol Rep. October 2010;12(5):379-385.
Schäfer VS, Kermani TA, Crowson CS, Hunder GG, Gabriel SE, Ytterberg SR, Matteson EL, Warrington KJ. Incidence of
herpes zoster in patients with giant cell arteritis: a population-based cohort study. Rheumatology (Oxford). November
2010;49(11):2104-2108.
Kremers HM, Myasoedova E, Crowson CS, Savova G, Gabriel SE, Matteson EL. The Rochester Epidemiology Project: exploiting
the capabilities for population-based research in rheumatic diseases. Rheumatology (Oxford). January 2011;50(1):6-15.
Liang KP, Myasoedova E, Crowson CS, Davis JM, Roger VL, Karon BL, Borgeson DD, Therneau TM, Rodeheffer RJ, Gabriel SE.
Increased prevalence of diastolic dysfunction in rheumatoid arthritis. Ann Rheum Dis. September 2010;69(9):1665-1670.
18

Davis JM III, Knutson KL, Strausbauch MA, Crowson CS, Therneau TM, Wettstein PJ, Matteson EL, Gabriel SE. Analysis of
complex biomarkers for human immune-mediated disorders based on cytokine responsiveness of peripheral blood cells.
J Immunol. June 2010;184(12):7297-7304.

Biography: Sherine Gabriel, MD, MSc


Singh JA, Gabriel SE, Lewallen DG. Higher body mass index is not associated with worse pain outcomes after primary or
revision total knee arthroplasty. J Arthroplasty. April 2010.
Kermani TA, Schäfer VS, Crowson CS, Hunder GG, Ytterberg SR, Matteson EL, Gabriel SE, Warrington KJ. Cancer preceding
giant cell arteritis: a case-control study. Arthritis Rheum. June 2010;62(6):1763-1769.
Myasoedova E, Crowson CS, Kremers HM, Therneau TM, Gabriel SE. Is the incidence of rheumatoid arthritis rising?: results
from Olmsted County, Minnesota, 1955-2007. Arthritis Rheum. June 2010;62(6):1576-1582.
Kermani TA, Schäfer VS, Crowson CS, Hunder GG, Gabriel SE, Ytterberg SR, Matteson EL, Warrington KJ. Malignancy risk in
patients with giant cell arteritis: a population-based cohort study. Arthritis Care Res (Hoboken). February 2010;62(2):149-154.
Myasoedova E, Gabriel SE. Cardiovascular disease in rheumatoid arthritis: a step forward. Curr Opin Rheumatol. May
2010;22(3):342-347.
Bongartz T, Nannini C, Medina-Velasquez YF, Achenbach SJ, Crowson CS, Ryu JH, Vassallo R, Gabriel SE, Matteson EL.
Incidence and mortality of interstitial lung disease in rheumatoid arthritis: a population-based study. Arthritis Rheum. June
2010;62(6):1583-1591.
Crowson CS, Liang KP, Therneau TM, Kremers HM, Gabriel SE. Could accelerated aging explain the excess mortality in patients
with seropositive rheumatoid arthritis? Arthritis Rheum. February 2010;62(2):378-382.
Gabriel SE. Heart disease and rheumatoid arthritis: understanding the risks. Ann Rheum Dis. January 2010;69(Suppl 1):i61-i64.
Myasoedova E, Crowson CS, Kremers HM, Fitz-Gibbon PD, Therneau TM, Gabriel SE. Total cholesterol and LDL levels decrease
before rheumatoid arthritis. Ann Rheum Dis. July 2010;69(7):1310-1314.

About Mayo Clinic:


Mayo Clinic is a non-profit worldwide leader in medical care, research and education for people from all walks of life. More than
55,000 doctors, scientists, students and allied health staff work and study at Mayo Clinic campuses in Rochester, Minnesota;
Jacksonville, Florida; and Scottsdale/Phoenix, Arizona. Collectively, Mayo Clinic cares for more than half a million people each year.
Mayo Clinic also serves more than 70 communities in the upper Midwest through the Mayo Health System.

Additional Information:
● Mayo Clinic. http://www.mayoclinic.org

Sources:
Appointments announced to Methodology Committee of the Patient-Centered Outcomes Research Institute (PCORI) [press
release]. Washington, DC: US Government Accountability Office; January 21, 2011.
http://www.gao.gov/press/pcori_2011jan21.html. Accessed January 30, 2011.
Doctors and departments, Sherine E. Gabriel, MD. Mayo Clinic Web site. http://www.mayoclinic.org/bio/10293850.html.
Accessed January 30, 2011.
News from Mayo Clinic. Mayo Clinic Web site. http://www.mayoclinic.org/news. Accessed January 31, 2011.
Medical experts for media—epidemiology. Mayo Clinic News Blog; September 2, 2010.
http://newsblog.mayoclinic.org/2010/09/02/medical-experts-for-media-epidemiology. Accessed January 30, 2011.
Mayo Clinic Facts—2009. Mayo Clinic Web site. http://www.mayoclinic.org/about/facts.html. Accessed January 30, 2011.
19

Steven Goodman, MD, MHS, PhD

Biography: Steven Goodman, MD, MHS, PhD


Professor of Oncology, of Pediatrics, of Epidemiology and of Biostatistics,
Johns Hopkins School of Medicine and Bloomberg School of Public Health

Biography:
Steven Goodman, MD, MHS, PhD, is professor of oncology in the Division of Biostatistics of the Johns Hopkins Kimmel Cancer
Center, with appointments in the Departments of Pediatrics, Biostatistics, and Epidemiology in the Johns Hopkins Schools of
Medicine and Public Health. He is also a core faculty member in the Johns Hopkins Bloomberg School of Public Health’s Center for
Clinical Trials and the Johns Hopkins Berman Institute of Bioethics.
Since 2004 he has been the editor of Clinical Trials: Journal of the Society for Clinical Trials, and has served as senior statistical
editor of the Annals of Internal Medicine since 1987.
He has served on the Institute of Medicine’s (IOM) Committees on Immunization Safety Review, Health Effects in Vietnam Veterans
of Exposure to Herbicides: Second Biennial Update, Agent Orange and Diabetes, and the Committee on Alternative Models to
Daubert Standards. He serves as co-chair of the IOM Ethical and Scientific Issues in Studying the Safety of Approved Drugs, as a
member and scientific advisor to the Medical Advisory Panel of the National Blue Cross Blue Shield Technology Evaluation program,
and served as a consultant to the President’s Advisory Commission on Human Radiation Experiments. He was the lead author of the
chapter on causal assessment in the Surgeon General’s 2004 report on Smoking and Health.
He was co-director of the Johns Hopkins Evidence-based Practice Center from 2000-2004, and served for three years on the Medical
Coverage Advisory Commission. He teaches and writes extensively on evidence evaluation and inferential, methodological, and
ethical issues in epidemiology and clinical research.
Dr. Goodman received an AB from Harvard, an MD from New York University, trained in pediatrics at Washington University in St.
Louis, and received his MHS in biostatistics and his PhD in epidemiology from Johns Hopkins Bloomberg School of Public Health.

Selected Publications:
Robinson KA, Goodman SN. A systematic examination of the citation of prior research in reports of randomized, controlled
trials. Ann Intern Med. 2011;154(1):50-55.
Goodman SN. On making clinical trials possible. Clin Trials. 2010;7(6):621.
Gerson J, Goodman SN. An absence of evidence in “evidence-based rulemaking.” Am J Bioeth. 2010;10(6):22-23.
Brodsky RA, Chen AR, Dorr D, Fuchs EJ, Huff CA, Luznik L, Smith BD, Matsui WH, Goodman SN, Ambinder RF, Jones RJ. High-
dose cyclophosphamide for severe aplastic anemia: long-term follow-up. Blood. 2010;115(11):2136-2141.
Dawson L, Zarin DA, Emanuel EJ, Friedman LM, Chaudhari B, Goodman SN. Considering usual medical care in clinical trial
design. PLoS Med. 2009;6(9):e1000111.
Luce BR, Kramer JM, Goodman SN, Connor JT, Tunis S, Whicher D, Schwartz JS. Rethinking randomized clinical trials for
comparative effectiveness research: the need for transformational change. Ann Intern Med. 2009;151(3):206-209.
Marchionni L, Wilson RF, Wolff AC, Marinopoulos S, Parmigiani G, Bass EB, Goodman SN. Systematic review: gene expression
profiling assays in early-stage breast cancer. Ann Intern Med. 2008;148(5):358-369.
Goodman SN. Systematic reviews are not biased by results from trials stopped early for benefit. J Clin Epidemiol.
2008;61(1):95-96.
20

Goodman SN. Stopping at nothing? Some dilemmas of data monitoring in clinical trials. Ann Intern Med.
2007;146(12):882-887.

Biography: Steven Goodman, MD, MHS, PhD


Laine C, Goodman SN, Griswold ME, Sox HC. Reproducible research: moving toward research the public can really trust.
Ann Intern Med. 2007;146(6):450-453.
Ioannidis JP, Mulrow CD, Goodman SN. Adverse events: the more you search, the more you find. Ann Intern Med.
2006;144(4):298-300.
Laowattana S, Zeger SL, Lima JA, Goodman SN, Wittstein IS, Oppenheimer SM. Left insular stroke is associated with adverse
cardiac outcome. Neurology. 2006;66(4):477-483.
Burger I, Sugarman J, Goodman SN. Ethical issues in evidence-based surgery. Surg Clin North Am. 2006;86(1):151-168.
Goodman SN, Sladky JT. A Bayesian approach to randomized controlled trials in children utilizing information from adults: the
case of Guillain-Barré syndrome. Clin Trials. 2005;2(4):305-310.
Goodman SN. Introduction to Bayesian methods I: measuring the strength of evidence. Clin Trials. 2005;2(4):282-290.

About the Johns Hopkins University School of Medicine and Bloomberg School
of Public Health:
From the 1889 opening of the Johns Hopkins Hospital, to the opening of the School of Medicine four years later, there emerged the
concept of combining research, teaching and patient care. This model, the first of its kind, would lead to a national and international
reputation for excellence and discovery.
Today, Johns Hopkins uses one overarching name—Johns Hopkins Medicine—to identify its entire medical enterprise. This $5 billion
system unites the physicians and scientists of the Johns Hopkins University School of Medicine with the health professionals and
facilities that make up the broad, integrated Johns Hopkins Health System.
The Johns Hopkins Bloomberg School of Public Health is dedicated to the education of a diverse group of research scientists and
public health professionals, a process inseparably linked to the discovery and application of new knowledge, and through these
activities, to the improvement of health and prevention of disease and disability around the world.

