Speeding Therapy Access Today Act of 2021

I 117TH CONGRESS 1ST SESSION H. R. 1730 To amend the Federal Food, Drug, and Cosmetic Act to accelerate develop- ment of therapies across the spectrum of rare diseases and conditions and facilitate patient access to such therapies, and for other purposes. IN THE HOUSE OF REPRESENTATIVES MARCH 10, 2021 Mr. BILIRAKIS (for himself and Mr. BUTTERFIELD) 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 accelerate development of therapies across the spectrum of rare diseases and conditions and facilitate patient access to such therapies, 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 ‘‘Speeding Therapy Ac- 4 cess Today Act of 2021’’. 5 SEC. 2. TABLE OF CONTENTS. 6 The table of contents of this Act is as follows: 7 Sec. 1. Short title. Sec. 2. Table of contents. Sec. 3. Intercenter Institute on Rare Diseases and Conditions. VerDate Sep 11 2014 01:39 May 07, 2021 Jkt 019200 PO 00000 Frm 00001 Fmt 6652 Sfmt 6211 E:\BILLS\H1730.IH H1730 pamtmann on DSKBC07HB2PROD with BILLS 2 •HR 1730 IH Sec. 4. Rare Disease and Condition Drug Advisory Committee. Sec. 5. Grants and contracts for development of drugs for rare diseases and conditions. SEC. 3. INTERCENTER INSTITUTE ON RARE DISEASES AND 1 CONDITIONS. 2 (a) ESTABLISHMENT REQUIRED.—The first sentence 3 of section 1014(a) of the Federal Food, Drug, and Cos- 4 metic Act (21 U.S.C. 399g(a)) is amended by inserting 5 ‘‘, at least one of which shall be focused on rare diseases 6 and conditions’’ before the period at the end of the sen- 7 tence. 8 (b) TIMING OF ESTABLISHMENT.—Subsection (c) of 9 section 1014 of the Federal Food, Drug, and Cosmetic 10 Act (21 U.S.C. 399g) is amended to read as follows: 11 ‘‘(c) TIMING.—Not later than the date that is 1 year 12 after the date of enactment of the Speeding Therapy Ac- 13 cess Today Act of 2021, the Secretary shall establish, in 14 accordance with this section and section 529B, an Insti- 15 tute under subsection (a) focused on rare diseases and 16 conditions, to be known as the Intercenter Institute on 17 Rare Diseases and Conditions.’’. 18 (c) RESPONSIBILITIES.—Subchapter B of chapter V 19 of the Federal Food, Drug, and Cosmetic Act (relating 20 to drugs for rare diseases or conditions) is amended by 21 inserting after section 529A of such Act (21 U.S.C. 360ff– 22 1) the following new section: 23 VerDate Sep 11 2014 01:39 May 07, 2021 Jkt 019200 PO 00000 Frm 00002 Fmt 6652 Sfmt 6201 E:\BILLS\H1730.IH H1730 pamtmann on DSKBC07HB2PROD with BILLS 3 •HR 1730 IH ‘‘SEC. 529B. INTERCENTER INSTITUTE ON RARE DISEASES 1 AND CONDITIONS. 2 ‘‘(a) RESPONSIBILITIES.—In addition to carrying out 3 activities listed in section 1014(a), the Intercenter Insti- 4 tute on Rare Diseases and Conditions shall— 5 ‘‘(1) serve as the Food and Drug Administra- 6 tion’s coordinating office for engagement with rare 7 disease and condition stakeholders, complementing 8 but not supplanting engagement activities between 9 stakeholders and the review divisions; 10 ‘‘(2) build, within the Food and Drug Adminis- 11 tration, knowledge and understanding associated 12 with the review of medical products to treat rare dis- 13 eases and conditions, including advancements in trial 14 design, statistical analysis, regulatory science, prod- 15 uct manufacturing, and other topics as determined 16 by the Secretary; 17 ‘‘(3) implement cross-center rare disease and 18 condition-focused meetings and policy development; 19 ‘‘(4) coordinate rare disease and condition-spe- 20 cific regulatory science initiatives; 21 ‘‘(5) facilitate stakeholder engagement to the 22 external community and international regulatory 23 agencies on rare disease and condition product devel- 24 opment; 25 VerDate Sep 11 2014 01:39 May 07, 2021 Jkt 019200 PO 00000 Frm 00003 Fmt 6652 Sfmt 6201 E:\BILLS\H1730.IH H1730 pamtmann on DSKBC07HB2PROD with BILLS 4 •HR 1730 IH ‘‘(6) establish and implement the Accelerating 1 Lifesavings Therapies in Treating Ultra-rare Dis- 2 ease Entities Program under subsection (b); and 3 ‘‘(7) establish and carry out the rare disease 4 and condition third-party payor program under sub- 5 section (d). 