II 

117TH CONGRESS 

1ST SESSION S. 670 

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. 

IN THE SENATE OF THE UNITED STATES 

MARCH 10, 2021 

Ms. KLOBUCHAR (for herself and Mr. WICKER) introduced the following bill; 

which was read twice and referred to the Committee on Health, Education, Labor, and Pensions 

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. 

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 ‘‘Speeding Therapy Ac5 cess Today Act of 2021’’. 

6 SEC. 2. TABLE OF CONTENTS. 

7 The table of contents of this Act is as follows: 

Sec. 1. Short title. 


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Sec. 2. Table of contents. 

Sec. 3. Intercenter Institute on Rare Diseases and Conditions. 

Sec. 4. Rare Disease and Condition Drug Advisory Committee. 

Sec. 5. Grants and contracts for development of drugs for rare diseases and 

conditions. 

1 SEC. 3. INTERCENTER INSTITUTE ON RARE DISEASES AND 

2 CONDITIONS. 

3 (a) ESTABLISHMENT REQUIRED.—The first sentence 

4 of section 1014(a) of the Federal Food, Drug, and Cos5 metic Act (21 U.S.C. 399g(a)) is amended by inserting 

6 ‘‘, at least one of which shall be focused on rare diseases 

7 and conditions’’ before the period at the end of the sen8 tence. 

9 (b) TIMING OF ESTABLISHMENT.—Subsection (c) of 

10 section 1014 of the Federal Food, Drug, and Cosmetic 

11 Act (21 U.S.C. 399g) is amended to read as follows: 

12 ‘‘(c) TIMING.—Not later than the date that is 1 year 

13 after the date of enactment of the Speeding Therapy Ac14 cess Today Act of 2021, the Secretary shall establish, in 

15 accordance with this section and section 529B, an Insti16 tute under subsection (a) focused on rare diseases and 

17 conditions, to be known as the Intercenter Institute on 

18 Rare Diseases and Conditions.’’. 

19 (c) RESPONSIBILITIES.—Subchapter B of chapter V 

20 of the Federal Food, Drug, and Cosmetic Act (relating 

21 to drugs for rare diseases or conditions) is amended by 

22 inserting after section 529A of such Act (21 U.S.C. 360ff– 

23 1) the following new section: 


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1 ‘‘SEC. 529B. INTERCENTER INSTITUTE ON RARE DISEASES 

2 AND CONDITIONS. 

3 ‘‘(a) RESPONSIBILITIES.—In addition to carrying out 

4 activities listed in section 1014(a), the Intercenter Insti5 tute on Rare Diseases and Conditions shall— 

6 ‘‘(1) serve as the Food and Drug Administra7 tion’s coordinating office for engagement with rare 

8 disease and condition stakeholders, complementing 

9 but not supplanting engagement activities between 

10 stakeholders and the review divisions; 

11 ‘‘(2) build, within the Food and Drug Adminis12 tration, knowledge and understanding associated 

13 with the review of medical products to treat rare dis14 eases and conditions, including advancements in trial 

15 design, statistical analysis, regulatory science, prod16 uct manufacturing, and other topics as determined 

17 by the Secretary; 

18 ‘‘(3) implement cross-center rare disease and 

19 condition-focused meetings and policy development; 

20 ‘‘(4) coordinate rare disease and condition-spe21 cific regulatory science initiatives; 

22 ‘‘(5) facilitate stakeholder engagement to the 

23 external community and international regulatory 

24 agencies on rare disease and condition product devel25 opment; 


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1 ‘‘(6) establish and implement the Accelerating 

2 Lifesavings Therapies in Treating Ultra-rare Dis3 ease Entities Program under subsection (b); and 

4 ‘‘(7) establish and carry out the rare disease 

5 and condition third-party payor program under sub6 section (d). 

7 ‘‘(b) ALTITUDE PROGRAM.— 

8 ‘‘(1) IN GENERAL.—The Intercenter Institute 

9 shall establish and implement a program, to be 

10 known as the Accelerating Lifesavings Therapies in 

11 Treating Ultra-rare Disease Entities Program, to 

12 identify and make recommendations to address cur13 rent and emerging regulatory science and public pol14 icy challenges associated with developing medical 

15 products to treat rare diseases or conditions in an 

16 individual or very small populations. 