Additional Information:
● The Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins: Baltimore, MD.
http://www.hopkinsmedicine.org/kimmel_cancer_center/
● Johns Hopkins University School of Medicine. http://www.hopkinsmedicine.org/
● Johns Hopkins Bloomberg School of Public Health. http://www.jhsph.edu/

Sources:
Appointments announced to Methodology Committee of the Patient-Centered Outcomes Research Institute (PCORI)
[press release]. Washington, DC: US Government Accountability Office; January 21, 2011.
http://www.gao.gov/press/pcori_2011jan21.html. Accessed January 30, 2011.
Steven Goodman. Johns Hopkins Bloomberg School of Public Health Faculty Web site.
http://faculty.jhsph.edu/default.cfm?id=289&Lastname=Goodman&Firstname=Steven. Accessed January 30, 2011.
Oncology Biostatistics: Biostatisticians: Steven Goodman, MD, MHS, PhD Oncology Biostatistics Web site.
http://www.cancerbiostats.onc.jhmi.edu/Goodman.cfm. Accessed February 1, 2011.
The PACE Initiative: Advisory Board. The PACE Initiative Web site. http://www.paceinitiative.org/about_board.html.
Accessed February 1, 2011.
21

Johns Hopkins epidemiologist named to Patient-Centered Outcomes Research Institute committee [press release]. Baltimore,
MD: Johns Hopkins Bloomberg School of Public Health; January 28, 2011.

Biography: Steven Goodman, MD, MHS, PhD


http://www.jhsph.edu/publichealthnews/articles/2011/goodman_pcori.html. Accessed February 1, 2011.
22

Mark Helfand, MD, MS, MPH

Professor of Medicine and of Medical Informatics and Clinical Epidemiology, Oregon Health

Biography: Mark Helfand, MD, MS, MPH


and Science University; Staff Physician, Portland VA Medical Center

Biography:
Mark Helfand, MD, MS, MPH, is the founder and director of the Oregon Evidence-based Practice Center, and also directs the
Veterans Affairs Evidence-based Synthesis Program and the Scientific Resource Center for the Agency for Healthcare Research and
Quality (AHRQ) Effective Healthcare Program. Dr. Helfand has been a member of the Institute of Medicine Committee on Standards
for Systematic Reviews of Comparative Effectiveness Research and the IOM Committee on Comparative Effectiveness Research
Prioritization. From 1998-2002, he led a team that helped the US Preventive Services Task Force prioritize topics and develop
evidence-based guidelines. Dr. Helfand is certified by the American Board of Internal Medicine and the American Board of Critical
Care Medicine. He is a health and medical consultant with Consumers Union Reports’ Best Buy Drugs Project and is editor-in-chief
of Medical Decision Making. Dr. Helfand received an AB and BS from Stanford University, an MD and MPH from the University of
Illinois Medical School, and an MS in health services research from Stanford University.

Selected Publications:
BroŽek JL, Akl EA, Compalati E, Kreis J, Terracciano L, Fiocchi A, Ueffing E, Andrews J, Alonso-Coello P, Meerpohl JJ, Lang DM,
Jaeschke R, Williams JW Jr, Phillips B, Lethaby A, Bossuyt P, Glasziou P, Helfand M, Watine J, Afilalo M, Welch V, Montedori A,
Abraha I, Horvath AR, Bousquet J, Guyatt GH, Schünemann HJ; for the GRADE Working Group. Grading quality of evidence and
strength of recommendations in clinical practice guidelines, Part 3 of 3. The GRADE approach to developing recommendations.
Allergy. January 17, 2010:1398-9995.
Balshem H, Helfand M, Schunemann HJ, Oxman AD, Kunz R, Brozek J, Vist GE, Falck-Ytter Y, Meerpohl J, Norris S, Guyatt GH.
GRADE guidelines: 3. Rating the quality of evidence. J Clin Epidemiol. January 5, 2011.
Kroenke K, Kapoor W, Helfand M, Meltzer DO, McDonald MA, Selker H. Training and career development for comparative
effectiveness research workforce development: CTSA Consortium Strategic Goal Committee on Comparative Effectiveness
Research Workgroup on Workforce Development. Clin Transl Sci. October 2010;3(5):258-262.
Sox HC, Helfand M, Grimshaw J, Dickersin K; PLoS Medicine Editors, Tovey D, Knottnerus JA, Tugwell P. Comparative
effectiveness research: challenges for medical journals. J Clin Epidemiol. August 2010;63(8):862-864.
Sox HC, Helfand M, Grimshaw J, Dickersin K, Tovey D, Knottnerus JA, Tugwell P. Comparative effectiveness research: challenges
for medical journals. Croat Med J. June 2010;51(3):191-194.
Sox HC, Helfand M, Grimshaw J, Dickersin K; PLoS Medicine Editors, Tovey D, Knottnerus JA, Tugwell P. Comparative
effectiveness research: challenges for medical journals. Am J Manag Care. May 1, 2010;16(5):e131-e133.
Helfand M, Balshem H. AHRQ series paper 2: principles for developing guidance: AHRQ and the effective health-care program.
J Clin Epidemiol. May 2010;63(5):484-490.
Whitlock EP, Lopez SA, Chang S, Helfand M, Eder M, Floyd N. AHRQ series paper 3: Identifying, selecting, and refining
topics for comparative effectiveness systematic reviews: AHRQ and the Effective Health-Care Program. J Clin Epidemiol. May
2010;63(5):491-501.
Sox HC, Helfand M, Grimshaw J, Dickersin K, Tovey D, Knottnerus JA, Tugwell P. Comparative effectiveness research: challenges
for medical journals. Med Decis Making. May-June 2010;30(3):301-303.
Sox HC, Helfand M, Grimshaw J, Dickersin K; PLoS Medicine Editors, Tovey D, Knottnerus JA, Tugwell P. Comparative
effectiveness research: challenges for medical journals. Trials. April 28, 2010;11:45.
23

Sox HC, Helfand M, Grimshaw J, Dickersin K; PLoS Medicine Editors, Tovey D, Knottnerus JA, Tugwell P. Comparative
effectiveness research: challenges for medical journals. PLoS Med. April 27, 2010;7(4):e1000269.
Helfand M, Aguilar-Gaxiola SA, Selker HP. Comparative effectiveness research: an approach to avoiding “overgeneralized

Biography: Mark Helfand, MD, MS, MPH


medicine.” Clin Transl Sci. December 2009;2(6):444-445.

About the Oregon Evidence-based Practice Center:


The Oregon Evidence-based Practice Center (Oregon EPC) conducts systematic reviews of health care topics for federal and state
agencies and private foundations. These reviews report the evidence from clinical research studies and the quality of that evidence
for use by policymakers in decisions on guidelines and coverage. The Center is one of 14 EPCs sponsored by the Agency for
Healthcare Research and Quality.
Investigators with the Evidence-based Practice Center have a particular interest in diagnostic technology assessment, prevention
effectiveness, evidence-based informatics, research in managed care, and critical appraisal of cost-effectiveness analysis and decision
analysis. In the past, faculty affiliated with the center have investigated areas such as acute head injury, pain management, drug
effectiveness, thyroid function tests, cancer screening, diagnostic use of upper gastrointestinal endoscopy, asthma diagnosis and
management, telemedicine, menopausal symptoms, osteoporosis, vaginal birth after cesarean section, and statewide
trauma systems.

Additional Information:
● Oregon Evidence-based Practice Center, Oregon Health and Science University.
http://www.ohsu.edu/xd/research/centers-institutes/evidence-based-practice-center/
● Evidence-based Synthesis Program. http://www.hsrd.research.va.gov/publications/esp

Sources:
AcademyHealth Methods Council members. Mark Helfand, MD, MPH, MS AcademyHealth Web site.
http://www.academyhealth.org/Training/ResourceDetail.cfm?ItemNumber=2428. Accessed January 30, 2011.
Oregon Evidence-based Practice Center, Oregon Health and Science University Web site.
http://www.ohsu.edu/xd/research/centers-institutes/evidence-based-practice-center/. Accessed January 30, 2011.
CMTP Policy Leadership Briefings on Comparative Effectiveness Research. Panelist Biographies. Center for Medical Technology
Policy Web site. http://www.cmtpnet.org/cmtp-research/what-is-cer-presentations/Panelist%20Bios.pdf.
Accessed January 21, 2011.
24

John Ioannidis, MD, DSc

C.F. Rehnborg Professor in Disease Prevention, Professor of Medicine and


Director, Stanford Prevention Research Center, Stanford University School of

Biography: John Ioannidis, MD, DSc


Medicine

Biography:
John Ioannidis, MD, DSc, is the C.F. Rehnborg Professor in Disease Prevention, professor of medicine and director of the Stanford
Prevention Research Center at the Stanford University School of Medicine. He is an adjunct professor at the Harvard School of
Public Health and Tufts University School of Medicine and a visiting professor at Imperial College London. He previously chaired
the Department of Hygiene and Epidemiology at the University of Ioannina School of Medicine in Greece. Since 2008, Dr. Ioannidis
has led the Genetics/Genomics component of the Tufts Clinical and Translational Science Institute (CTSI) and the Center for
Genetic Epidemiology and Modeling of the Tufts Institute for Clinical Research and Health Policy Studies at Tufts Medical Center.
Dr. Ioannidis serves on the executive board of the Human Genome Epidemiology Network and has served as president of the
Society for Research Synthesis Methodology. He has been a member of the editorial boards of 26 international journals, including
Lancet, Annals of Internal Medicine, PLoS Medicine, Journal of the National Cancer Institute, Journal of Clinical Epidemiology, and
Clinical Trials, and is editor-in-chief of the European Journal of Clinical Investigation. Much of Dr. Ioannidis’ own work involves
strengthening the way that research is planned, carried out and reported. Dr. Ioannidis graduated from Athens College and received
an MD and DSc in biopathology from the University of Athens School of Medicine.

