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. 

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’’. 

7 SEC. 2. EXPANDED ACCESS POLICY AS CONDITION OF EX8 PEDITED APPROVAL. 

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

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


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

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

3 (2) by inserting after subsection (c) the fol4 lowing new subsection: 

5 ‘‘(d) EXPANDED ACCESS POLICY REQUIRED FOR

6 COVERED BREAKTHROUGH DRUGS.— 

7 ‘‘(1) IN GENERAL.—With respect to a qualified 

8 breakthrough drug, not later than 30 days after the 

9 date on which the drug meets the definition of a cov10 ered breakthrough drug (as specified in paragraph 

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

12 shall submit to the Secretary and make publicly 

13 available the policy of the sponsor with respect to re14 quests submitted under subsection (b). In the case 

15 of such a policy under which the sponsor accepts 

16 such requests, such policy shall include— 

17 ‘‘(A) a single point of contact who receives 

18 and processes such requests; 

19 ‘‘(B) procedures for making such requests; 

20 ‘‘(C) the minimum criteria for the spon21 sor’s consideration or approval of such requests; 

22 and 

23 ‘‘(D) the amount of time the sponsor an24 ticipates will be necessary to make a decision on 

25 such requests. 


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1 ‘‘(2) COVERED BREAKTHROUGH DRUG.—In this 

2 subsection, the term ‘covered breakthrough drug’ 

3 means a drug— 

4 ‘‘(A) that is designated as a breakthrough 

5 therapy or as a fast track product or is ap6 proved under accelerated approval under section 

7 506; 

8 ‘‘(B) that is designated under section 

9 505E(d) as a qualified infectious disease prod10 uct; or 

11 ‘‘(C) the sponsor of which is awarded a 

12 priority review voucher under section 524 or 

13 529.’’. 

14 SEC. 3. NOTIFICATION OF SUBMITTERS OF COMPAS15 SIONATE USE REQUESTS. 

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

17 Act (21 U.S.C. 360bbb), as amended by section 2, is fur18 ther amended— 

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

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

21 (g), respectively; and 

22 (2) by inserting after subsection (d) (as in23 serted by section 2(2)) the following new subsection: 

24 ‘‘(e) NOTIFICATION OF SUBMITTERS OF RE25 QUESTS.—In the case of the denial by a manufacturer or 


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

2 than 5 days after the date of such denial, the manufac3 turer or distributor, as applicable, shall submit to the per4 son (or physician) who made the request written notice 

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

6 SEC. 4. GAO QUALITATIVE ANALYSIS ON INDIVIDUAL PA7 TIENT ACCESS TO UNAPPROVED THERAPIES 

8 AND DIAGNOSTICS. 

9 Not later than 180 days after the date of the enact10 ment of this Act and each year thereafter, the Comptroller 

11 General of the United States shall submit to the Com12 mittee on Energy and Commerce of the House of Rep13 resentatives and the Committee on Health, Education, 

14 Labor and Pensions of the Senate a report containing a 

15 qualitative analysis of the extent to which individual pa16 tients have access to investigational drugs pursuant to 

17 subsection (b) of section 561 of the Federal Food, Drug, 

18 and Cosmetic Act (21 U.S.C. 360bbb) and recommenda19 tions for improving such access. In preparing such report, 

20 the Comptroller General shall conduct a qualitative anal21 ysis of the following: 

22 (1) Whether there are any identifiable patterns 

23 in requests submitted under subsection (b) of such 

24 section, such as the types of indications for which 


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1 requests for individual patient access are sought or 

2 the reasons for the denial of such requests. 

3 (2) What the primary barriers are to drug 

4 sponsors granting requests for individual patient ac5 cess. 

6 (3) How the Secretary evaluates safety and effi7 cacy data submitted in connection with such re8 quests. 

9 (4) The amount of time that— 

10 (A) a physician typically takes to complete 

11 the paperwork necessary to make such a re12 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 prac20 tices may be modified, streamlined, expanded, or dis21 continued to reduce or prevent delays in approving 

22 such requests. 

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

24 period covered by the report— 


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1 (A) were approved by drug sponsors and 

2 the Food and Drug Administration; 

3 (B) were approved by drug sponsors but 

4 denied by the Food and Drug Administration; 

5 and 

6 (C) were denied by drug sponsors. 

7 (7) How to encourage drug sponsors to grant 

8 requests for expanded access under such section 

9 561, including requests for emergency use, inter10 mediate-size patient populations, and large patient 

11 populations under a specified indication. 

