I 
113TH CONGRESS 
2D SESSION 
H. R. 5805 
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 
DECEMBER 8, 2014 
Mr. MCCAUL introduced the following bill; which was referred to the 
Committee on Energy and Commerce 
A BILL 
To amend the Federal Food, Drug, and Cosmetic Act with 
respect to expanding access for breakthrough drugs, and 
for other purposes. 
Be it enacted by the Senate and House of Representa-
tives of the United States of America in Congress assembled, 
SECTION 1. SHORT TITLE. 
This Act may be cited as the ‘‘Andrea Sloan Compas-
sionate Use Reform and Enhancement Act’’ or the ‘‘An-
drea Sloan CURE Act’’. 
SEC. 2. EXPANDED ACCESS POLICY AS CONDITION OF EX-
PEDITED APPROVAL. 
Section 561 of the Federal Food, Drug, and Cosmetic 
Act (21 U.S.C. 360bbb) is amended— 
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(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 
COVERED BREAKTHROUGH DRUGS.— 
‘‘(1) IN GENERAL.—With respect to a qualified 
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 subsection (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-
sor’s consideration or approval of such requests; 
and 
‘‘(D) the amount of time the sponsor an-
ticipates will be necessary to make a decision on 
such requests. 
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‘‘(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 
506; 
‘‘(B) that is designated under section 
505E(d) as a qualified infectious disease prod-
uct; or 
‘‘(C) the sponsor of which is awarded a 
priority review voucher under section 524 or 
529.’’. 
SEC. 3. NOTIFICATION OF SUBMITTERS OF COMPAS-
SIONATE USE REQUESTS. 
Section 561 of the Federal Food, Drug, and Cosmetic 
Act (21 U.S.C. 360bbb), as amended by section 2, is fur-
ther amended— 
(1) by redesignating subsections (e) and (f) (as 
redesignated by section 2(1)) as subsections (f) and 
(g), respectively; and 
(2) by inserting after subsection (d) (as in-
serted by section 2(2)) the following new subsection: 
‘‘(e) NOTIFICATION
OF
SUBMITTERS
OF
RE-
QUESTS.—In the case of the denial by a manufacturer or 
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distributor of a request under subsection (b), not later 
than 5 days after the date of such denial, the manufac-
turer or distributor, as applicable, shall submit to the per-
son (or physician) who made the request written notice 
of the denial, including an explanation for the denial.’’. 
SEC. 4. GAO QUALITATIVE ANALYSIS ON INDIVIDUAL PA-
TIENT ACCESS TO UNAPPROVED THERAPIES 
AND DIAGNOSTICS. 
Not later than 180 days after the date of the enact-
ment of this Act and each year thereafter, the Comptroller 
General of the United States shall submit to the Com-
mittee on Energy and Commerce of the House of Rep-
resentatives and the Committee on Health, Education, 
Labor and Pensions of the Senate a report containing a 
qualitative analysis of the extent to which individual pa-
tients have access to investigational drugs pursuant to 
subsection (b) of section 561 of the Federal Food, Drug, 
and Cosmetic Act (21 U.S.C. 360bbb) and recommenda-
tions for improving such access. In preparing such report, 
the Comptroller General shall conduct a qualitative anal-
ysis of the following: 
(1) Whether there are any identifiable patterns 
in requests submitted under subsection (b) of such 
section, such as the types of indications for which 
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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— 
(A) a physician typically takes to complete 
the paperwork necessary to make such a re-
quest; 
(B) a drug sponsor takes to process such 
a request and to issue a decision with respect 
to the request; and 
(C) the Secretary takes to process such a 
request and to issue a decision with respect to 
the request. 
(5) How regulations, guidance, policies, or prac-
tices may be modified, streamlined, expanded, or dis-
continued to reduce or prevent delays in approving 
such requests. 
(6) The number of such requests that, for the 
period covered by the report— 
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(A) were approved by drug sponsors and 
the Food and Drug Administration; 
(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. 
(8) Whether and to what extent adverse events 
reported to the Secretary as a result of individual 
use of an investigational drug or investigational de-
vice under such section 561 affected the development 
or approval of any drug or device. 
SEC. 5. EXPANDED ACCESS TASK FORCE. 
(a) ESTABLISHMENT.—The Secretary of Health and 
Human Services shall establish a task force within the De-
partment of Health and Human Services to explore mech-
anisms for improving the access individual patients have 
to investigational drugs pursuant to subsection (b) of sec-
tion 561 of the Federal Food, Drug, and Cosmetic Act 
(21 U.S.C. 360bbb), to be known as the ‘‘Expanded Ac-
cess Task Force’’ (in this section referred to as the ‘‘Task 
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Force’’). Not later than 90 days after the date on which 
the Comptroller General of the United States submits the 
first report required under section 4, the Task Force shall 
be convened. 
(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. 
(B) One representative from the Depart-
ment of Health and Human Services, appointed 
by the Secretary of Health and Human Serv-
ices. 
(C) Four representatives appointed by the 
Majority Leader of the House of Representa-
tives, in consultation with the Minority Leader 
of the House of Representatives, and the Chair-
man and the Ranking Member of the Com-
mittee on Energy and Commerce of the House 
of Representatives, including— 
(i) one representative of a biopharma-
ceutical company of less than 250 full-time 
employees; 
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(ii) one representative of the rare dis-
ease patient community; 
(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— 
(i) one representative of the bio-
pharmaceutical industry; 
(ii) one representative of the patient 
community; and 
(iii) one representative of the health 
care payor community. 
(2) COMPENSATION.—Members of the Task 
Force shall serve without compensation. 
(c) DUTIES.—The Task Force shall comprehensively 
evaluate the access individual patients have to investiga-
tional drugs pursuant to subsection (b) of section 561 of 
the Federal Food, Drug, and Cosmetic Act (21 U.S.C. 
360bbb), taking into account— 
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