Selected Publications:
Ioannidis JP, Castaldi P, Evangelou E. A compendium of genome-wide associations for cancer: critical synopsis and reappraisal.
Natl Cancer Inst. 2010;102(12):846-858.
Galvan A, Ioannidis JP, Dragani TA. Beyond genome-wide association studies: genetic heterogeneity and individual
predisposition to cancer. Trends Genet. 2010;26(3):132-141.
Salanti G, Dias S, Welton NJ, Ades AE, Golfinopoulos V, Kyrgiou M, Mauri D, Ioannidis JP. Evaluating novel agent effects in
multiple-treatments meta-regression. Stat Med. 2010;29(23):2369-2383.
Salanti G, Ades AE, Ioannidis JP. Graphical methods and numerical summaries for presenting results from multiple-treatment
meta-analysis: an overview and tutorial. J Clin Epidemiol. February 2011;64(2):163-171. Epub August 5, 2010.
Khoury MJ, Gwinn M, Ioannidis JP. The emergence of translational epidemiology: from scientific discovery to population health
impact. Am J Epidemiol. 2010;172(5):517-524.
Ioannidis JP, Karassa FB. The need to consider the wider agenda in systematic reviews and meta-analyses: breadth, timing, and
depth of the evidence. BMJ. September 13, 2010:341:c4875.
Ioannidis JP, Tatsioni A, Karassa FB. A vision for the European journal of clinical investigation: note from the new editors. Eur J
Clin Invest. 2010;40(1):1-3.
Kent DM, Rothwell PM, Ioannidis JP, Altman DG, Hayward RA. Assessing and reporting heterogeneity in treatment effects in
clinical trials: a proposal. Trials. August 12, 2010;11:85.
Tsianos GI, Evangelou E, Boot A, Zillikens MC, van Meurs JB, Uitterlinden AG, Ioannidis JP. Associations of polymorphisms
of eight muscle- or metabolism-related genes with performance in Mount Olympus marathon runners. J Appl Physiol.
2010;108(3):567-574.
25

Contopoulos-Ioannidis DG, Baltogianni MS, Ioannidis JP. Comparative effectiveness of medical interventions in adults versus
children. J Pediatr. 2010;157(2):322-330,e17.
Ioannidis JP. Why most published research findings are false. PLoS Med. 2005;2(8):e124.

Biography: John Ioannidis, MD, DSc


About Stanford Medicine:
Vast in both its physical scale and its impact on human health—locally, nationally, and globally—Stanford Medicine is composed of
three main components:
• Stanford School of Medicine, a premier research-intensive medical school that improves health through leadership and
collaborative discoveries and innovation in patient care, education, and research.
• Stanford Hospital and Clinics, consistently ranked among the top hospitals in the nation for advanced care in such areas as
cancer treatment, cardiac care, neurology, orthopedic surgery, and organ transplantation.
• Lucile Packard Children’s Hospital, internationally recognized for advancing family-centered care of children and
expectant mothers.
The Medical Center is located on the main Stanford University campus, adjacent to top-ranked programs in engineering, physical
and biological sciences, computer science, ethics, and other disciplines. This close proximity—coupled with Stanford’s long tradition
of encouraging collaboration—fosters multidisciplinary research and ultimately accelerates the pace at which new knowledge can be
translated into new ways to prevent, diagnose, and treat disease.

Additional Information:
● Stanford Medicine. http://stanfordmedicine.org

Sources:
Profile, John Ioannidis. Stanford School of Medicine Web site.
http://med.stanford.edu/profiles/medicine/researcher/John_Ioannidis/. Accessed January 30, 2011.
About us. Stanford Medicine Web site. http://stanfordmedicine.org/about/. Accessed January 30, 2011.
John Ioannidis, Adjunct Professor of Epidemiology. Harvard School of Public Health Web site.
http://www.hsph.harvard.edu/faculty/john-ioannidis/. Accessed January 30, 2011.
26

Michael Lauer, MD
NIH Designee to the Methodology Committee
Director, Division of Cardiovascular Sciences, National Heart, Lung, and Blood
Institute, National Institutes of Health

Biography: Michael Lauer, MD


Biography:
Michael Lauer, MD, is the director of the Division of Cardiovascular Sciences at the National Heart, Lung, and Blood Institute
(NHLBI), part of the National Institutes of Health (NIH). In this position, Dr. Lauer provides leadership for the Institute’s national
program for research on the causes, prevention, and treatment of cardiovascular (basic, clinical, population, and health sciences)
diseases. Dr. Lauer joined the NHLBI in July 2007.
Dr. Lauer’s primary research interests include cardiovascular clinical epidemiology and comparative effectiveness, with a focus on
diagnostic testing. He also has a strong background in leadership of the cardiovascular community and longstanding interests in
medical editing—for seven years he was a contributing editor for Journal of the American Medical Association—and human subjects
protection.
Prior to joining the NHLBI, Dr. Lauer served as the director of the Cleveland Clinic Foundation Exercise Laboratory and vice chair
of the clinic’s Institutional Review Board. He also served as co-director of the Coronary Intensive Care Unit and director of clinical
research in the clinic’s department of cardiology.
Dr. Lauer earned his Bachelor of Science degree in biology, summa cum laude, from Rensselaer Polytechnic Institute in 1983
and his Doctor of Medicine, magna cum laude, from Albany Medical College in 1985. Following internal medical training at the
Massachusetts General Hospital, Harvard Medical School, he completed a clinical fellowship in cardiology at the Boston Beth Israel
Hospital, Harvard Medical School. His further training in epidemiology included a research fellowship at the NHLBI’s Framingham
Heart Study, Boston University; the program in clinical effectiveness, Harvard School of Public Health, Harvard University; and the
Program for Physician Educators, Harvard Macy Institute.
Dr. Lauer is an elected fellow of the American College of Cardiology and American Heart Association, and has been elected to
membership in the American Society for Clinical Investigation. He also served as chairman of the Exercise, Cardiac Rehabilitation,
and Prevention Committee of the American Heart Association’s Council of Clinical Cardiology, and has received numerous awards in
recognition of his scientific and teaching accomplishments.

Selected Publications:
Lauer MS. Thought Exercises on Accountability and Performance Measures at the National Heart, Lung, and Blood Institute
(NHLBI): An Invited Commentary for Circulation Research. Circ Res. 2011 Feb 18;108(4):405-409.
Hsich E, Gorodeski EZ, Blackstone EH, Ishwaran H, Lauer MS. Identifying important risk factors for survival in patient with
systolic heart failure using random survival forests. Circ Cardiovasc Qual Outcomes. 2011 Jan 1;4(1):39-45. Epub November
23, 2010.
Lauer MS. The historical and moral imperatives of comparative effectiveness research. Stat Med. 2010 Aug 30;29(19):1982-
1984; discussion 1996-1997.
Lauer MS. Risk for venous thromboembolism in patients with superficial venous thrombosis. Ann Intern Med. 2010 Jul
6;153(1):62; author reply 63.
Lauer MS. Screening asymptomatic subjects for subclinical atherosclerosis: not so obvious. J Am Coll Cardiol. 2010 Jul
6;56(2):106-108.
27

Lauer MS, Collins FS. Using science to improve the nation’s health system: NIH’s commitment to comparative effectiveness
research. JAMA. 2010 Jun 2;303(21):2182-2183.
Lauer MS, Skarlatos S. Translational research for cardiovascular diseases at the National Heart, Lung, and Blood Institute:
moving from bench to bedside and from bedside to community. Circulation. 2010 Feb 23;121(7):929-933.
Hashkes PJ, Wright BM, Lauer MS, Worley SE, Tang AS, Roettcher PA, Bowyer SL. Mortality outcomes in pediatric rheumatology

Biography: Michael Lauer, MD


in the US. Arthritis Rheum. 2010 Feb;62(2):599-608.
Lauer MS. Elements of danger–the case of medical imaging. Minn Med. 2009 Dec;92(12):40-41.

About the National Heart, Lung and Blood Institute:


The National Heart, Lung, and Blood Institute (NHLBI) provides global leadership for a research, training, and education program to
promote the prevention and treatment of heart, lung, and blood diseases and enhance the health of all individuals so that they can
live longer and more fulfilling lives.
The NHLBI stimulates basic discoveries about the causes of disease, enables the translation of basic discoveries into clinical practice,
fosters training and mentoring of emerging scientists and physicians, and communicates research advances to the public. It creates
and supports a robust, collaborative research infrastructure in partnership with private and public organizations, including academic
institutions, industry, and other government agencies. The Institute collaborates with patients, families, health care professionals,
scientists, professional societies, patient advocacy groups, community organizations, and the media to promote the application of
research results and leverage resources to address public health needs. The NHLBI also collaborates with international organizations
to help reduce the burden of heart, lung, and blood diseases worldwide.

Additional Information:
● National Heart, Lung and Blood Institute. www.nhlbi.nih.gov

Sources:
National Heart, Lung and Blood Institute, National Institutes of Health Website.
http://public.nhlbi.nih.gov/Newsroom/home/ShowBio.aspx?PID=2724. Accessed February 23, 2011.
NHLBI Mission Statement. http://www.nhlbi.nih.gov/about/org/mission.htm. Accessed March 1, 2011.
28

David Meltzer, MD, PhD

Director, Center for Health and the Social Sciences, Chief of the Section
of Hospital Medicine, and Associate Professor, Department of Medicine,

Biography: David Meltzer, MD, PhD


Department of Economics, and Graduate School of Public Policy Studies,
University of Chicago

Biography:
David Meltzer, MD, PhD, is director of the Center for Health and the Social Sciences and chief of the Section of Hospital Medicine at
the University of Chicago. He is also an associate professor in the Department of Medicine, Department of Economics, and Graduate
School of Public Policy Studies at the University of Chicago. Dr. Meltzer also serves as co-director of the Program on Outcomes
Research Training and the MD/PhD program in the social sciences. In addition, he is on the faculty of the Graduate Program in
Health Administration and Policy, the Population Research Center, the Center on Aging, and the Hospital Medicine and Economic
Center for Education and Research in Therapeutics, which is funded by the Agency for Healthcare Research and Quality.
Dr. Meltzer’s research explores problems in health economics and public policy with a focus on the theoretical foundations of
medical cost-effectiveness analysis and the determinants of the cost and quality of care, especially in teaching hospitals. He is
currently completing a randomized trial comparing the use of doctors who specialize in inpatient care (“hospitalists”) with traditional
physicians in six academic medical centers.
Dr. Meltzer is the recipient of numerous awards, including the National Institute of Health Medical Scientist Training Program
Fellowship, the National Science Foundation Graduate Fellowship in Economics, the University of Chicago Searle Fellowship, the
Lee Lusted Prize of the Society for Medical Decision Making, the Health Care Research Award of the National Institute for Health
Care Management, the Eugene Garfield Award from Research!America, and the Robert Wood Johnson Generalist Physician Award.
Dr. Meltzer is a research associate of the National Bureau of Economic Research, an elected member of the American Society for
Clinical Investigation, and past president of the Society for Medical Decision Making. He has served on panels examining the future
of Medicare for the National Academy of Social Insurance and the Department of Health and Human Services (HHS), and US
organ allocation policy for the Institute of Medicine (IOM). He recently served on an IOM panel examining the effectiveness of the
US drug safety system and currently serves on the HHS Secretary’s Advisory Committee on Healthy People 2020, which aims to
establish health objectives for the US population.

