I 
114TH CONGRESS 
1ST SESSION 
H. R. 909 
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 
FEBRUARY 12, 2015 
Mr. MCCAUL (for himself, Mr. BUTTERFIELD, Mr. BURGESS, Mr. GRIFFITH, 
Ms. MATSUI, and Mr. LANCE) 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’’. 
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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— 
(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 INVESTIGATIONAL DRUGS.— 
‘‘(1) IN GENERAL.—With respect to a covered 
investigational drug, not later than 30 days after the 
date on which the drug meets the definition of a cov-
ered investigational drug (as specified in paragraph 
(2)), the sponsor of the covered investigational drug 
shall submit to the Secretary, and make publicly 
available, the policy of the sponsor with respect to 
requests 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 general criteria for the sponsor’s 
consideration or approval of such requests; and 
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‘‘(D) the amount of time the sponsor an-
ticipates will be necessary to respond to such 
requests. 
‘‘(2) COVERED
INVESTIGATIONAL
DRUG.—In 
this subsection, the term ‘covered investigational 
drug’ means a drug that— 
‘‘(A) is designated as a breakthrough ther-
apy or as a fast track product; 
‘‘(B) is designated under section 505E(d) 
as a qualified infectious disease product; or 
‘‘(C) is designated under section 526 as a 
drug for a rare disease or condition.’’. 
SEC. 3. NOTIFICATION OF SUBMITTERS OF EXPANDED AC-
CESS 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 
distributor of a request under subsection (b), not later 
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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 every two years thereafter through 
2023, the Comptroller General of the United States shall 
submit to the Committee on Energy and Commerce of the 
House of Representatives and the Committee on Health, 
Education, Labor and Pensions of the Senate a report 
containing a qualitative analysis of the extent to which in-
dividual patients have access to investigational drugs pur-
suant to subsection (b) of section 561 of the Federal Food, 
Drug, and Cosmetic Act (21 U.S.C. 360bbb) and rec-
ommendations for improving such access. In preparing 
such report, the Comptroller General shall conduct a qual-
itative analysis 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 
requests for individual patient access are sought or 
the reasons for the denial of such requests. 
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(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— 
(A) were approved by drug sponsors and 
the Food and Drug Administration; 
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(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 
Force’’). Not later than 90 days after the date on which 
the Comptroller General of the United States submits the 
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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 13 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) Six 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 chairman 
and the ranking member of the Committee on 
Energy and Commerce of the House of Rep-
resentatives, including— 
(i) one current or former representa-
tive of the biopharmaceutical industry of 
not less than 250 full-time employees; 
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(ii) one representative of a biopharma-
ceutical company of less than 250 full-time 
employees; 
(iii) one representative of the patient 
community; 
(iv) one representative of the rare dis-
ease patient community; 
(v) one representative of the health 
care provider community; and 
(vi) one bioethicist. 
(D) Five representatives appointed by ma-
jority leader of the Senate, in consultation with 
the minority leader of the Senate, and the 
chairman and the ranking member of the Com-
mittee on Health, Education, Labor and Pen-
sions of the Senate, including— 
(i) one representative of the bio-
pharmaceutical industry of not less than 
250 full-time employees; 
(ii) one current or former representa-
tive of a biopharmaceutical company of 
less than 250 full-time employees; 
(iii) one representative of the patient 
community; 
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(iv) one representative of the rare dis-
ease patient community; and 
(v) 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— 
(1) the unique challenges faced by children with 
likely fatal diseases for which there is not a com-
parable or satisfactory alternative therapy available; 
(2) possible incentives for biopharmaceutical 
companies and providers to approve requests sub-
mitted under such subsection; 
(3) ways to improve followup reporting of ad-
verse event data and compliance with such reporting 
requirements; 
(4) 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-
section; 
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(5) ways to streamline and standardize the 
process for submitting requests under such sub-
section; and 
(6) the costs incurred by biopharmaceutical 
companies for the time, effort, and delivery of inves-
tigational drugs to patients for the diagnosis, moni-
toring, or treatment of a serious disease or condition 
under such subsection. 
(d) REPORT.—Not later than 180 days after the date 
on which the Task Force is convened, the Task Force shall 
submit to the Committee on Energy and Commerce of the 
House of Representatives and the Committee on Health, 
Education, Labor and Pensions of the Senate a report in 
an electronic format describing the specific recommenda-
tions of the Task Force for improving the access individual 
patients have to investigational drugs pursuant to sub-
section (b) of section 561 of the Federal Food, Drug, and 
Cosmetic Act (21 U.S.C. 360bbb). 
(e) TERMINATION.—The task force shall terminate 
upon submission of the report required under subsection 
(d). 
SEC. 6. FINALIZING DRAFT GUIDANCE ON EXPANDED AC-
CESS. 
(a) IN GENERAL.—Not later than 180 days after the 
date on which the Expanded Access Task Force estab-
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lished under section 5 submits the report under subsection 
(d) of such section, the Secretary of Health and Human 
Services shall finalize the draft guidance entitled ‘‘Ex-
panded Access to Investigational Drugs for Treatment 
Use—Qs & As’’ and dated May 2013. 
(b) CONTENTS.—The final guidance referred to in 
subsection (a) shall— 
(1) clearly define how the Secretary interprets 
and uses adverse drug event data reported by inves-
tigators in the case of data reported from use under 
a request submitted under section 561(b) of the 
Federal Food, Drug, and Cosmetic Act (21 U.S.C. 
360bbb(b)); and 
(2) take into account the report of the Ex-
panded Access Task Force submitted under section 
5(d) and the first report of the Comptroller General 
of the United States submitted under section 4. 
Æ 
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