6 ‘‘(b) ALTITUDE PROGRAM.— 7 ‘‘(1) IN GENERAL.—The Intercenter Institute 8 shall establish and implement a program, to be 9 known as the Accelerating Lifesavings Therapies in 10 Treating Ultra-rare Disease Entities Program, to 11 identify and make recommendations to address cur- 12 rent and emerging regulatory science and public pol- 13 icy challenges associated with developing medical 14 products to treat rare diseases or conditions in an 15 individual or very small populations. 16 ‘‘(2) ISSUES.—The program under paragraph 17 (1) shall focus on issues including— 18 ‘‘(A) manufacturing standards for thera- 19 pies described in such paragraph, including in 20 non-industry settings; 21 ‘‘(B) trial designs and metrics; 22 ‘‘(C) regulatory flexibilities for abbreviated 23 toxicology studies, overlapping animal studies, 24 and patient dosing; 25 VerDate Sep 11 2014 01:39 May 07, 2021 Jkt 019200 PO 00000 Frm 00004 Fmt 6652 Sfmt 6201 E:\BILLS\H1730.IH H1730 pamtmann on DSKBC07HB2PROD with BILLS 5 •HR 1730 IH ‘‘(D) regulatory science, chemistry, manu- 1 facturing, and other needs associated with de- 2 veloping such therapies; and 3 ‘‘(E) other issues as determined by the 4 Secretary. 5 ‘‘(c) PROPOSALS FOR AMENDING LABELS.— 6 ‘‘(1) STAKEHOLDER GROUP.—Not later than 7 180 days after the date of enactment of this section, 8 the Intercenter Institute shall convene a meeting of 9 stakeholders from the rare disease community, in- 10 cluding patients, caregivers, product manufacturers, 11 third-party payors, and others, to consider potential 12 amendments to labels for medical products to treat 13 rare diseases or conditions approved pursuant to a 14 pathway under section 506. 15 ‘‘(2) GUIDANCE.—Not later than 90 days after 16 the date of the meeting under paragraph (1), the 17 Secretary shall issue guidance to propose changes to 18 how the labels of medical products to treat rare dis- 19 eases or conditions demonstrate clinical benefits and 20 reflect relevant scientific data including surrogate 21 endpoints. 22 ‘‘(d) RARE DISEASE AND CONDITION THIRD-PARTY 23 PAYOR PROGRAM.— 24 VerDate Sep 11 2014 01:39 May 07, 2021 Jkt 019200 PO 00000 Frm 00005 Fmt 6652 Sfmt 6201 E:\BILLS\H1730.IH H1730 pamtmann on DSKBC07HB2PROD with BILLS 6 •HR 1730 IH ‘‘(1) IN GENERAL.—The Intercenter Institute 1 shall establish and carry out a voluntary rare disease 2 and condition early third-party payor feedback pro- 3 gram— 4 ‘‘(A) to inform coverage policies for rare 5 disease therapies; and 6 ‘‘(B) to inform clinical trial design, patient 7 engagement, and other data collections. 8 ‘‘(2) PROGRAM REQUIREMENTS.—The program 9 under paragraph (1) shall— 10 ‘‘(A) facilitate voluntary communication 11 between sponsors of medical products to treat 12 rare diseases and conditions and third-party 13 payors; and 14 ‘‘(B) require participation of the Centers 15 for Medicare & Medicaid Services with rep- 16 resentation from— 17 ‘‘(i) the Center for Medicare; and 18 ‘‘(ii) the Center for Medicaid and 19 CHIP Services. 20 ‘‘(3) ANNUAL REPORT.