Selected Publications:
Huang E, Shook M, Jin L, Chin M, Meltzer D. The impact of patient preferences on the cost-effectiveness of intensive glucose
control in older patients with new onset diabetes. Diabetes Care. 2006;29:259-264.
Basu A, Meltzer D. Implications of spillover effects within the family for medical cost-effectiveness analysis. J Health Econ.
2005;24:751-773.
Manns B, Meltzer D, Taub K, Donaldson C. Illustrating the impact of including future costs in economic evaluations: an
application to end-stage renal disease care. Health Econ. 2003;12(11):949-958.
Meltzer D, Manning W, Morrison J, Shah M, Jin L, Guth T, Levinson W. Effects of physician experience on costs and outcomes
on an academic general medicine service: results of a trial of hospitalists. Ann Intern Med. 2002;137:866-874.
Meltzer, D. Addressing uncertainty in medical cost-effectiveness analysis: implications of expected utility maximization for
methods to perform sensitivity analysis and the use of cost-effectiveness analysis to set priorities for medical research. J Health
Econ. 2001;20(1):109-129.
Meltzer, D. Accounting for future costs in medical cost-effectiveness analysis. J Health Econ. 1997;16(1):33-64.
29

About the University of Chicago Center for Health and the Social Sciences:
The University of Chicago Center for Health and the Social Sciences provides needed infrastructure to support and encourage new
research initiatives at the interface of health and the social sciences. Such infrastructure includes: coordination of collaborative efforts
in research and training; shared space; grant support and fundraising; and computer, data, and focused methodological support.

Biography: David Meltzer, MD, PhD


The center also provides a venue to promote interdisciplinary collaboration for research and training at the university. The center
complements the resources of existing centers, functioning not only as an independent center in the usual sense, but also as a
collaborator with existing centers. The center aims to facilitate the development of additional new centers in focused related areas
as faculty interest and funding opportunities arise. This is to be accomplished partially through the creation of program areas that
organize faculty interested in specific topic areas. Ultimately, this arrangement will allow the center to act as an incubator to facilitate
the further growth of innovative research in health and the social sciences both within and across the units of the university.

Additional Information:
● The University of Chicago Center for Health and the Social Sciences. http://chess.uchicago.edu/index.html
● Centers for Education and Research on Therapeutics (CERTs), University of Chicago. http://chess.uchicago.edu/cert/cert.html
● The Harris School of Public Policy Studies, University of Chicago. http://harrisschool.uchicago.edu/
● The University of Chicago. http://www.uchicago.edu/index.shtml

Sources:
Appointments Announced to Methodology Committee of the Patient-Centered Outcomes Research Institute (PCORI) [press
release]. Washington, DC: US Government Accountability Office; January 21, 2011.
http://www.gao.gov/press/pcori_2011jan21.html. Accessed January 30, 2011.
Faculty: David O. Meltzer, MD, PhD. The Harris School, University of Chicago, Faculty Web site.
http://harrisschool.uchicago.edu/faculty/web-pages/david-meltzer.asp. Accessed January 30, 2011.
Physician Faculty Scholars: David O. Meltzer, MD, PhD. Robert Wood Johnson Foundation Web site.
http://rwjfpfsp.stanford.edu/nac/meltzer.html. Accessed January 30, 2011.
About CHeSS. The University of Chicago Center for Health and the Social Sciences Web site.
http://chess.uchicago.edu/about.html. Accessed January 30, 2011.
30

Brian Mittman, PhD

Director, VA Center for Implementation Practice and Research Support,


Department of Veterans Affairs Greater Los Angeles Healthcare System

Biography: Brian Mittman, PhD


Biography:
Brian Mittman, PhD, is the director of the Veterans Affairs (VA) Center for Implementation Practice and Research Support and
senior social scientist at the VA/UCLA/RAND Center for the Study of Healthcare Provider Behavior, both at the VA Greater Los
Angeles Healthcare System. From 2002 to 2004 he served as interim associate director of the VA’s Health Services Research and
Development Service, directing the VA Quality Enhancement Research Initiative (QUERI). Dr. Mittman served as a visiting professor
in the Department of Health Services, UCLA School of Public Health, from 2003 to 2006, and taught at the UCLA Anderson
Graduate School of Management (visiting lecturer to visiting associate professor) from 1986 to 1993.
Dr. Mittman’s research interests include implementation science, health care quality improvement, and health care management.
He convened and chaired the planning committee that launched the journal Implementation Science and serves as co-editor-in-
chief of the journal. He was a founding member of the Institute of Medicine Forum on the Science of Quality Improvement and
Implementation (2006–2008) and is a consultant and member of the editorial board for the Agency for Healthcare Research and
Quality (AHRQ) Health Care Innovations Exchange. This initiative classifies innovative strategies to increase implementation of
evidence-based clinical practices and enhance the efficiency and effectiveness of health care delivery.
Dr. Mittman served on the National Institutes of Health (NIH) review committee (Special Emphasis Panel) on Dissemination and
Implementation Research in Health in 2006 and chaired the panel in March and October of 2007. His published research appears
in the Journal of the American Medical Association, Annals of Internal Medicine, Medical Care, Health Services Research, and other
journals. He is a frequent speaker on implementation research.

Selected Publications:
Yano EM, Rubenstein LV, Farmer MM, Chernof BA, Mittman BS, Lanto AB, Simon BF, Lee ML, Sherman SE. Targeting primary
care referrals to smoking cessation clinics does not improve quit rates: implementing evidence-based interventions into
practice. Health Serv Res. 2008;43(5p1):1637-1661.
Stetler CB, McQueen L, Demakis J, Mittman BS. An organizational framework and strategic implementation for system-level
change to enhance research-based practice: QUERI Series. Implement Sci. May 29, 2008;3:30.
Stetler CB, Mittman BS, Francis J. Overview of the VA Quality Enhancement Research Initiative (QUERI) and QUERI theme
articles: QUERI series. Implement Sci. February 15, 2008;3:8.
Chodosh J, Mittman BS, Connor KI, Vassar SD, Lee ML, DeMonte RW, Ganiats TG, Heikoff LE, Rubenstein LZ, Della Penna RD,
Vickrey BG. Caring for patients with dementia: how good is the quality of care? Results from three health systems. J Am Geriatr
Soc. August 2007;55(8):1260-1268.
Vickrey BG, Mittman BS, Connor KI, Pearson ML, Della Penna RD, Ganiats TG, Demonte RW Jr, Chodosh J, Cui X, Vassar S,
Duan N, Lee M. The effect of a disease management intervention on quality and outcomes of dementia care: a randomized,
controlled trial. Ann Intern Med. November 21, 2006;145(10):713-726.
McQueen L, Mittman BS, Demakis JG. Overview of the Veterans Health Administration (VHA) Quality Enhancement Research
Initiative (QUERI). J Am Med Inform Assoc. September-October 2004;11(5):339-343. Epub June 7, 2004.
31

Mittman BS. Creating the evidence base for quality improvement collaboratives. Ann Intern Med. June 1,2004;140(11):897-901.
Liu H, Harker JO, Wong AL, Maclean CH, Bulpitt KJ, Mittman BS, Fitzgerald J, Grossman J, Rubenstein LZ, Hahn B, Paulus
HE, Kahn KL. Case finding for population-based studies of rheumatoid arthritis: comparison of patient self-reported ACR
criteria-based algorithms to physician-implicit review for diagnosis of rheumatoid arthritis. Semin Arthritis Rheum. April
2004;33(5):302-310.

Biography: Brian Mittman, PhD


Switzer GE, Halm EA, Chang CC, Mittman BS, Walsh MB, Fine MJ. Physician awareness and self-reported use of local and
national guidelines for community-acquired pneumonia. J Gen Intern Med. October 2003;18(10):816-823.
Berlowitz DR, Young GJ, Hickey EC, Saliba D, Mittman BS, Czarnowski E, Simon B, Anderson JJ, Ash AS, Rubenstein LV,
Moskowitz MA. Quality improvement implementation in the nursing home. Health Serv Res. February 2003;38(1)(pt 1):65-83.

About the VA Greater Los Angeles Healthcare System:


The VA Greater Los Angeles (GLA) Healthcare System is the largest, most complex health care system within the Department of
Veterans Affairs. It is one component of the VA Desert Pacific Healthcare Network (VISN22) offering services to veterans residing in
Southern California and Southern Nevada. GLA consists of three ambulatory care centers, a tertiary care facility and 10 community
based outpatient clinics. GLA serves veterans residing throughout five counties: Los Angeles, Ventura, Kern, Santa Barbara, and
San Luis Obispo. There are 1.4 million veterans in the GLA service area. GLA is affiliated with both UCLA School of Medicine and
University of Southern California School of Medicine, as well as more than 45 colleges, universities and vocational schools in 17
different medical, nursing, paramedical and administrative programs.

Additional Information:
● VA Greater Los Angeles Healthcare System. http://www.losangeles.va.gov/
● US Department of Veterans Affairs. http://www.va.gov/landing2_about.htm
● Implementation Science. www.ImplementationScience.com

Sources:
Meet Our Experts: Brian Mittman, PhD. Improvement Science Research Network Web site.
http://www.improvementscienceresearch.net/about/experts/mittman.asp. Accessed January 30, 2011.
About the VA Greater Los Angeles Healthcare System. VA Greater Los Angeles Healthcare System Web site.
http://www.losangeles.va.gov/about/. Accessed January 30, 2011.
32

Robin Newhouse, PhD, RN

Assistant Dean, Doctor of Nursing Practice Program and Associate Professor,


Organizational Systems and Adult Health, University of Maryland School

Biography: Robin Newhouse, PhD, RN


of Nursing

Biography:
Robin Newhouse, PhD, RN, is assistant dean for the Doctor of Nursing Practice Program and an associate professor for
organizational systems and adult health at the University of Maryland School of Nursing. She conducts randomized controlled trials
and systematic reviews with research focusing on quality of care, evidence-based practice among clinicians, and outcomes research
in health care delivery systems. Dr. Newhouse has served as chair of the Johns Hopkins Nursing Evidence Based Practice Model and
implemented a strategic plan for evidence-based practice and research at the Johns Hopkins Hospital. She is a peer reviewer for
the Journal of Nursing Administration and for the Journal of Nursing Scholarship. Dr. Newhouse received her BS and her PhD in
nursing from the University of Maryland School of Nursing.

