Pharmacoeconomics & Health Outcomes

Health & Economic Data Sources

Leon E. Cosler, R.Ph., Ph.D.

Associate Professor of Pharmacoeconomics
Albany College of Pharmacy
Road Map: Data Sources
1. Identify various data sources useful for this field

2. Advantages & disadvantages of different types

3. List several government databases

- Selected examples

4. Current laws / regulations in human research

- Patient informed consent process

“Where’s he getting this?”

• Parts of Bootman Text Ch. 12
Recommended Steps for P-E study

• Well defined hypothesis

• Perspective is specified
• Alternative therapies / options considered
• Clinical effectiveness is considered
• All relevant costs / outcomes identified
• and accurately measured

• Study design / methodology appropriate

• Results interpreted and communicated
Data Sources
• Selecting source for data depends on:
• The type of data needed
• The research question(s)
• The study perspective
• Access to the data
• Resources ($$$) you have to spend
• Common sources include:
• Nationally collected data
• Payers (insurers, medicaid, medicare)
• Providers (inpatient*, MCOs, PBMs)
• Rx clinical trials (more on this later…)
• Patient surveys
The type of data needed:

- Economic data ($$$$)

• Direct Medical Costs (easiest)

- hospitalizations
- visits: MD, clinic, ED, DDS,
- diagnostic procedures
- lab tests
- medications
- home health care
- medical equipment / supplies
The type of data needed:

- Economic data ($$$$)

• Some $$$ only available from Pt.:
- out-of-pocket $$$ (co-pays)
> OOP or TrOOP (true out of pocket) costs

- non-medical direct $$$

• Requires surveying the patient

- Pt. diaries or journals
- difficult, but can be very important
- driven by study perspective
The type of data needed:

- Patient Outcomes data

• clinical outcomes get from:

- Ex: medical records

• humanistic outcomes get from:

- Ex: patient surveys or questionnaires
Data Sources

• The research question(s):

- Specifically defined study objective(s):
• clearly drives the data you will need

• The study perspective determines:

- “costs” or “charges”
- which categories are needed?
• and suggests where to get them...
Access to health databases:

• Provider / Payer cost data

• often considered proprietary info.
• also private medical info. restrictions
• “willingness to share” differs

• Surveying patients
• often requires “informed consent”
• IRB review of protocol

• Release of medical information

• HIPAA & PHI (protected health information)
http://www.hhs.gov/ocr/hipaa/
Resources ($$$) for data:

- Some organizations charge for data

- Researchers (i.e.: you) may need:

• computer programming assistance

• technical support to understand the data

• extra computer equipment / software

• statistical consultants
External Data Sources

- already published studies

• comparable therapy to your objective?
• quality study?
• “meta-analysis”

- Medical claims and databases

• ex: Medicare / Medicaid / Insurers
• very limited clinical measures
• rarely any patient outcomes
• no humanistic outcomes
Internal data

• inside your (hospital / medical center)

- requires sufficient # of cases
- generalizable only to your situation

• Medical Records have rich clinical info.

• difficult to extract & recompile
• difficult to get large samples
• often a costly source of data
• New trends toward electronic medical
records (EMRs)
Frequently Used Data Sources

• Hospital based data

Medical Record Billing Data Admission / Discharge
Information
Pt. Demographics No. of products received Length of stay
Diagnosis Codes No. of tests Admission type
Procedure Codes Department costs Place of admission
MD information Total hospital costs Discharge status
Severity of Illness Total hospital charges

- Remember these are different:

• hospital “cost”
• hospital “charges”
• Requires permission to access records
Pharmacoeconomics & Health Outcomes

Sources of Health Care Data

Leon E. Cosler, R.Ph., Ph.D.

Associate Professor of Pharmacoeconomics
Albany College of Pharmacy
http://www.nlm.nih.gov/hsrinfo/datasites.html
Government Databases
Census Data (www.census.gov)
Census Data
Government Databases: CMS
Government Databases: CDC - NCHS
Government Databases: CDC - NCHS
Government Databases: AHRQ
Pharmacoeconomics & Health Outcomes

Human Subjects in Research

Research Designs

Leon E. Cosler, R.Ph., Ph.D.