17 ‘‘(2) ISSUES.—The program under paragraph 

18 (1) shall focus on issues including— 

19 ‘‘(A) manufacturing standards for thera20 pies described in such paragraph, including in 

21 non-industry settings; 

22 ‘‘(B) trial designs and metrics; 

23 ‘‘(C) regulatory flexibilities for abbreviated 

24 toxicology studies, overlapping animal studies, 

25 and patient dosing; 


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1 ‘‘(D) regulatory science, chemistry, manu2 facturing, and other needs associated with de3 veloping such therapies; and 

4 ‘‘(E) other issues as determined by the 

5 Secretary. 

6 ‘‘(c) PROPOSALS FOR AMENDING LABELS.— 

7 ‘‘(1) STAKEHOLDER GROUP.—Not later than 

8 180 days after the date of enactment of this section, 

9 the Intercenter Institute shall convene a meeting of 

10 stakeholders from the rare disease community, in11 cluding patients, caregivers, product manufacturers, 

12 third-party payors, and others, to consider potential 

13 amendments to labels for medical products to treat 

14 rare diseases or conditions approved pursuant to a 

15 pathway under section 506. 

16 ‘‘(2) GUIDANCE.—Not later than 90 days after 

17 the date of the meeting under paragraph (1), the 

18 Secretary shall issue guidance to propose changes to 

19 how the labels of medical products to treat rare dis20 eases or conditions demonstrate clinical benefits and 

21 reflect relevant scientific data including surrogate 

22 endpoints. 

23 ‘‘(d) RARE DISEASE AND CONDITION THIRD-PARTY

24 PAYOR PROGRAM.— 


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1 ‘‘(1) IN GENERAL.—The Intercenter Institute 

2 shall establish and carry out a voluntary rare disease 

3 and condition early third-party payor feedback pro4 gram— 

5 ‘‘(A) to inform coverage policies for rare 

6 disease therapies; and 

7 ‘‘(B) to inform clinical trial design, patient 

8 engagement, and other data collections. 

9 ‘‘(2) PROGRAM REQUIREMENTS.—The program 

10 under paragraph (1) shall— 

11 ‘‘(A) facilitate voluntary communication 

12 between sponsors of medical products to treat 

13 rare diseases and conditions and third-party 

14 payors; and 

15 ‘‘(B) require participation of the Centers 

16 for Medicare & Medicaid Services with rep17 resentation from— 

18 ‘‘(i) the Center for Medicare; and 

19 ‘‘(ii) the Center for Medicaid and 

20 CHIP Services. 

21 ‘‘(3) ANNUAL REPORT.—The Intercenter Insti22 tute shall— 

23 ‘‘(A) on an annual basis, submit a report 

24 to that Congress on— 


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1 ‘‘(i) the participation within the pro2 gram under paragraph (1); and 

3 ‘‘(ii) the impacts of the program 

4 under paragraph (1); and 

5 ‘‘(B) post each such report on the public 

6 website of the Intercenter Institute. 

7 ‘‘(4) BULLETIN TO MEDICAID DIRECTORS.— 

8 Following the approval, clearance, or authorization 

9 by the Food and Drug Administration of a medical 

10 product to treat a rare disease or condition, the Sec11 retary shall issue a bulletin to State Medicaid direc12 tors containing information to help inform coverage 

13 decisions on the product by State Medicaid and Chil14 dren’s Health Insurance programs. 

15 ‘‘(e) DEFINITION.—In this section, the terms ‘Inter16 center Institute on Rare Diseases and Conditions’ and 

17 ‘Intercenter Institute’ refer to the Intercenter Institute on 

18 Rare Diseases and Conditions established pursuant to sec19 tion 1014.’’. 

20 SEC. 4. RARE DISEASE AND CONDITION DRUG ADVISORY 

21 COMMITTEE. 

22 Subchapter B of chapter V of the Federal Food, 

23 Drug, and Cosmetic Act is further amended by inserting 

24 after section 529B of such Act, as inserted by section 3, 

25 the following new section: 


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1 ‘‘SEC. 529C. RARE DISEASE AND CONDITION DRUG ADVI2 SORY COMMITTEE. 