Selected Publications:
Newhouse, RP. Instruments to assess organizational readiness for evidence-based practice. J Nurs Adm. 2010;40(10):404-407.
Newhouse, RP. Do we know how much the evidence-based intervention cost? J Nurs Adm. 2010;40(7/8):296-299.
Satterfield J, Spring B, Brownson RC, Mullen E, Newhouse R, Walker B, Whitlock E. Toward a transdisciplinary model of
evidence-based practice. Milbank Q. 2009;87(2):368-390.
Newhouse RP, Morlock L, Pronovost P, Colantuoni E, Johantgen M. Rural hospital nursing: better environments = higher
shared vision and higher quality and safety engagement. J Nurs Adm. 2009;39(4):189-195.
Newhouse RP. Agency for Healthcare Research and Quality Health Care Innovations Exchange. J Nurs Adm. 2009;39(3):103-105.
Newhouse RP. Evidence-based behavioral practice: an exemplar of interprofessional collaboration. J Nurs Adm.
2008;38(10):414-416.
Newhouse RP. Evidence driving quality initiatives: the Maryland Hospital Association collaborative on nurse retention.
J Nurs Adm. 2008;38(6):268-271.

About the University of Maryland School of Nursing:


The University of Maryland School of Nursing shares a 61-acre campus in downtown Baltimore with the University of Maryland,
Baltimore’s (UMB) six other professional schools—Dentistry, Law, Medicine, Pharmacy, Social Work, and the Graduate School—and
is in close proximity to the University of Maryland Medical Center, University of Maryland Biotechnology Institute, University of
Maryland Biopark, and the Baltimore VA Medical Center. The School of Nursing also maintains facilities and conducts classes at the
University System of Maryland regional center at Shady Grove in Rockville, Maryland. The School is dedicated to creating a research-
intensive environment that will advance the science of nursing through research and scholarship of the highest quality.

Additional Information:
● University of Maryland School of Nursing. http://nursing.umaryland.edu/
33

Sources:
Appointments Announced to Methodology Committee of the Patient-Centered Outcomes Research Institute (PCORI)
[press release]. Washington, DC: US Government Accountability Office; January 21, 2011.

Biography: Robin Newhouse, PhD, RN


http://www.gao.gov/press/pcori_2011jan21.html. Accessed January 30, 2011.
About Us. University of Maryland School of Nursing Web site. http://nursing.umaryland.edu/about. Accessed January 30, 2011.
34

Sharon-Lise Normand, MSc, PhD


Methodology Committee Vice Chair

Biography: Sharon-Lise Normand, MSc, PhD


Professor of Health Care Policy, Harvard Medical School; Professor of
Biostatistics, Harvard School of Public Health

Biography:
Sharon-Lise T. Normand, PhD, is a professor of health care policy (biostatistics) in the Department of Health Care Policy at Harvard
Medical School and in the Department of Biostatistics at the Harvard School of Public Health. Dr. Normand’s research focuses on the
development of statistical methods for health services research, primarily using Bayesian approaches to problem solving, including
assessment of quality of care, methods for causal inference, provider profiling, meta-analysis, and latent variable modeling. She
has developed a long line of research on methods for the analysis of patterns of treatment and quality of care for patients with
cardiovascular disease and with mental disorders in particular.
Dr. Normand earned her BSc and MSc degrees in statistics from the University of Western Ontario and her PhD in biostatistics from
the University of Toronto. She is a fellow of the American Statistical Association, a fellow of the American College of Cardiology,
and an associate member of the Society of Thoracic Surgeons. She was president of the Eastern North American Region of
the International Biometrics Society in 2010; serves on task forces for the American Heart Association, the American College
of Cardiology, and the Society of Thoracic Surgeons; and is currently a member of two Institute of Medicine Committees: the
Committee on Aerospace Medicine and the Medicine of Extreme Environments; and the Committee on Future Directions for the
National Healthcare Quality and Disparities Reports.

Selected Publications:
Ross JS, Maynard C, Krumholz HM, Sun H, Rumsfeld JS, Normand SL, Wang Y, Fihn SD. Use of administrative claims models to
assess 30-day mortality among veterans’ health administration hospitals. Med Care. July 2010;48(7):652-658.
Bueno H, Ross JS, Wang Y, Chen J, Vidán W, Normand SL, Curtis JP, Drye EE, Lichtman J, Keenan PS, Kosiborod M, Krumholtz HM.
Trends in length of stay and short-term outcomes among Medicare patients hospitalized for heart failure, 1993-2006. JAMA. June 2,
2010;303(21):2141-2147.
Ross JS, Normand SL, Wang Y, Ko DT, Chen J, Drye EE, Keenan PS, Lichtman JH, Bueno H, Schreiner GC, Krumholz, HM. Hospital
volume and 30-day mortality for three common medical admissions. N Engl J Med. March 25, 2010;362(12):1110-1118.
Chen J, Krumholz HM, Wang Y, Curtis JP, Rathore SS, Ross JS, Normand SL, Schreiner G, Mulvey G, Nallamothu BK. Differences
in patient survival after acute myocardial infarction by hospital capability of performing percutaneous coronary intervention:
implications for regionalization. Arch Intern Med. March 8, 2010;170(5):433-439.
Shahian DM, O’Brien SM, Normand SL, Peterson ED, Edwards FH. Association of hospital coronary artery bypass volume with
processes of care, mortality, morbidity, and the Society of Thoracic Surgeons composite quality score. J Thorac Cardiovasc Surg.
February 2010;139(2):273-282.
Castellanos LR, Normand SL, Ayanian JZ. Racial and ethnic disparities in access to higher and lower quality cardiac surgeons for
coronary artery bypass grafting. Amer J Cardiol. June 15, 2009; 103(12):1682-1686. Epub April 16, 2009.
Krumholz HM, Wang Y, Chen J, Drye EE, Radford MJ, Havranek DP, Masoudi FA, Nallamothu BK, Spertus JA, Ross JS, Lichtman JH,
Curtis JP, Han LF, Rapp MT, Straube BM, Normand S-LT. Reduction in acute myocardial infarction mortality in the United States: risk-
standardized mortality rates from 1995-2006. JAMA. August 19, 2009;302(7):767-773.
35

Mulvey GK, Wang Y, Lin Z, Wang OJ, Chen J, Keenan PS, Drye EE, Rathore SS, Normand SL, Krumholz HM. Mortality and
readmission for patients with heart failure among U.S. News & World Report’s top heart hospitals. Circ Cardiovasc Qual Outcomes.

Biography: Sharon-Lise Normand, MSc, PhD


November 2009;2(6):558-565.

About the Department of Health Care Policy at Harvard Medical School:


The Department of Health Care Policy at Harvard Medical School is one of only a few academic departments of health policy
nationwide located in a medical school. Research includes broad topics on financing and delivery of health care, quality of care,
studies on special and disadvantaged populations (including those with mental disorders), and access to care.

About the Harvard School of Public Health:


The Harvard School of Public Health is dedicated to advancing the public’s health through learning, discovery, and communication.
More than 400 faculty members are engaged in teaching and training the 1,000-plus student body in a broad spectrum of
disciplines crucial to the health and well being of individuals and populations around the world. Programs and projects range from
the molecular biology of AIDS vaccines to the epidemiology of cancer; from risk analysis to violence prevention; from maternal and
children’s health to quality of care measurement; and from health care management to international health and human rights.

Additional Information:
● Department of Health Care Policy, Harvard Medical School. http://www.hcp.med.harvard.edu/about
● Harvard School of Public Health. http://www.hsph.harvard.edu/

Sources:
About HCP. Department of Health Care Policy, Harvard Medical School Web site. http://www.hcp.med.harvard.edu/about.
Accessed January 30, 2011.
Sharon-Lise T. Normand, PhD. Department of Health Care Policy, Harvard Medical School Web site.
http://www.hcp.med.harvard.edu/people/hcp_core_faculty/sharon-lise_normand. Accessed January 30, 2011.
Exercise may lower risk of death for men with prostate cancer (press release). Boston, MA: Harvard School of Public Health;
January 5, 2011. http://www.hsph.harvard.edu/news/press-releases/2011-releases/exercise-prostate-cancer-death.html.
Accessed January 30, 2011.
36

Sebastian Schneeweiss, MD, ScD

Biography: Sebastian Schneeweiss, MD, ScD


Associate Professor, Department of Medicine, Harvard Medical School;
Associate Professor, Department of Epidemiology, Harvard School of Public
Health; Vice Chief and Director, Drug Evaluation and Outcomes Research,
Division of Pharmacoepidemiology and Pharmacoeconomics, Brigham and
Women’s Hospital

Biography:
Sebastian Schneeweiss, MD, ScD, is an associate professor in the Department of Medicine at Harvard Medical School and in the
Department of Epidemiology at the Harvard School of Public Health. He also serves as vice chief and director of drug evaluation and
outcomes research for the Division of Pharmacoepidemiology and Pharmacoeconomics at Brigham and Women’s Hospital (BWH).
In addition, Dr. Schneeweiss is the director and chair of the Executive Board of the DEcIDE Research Center (Developing Evidence to
Inform Decisions about Effectiveness) at Brigham and Women’s Hospital and of the DEcIDE Methods Center. His research interests
include pharmacoepidemiology and pharmaceutical outcomes research, with particular interest in the comparative safety and
effectiveness of pharmaceuticals and biotech products, drug policy and risk management program evaluation, and epidemiologic
methods using electronic health care databases. An important part of Dr. Schneeweiss’s teaching is the development and testing
of new pharmacoepidemiologic methods that rely on large computerized claims databases. Dr. Schneeweiss received a BA from
Gymnasium Icking in Germany, an MD from Ludwig-Maximilians-University Medical School, and an ScD in Epidemiology from
Harvard School of Public Health. 

Selected Publications:
Brookhart MA, Rassen JA, Schneeweiss S. Instrumental variable methods in comparative safety and effectiveness research.
Pharmacoepidemiol Drug Saf. June 2010;19(6):537-554.
Brookhart MA, Stürmer T, Glynn RJ, Rassen J, Schneeweiss S. Confounding control in healthcare database research: challenges
and potential approaches. Med Care. June 2010;48(6)(suppl):S114-S120.
Rassen JA, Solomon DH, Curtis JR, Herrinton L, Schneeweiss S. Privacy-maintaining propensity score-based pooling of multiple
databases applied to a study of biologics. Med Care. June 2010;48(6)(suppl):S83-S89.
Dreyer NA, Schneeweiss S, McNeil BJ, Berger ML, Walker AM, Ollendorf DA, Gliklich RE. GRACE principles: recognizing high-
quality observational studies of comparative effectiveness. Am J Manag Care. June 2010;16(6):467-471.
Schneeweiss S. A basic study design for expedited safety signal evaluation based on electronic healthcare data.
Pharmacoepidemiol Drug Saf. 2010;19 (8):858-868. Epub ahead of print May 19, 2010.
Schneeweiss S, Patrick AR, Solomon DH, Metha J, Dormuth C, Miller M, Lee J, Wang PS. Variation in the risk of suicide attempts
and completed suicides by antidepressant agent in adults: A propensity score-adjusted analysis of 9 years of data. Arch Gen
Psychiatry. 2010;67:497-506.
Patorno E, Bohn RL, Wahl PM, Avorn J, Patrick AR, Liu J, Schneeweiss S. Anticonvulsant medications and the risk of suicide,
attempted suicide, or violent death. JAMA. 2010;303:1401-1409.
Schneeweiss S, Patrick AR, Solomon DH, Metha J, Dormuth C, Miller M, Lee J, Wang PS. The comparative safety of
antidepressant agents in children regarding suicidal acts. Pediatrics. 2010;125:876–888.
Rassen JA, Choudhry N, Avorn J, Schneeweiss S. Cardiovascular outcomes and mortality in patients using clopidogrel with
proton pump inhibitors after percutaneous coronary intervention. Circulation. 2009;120:2322-2329.
37

Schneeweiss S, Avorn J. Antipsychotic agents and sudden cardiac death—how to manage the risk. N Engl J Med.
2009;360:294-296.

