Helping Experts Accelerate Rare Treatments Act of 2020

I 116TH CONGRESS 2D SESSION H. R. 7567 To amend the Federal Food, Drug, and Cosmetic Act to improve the treatment of rare diseases and conditions, and for other purposes. IN THE HOUSE OF REPRESENTATIVES JULY 9, 2020 Mr. TONKO (for himself and Mr. MCKINLEY) 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 to improve the treatment of rare diseases and conditions, and for other purposes. Be it enacted by the Senate and House of Representa- 1 tives of the United States of America in Congress assembled, 2 SECTION 1. SHORT TITLE. 3 This Act may be cited as the ‘‘Helping Experts Accel- 4 erate Rare Treatments Act of 2020’’. 5 SEC. 2. IMPROVING THE TREATMENT OF RARE DISEASES 6 AND CONDITIONS. 7 (a) ANNUAL REPORT ON ORPHAN DRUG PRO- 8 GRAM.—Subchapter B of chapter V of the Federal Food, 9 VerDate Sep 11 2014 00:03 Aug 07, 2020 Jkt 099200 PO 00000 Frm 00001 Fmt 6652 Sfmt 6201 E:\BILLS\H7567.IH H7567 pbinns on DSKJLVW7X2PROD with BILLS 2 •HR 7567 IH Drug, and Cosmetic Act (21 U.S.C. 360aa et seq.) is 1 amended by adding at the end the following new section: 2 ‘‘SEC. 529B. ANNUAL REPORT ON ORPHAN DRUG PROGRAM. 3 ‘‘(a) IN GENERAL.—Not later than 1 year after the 4 date of enactment of the Helping Experts Accelerate Rare 5 Treatments Act of 2020, and not less than annually there- 6 after, the Secretary shall submit to the Congress a report 7 summarizing the activities of the Food and Drug Adminis- 8 tration for designating drugs under section 526 for a rare 9 disease or condition and approving such drugs under sec- 10 tion 505 of this Act or licensing such drugs under section 11 351 of the Public Health Service Act, including— 12 ‘‘(1) the number of applications for such drugs 13 under sections 505 of this Act and section 351 of 14 the Public Health Service Act received by the Food 15 and Drug Administration, the number of such appli- 16 cations considered by each Food and Drug Adminis- 17 tration division, and the number of such applications 18 pending and approved; 19 ‘‘(2) the number of applications described in 20 paragraph (1) disaggregated by— 21 ‘‘(A) the disease or condition to be ad- 22 dressed; and 23 ‘‘(B) the target subpopulations identified 24 in such applications; 25 VerDate Sep 11 2014 00:03 Aug 07, 2020 Jkt 099200 PO 00000 Frm 00002 Fmt 6652 Sfmt 6201 E:\BILLS\H7567.IH H7567 pbinns on DSKJLVW7X2PROD with BILLS 3 •HR 7567 IH ‘‘(3) the prevalence in the United States of each 1 disease or condition addressed by an application de- 2 scribed in paragraph (1); 3 ‘‘(4) integration of Food and Drug Administra- 4 tion staff with expertise in rare diseases and condi- 5 tions in each review of an application described in 6 paragraph (1); 7 ‘‘(5) identification of all external experts with 8 expertise in rare diseases and conditions who are 9 consulted in connection with, or who otherwise par- 10 ticipate in, each review of an application described in 11 paragraph (1); and 12 ‘‘(6) the status of each application described in 13 paragraph (1). 14 ‘‘(b) PUBLIC AVAILABILITY.—The Secretary shall 15 make each report under subsection (a) available to the 16 public, including by posting the report on the website of 17 the Food and Drug Administration.’’. 18 (b) STUDY ON EUROPEAN UNION SAFETY AND EFFI- 19 CACY REVIEWS OF DRUGS FOR RARE DISEASES AND CON- 20 DITIONS.— 21 (1) IN GENERAL.