3 ‘‘(a) IN GENERAL.—The Secretary shall establish 

4 and maintain a committee, to be known as the Rare Dis5 ease and Condition Drug Advisory Committee (in this sec6 tion referred to as the ‘Advisory Committee’). 

7 ‘‘(b) DUTY OF COMMITTEE.—The Advisory Com8 mittee shall advise the Secretary on issues associated with 

9 development of therapies to treat rare diseases or condi10 tions. 

11 ‘‘(c) SPECIFIC ISSUES.—In advising the Secretary, 

12 the Advisory Committee may address issues including— 

13 ‘‘(1) modified or new regulatory pathways to 

14 support review of therapies; 

15 ‘‘(2) clinical trial design needs, including devel16 opment of innovative approaches to clinical trials; 

17 ‘‘(3) qualifications of biomarkers or other drug 

18 development tools for use in reviews; 

19 ‘‘(4) modified or new standards to support the 

20 review of already marketed drugs being evaluated 

21 for repurposing to treat a rare disease or condition; 

22 and 

23 ‘‘(5) issues— 

24 ‘‘(A) that pertain to an application for ap25 proval of a therapy to treat a rare disease or 

26 condition; and 


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1 ‘‘(B) with respect to which a review divi2 sion has requested that the Advisory Committee 

3 provide advice. 

4 ‘‘(d) MEMBERSHIP.— 

5 ‘‘(1) IN GENERAL.—The Advisory Committee 

6 shall consist of— 

7 ‘‘(A) not more than 15 members appointed 

8 by the Secretary in accordance with paragraph 

9 (2); and 

10 ‘‘(B) the nonvoting ex officio members 

11 under paragraph (3). 

12 ‘‘(2) APPOINTED MEMBERS.— 

13 ‘‘(A) SPECIAL GOVERNMENT EMPLOY14 EES.—Members of the Advisory Committee ap15 pointed pursuant to paragraph (1)(A) shall 

16 serve as special Government employees (as de17 fined in section 202(a) of title 18, United 

18 States Code). 

19 ‘‘(B) ELIGIBILITY.—To be eligible for ap20 pointment pursuant to paragraph (1)(A), an in21 dividual shall— 

22 ‘‘(i) be eligible to serve as special Gov23 ernment employee (as defined in section 

24 202(a) of title 18, United States Code); 

25 and 


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1 ‘‘(ii) have expertise in the fields of 

2 public policy, law, regulatory policy, eco3 nomics, patient-focused product develop4 ment, or patient advocacy. 

5 ‘‘(C) COMPOSITION.—Of the members of 

6 the Advisory Committee appointed pursuant to 

7 paragraph (1)(A)— 

8 ‘‘(i) up to 10 shall be selected from 

9 among experts in the disciplines relevant to 

10 the activities of the Intercenter Institute 

11 on Rare Diseases and Conditions, to in12 clude at least one expert in each of— 

13 ‘‘(I) rare disease product develop14 ment; 

15 ‘‘(II) conducting clinical trials 

16 with respect to rare diseases and con17 ditions, including with respect to very 

18 small patient populations; 

19 ‘‘(III) rare disease and condition 

20 natural history and related studies; 

21 ‘‘(IV) health economics per22 taining to the development of medical 

23 products for rare diseases or condi24 tions; 


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1 ‘‘(V) manufacturing and related 

2 needs associated with medical prod3 ucts for rare diseases or conditions; 

4 and 

5 ‘‘(VI) patient experience data col6 lection; and 

7 ‘‘(ii) up to 5 shall be selected from the 

8 public, to include— 

9 ‘‘(I) at least 4 individuals who 

10 are representatives of the rare disease 

11 patient community; 

12 ‘‘(II) at least one individual who 

13 is directly impacted by a rare disease 

14 or condition; and 

15 ‘‘(III) at least one person who 

16 serves as a family caregiver to a per17 son diagnosed with a rare disease or 

18 condition. 