Biography: Sebastian Schneeweiss, MD, ScD


Schneeweiss S, Seeger CD, Landon J, Walker AM. Aprotinin during coronary-artery bypass grafting and risk of death. N Engl J
Med. 2008;358:771-783.
Brookhart MA, Schneeweiss S. Preference-based instrumental variable methods for the estimation of treatment effects:
assessing validity and interpreting results. Int J Biostat. 2007;3:14.
Schneeweiss S, Patrick AR, Stürmer T, Brookhart MA, Avorn J, Maclure M, Rothman K, Glynn RJ. Increasing levels of
restriction in pharmacoepidemiologic database studies of elderly and comparison with randomized trial results. Med Care.
2007;45:S131-S142.
Schneeweiss S. Developments in post-marketing comparative effectiveness research. Clin Pharm Ther. 2007;82:143-156.

About the Harvard School of Public Health:


The Harvard School of Public Health is dedicated to advancing the public’s health through learning, discovery, and communication.
More than 400 faculty members are engaged in teaching and training the 1,000-plus student body in a broad spectrum of
disciplines crucial to the health and well being of individuals and populations around the world. Programs and projects range from
the molecular biology of AIDS vaccines to the epidemiology of cancer; from risk analysis to violence prevention; from maternal and
children’s health to quality of care measurement; and from health care management to international health and human rights.

About Brigham and Women’s Hospital:


Recognized internationally for its excellence in patient care, its outstanding reputation in biomedical research, and its commitment
to educating and training physicians, research scientists and other health care professionals, Brigham and Women’s Hospital (BWH)
is a 793-bed teaching affiliate of Harvard Medical School located in the heart of Boston’s renowned Longwood Medical Area. Along
with its modern inpatient facilities, BWH boasts extensive outpatient services and clinics, neighborhood primary care health centers,
state-of-the art diagnostic and treatment technologies and research laboratories.

Additional Information:
● Harvard School of Public Health. http://www.hsph.harvard.edu
● Brigham and Women’s Hospital. http://www.brighamandwomens.org

Sources:
About HSPH. Harvard School of Public Health Web site. http://www.hsph.harvard.edu/about/. Accessed January 30, 2011.
Publications: Sebastian Schneeweiss. Department of Epidemiology, Harvard School of Public Health Web site.
http://www.hsph.harvard.edu/faculty/sebastian-schneeweiss/publications/. Accessed January 30, 2011.
Harvard Catalyst Profiles: Sebastian Schneeweiss, SD, MD, Harvard Catalyst Web site.
http://connects.catalyst.harvard.edu/profiles/profile/person/64475. Accessed January 30, 2011.
Faculty: Sebastian Schneeweiss, MD, ScD, Division of Pharmacoepidemiology and Pharmacoeconomics, Brigham and Women’s
Hospital, Department of Medicine Web site. http://www.drugepi.org/staff/faculty/faculty_schneeweiss.php.
Accessed January 30, 2011.
About BWH. Brigham and Women’s Hospital Web site. http://www.brighamandwomens.org/about_bwh/about_us.aspx.
Accessed January 30, 2011.
38

Mary Tinetti, MD

Professor of Medicine, Epidemiology, and Public Health, Division of Geriatrics,


Yale University School of Medicine; Director, Program on Aging, Yale University
School of Medicine

Biography: Mary Tinetti, MD


Biography:
Mary Tinetti, MD, is a professor of medicine, epidemiology and public health in the Division of Geriatrics at Yale University School
of Medicine. She also serves as director for both the Program on Aging and the Hartford Center of Excellence in Aging at Yale
University School of Medicine.
Dr. Tinetti has conducted extensive research in the field of aging and geriatrics and served as chair of the National Institute on Aging
(NIA) Advisory Council review of the NIA Gerontology and Clinical Geriatrics Program. Dr. Tinetti has been elected to the Institute of
Medicine and was named a 2009 MacArthur Foundation Fellow for her work in the area of morbidity due to falls by elderly people.
Dr. Tinetti’s research focus includes the health effects of multiple chronic conditions, the relative and absolute benefits versus risks
of comparative treatments, morbidity due to falling, and cross-disease health outcomes and measurement for older adults. She has
conducted pioneering work in diagnosis, treatment and outcomes of elderly patients.
Dr. Tinetti received a BA from the University of Michigan at Ann Arbor and an MD from the University of Michigan Medical School.

Selected Publications:
Chang SS, Vaz Fragoso CA, Van Ness PH, Fried LP, Tinetti ME. Association between combined interleukin-6 and c-reactive
protein levels and pulmonary function in older women: results from the women’s health and aging studies I and II. Am Geriatr
Soc. January 2011;59(1):113-119.
Fried TR, Tinetti ME, Iannone L. Primary care clinicians’ experiences with treatment decision making for older persons with
multiple conditions. Arch Intern Med. January 10, 2011;171(1):75-80.
Tinetti ME. Making prevention recommendations relevant for an aging population. Ann Intern Med. December 21,
2010;153(12):843-844.
Tinetti ME, Kumar C. The patient who falls: “It’s always a trade-off.” JAMA. January 20, 2010;303(3):258-266.
Mendu ML, McAvay G, Lampert R, Stoehr J, Tinetti ME. Yield of diagnostic tests in evaluating syncopal episodes in older
patients. Arch Intern Med. July 27, 2009;169(14):1299-1305.
Inouye SK, Brown CJ, Tinetti ME. Medicare nonpayment, hospital falls, and unintended consequences. N Engl J Med. June 4,
2009;360(23):2390-2393.
Tinetti ME, McAvay G, Fried TR, Allore HG, Salmon J, Foody JM, Bianco L, Ginter S, Fraenkel L. Variable priorities in the face
of competing outcomes: the tradeoff among cardiovascular events, medication symptoms, and fall injuries. J Am Geriatr Soc.
2008;56:1409-1416.
Tinetti ME, Baker DI, King M, Gottschalk M, Murphy T, Acampora D, Leo-Summers L, Allore H. Effect of translating evidence into
practice on rate of serious fall injury. New Engl J Med. 2008;359:252-261.
Tinetti ME. Over-the-counter sales of statins and other drugs for asymptomatic conditions. New Engl J Med. 2008;358:2728-2732.
Belcher V, Fried TR, Agostini JV, Tinetti ME: Views from older adults on patient participation in medication-related decision-
making. J Gen Intern Med. 2006;21:298-303.
39

Tinetti ME, Gordon C, Sogolow E, Lapin P, Bradley E. Fall prevention: A case study of the challenges in adopting evidence-based
geriatric care practices. Gerontologist. 2006;46:717-725.

About the Yale University School of Medicine:


Located in New Haven, Connecticut, the Yale School of Medicine is a world-renowned leader in biomedical research, education and

Biography: Mary Tinetti, MD


advanced health care and is one of the nation’s oldest and largest schools of medicine and public health. It is preeminent in medical
studies and has long been regarded as one of the world’s foremost medical institutions.
Yale School of Medicine consists of 28 departments, is one of 41 comprehensive cancer centers designated by the National Cancer
Institute, and is a member of the Association of American Medical Colleges (AAMC) and the Association of Academic Health Centers
(AAHC). Yale is a leading recipient of research funding from the National Institutes of Health.

Additional Information:
● Yale University School of Medicine. http://medicine.yale.edu
● Yale University School of Medicine Program on Aging. http://medicine.yale.edu/intmed/geriatrics/research/index.aspx

Sources:
Mary Elizabeth Tinetti, MD. Internal Medicine Faculty, Yale School of Medicine Web site.
http://medicine.yale.edu/intmed/people/mary_tinetti-3.profile. Accessed January 30, 2011.
2009 MacArthur Fellows: Mary Tinetti. The John D. and Catherine T. MacArthur Foundation Web site.
http://www.macfound.org/site/c.lkLXJ8MQKrH/b.5458043/k.936A/Mary_Tinetti.htm. Accessed January 30, 2011.
Mary Tinetti publications search results. National Center for Biotechnology Information, US National Library of Medicine,
PubMed Web site. http://www.ncbi.nlm.nih.gov/sites/entrez?db=pubmed&cmd=DetailsSearch&term=mary+tinetti.
Accessed January 30, 2011.
About YSM. Yale School of Medicine Web site. http://medicine.yale.edu/about/index.aspx. Accessed January 30, 2011.
Appointments announced to Methodology Committee of the Patient-Centered Outcomes Research Institute (PCORI) [press
release]. Washington, DC: US Government Accountability Office; January 21, 2011.
http://www.gao.gov/press/pcori_2011jan21.html. Accessed January 30, 2011.
40

Clyde Yancy, MD, MSc

Chief, Cardiology, Northwestern University Feinberg School of Medicine;


Associate Director, The Bluhm Cardiovascular Institute, Northwestern Memorial
Hospital

Biography: Clyde Yancy, MD, MSc


Biography:
Clyde Yancy, MD, MSc, was recently appointed Magerstadt professor and chief of cardiology at the Northwestern University Feinberg
School of Medicine. He is also currently serving as the associate director of the Bluhm Cardiovascular Institute at Northwestern
Memorial Hospital.
During the 2009-2010 fiscal year, Dr. Yancy served as president of the American Heart Association (AHA), and prior to his position
at Northwestern he was the medical director of the Baylor Heart and Vascular Institute and chief of cardiothoracic transplantation at
Baylor University Medical Center.
Dr. Yancy is a fellow of the American College of Cardiology (ACC) and the American College of Physicians and is a member of
the ACC/AHA guideline writing committee for chronic heart failure. Dr. Yancy has served on the executive committee for the Heart
Failure Society of America and has also served as the chair of the Food and Drug Administration’s cardiovascular device panel.
Dr. Yancy’s work has focused on areas including hypertension, heart failure and heart transplantation, and ethnic and racial
disparities in cardiovascular disease. He has authored or co-authored more than 200 papers and has been honored by his
colleagues through multiple awards including the “Cardiologists-In-Training” hero award from the National Association of Black
Cardiologists, Inc, in 2006 and American Heart Association’s “Physician of the Year” award in 2003.
Dr. Yancy earned his medical degree from Tulane University School of Medicine in 1982 and completed an internship and residency
in internal medicine at Parkland Memorial Hospital in Dallas, Texas in 1985. He completed a fellowship in cardiology at the
University of Texas Southwestern Medical Center and subsequently led the institution’s heart-transplant and heart-failure programs.