—The Intercenter Insti- 21 tute shall— 22 ‘‘(A) on an annual basis, submit a report 23 to that Congress on— 24 VerDate Sep 11 2014 01:39 May 07, 2021 Jkt 019200 PO 00000 Frm 00006 Fmt 6652 Sfmt 6201 E:\BILLS\H1730.IH H1730 pamtmann on DSKBC07HB2PROD with BILLS 7 •HR 1730 IH ‘‘(i) the participation within the pro- 1 gram under paragraph (1); and 2 ‘‘(ii) the impacts of the program 3 under paragraph (1); and 4 ‘‘(B) post each such report on the public 5 website of the Intercenter Institute. 6 ‘‘(4) BULLETIN TO MEDICAID DIRECTORS.— 7 Following the approval, clearance, or authorization 8 by the Food and Drug Administration of a medical 9 product to treat a rare disease or condition, the Sec- 10 retary shall issue a bulletin to State Medicaid direc- 11 tors containing information to help inform coverage 12 decisions on the product by State Medicaid and Chil- 13 dren’s Health Insurance programs. 14 ‘‘(e) DEFINITION.—In this section, the terms ‘Inter- 15 center Institute on Rare Diseases and Conditions’ and 16 ‘Intercenter Institute’ refer to the Intercenter Institute on 17 Rare Diseases and Conditions established pursuant to sec- 18 tion 1014.’’. 19 SEC. 4. RARE DISEASE AND CONDITION DRUG ADVISORY 20 COMMITTEE. 21 Subchapter B of chapter V of the Federal Food, 22 Drug, and Cosmetic Act is further amended by inserting 23 after section 529B of such Act, as inserted by section 3, 24 the following new section: 25 VerDate Sep 11 2014 01:39 May 07, 2021 Jkt 019200 PO 00000 Frm 00007 Fmt 6652 Sfmt 6201 E:\BILLS\H1730.IH H1730 pamtmann on DSKBC07HB2PROD with BILLS 8 •HR 1730 IH ‘‘SEC. 529C. RARE DISEASE AND CONDITION DRUG ADVI- 1 SORY COMMITTEE. 2 ‘‘(a) IN GENERAL.—The Secretary shall establish 3 and maintain a committee, to be known as the Rare Dis- 4 ease and Condition Drug Advisory Committee (in this sec- 5 tion referred to as the ‘Advisory Committee’). 6 ‘‘(b) DUTY OF COMMITTEE.—The Advisory Com- 7 mittee shall advise the Secretary on issues associated with 8 development of therapies to treat rare diseases or condi- 9 tions. 10 ‘‘(c) SPECIFIC ISSUES.—In advising the Secretary, 11 the Advisory Committee may address issues including— 12 ‘‘(1) modified or new regulatory pathways to 13 support review of therapies; 14 ‘‘(2) clinical trial design needs, including devel- 15 opment of innovative approaches to clinical trials; 16 ‘‘(3) qualifications of biomarkers or other drug 17 development tools for use in reviews; 18 ‘‘(4) modified or new standards to support the 19 review of already marketed drugs being evaluated 20 for repurposing to treat a rare disease or condition; 21 and 22 ‘‘(5) issues— 23 ‘‘(A) that pertain to an application for ap- 24 proval of a therapy to treat a rare disease or 25 condition; and 26 VerDate Sep 11 2014 01:39 May 07, 2021 Jkt 019200 PO 00000 Frm 00008 Fmt 6652 Sfmt 6201 E:\BILLS\H1730.IH H1730 pamtmann on DSKBC07HB2PROD with BILLS 9 •HR 1730 IH ‘‘(B) with respect to which a review divi- 1 sion has requested that the Advisory Committee 2 provide advice. 3 ‘‘(d) MEMBERSHIP.— 4 ‘‘(1) IN GENERAL.—The Advisory Committee 5 shall consist of— 6 ‘‘(A) not more than 15 members appointed 7 by the Secretary in accordance with paragraph 8 (2); and 9 ‘‘(B) the nonvoting ex officio members 10 under paragraph (3). 11 ‘‘(2) APPOINTED MEMBERS.— 12 ‘‘(A) SPECIAL GOVERNMENT EMPLOY- 13 EES.—Members of the Advisory Committee ap- 14 pointed pursuant to paragraph (1)(A) shall 15 serve as special Government employees (as de- 16 fined in section 202(a) of title 18, United 17 States Code). 18 ‘‘(B) ELIGIBILITY.