Andrea Sloan CURE Act

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.....................................................................
(Original Signature of Member)
113TH CONGRESS
2D SESSION
H. R. ll
To amend the Federal Food, Drug, and Cosmetic Act with respect to
expanding access for breakthrough drugs, and for other purposes.
IN THE HOUSE OF REPRESENTATIVES

Mr. MCCAUL introduced the following bill; which was referred to the
Committee on llllllllllllll
A BILL
To amend the Federal Food, Drug, and Cosmetic Act with
respect to expanding access for breakthrough drugs, and
for other purposes.
1
Be it enacted by the Senate and House of Representa-
2 tives of the United States of America in Congress assembled,

3
SECTION 1. SHORT TITLE.
This Act may be cited as the Andrea Sloan Compas-
5 sionate Use Reform and Enhancement Act or the An6 drea Sloane CURE Act.
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1
SEC. 2. EXPANDED ACCESS POLICY AS CONDITION OF EX-
2
3
PEDITED APPROVAL.
Section 561 of the Federal Food, Drug, and Cosmetic
4 Act (21 U.S.C. 360bbb) is amended

5
6
(1) by redesignating subsections (d) and (e) as

subsections (e) and (f), respectively; and
(2) by inserting after subsection (c) the fol-
lowing new subsection:
(d) EXPANDED ACCESS POLICY REQUIRED
FOR
10 QUALIFIED BREAKTHROUGH DRUGS.

11
(1) IN
respect to a qualified
12
breakthrough drug, not later than 30 days after the
13
date on which the drug meets the definition of a cov-
14
ered breakthrough drug (as specified in paragraph
15
(2)), the sponsor of the covered breakthrough drug
16
shall submit to the Secretary and make publicly
17
available the policy of the sponsor with respect to re-
18
quests submitted under section 561(b). In the case
19
of such a policy under which the sponsor accepts
20
such requests, such policy shall include
21
(A) a single point of contact who receives
22
and processes such requests;
23
(B) procedures for making such requests;
24
(C) the minimum criteria for the spon-
25
sors consideration or approval of such requests;
26
and
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1
(D) the amount of time the sponsor an-
ticipates will be necessary to make a decision on
such requests.
(2) COVERED
BREAKTHROUGH DRUG.In
this
subsection, the term covered breakthrough drug
means a drug
(A) that is designated as a breakthrough
therapy or as a fast track product or is ap-
proved under accelerated approval under section
10
506;
11
(B) that is designated under section
12
505E(d) as a qualified infectious disease prod-
13
uct; or
14
(C) the sponsor of which is awarded a
15
priority review voucher under section 524 or
16
529..
17
SEC.
3.
18
19
NOTIFICATION
OF
SUBMITTERS
OF
COMPAS-
SIONATE USE REQUESTS.
Section 561 of the Federal Food, Drug, and Cosmetic
20 Act (21 U.S.C. 360bbb), as amended by section 2, is fur21 ther amended

22
(1) by redesignating subsections (e) and (f) (as
23
redesignated by section 2(1)) as subsections (f) and
24
(g), respectively; and
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(2) by inserting after subsection (d) (as in-
serted by section 2(2)) the following new subsection:
(e)
QUESTS.In
NOTIFICATION
OF
SUBMITTERS
OF
RE-
the case of the denial by a manufacturer or
5 distributor of a request under subsection (b), not later

6 than 5 days after the date of such denial, the manufac7 turer or distributor, as applicable, shall submit to the per8 son (or physician) who made the request written notice
9 of the denial, including an explanation for the denial..
10
SEC. 4. GAO QUALITATIVE ANALYSIS ON INDIVIDUAL PA-
11
TIENT ACCESS TO UNAPPROVED THERAPIES
12
AND DIAGNOSTICS.
13
Not later than 180 days after the date of the enact-
14 ment of this Act and each year thereafter, the Comptroller