Selected Publications:
Ambardekar AV, Fonarow GC, Hernandez AF, Pan W, Yancy CW, Krantz MJ. Characteristics and in-hospital outcomes for
nonadherent patients with heart failure: findings from Get With The Guidelines-Heart Failure (GWTG-HF). Get With the
Guidelines Steering Committee and Hospitals. Am Heart J. October, 2009;158(4):644-652.
Albert NM, Fonarow GC, Yancy CW, Curtis AB, Stough WG, Gheorghiade M, Heywood JT, McBride M, Mehra MR, O’Connor
CM, Reynolds D, Walsh MN. Outpatient cardiology practices with advanced practice nurses and physician assistants provide
similar delivery of recommended therapies (findings from IMPROVE HF). Am J Cardiol. June 15, 2010;105(12):1773-1779.
Albert NM, Fonarow GC, Yancy CW, Curtis AB, Stough WG, Gheorghiade M, Heywood JT, McBride M, Mehra MR, O’Connor
CM, Reynolds D, Walsh MN. Influence of dedicated heart failure clinics on delivery of recommended therapies in outpatient
cardiology practices: findings from the Registry to Improve the Use of Evidence-Based Heart Failure Therapies in the Outpatient
Setting (IMPROVE HF). Am Heart J. February 2010;159(2):238-244.
Ballard DJ, Ogola G, Fleming NS, Stauffer BD, Leonard BM, Khetan R, Yancy CW. Impact of a standardized heart failure order
set on mortality, readmission, and quality and costs of care. Int J Qual Health Care. December 2010;22(6):437-444.
Ezekowitz JA, Hernandez AF, Starling RC, Yancy CW, Massie B, Hill JA, Krum H, Diaz R, Ponikowski P, Metra M, Howlett JG,
Gennevois D, O’Connor CM, Califf RM, Fonarow GC. Standardizing care for acute decompensated heart failure in a large
megatrial: the approach for the Acute Studies of Clinical Effectiveness of Nesiritide in Subjects with Decompensated Heart
Failure (ASCEND-HF). Am Heart J. February 2009;157(2):219-228.
41

About the Northwestern University Feinberg School of Medicine:


Northwestern University Feinberg School of Medicine was founded in 1859 and initially led by Nathan Smith Davis, who was
instrumental in the founding of the American Medical Association. Part of Northwestern University, an independent private
institution founded in 1851, Feinberg School of Medicine is widely considered one of the world’s premier medical research and

Biography: Clyde Yancy, MD, MSc


educational institutions.
Located on a 20-acre campus in Chicago, Illinois, Feinberg School of Medicine retains more than 4,000 leading research and
educational faculty members. With over 2,000 students from all over the world currently enrolled, Feinberg School of Medicine
seeks to prepare the next generation of doctors while conducting groundbreaking research in a variety of fields.

About the Bluhm Cardiovascular Institute at Northwestern Memorial Hospital:


The Bluhm Cardiovascular Institute is Northwestern Memorial Hospital’s heart and vascular division. The Bluhm Institute is a
teaching clinic, offering world-class treatment to patients and a hands-on learning environment for Northwestern University
medical students.
The Bluhm Institute is separated into six centers: the Center for Atrial Fibrillation, the Center for Coronary Disease, the Center for
Heart Failure, the Center for Heart Valve Disease, the Center for Vascular Disease, and the Center for Women’s Cardiovascular
Health.

Additional Information:
● Northwestern University Feinberg School of Medicine. http://www.feinberg.northwestern.edu/
● The Bluhm Cardiovascular Institute at Northwestern Memorial Hospital, Chicago.
http://www.nmh.org/nm/bluhm+cardiovascular+institute

Sources:
Meltzer and Yancy to lead American Heart Association in 2009–10 [news release]. Dallas, TX: American Heart Association;
March 16, 2009. http://www.newsroom.heart.org/index.php?s=43&item=697. Accessed January 30, 2011.
Northwestern names new chief of cardiology [press release]. Chicago, IL: Northwestern Memorial Hospital; January 17, 2011.
http://www.nmh.org/nm/clyde+yancy+announcement. Accessed January 30, 2011.
Clyde W. Yancy, MD, FACC, FAHA, MACP. American Heart Association Web site.
http://www.americanheart.org/presenter.jhtml?identifier=3040920. Accessed January 30, 2011.
Dr. Clyde Yancy to join Northwestern University. The Heart.Org; January 12, 2011. http://www.theheart.org/article/1170795.do.
Accessed January 21, 2011.
Dr. Clyde Yancy, chief of cardiology, Feinberg School of Medicine at Northwestern University. Chicago Tribune. January 13,
2011:Business. http://www.chicagotribune.com/business/ct-biz-mou-yancy-clyde,0,6902195.photo. Accessed January 30, 2011.
About Northwestern University Feinberg School of Medicine. Northwestern University Feinberg School of Medicine Web site.
http://www.feinberg.northwestern.edu/about/index.html. Accessed January 30, 2011.
Bluhm Cardiovascular Institute, Northwestern Memorial Hospital Web site.
http://www.nmh.org/nm/bluhm+cardiovascular+institute. Accessed January 30, 2011.
42

Why Methods Are Important


Decisions made by patients and providers about the care that the patient will receive frequently involve choices among
available therapies. Commonly, there are few research studies that compare alternative treatments or treatment strategies
in a “head-to-head” fashion. For this reason, it can be challenging for clinicians and patients to make care decisions.
Even less plentiful are studies that compare treatment options in real-world settings, where results may differ from those
observed in the well-controlled experimental clinical trials. As the realization of this need for enhanced comparative
information to achieve best outcomes has grown, attention has focused on comparative effectiveness research (CER).
Although there are many types of biomedical research that are very useful in understanding disease and developing new
treatments, the three most commonly utilized types of CER are 1) randomized controlled trials (RCTs) which directly
compare two (or more) approaches to diagnosing or treating patients, 2) meta-analysis, or systematic reviews, and 3)
observational or “non-experimental” studies. If conducted and interpreted correctly, these types of research can help to
inform health care decision making. If, however, such studies are conducted or interpreted incorrectly, the comparative
answers from these studies may be inaccurate, or worse, misleading.

Understanding the Types of Study Designs


Randomized Controlled Trials
Given the wide range of research questions,
one method may not be suitable for all RCTs are experiments wherein subjects in a population are
situations. Below are examples of research randomly allocated into study and control groups to receive
priorities, per the Institute of Medicine. or not to receive an experimental intervention such as a new
therapeutic biopharmaceutical or medical device. Although
• What is the best treatment (assistive listening devices,
some RCTs include large numbers of patients to address
cochlear implants, rehabilitation) for hearing loss in
children and adults?
broad population questions about competing therapies,
studies of this type often apply the intervention to a carefully
• What is the best primary prevention to prevent falls in
selected population while reducing the impact of external
older adults? Exercise or balance training?
factors (such as comorbidities) that could also influence the
• What is the most effective treatment for atrial outcome of interest. The controls required in such a study
fibrillation? Surgery, catheter ablation or pharmacologic type sometimes impact whether the findings may apply to a
treatment?
broader population. Even so, the strengths associated with
• What is the best approach to screen, treat, RCT methodology, particularly in its lowered risk of bias and
and intervene to eradicate methicillin resistant confounding, have allowed this type of study design to gain
staphylococcus aureus in communities, institutions, sufficient credibility that many now consider it to be the “gold
and hospitals? standard” of clinical research.
43

Randomized Controlled Trials


• RCTs can determine whether an intervention provides benefit in a very controlled environment.
• The controlled nature of an RCT may limit its generalizability to a broader population.
• T argeted therapy illuminated by carefully thought out subgroup analyses can improve the efficacious and safe use of
an intervention.

There are several kinds of randomized controlled trials that can be matched to potential research questions. These
include:
• P ragmatic clinical trials, which are designed to demonstrate how a medical intervention works in a typical, real-
world setting;
• Cluster RCTs, in which groups are randomized to an intervention instead of randomizing individuals;
• B ayesian, or adaptive trials, which makes use of prior information on a medical intervention to estimate a prior
distribution. This prior information is then combined with trial data to create a posterior distribution;
•N
 -of-1 trials, which are single event case studies to look at the effect of an intervention in an individual;
•D
 elayed-design or “advance coverage” trials, which have many variations, but the most common version of
this design randomizes participants to either receive the intervention from the start of the trial, or have the
intervention withheld for a pre-specified amount of time. By the end of the trial, both study groups have
received the intervention.

Meta-Analyses
Meta-analyses synthesize existing data across a series of similar studies, generally RCTs. This study design requires careful
selection of which studies to include and which statistical methods to employ. Publication bias, which occurs when
positive studies are more abundantly available on a topic due to negative studies not being published, can significantly
impair the validity of this study type. However, a balanced meta-analysis can produce a summary estimate of the medical
literature that underpins benchmarks of clinical decision making.

Meta-Analyses
• The results of a meta-analysis are highly dependent on the studies included (and excluded). Are these criteria properly defined and
relevant to the purposes of the meta-analysis? Were the combined studies sufficiently similar?
• The statistical methodology can impact study results. Have there been reviews critiquing the methods used in the meta-analysis? The
statistical methodology can impact study results. Have there been reviews critiquing the methods used in the meta-analysis?
• N
 othing is permanent. Emerging data may change the playing field, and meta-analysis results are only as good as the data and
statistics from which they are derived.
44

Observational Studies
Observational studies follow participants over a period of time to examine the potential associations between patients’
exposure to treatment and health outcomes. These studies can be performed prospectively, observing patients in real
time, or they can be retrospective analyses of existing databases. Given the abundance of billing or administrative
databases, comparisons of therapies and their outcomes will frequently use these databases, as they embody the
experience of patients in real-world settings. While results from observational studies tend to generate hypotheses and
may require the more rigorous RCT for confirmation, long-term observational studies have provided substantial medical
knowledge (e.g., demonstrating the link between elevated blood cholesterol and the development of heart disease).

