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. 

1 Be it enacted by the Senate and House of Representa2 tives of the United States of America in Congress assembled, 

3 SECTION 1. SHORT TITLE. 

4 This Act may be cited as the ‘‘Andrea Sloan Compas5 sionate Use Reform and Enhancement Act’’ or the ‘‘An6 drea Sloan CURE Act’’. 


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1 SEC. 2. EXPANDED ACCESS POLICY AS CONDITION OF EX2 PEDITED APPROVAL. 

3 Section 561 of the Federal Food, Drug, and Cosmetic 

4 Act (21 U.S.C. 360bbb) is amended— 

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

6 subsections (e) and (f), respectively; and 

7 (2) by inserting after subsection (c) the fol8 lowing new subsection: 

9 ‘‘(d) EXPANDED ACCESS POLICY REQUIRED FOR

10 COVERED INVESTIGATIONAL DRUGS.— 

11 ‘‘(1) IN GENERAL.—With respect to a covered 

12 investigational drug, not later than 30 days after the 

13 date on which the drug meets the definition of a cov14 ered investigational drug (as specified in paragraph 

15 (2)), the sponsor of the covered investigational drug 

16 shall submit to the Secretary, and make publicly 

17 available, the policy of the sponsor with respect to 

18 requests submitted under subsection (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 general criteria for the sponsor’s 

25 consideration or approval of such requests; and 


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1 ‘‘(D) the amount of time the sponsor an2 ticipates will be necessary to respond to such 

3 requests. 

4 ‘‘(2) COVERED INVESTIGATIONAL DRUG.—In 

5 this subsection, the term ‘covered investigational 

6 drug’ means a drug that— 

7 ‘‘(A) is designated as a breakthrough ther8 apy or as a fast track product; 

9 ‘‘(B) is designated under section 505E(d) 

10 as a qualified infectious disease product; or 

11 ‘‘(C) is designated under section 526 as a 

12 drug for a rare disease or condition.’’. 

13 SEC. 3. NOTIFICATION OF SUBMITTERS OF EXPANDED AC14 CESS REQUESTS. 

15 Section 561 of the Federal Food, Drug, and Cosmetic 

16 Act (21 U.S.C. 360bbb), as amended by section 2, is fur17 ther amended— 

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

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

20 (g), respectively; and 

21 (2) by inserting after subsection (d) (as in22 serted by section 2(2)) the following new subsection: 

23 ‘‘(e) NOTIFICATION OF SUBMITTERS OF RE24 QUESTS.—In the case of the denial by a manufacturer or 

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


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1 than 5 days after the date of such denial, the manufac2 turer or distributor, as applicable, shall submit to the per3 son (or physician) who made the request written notice 

4 of the denial, including an explanation for the denial.’’. 

5 SEC. 4. GAO QUALITATIVE ANALYSIS ON INDIVIDUAL PA6 TIENT ACCESS TO UNAPPROVED THERAPIES 

7 AND DIAGNOSTICS. 

8 Not later than 180 days after the date of the enact9 ment of this Act and every two years thereafter through 

10 2023, the Comptroller General of the United States shall 

11 submit to the Committee on Energy and Commerce of the 

12 House of Representatives and the Committee on Health, 

13 Education, Labor and Pensions of the Senate a report 

14 containing a qualitative analysis of the extent to which in15 dividual patients have access to investigational drugs pur16 suant to subsection (b) of section 561 of the Federal Food, 

17 Drug, and Cosmetic Act (21 U.S.C. 360bbb) and rec18 ommendations for improving such access. In preparing 

19 such report, the Comptroller General shall conduct a qual20 itative analysis of the following: 

21 (1) Whether there are any identifiable patterns 

22 in requests submitted under subsection (b) of such 

23 section, such as the types of indications for which 

24 requests for individual patient access are sought or 

25 the reasons for the denial of such requests. 


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1 (2) What the primary barriers are to drug 

2 sponsors granting requests for individual patient ac3 cess. 

4 (3) How the Secretary evaluates safety and effi5 cacy data submitted in connection with such re6 quests. 

7 (4) The amount of time that— 

8 (A) a physician typically takes to complete 

9 the paperwork necessary to make such a re10 quest; 

11 (B) a drug sponsor takes to process such 

12 a request and to issue a decision with respect 

13 to the request; and 

14 (C) the Secretary takes to process such a 

15 request and to issue a decision with respect to 

16 the request. 

17 (5) How regulations, guidance, policies, or prac18 tices may be modified, streamlined, expanded, or dis19 continued to reduce or prevent delays in approving 

20 such requests. 

21 (6) The number of such requests that, for the 

22 period covered by the report— 

23 (A) were approved by drug sponsors and 

24 the Food and Drug Administration; 


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1 (B) were approved by drug sponsors but 

2 denied by the Food and Drug Administration; 

3 and 

4 (C) were denied by drug sponsors. 

5 (7) How to encourage drug sponsors to grant 

6 requests for expanded access under such section 

7 561, including requests for emergency use, inter8 mediate-size patient populations, and large patient 

9 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 de13 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 first report required under section 4, the Task Force shall 

2 be convened. 