—To be eligible for ap- 19 pointment pursuant to paragraph (1)(A), an in- 20 dividual shall— 21 ‘‘(i) be eligible to serve as special Gov- 22 ernment employee (as defined in section 23 202(a) of title 18, United States Code); 24 and 25 VerDate Sep 11 2014 01:39 May 07, 2021 Jkt 019200 PO 00000 Frm 00009 Fmt 6652 Sfmt 6201 E:\BILLS\H1730.IH H1730 pamtmann on DSKBC07HB2PROD with BILLS 10 •HR 1730 IH ‘‘(ii) have expertise in the fields of 1 public policy, law, regulatory policy, eco- 2 nomics, patient-focused product develop- 3 ment, or patient advocacy. 4 ‘‘(C) COMPOSITION.—Of the members of 5 the Advisory Committee appointed pursuant to 6 paragraph (1)(A)— 7 ‘‘(i) up to 10 shall be selected from 8 among experts in the disciplines relevant to 9 the activities of the Intercenter Institute 10 on Rare Diseases and Conditions, to in- 11 clude at least one expert in each of— 12 ‘‘(I) rare disease product develop- 13 ment; 14 ‘‘(II) conducting clinical trials 15 with respect to rare diseases and con- 16 ditions, including with respect to very 17 small patient populations; 18 ‘‘(III) rare disease and condition 19 natural history and related studies; 20 ‘‘(IV) health economics per- 21 taining to the development of medical 22 products for rare diseases or condi- 23 tions; 24 VerDate Sep 11 2014 01:39 May 07, 2021 Jkt 019200 PO 00000 Frm 00010 Fmt 6652 Sfmt 6201 E:\BILLS\H1730.IH H1730 pamtmann on DSKBC07HB2PROD with BILLS 11 •HR 1730 IH ‘‘(V) manufacturing and related 1 needs associated with medical prod- 2 ucts for rare diseases or conditions; 3 and 4 ‘‘(VI) patient experience data col- 5 lection; and 6 ‘‘(ii) up to 5 shall be selected from the 7 public, to include— 8 ‘‘(I) at least 4 individuals who 9 are representatives of the rare disease 10 patient community; 11 ‘‘(II) at least one individual who 12 is directly impacted by a rare disease 13 or condition; and 14 ‘‘(III) at least one person who 15 serves as a family caregiver to a per- 16 son diagnosed with a rare disease or 17 condition. 18 ‘‘(3) NONVOTING EX OFFICIO MEMBERS.—The 19 nonvoting ex officio members of the Advisory Com- 20 mittee under paragraph (1)(B) shall consist of the 21 following: 22 ‘‘(A) The Secretary (or the Secretary’s 23 designee). 24 VerDate Sep 11 2014 01:39 May 07, 2021 Jkt 019200 PO 00000 Frm 00011 Fmt 6652 Sfmt 6201 E:\BILLS\H1730.IH H1730 pamtmann on DSKBC07HB2PROD with BILLS 12 •HR 1730 IH ‘‘(B) The Director of the Intercenter Insti- 1 tute on Rare Diseases and Conditions. 2 ‘‘(C) The Director of the Center for Bio- 3 logics Evaluation and Research (or the Direc- 4 tor’s designee). 5 ‘‘(D) The Director of the Center for Drug 6 Evaluation and Research (or the Director’s des- 7 ignee). 8 ‘‘(E) The Director of the Center for De- 9 vices and Radiological Health (or the Director’s 10 designee). 11 ‘‘(F) The Director of the National Center 12 for the Advancing Translational Sciences of the 13 National Institutes of Health (or the Director’s 14 designee). 15 ‘‘(G) The Administrator of the Centers for 16 Medicare & Medicaid Services (or the Adminis- 17 trator’s designee). 18 ‘‘(H) Any additional officers or employees 19 of the Department of Health and Human Serv- 20 ices as the Secretary determines necessary for 21 the Advisory Committee to effectively carry out 22 its functions. 23 VerDate Sep 11 2014 01:39 May 07, 2021 Jkt 019200 PO 00000 Frm 00012 Fmt 6652 Sfmt 6201 E:\BILLS\H1730.IH H1730 pamtmann on DSKBC07HB2PROD with BILLS 13 •HR 1730 IH ‘‘(4) CHAIR.—The Chair of the Advisory Com- 1 mittee shall be the Director of the Intercenter Insti- 2 tute for Rare Diseases and Conditions. 3 ‘‘(5) TERMS.— 4 ‘‘(A) MEMBERS.— 5 ‘‘(i) IN GENERAL.—The term of a 6 member of the Advisory Committee ap- 7 pointed pursuant to paragraph (1)(A) shall 8 be 4 ye [Text truncated for display. 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