Observational Studies
• Observational studies provide an understanding of real-world care and its impact, but can be biased due to uncontrolled factors.
• Before accepting the findings from an observational study, consider whether confounding factors may have influenced the results.
• Observational studies can identify associations but cannot prove cause-and-effect relationships.
• The GRACE Principles (www.graceprinciples.org) are an excellent source of further information about observational studies and their
application in CER.

How Could These Designs Be Used?


One of the PCORI Methodology Committee’s first tasks will be to prepare a translation table to assist in the development
of research designs, which will help researchers match the research method to the question at hand. Certain methods
are better suited for certain designs than others. For example, a translation table will indicate the types of studies (or
methods) that might be appropriate to assess real-world patient adherence to medications, which likely differ from the
study types appropriate to assess whether surgical or non-surgical therapy provides the best outcomes for low back
pain. Other considerations for choosing a particular method include its strengths and limitations, such as the internal
validity (strength of cause and effect), the generalizability (ability to extend research findings to other groups, patients, or
settings), feasibility (to understand the conduct of the research or the costs to conduct), and the timeliness of research
findings (how long it will take to conduct a study).

Sources:
Tunis SR, Benner J, McClellan M. Stat Med. 2010 Aug 30;29(19):1963-1976. PMID: 20564311
Dubois RW, Kindermann SL. Demystifing comparative effectiveness research: a case study learning guide.
November 2009.
45

Existing Methodological Standards and Good Practices


There are a number of existing methodological standards and good practices for randomized clinical trials, systematic
reviews, meta-analyses and observational studies. The following list highlights some of the most widely recognized
approaches.

For a broader listing, view the online library maintained by the Enhancing the Quality of Transparency of Health Research
(EQUATOR) Network at www.equator-network.org.
Bodies of Evidence
Systematic Reviews and Meta-Analysis
Meta-analysis Of Observational Studies in Epidemiology (MOOSE). http://www.consort-statement.org/mod_product/
uploads/MOOSE%20Statement%202000.pdf
Preferred Reporting Items for Systematic Reviews and Meta-Analyses (PRISMA). http://www.prisma-statement.org/index.
htm
Cochrane Collaboration Reviews. www.cochrane.org
Agency for Healthcare Research and Quality (AHRQ) Methods Reference Guide for Effectiveness and Comparative
Effectiveness Reviews. effectivehealthcare.ahrq.gov
Grading Systems and Tools for Studies
Grading of Recommendations Assessment, Development and Evaluation (GRADE). www.gradeworkinggroup.org
A Measurement Tool to Assess Systematic Reviews (AMSTAR).
http://www.evipnet.org/php/level.php?lang=en&component=103&item=16
Appraisal of Guidelines, Research and Evaluation (AGREE). www.agreecollaboration.org
Conference on Guideline Standardization (COGS). gem.med.yale.edu/cogs
Individual Study Types
Randomized Controlled Trials
Consolidated Standards of Reporting Trials (CONSORT). www.consort-statement.org
Consolidated Standards of Reporting Trials (CONSORT)-Pragmatic Trials.
www.consort-statement.org/extensions/designs/pragmatic-trials
Pragmatic-Explanatory Continuum Indicator Summary (PRECIS). www.support-collaboration.org/precis.pdf
Observational Studies
Good ReseArch for Comparative Effectiveness (GRACE) Principles. www.graceprinciples.org
Strengthening the Reporting of Observational Studies in Epidemiology (STROBE). www.strobe-statement.org
International Society for Pharmacoeconomics and Outcomes Research (ISPOR) Good Research Practices for CER Using
Secondary Databases. http://www.ispor.org/workpaper/practices_index.asp
A Checklist for Retrospective Database Studies—Report of the ISPOR Task Force on Retrospective Database Studies.
www.ispor.org/workpaper/healthscience/ret_dbtfr0203.asp
A Checklist for Medication Compliance and Persistence Studies Using Retrospective Databases.
http://www.ispor.org/workpaper/medcompliancechecklist.asp
AHRQ’s Registries for Evaluating Patient Outcomes: A User’s Guide.
www.effectivehealthcare.ahrq.gov/index.cfm/search-for-guides-reviews-and-reports/?productid=12&pageaction=displayproduct
46

Other Applications
Sox, et al, Editorial Standards for the Conduct and Reporting of CER. www.ncbi.nlm.nih.gov/pmc/articles/PMC2860496
Standards for the Reporting of Diagnostic Accuracy Studies (STARD). www.stard-statement.org
Strengthening the Reporting of Genetic Association Studies (STREGA).
www.medicine.uottawa.ca/public-health-genomics/web/eng/strega.html
Standards for Quality Improvement Reporting Excellence (SQUIRE). www.squire-statement.org
Reporting Recommendations for Tumor Marker Prognostic Studies (REMARK).
www.equator-network.org/resource-centre/library-of-health-research-reporting/reporting-guidelines/remark/
Transparent Reporting of Evaluations With Nonrandomized Designs (TREND). www.cdc.gov/trendstatement/
European Network of Centres for Pharmacoepidemiology and Pharmacovigilance Checklist for Methodologic Standards
for ENCePP Study Protocols. www.encepp.eu/public_consultation/documents/ChecklistofMSforENCePPStudies_
consultationversion.pdf
Guidelines for Good Pharmacoepidemiology Practices (GPP). www.pharmacoepi.org/resources/guidelines_08027.cfm

Source:
List adapted from Dreyer NA, Tunis SR, Berger M, Ollendorf D, Mattox P, Gliklich R. Why observational
studies should be among the tools used in comparative effectiveness research. Health Aff (Millwood).
2010;29(10):1818-1825.
http://content.healthaffairs.org/cgi/data/29/10/1818/DC1/1. Accessed December 2, 2010.
47

Additional Resources
Many organizations—including, but not limited to those listed below—are taking an active role in examining research
methods. There is also a wide variety of collaborative efforts among academic researchers, many of which are listed in
the preceding section of this resource guide.

Agency for Healthcare Research and Quality Effective Healthcare Program. effectivehealthcare.ahrq.gov
Center for Medical Technology Policy (CMTP). www.cmtpnet.org
Cochrane Collaboration. www.cochrane.org
ECRI. www.ecri.org
European Network of Centres for Pharmacoepidemiology and Pharmacovigilance (ENCePP). www.encepp.eu
Hayes, Inc. www.hayesinc.com
Institute for Clinical and Economic Review (ICER). www.icer-review.org
Institute of Medicine. www.iom.gov
International Network of Agencies for Health Technology Assessment. www.inahta.org
International Society for Pharmacoeconomics and Outcomes Research (ISPOR). www.ispor.org
International Society for Pharmacoepidemiology (ISPE). www.pharmacoepi.org
James Lind Library. www.jameslindlibrary.org
National Pharmaceutical Council. www.npcnow.org
Patient-Centered Outcomes Research Institute (PCORI). www.pcori.org
Pragmatic Approaches to Comparative Effectiveness (PACE) Initiative. www.paceinitiative.org/index.html

The National Pharmaceutical Council plans to regularly update this list on its Web site. To be considered for inclusion,
please send an email with the organization’s name and Web address to info@npcnow.org.
48

Appendix
Excerpted from The Patient Protection and Affordable Care Act, PL 111-148.

Subtitle D--Patient-Centered Outcomes Research, SEC. 6301. PATIENT-CENTERED OUTCOMES RESEARCH

CARRYING OUT RESEARCH PROJECT AGENDA.-

‘’(A) RESEARCH.-The Institute shall carry out the research project agenda established under paragraph (1)(B) in
accordance with the methodological standards adopted under paragraph (9) using methods, including the following:

‘’(i) Systematic reviews and assessments of existing and future research and evidence including original research
conducted subsequent to the date of the enactment of this section.

‘’(ii) Primary research, such as randomized clinical trials, molecularly informed trials, and observational studies.

‘’(iii) Any other methodologies recommended by the methodology committee established under paragraph (6) that are
adopted by the Board under paragraph (9).

(6) ESTABLISHING METHODOLOGY COMMITTEE-

`(A) IN GENERAL- The Institute shall establish a standing methodology committee to carry out the functions described in
subparagraph (C).

`(B) APPOINTMENT AND COMPOSITION- The methodology committee established under subparagraph (A) shall
be composed of not more than 15 members appointed by the Comptroller General of the United States. Members
appointed to the methodology committee shall be experts in their scientific field, such as health services research, clinical
research, comparative clinical effectiveness research, biostatistics, genomics, and research methodologies. Stakeholders
with such expertise may be appointed to the methodology committee. In addition to the members appointed under the
first sentence, the Directors of the National Institutes of Health and the Agency for Healthcare Research and Quality (or
their designees) shall each be included as members of the methodology committee.

`(C) FUNCTIONS- Subject to subparagraph (D), the methodology committee shall work to develop and improve the
science and methods of comparative clinical effectiveness research by, not later than 18 months after the establishment
of the Institute, directly or through subcontract, developing and periodically updating the following:

`(i) Methodological standards for research. Such methodological standards shall provide specific criteria for internal
validity, generalizability, feasibility, and timeliness of research and for health outcomes measures, risk adjustment,
and other relevant aspects of research and assessment with respect to the design of research. Any methodological
standards developed and updated under this subclause shall be scientifically based and include methods by which
new information, data, or advances in technology are considered and incorporated into ongoing research projects by
the Institute, as appropriate. The process for developing and updating such standards shall include input from relevant
experts, stakeholders, and decisionmakers, and shall provide opportunities for public comment. Such standards shall also
include methods by which patient subpopulations can be accounted for and evaluated in different types of research. As
appropriate, such standards shall build on existing work on methodological standards for defined categories of health
interventions and for each of the major categories of comparative clinical effectiveness research methods (determined as
of the date of enactment of the Patient Protection and Affordable Care Act).

`(ii) A translation table that is designed to provide guidance and act as a reference for the Board to determine research
methods that are most likely to address each specific research question.
49

`(D) CONSULTATION AND CONDUCT OF EXAMINATIONS- The methodology committee may consult and contract with
the Institute of Medicine of the National Academies and academic, nonprofit, or other private and governmental entities
with relevant expertise to carry out activities described in subparagraph (C) and may consult with relevant stakeholders
to carry out such activities.

`(E) REPORTS- The methodology committee shall submit reports to the Board on the committee’s performance
of the functions described in subparagraph (C). Reports shall contain recommendations for the Institute to adopt
methodological standards developed and updated by the methodology committee as well as other actions deemed
necessary to comply with such methodological standards.