15 General of the United States shall submit to Congress a
16 report containing an analysis of the extent to which indi17 vidual patients have access to investigational drugs pursu18 ant to subsection (b) of section 561 of the Federal Food,
19 Drug, and Cosmetic Act (21 U.S.C. 360bbb) and rec20 ommendations for improving such access. In preparing
21 such report, the Comptroller General shall conduct a qual22 itative analysis of the following:
23
(1) Whether there are any identifiable patterns
24
in requests submitted under subsection (b) of such
25
section, such as the types of indications for which
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1
requests for individual patient access are sought or
the reasons for the denial of such requests.
(2) What the primary barriers are to drug
sponsors granting requests for individual patient ac-
cess.
(3) How the Secretary evaluates safety and effi-
cacy data submitted in connection with such re-
quests.
(4) The amount of time that
10
(A) a physician typically takes to complete
11
the paperwork necessary to make such a re-
12
quest;
13
(B) a drug sponsor takes to process such
14
a request and to issue a decision with respect
15
to the request; and
16
(C) the Secretary takes to process such a
17
request and to issue a decision with respect to
18
the request.
19
(5) How regulations, guidance, policies, or prac-
20
tices may be modified, streamlined, expanded, or dis-
21
continued to reduce or prevent delays in approving
22
such requests.
23
(6) The number of such requests that
24
(A) were approved by drug sponsors and
25
the Food and Drug Administration;
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1
(B) were approved by drug sponsors but
denied by the Food and Drug Administration;
and
(C) were denied by drug sponsors.
(7) How to encourage drug sponsors to grant
requests for expanded access under such section
561, including requests for emergency use, inter-
mediate-size patient populations, and large patient
populations under a specified indication.
10
(8) Whether and to what extent adverse events
11
reported to the Secretary as a result of individual
12
use of an investigational drug or investigational de-
13
vice under such section 561 affected the development
14
or approval of any drug or device.
15
SEC. 5. EXPANDED ACCESS TASK FORCE.
16
(a) ESTABLISHMENT.The Secretary of Health and
17 Human Services shall establish a task force within the De18 partment of Health and Human Services to explore mech19 anisms for improving the access individual patients have
20 to investigational drugs pursuant to subsection (b) of sec21 tion 561 of the Federal Food, Drug, and Cosmetic Act
22 (21 U.S.C. 360bbb), to be known as the Expanded Ac23 cess Task Force (in this section referred to as the Task
24 Force). Not later than 90 days after the date on which
25 the Comptroller General of the United States submits the
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1 report required under section 4, the Task Force shall be
2 convened.
3
(b) MEMBERSHIP.
(1) COMPOSITION.The Task Force shall be
composed of not more than 9 voting members ap-
pointed as follows:
(A) One member to serve as Chairman of
the Task Force, appointed by the Speaker of
the House of Representatives.
10
(B) One representative from the Depart-
11
ment of Health and Human Services, appointed
12
by the Secretary of Health and Human Serv-
13
ices.
14
(C) Four representatives appointed by the
15
Majority Leader of the House of Representa-
16
tives, in consultation with the Minority Leader
17
of the House of Representatives, and the Chair-
18
man and the Ranking Member of the Com-
19
mittee on Energy and Commerce of the House
20
of Representatives, including
21
(i) one representative of a biopharma-
22
ceutical company of less than 250 full-time
23
employees;
24
(ii) one representative of the rare dis-
25
ease patient community;
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1
(iii) one representative of the health
care provider community; and
(iv) one bioethicist.
(D) Three representatives appointed by
Majority Leader of the Senate, in consultation
with the Minority Leader of the Senate, and the
Chairman and the Ranking Member of the
Health, Education, Labor and Pensions Com-
mittee of the Senate, including
10
(i) one representative of the bio-
11
pharmaceutical industry;
12
(ii) one representative of the patient
13
community; and
14
(iii) one representative of the health
15
care payor community.
16
(2) COMPENSATION.Members of the Task
17
Force shall serve without compensation.
18
(c) FUNCTIONS.The Task Force shall comprehen-
19 sively evaluate the access individual patients have to inves20 tigational drugs pursuant to subsection (b) of section 561
21 of the Federal Food, Drug, and Cosmetic Act (21 U.S.C.
22 360bbb), taking into account
23
(1) the unique challenges faced by children with
24
likely fatal diseases and no comparable or satisfac-
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1
tory alternative therapy available to diagnose, mon-
itor, or treat the disease;
(2) possible incentives for biopharmaceutical
companies and providers to approve requests sub-
mitted under such subsection;
(3) how the Secretary of Health and Human
Services interprets and takes into consideration ad-
verse event data reported in the case of data from
use under a request submitted under such sub-
10
section;
11
(4) ways to streamline and standardize the
12
process for submitting requests under such sub-
13
section; and
14
(5) the costs incurred by biopharmaceutical
15
companies for the time, effort, and delivery of inves-
16
tigational drugs to patients for the diagnosis, moni-
17
toring, or treatment of a serious disease or condition
18
under such subsection.
19
(d) REPORT.Not later than 180 days after the date
20 on which the Task Force is convened, the Task Force shall