Associate Professor of Pharmacoeconomics
Albany College of Pharmacy
Nazi Medical Experiments 1940s
Key Historical Events...

• Nazi Medical Experiments 1940s

- Nuremberg Medical Trials 1946 - 47
- Nuremberg Code 1947

• Declaration of Helsinki 1964

• Belmont Report 1978 *

• National Research Act 1974

- 45 CFR 46 (PHS) 1974+
- 21 CFR 50, 56, 315, 812 (FDA) 1981+
Key Historical Events...

The Nuremberg Code: 1947

1. Voluntary consent essential

2. Legal capacity to give consent
3. Without force, deceit or coercion
4. Have knowledge & comprehension of
the experiment’s elements to make an
informed decision
Tuskegee Syphilis Study ( 1932 - 72 )
Tuskegee Syphilis Study (1932 - 72)

• US PHS funded study of untreated syphilis

- re-reviewed in 1969 “justified & beneficial”

• Tuskegee Syphilis Review Panel (1972)

- found PHS study “ethically unjustified”

• Called for new federal office (the office of

human protections)
“… with the authority to regulate all federally
supported research involving human subjects…”
Key Historical Events...

• National Research Act of 1974:

- National Commission for the Protection of

Human Subjects of Biomedical and
Behavioral Research

- Published “Ethical Principles and

Guidelines for the Protection of Human
Subjects (aka The Belmont Report, 1978)
The Belmont Report (1978) :

• Defines “research”

• 3 Basic ethical principles

• Respect for persons
• Beneficence – cannot lie to them…full
disclosure…do not do experiments that
will hurt them
• Justice – pick patients fairly…don’t pick
poor men in Tuskegee b/c they have
syphilis..dont pick prisoners
• “Informed Consent”
CFR Title 45 Part 46 (1991):

“Protection of Human Subjects”

• all research involving humans supported
by any Federal agency
• established the role of Institutional Review
Boards (IRBs)
• Defines
• “research”
• Human subjects
• “informed consent”
CFR Title 45 Part 46 (1991):
Research
• “…(d) Research means a systematic investigation, including research
development, testing and evaluation, designed to develop or
contribute to generalizable knowledge. Activities which meet this
definition constitute research for purposes of this policy, whether or
not they are conducted or supported under a program which is
considered research for other purposes. For example, some
demonstration and service programs may include research
activities…”

Human Subject
• “… (f) Human subject means a living individual about whom an
investigator (whether professional or student) conducting research
obtains:
(1) Data through intervention or interaction with the individual, or
(2) Identifiable private information….”
http://www.hhs.gov/ohrp/
FDA - Regulated Research

• INDs / IDEs in addition to IRB review

• Additional Laws:
- 21 CFR 312, 314, 600, 812, 814
• Investigators required to sign FDA 1572
• Obligations of the study sponsor
- industry-sponsored studies
• Safety Reports: SAEs reported promptly
http://www.fda.gov/oc/ohrt/irbs/default.htm
Institutional Review Boards (IRBs)

• Must have at least 5 members

- diverse with at least one “non-scientist”
- one outside the organization
- Three “levels” of protocol review:
a. “exempted” from review
b. “expedited” review
c. “full” board review if it is high risk study
IRB reviews the following:

2. Assess risks to patients

3. Equitable selection of patients

4. Informed Consent

5. Adequate monitoring of patient safety

6. Adequate protection of privacy (PHI)

Informed Consent components

1. Declaration statement
- Risks / benefits
- Alternative procedures
- Confidentiality procedures
- Medical treatment if needed
• Usually responsibility of sponsor
- “Who do you call… “
- Voluntary statement
• Says you are volunartily being asked to
patricipate…may leave study at any time you want
Jesse Gelsinger (1981 - 1999)
Current Developments (2000 +)

• Federal Office Renamed: OHRP

- All P.I.s must be trained
- NIH considers adding funds for IRBs
- FDA updating information sheets and
adverse event monitoring
- changes to 45 CFR 46 considered
Ellen Roche (1977 - 2001)
Important Web sites

http://www.hhs.gov/ohrp/

http://www.primr.org/

http://www.fda.gov/oc/ohrt/irbs/default.htm

http://www.nih.gov/sigs/bioethics/IRB.html
That’s all for today… !