19 ‘‘(3) NONVOTING EX OFFICIO MEMBERS.—The 

20 nonvoting ex officio members of the Advisory Com21 mittee under paragraph (1)(B) shall consist of the 

22 following: 

23 ‘‘(A) The Secretary (or the Secretary’s 

24 designee). 


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1 ‘‘(B) The Director of the Intercenter Insti2 tute on Rare Diseases and Conditions. 

3 ‘‘(C) The Director of the Center for Bio4 logics Evaluation and Research (or the Direc5 tor’s designee). 

6 ‘‘(D) The Director of the Center for Drug 

7 Evaluation and Research (or the Director’s des8 ignee). 

9 ‘‘(E) The Director of the Center for De10 vices and Radiological Health (or the Director’s 

11 designee). 

12 ‘‘(F) The Director of the National Center 

13 for the Advancing Translational Sciences of the 

14 National Institutes of Health (or the Director’s 

15 designee). 

16 ‘‘(G) The Administrator of the Centers for 

17 Medicare & Medicaid Services (or the Adminis18 trator’s designee). 

19 ‘‘(H) Any additional officers or employees 

20 of the Department of Health and Human Serv21 ices as the Secretary determines necessary for 

22 the Advisory Committee to effectively carry out 

23 its functions. 


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1 ‘‘(4) CHAIR.—The Chair of the Advisory Com2 mittee shall be the Director of the Intercenter Insti3 tute for Rare Diseases and Conditions. 

4 ‘‘(5) TERMS.— 

5 ‘‘(A) MEMBERS.— 

6 ‘‘(i) IN GENERAL.—The term of a 

7 member of the Advisory Committee ap8 pointed pursuant to paragraph (1)(A) shall 

9 be 4 years, except that any member ap10 pointed to fill a vacancy in an unexpired 

11 term shall be appointed for the remainder 

12 of that term. 

13 ‘‘(ii) CONTINUED SERVICE.—A mem14 ber appointed pursuant to paragraph 

15 (1)(A) may continue serving as a member 

16 of the Advisory Committee for up to 180 

17 days after the expiration of that member’s 

18 term if a successor has not been appointed. 

19 ‘‘(B) REAPPOINTMENT.—A member of the 

20 Advisory Committee who has been appointed 

21 pursuant to paragraph (1)(A) for a term of 4 

22 years may not be reappointed to serve as a 

23 member of the Advisory Committee before the 

24 date that is 2 years after the date of expiration 

25 of that member’s term. 


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1 ‘‘(e) QUORUM.—A majority of the appointed mem2 bers of the Advisory Committee shall constitute a quorum 

3 for the conduct of business.’’. 

4 SEC. 5. GRANTS AND CONTRACTS FOR DEVELOPMENT OF 

5 DRUGS FOR RARE DISEASES AND CONDI6 TIONS. 

7 (a) AUTHORITY OF SECRETARY.—Section 5(a) of the 

8 Orphan Drug Act (21 U.S.C. 360ee(a)) is amended— 

9 (1) in paragraph (2), by striking ‘‘and’’ at the 

10 end; and 

11 (2) by inserting before the period at the end ‘‘, 

12 and (4) developing practices pertaining to the chem13 istry, manufacturing, regulatory approval of, and 

14 controls of individualized therapies or therapies to 

15 treat very small populations’’. 

16 (b) ALTITUDE PROGRAM.—In supporting grants 

17 and contracts under section 5(a)(4) of the Orphan Drug 

18 Act, as added by subsection (a), the Secretary of Health 

19 and Human Services shall consult with the Director of the 

20 Intercenter Institute on Rare Diseases and Conditions re21 garding the Accelerating Lifesavings Therapies in Treat22 ing Ultra-rare Disease Entities Program established under 

23 section 529B(b) of the Federal Food, Drug, and Cosmetic 

24 Act, as added by section 3(c) of this Act, to— 


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1 (1) identify the regulatory science and related 

2 challenges and needs associated with developing indi3 vidualized therapies or therapies to treat very small 

4 patient populations; and 

5 (2) support research to address such challenges. 

Æ 


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