3 (b) MEMBERSHIP.— 

4 (1) COMPOSITION.—The Task Force shall be 

5 composed of not more than 13 voting members ap6 pointed as follows: 

7 (A) One member to serve as Chairman of 

8 the Task Force, appointed by the Speaker of 

9 the House of Representatives. 

10 (B) One representative from the Depart11 ment of Health and Human Services, appointed 

12 by the Secretary of Health and Human Serv13 ices. 

14 (C) Six representatives appointed by the 

15 majority leader of the House of Representa16 tives, in consultation with the minority leader of 

17 the House of Representatives, and the chairman 

18 and the ranking member of the Committee on 

19 Energy and Commerce of the House of Rep20 resentatives, including— 

21 (i) one current or former representa22 tive of the biopharmaceutical industry of 

23 not less than 250 full-time employees; 


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1 (ii) one representative of a biopharma2 ceutical company of less than 250 full-time 

3 employees; 

4 (iii) one representative of the patient 

5 community; 

6 (iv) one representative of the rare dis7 ease patient community; 

8 (v) one representative of the health 

9 care provider community; and 

10 (vi) one bioethicist. 

11 (D) Five representatives appointed by ma12 jority leader of the Senate, in consultation with 

13 the minority leader of the Senate, and the 

14 chairman and the ranking member of the Com15 mittee on Health, Education, Labor and Pen16 sions of the Senate, including— 

17 (i) one representative of the bio18 pharmaceutical industry of not less than 

19 250 full-time employees; 

20 (ii) one current or former representa21 tive of a biopharmaceutical company of 

22 less than 250 full-time employees; 

23 (iii) one representative of the patient 

24 community; 


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1 (iv) one representative of the rare dis2 ease patient community; and 

3 (v) one representative of the health 

4 care payor community. 

5 (2) COMPENSATION.—Members of the Task 

6 Force shall serve without compensation. 

7 (c) DUTIES.—The Task Force shall comprehensively 

8 evaluate the access individual patients have to investiga9 tional drugs pursuant to subsection (b) of section 561 of 

10 the Federal Food, Drug, and Cosmetic Act (21 U.S.C. 

11 360bbb), taking into account— 

12 (1) the unique challenges faced by children with 

13 likely fatal diseases for which there is not a com14 parable or satisfactory alternative therapy available; 

15 (2) possible incentives for biopharmaceutical 

16 companies and providers to approve requests sub17 mitted under such subsection; 

18 (3) ways to improve followup reporting of ad19 verse event data and compliance with such reporting 

20 requirements; 

21 (4) how the Secretary of Health and Human 

22 Services interprets and takes into consideration ad23 verse event data reported in the case of data from 

24 use under a request submitted under such sub25 section; 


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1 (5) ways to streamline and standardize the 

2 process for submitting requests under such sub3 section; and 

4 (6) the costs incurred by biopharmaceutical 

5 companies for the time, effort, and delivery of inves6 tigational drugs to patients for the diagnosis, moni7 toring, or treatment of a serious disease or condition 

8 under such subsection. 

9 (d) REPORT.—Not later than 180 days after the date 

10 on which the Task Force is convened, the Task Force shall 

11 submit to the Committee on Energy and Commerce of the 

12 House of Representatives and the Committee on Health, 

13 Education, Labor and Pensions of the Senate a report in 

14 an electronic format describing the specific recommenda15 tions of the Task Force for improving the access individual 

16 patients have to investigational drugs pursuant to sub17 section (b) of section 561 of the Federal Food, Drug, and 

18 Cosmetic Act (21 U.S.C. 360bbb). 

19 (e) TERMINATION.—The task force shall terminate 

20 upon submission of the report required under subsection 

21 (d). 

22 SEC. 6. FINALIZING DRAFT GUIDANCE ON EXPANDED AC23 CESS. 

24 (a) IN GENERAL.—Not later than 180 days after the 

25 date on which the Expanded Access Task Force estabVerDate Sep 11 2014 22:20 Feb 19, 2015 Jkt 049200 PO 00000 Frm 00010 Fmt 6652 Sfmt 6201 E:\BILLS\H909.IH H909

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1 lished under section 5 submits the report under subsection 

2 (d) of such section, the Secretary of Health and Human 

3 Services shall finalize the draft guidance entitled ‘‘Ex4 panded Access to Investigational Drugs for Treatment 

5 Use—Qs & As’’ and dated May 2013. 

6 (b) CONTENTS.—The final guidance referred to in 

7 subsection (a) shall— 

8 (1) clearly define how the Secretary interprets 

9 and uses adverse drug event data reported by inves10 tigators in the case of data reported from use under 

11 a request submitted under section 561(b) of the 

12 Federal Food, Drug, and Cosmetic Act (21 U.S.C. 

13 360bbb(b)); and 

14 (2) take into account the report of the Ex15 panded Access Task Force submitted under section 

16 5(d) and the first report of the Comptroller General 

17 of the United States submitted under section 4. 

Æ 


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