—The Comptroller General of 22 the United States shall conduct a study on Euro- 23 pean Union safety and efficacy reviews of drugs for 24 rare diseases and conditions, including— 25 VerDate Sep 11 2014 00:03 Aug 07, 2020 Jkt 099200 PO 00000 Frm 00003 Fmt 6652 Sfmt 6201 E:\BILLS\H7567.IH H7567 pbinns on DSKJLVW7X2PROD with BILLS 4 •HR 7567 IH (A) any differential prevalence-based mech- 1 anisms; and 2 (B) consideration and use of supplemental 3 data submitted during the review process, in- 4 cluding data associated with open label exten- 5 sion studies and expanded access programs. 6 (2) REPORT.—Not later than 1 year after the 7 date of enactment of this Act, the Comptroller Gen- 8 eral shall— 9 (A) complete the study under paragraph 10 (1); 11 (B) submit a report on the results of such 12 study to the Congress; and 13 (C) include in such report recommenda- 14 tions for changes to the processes and authori- 15 ties of the Food and Drug Administration to fa- 16 cilitate development of, and access to, treat- 17 ments for rare diseases and conditions. 18 (3) PUBLIC AVAILABILITY.—The Comptroller 19 General of the United States shall make each report 20 under paragraph (2) available to the public, includ- 21 ing by posting the report on the website of the Gov- 22 ernment Accountability Office. 23 VerDate Sep 11 2014 00:03 Aug 07, 2020 Jkt 099200 PO 00000 Frm 00004 Fmt 6652 Sfmt 6201 E:\BILLS\H7567.IH H7567 pbinns on DSKJLVW7X2PROD with BILLS 5 •HR 7567 IH (c) REVIEW PROCESS.—Subparagraph (A) of section 1 569(a)(2) of the Federal Food, Drug, and Cosmetic Act 2 (21 U.S.C. 360bbb–8(a)(2)) is amended to read as follows: 3 ‘‘(A) IN GENERAL.— 4 ‘‘(i) LIST.—The Secretary shall de- 5 velop and maintain a list of external ex- 6 perts who, because of their special exper- 7 tise, are qualified to provide advice on rare 8 disease and condition issues, including top- 9 ics described in subsection (b). 10 ‘‘(ii) AVAILABILITY AT MEETINGS.— 11 The Secretary shall ensure availability, for 12 consultation purposes, of an external ex- 13 pert on rare diseases, as described in sub- 14 paragraph (B), in connection with each 15 drug product advisory committee meeting 16 concerning a drug or biological product for 17 a rare disease or condition. 18 ‘‘(iii) REVIEW OF APPLICATIONS.— 19 The Secretary shall ensure that each re- 20 view of an application submitted under sec- 21 tion 505 of this Act or section 351 of the 22 Public Health Service Act for an indication 23 associated with a rare disease or condition 24 includes Food and Drug Administration 25 VerDate Sep 11 2014 00:03 Aug 07, 2020 Jkt 099200 PO 00000 Frm 00005 Fmt 6652 Sfmt 6201 E:\BILLS\H7567.IH H7567 pbinns on DSKJLVW7X2PROD with BILLS 6 •HR 7567 IH staff with expertise on rare diseases and 1 conditions as an integral part of the review 2 team.’’. 3 (d) RISK EVALUATION AND MITIGATION STRATEGY 4 ELEMENTS.—Subsection (a) of section 505–1 of the Fed- 5 eral Food, Drug, and Cosmetic Act (21 U.S.C. 355–1) is 6 amended by adding at the end the following new para- 7 graph: 8 ‘‘(5) ORPHAN DRUGS.—In making any deter- 9 mination regarding the inclusion, in the risk evalua- 10 tion and mitigation strategy for a drug that has 11 been designated as a drug for a rare disease or con- 12 dition pursuant to section 526, of an element that 13 requires patient action, restrictions on use of the 14 drug, or patient participation, the Secretary shall 15 consult with patients impacted by the rare disease or 16 condition in considering the burden of participation 17 and likely patient compliance.’’. 18 Æ VerDate Sep 11 2014 00:03 Aug 07, 2020 Jkt 099200 PO 00000 Frm 00006 Fmt 6652 Sfmt 6301 E:\BILLS\H7567.IH H7567 pbinns on DSKJLVW7X2PROD with BILLS