21 transmit to the Committee on Energy and Commerce of
22 the House of Representatives and the Committee on
23 Health, Education, Labor and Pensions of the Senate a
24 report in an electronic format describing the specific rec25 ommendations of the Task Force for improving the access
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1 individual patients have to investigational drugs pursuant
2 to subsection (b) of section 561 of the Federal Food,
3 Drug, and Cosmetic Act (21 U.S.C. 360bbb).
4
(e) TERMINATION.The task force shall terminate
5 upon transmittal of the report required under subsection

6 (d).
7
SEC. 6. FINALIZING DRAFT GUIDANCE ON EXPANDED AC-
8
9
CESS.
Not later than 180 days after the date of the enact-
10 ment of this Act, the Secretary of Health and Human

11 Services shall finalize the draft guidance entitled Ex12 panded Access to Investigational Drugs for Treatment
13 Use Qs & As and dated May 2013. The final guidance
14 shall clearly define how the Secretary interprets and uses
15 adverse drug event data reported by investigators in the
16 case of data reported from use under a request submitted
17 under section 561(b) of the Federal Food, Drug, and Cos18 metic Act (21 U.S.C. 360bbb(b)).
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F:\M13\MCCAUL\MCCAUL_051.

IN THE HOUSE OF REPRESENTATIVES

Be it enacted by the Senate and House of Representa-

2 tives of the United States of America in Congress assembled,

SECTION 1. SHORT TITLE.

This Act may be cited as the Andrea Sloan Compas-

18:31 Dec 05, 2014

SEC. 2. EXPANDED ACCESS POLICY AS CONDITION OF EX-

Section 561 of the Federal Food, Drug, and Cosmetic

4 Act (21 U.S.C. 360bbb) is amended

(1) by redesignating subsections (d) and (e) as

(2) by inserting after subsection (c) the fol-

lowing new subsection:

(d) EXPANDED ACCESS POLICY REQUIRED

10 QUALIFIED BREAKTHROUGH DRUGS.

breakthrough drug, not later than 30 days after the

date on which the drug meets the definition of a cov-

ered breakthrough drug (as specified in paragraph

(2)), the sponsor of the covered breakthrough drug

shall submit to the Secretary and make publicly

available the policy of the sponsor with respect to re-

quests submitted under section 561(b). In the case

of such a policy under which the sponsor accepts

such requests, such policy shall include

(A) a single point of contact who receives

and processes such requests;

(B) procedures for making such requests;

(C) the minimum criteria for the spon-

sors consideration or approval of such requests;

18:31 Dec 05, 2014

(D) the amount of time the sponsor an-

ticipates will be necessary to make a decision on

subsection, the term covered breakthrough drug

(A) that is designated as a breakthrough

therapy or as a fast track product or is ap-

proved under accelerated approval under section

(B) that is designated under section

505E(d) as a qualified infectious disease prod-

(C) the sponsor of which is awarded a

priority review voucher under section 524 or

SIONATE USE REQUESTS.

Section 561 of the Federal Food, Drug, and Cosmetic

20 Act (21 U.S.C. 360bbb), as amended by section 2, is fur21 ther amended

(1) by redesignating subsections (e) and (f) (as

redesignated by section 2(1)) as subsections (f) and

(g), respectively; and

18:31 Dec 05, 2014

(2) by inserting after subsection (d) (as in-

serted by section 2(2)) the following new subsection:

the case of the denial by a manufacturer or

5 distributor of a request under subsection (b), not later

SEC. 4. GAO QUALITATIVE ANALYSIS ON INDIVIDUAL PA-

TIENT ACCESS TO UNAPPROVED THERAPIES

14 ment of this Act and each year thereafter, the Comptroller

(1) Whether there are any identifiable patterns

in requests submitted under subsection (b) of such

section, such as the types of indications for which

18:31 Dec 05, 2014

requests for individual patient access are sought or

the reasons for the denial of such requests.

(2) What the primary barriers are to drug

sponsors granting requests for individual patient ac-

(3) How the Secretary evaluates safety and effi-

cacy data submitted in connection with such re-