12 (8) Whether and to what extent adverse events 

13 reported to the Secretary as a result of individual 

14 use of an investigational drug or investigational de15 vice under such section 561 affected the development 

16 or approval of any drug or device. 

17 SEC. 5. EXPANDED ACCESS TASK FORCE. 

18 (a) ESTABLISHMENT.—The Secretary of Health and 

19 Human Services shall establish a task force within the De20 partment of Health and Human Services to explore mech21 anisms for improving the access individual patients have 

22 to investigational drugs pursuant to subsection (b) of sec23 tion 561 of the Federal Food, Drug, and Cosmetic Act 

24 (21 U.S.C. 360bbb), to be known as the ‘‘Expanded Ac25 cess Task Force’’ (in this section referred to as the ‘‘Task 


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1 Force’’). Not later than 90 days after the date on which 

2 the Comptroller General of the United States submits the 

3 first report required under section 4, the Task Force shall 

4 be convened. 

5 (b) MEMBERSHIP.— 

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

7 composed of not more than 9 voting members ap8 pointed as follows: 

9 (A) One member to serve as Chairman of 

10 the Task Force, appointed by the Speaker of 

11 the House of Representatives. 

12 (B) One representative from the Depart13 ment of Health and Human Services, appointed 

14 by the Secretary of Health and Human Serv15 ices. 

16 (C) Four representatives appointed by the 

17 Majority Leader of the House of Representa18 tives, in consultation with the Minority Leader 

19 of the House of Representatives, and the Chair20 man and the Ranking Member of the Com21 mittee on Energy and Commerce of the House 

22 of Representatives, including— 

23 (i) one representative of a biopharma24 ceutical company of less than 250 full-time 

25 employees; 


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

3 (iii) one representative of the health 

4 care provider community; and 

5 (iv) one bioethicist. 

6 (D) Three representatives appointed by 

7 Majority Leader of the Senate, in consultation 

8 with the Minority Leader of the Senate, and the 

9 Chairman and the Ranking Member of the 

10 Health, Education, Labor and Pensions Com11 mittee of the Senate, including— 

12 (i) one representative of the bio13 pharmaceutical industry; 

14 (ii) one representative of the patient 

15 community; and 

16 (iii) one representative of the health 

17 care payor community. 

18 (2) COMPENSATION.—Members of the Task 

19 Force shall serve without compensation. 

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

21 evaluate the access individual patients have to investiga22 tional drugs pursuant to subsection (b) of section 561 of 

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

24 360bbb), taking into account— 


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1 (1) the unique challenges faced by children with 

2 likely fatal diseases for which there is not a com3 parable or satisfactory alternative therapy available; 

4 (2) possible incentives for biopharmaceutical 

5 companies and providers to approve requests sub6 mitted under such subsection; 

7 (3) how the Secretary of Health and Human 

8 Services interprets and takes into consideration ad9 verse event data reported in the case of data from 

10 use under a request submitted under such sub11 section; 

12 (4) ways to streamline and standardize the 

13 process for submitting requests under such sub14 section; and 

15 (5) the costs incurred by biopharmaceutical 

16 companies for the time, effort, and delivery of inves17 tigational drugs to patients for the diagnosis, moni18 toring, or treatment of a serious disease or condition 

19 under such subsection. 

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

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

22 submit to the Committee on Energy and Commerce of the 

23 House of Representatives and the Committee on Health, 

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

25 an electronic format describing the specific recommendaVerDate Sep 11 2014 02:10 Dec 09, 2014 Jkt 049200 PO 00000 Frm 00009 Fmt 6652 Sfmt 6201 E:\BILLS\H5805.IH H5805

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1 tions of the Task Force for improving the access individual 

2 patients have to investigational drugs pursuant to sub3 section (b) of section 561 of the Federal Food, Drug, and 

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

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

6 upon submission of the report required under subsection 

7 (d). 

8 SEC. 6. FINALIZING DRAFT GUIDANCE ON EXPANDED AC9 CESS. 

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

11 date on which the Expanded Access Task Force estab12 lished under section 5 submits the report under subsection 

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

14 Services shall finalize the draft guidance entitled ‘‘Ex15 panded Access to Investigational Drugs for Treatment 

16 Use—Qs & As’’ and dated May 2013. 

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

18 subsection (a) shall— 

19 (1) clearly define how the Secretary interprets 

20 and uses adverse drug event data reported by inves21 tigators in the case of data reported from use under 

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

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

24 360bbb(b)); and 


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1 (2) take into account the report of the Ex2 panded Access Task Force submitted under section 

3 5(d) and the first report of the Comptroller General 

4 of the United States submitted under section 4. 

Æ 


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