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-
tives of the United States of America in Congress assembled, 
SECTION 1. SHORT TITLE. 
This Act may be cited as the ‘‘Speeding Therapy Ac-
cess Today Act of 2021’’. 
SEC. 2. TABLE OF CONTENTS. 
The table of contents of this Act is as follows: 
Sec. 1. Short title. 
Sec. 2. Table of contents. 
Sec. 3. Intercenter Institute on Rare Diseases and Conditions. 
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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 
CONDITIONS. 
(a) ESTABLISHMENT REQUIRED.—The first sentence 
of section 1014(a) of the Federal Food, Drug, and Cos-
metic Act (21 U.S.C. 399g(a)) is amended by inserting 
‘‘, at least one of which shall be focused on rare diseases 
and conditions’’ before the period at the end of the sen-
tence. 
(b) TIMING OF ESTABLISHMENT.—Subsection (c) of 
section 1014 of the Federal Food, Drug, and Cosmetic 
Act (21 U.S.C. 399g) is amended to read as follows: 
‘‘(c) TIMING.—Not later than the date that is 1 year 
after the date of enactment of the Speeding Therapy Ac-
cess Today Act of 2021, the Secretary shall establish, in 
accordance with this section and section 529B, an Insti-
tute under subsection (a) focused on rare diseases and 
conditions, to be known as the Intercenter Institute on 
Rare Diseases and Conditions.’’. 
(c) RESPONSIBILITIES.—Subchapter B of chapter V 
of the Federal Food, Drug, and Cosmetic Act (relating 
to drugs for rare diseases or conditions) is amended by 
inserting after section 529A of such Act (21 U.S.C. 360ff– 
1) the following new section: 
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‘‘SEC. 529B. INTERCENTER INSTITUTE ON RARE DISEASES 
AND CONDITIONS. 
‘‘(a) RESPONSIBILITIES.—In addition to carrying out 
activities listed in section 1014(a), the Intercenter Insti-
tute on Rare Diseases and Conditions shall— 
‘‘(1) serve as the Food and Drug Administra-
tion’s coordinating office for engagement with rare 
disease and condition stakeholders, complementing 
but not supplanting engagement activities between 
stakeholders and the review divisions; 
‘‘(2) build, within the Food and Drug Adminis-
tration, knowledge and understanding associated 
with the review of medical products to treat rare dis-
eases and conditions, including advancements in trial 
design, statistical analysis, regulatory science, prod-
uct manufacturing, and other topics as determined 
by the Secretary; 
‘‘(3) implement cross-center rare disease and 
condition-focused meetings and policy development; 
‘‘(4) coordinate rare disease and condition-spe-
cific regulatory science initiatives; 
‘‘(5) facilitate stakeholder engagement to the 
external community and international regulatory 
agencies on rare disease and condition product devel-
opment; 
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‘‘(6) establish and implement the Accelerating 
Lifesavings Therapies in Treating Ultra-rare Dis-
ease Entities Program under subsection (b); and 
‘‘(7) establish and carry out the rare disease 
and condition third-party payor program under sub-
section (d). 
‘‘(b) ALTITUDE PROGRAM.— 
‘‘(1) IN GENERAL.—The Intercenter Institute 
shall establish and implement a program, to be 
known as the Accelerating Lifesavings Therapies in 
Treating Ultra-rare Disease Entities Program, to 
identify and make recommendations to address cur-
rent and emerging regulatory science and public pol-
icy challenges associated with developing medical 
products to treat rare diseases or conditions in an 
individual or very small populations. 
‘‘(2) ISSUES.—The program under paragraph 
(1) shall focus on issues including— 
‘‘(A) manufacturing standards for thera-
pies described in such paragraph, including in 
non-industry settings; 
‘‘(B) trial designs and metrics; 
‘‘(C) regulatory flexibilities for abbreviated 
toxicology studies, overlapping animal studies, 
and patient dosing; 
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‘‘(D) regulatory science, chemistry, manu-
facturing, and other needs associated with de-
veloping such therapies; and 
‘‘(E) other issues as determined by the 
Secretary. 
‘‘(c) PROPOSALS FOR AMENDING LABELS.— 
‘‘(1) STAKEHOLDER
GROUP.—Not later than 
180 days after the date of enactment of this section, 
the Intercenter Institute shall convene a meeting of 
stakeholders from the rare disease community, in-
cluding patients, caregivers, product manufacturers, 
third-party payors, and others, to consider potential 
amendments to labels for medical products to treat 
rare diseases or conditions approved pursuant to a 
pathway under section 506. 
‘‘(2) GUIDANCE.—Not later than 90 days after 
the date of the meeting under paragraph (1), the 
Secretary shall issue guidance to propose changes to 
how the labels of medical products to treat rare dis-
eases or conditions demonstrate clinical benefits and 
reflect relevant scientific data including surrogate 
endpoints. 
‘‘(d) RARE DISEASE AND CONDITION THIRD-PARTY 
PAYOR PROGRAM.— 
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‘‘(1) IN GENERAL.—The Intercenter Institute 
shall establish and carry out a voluntary rare disease 
and condition early third-party payor feedback pro-
gram— 
‘‘(A) to inform coverage policies for rare 
disease therapies; and 
‘‘(B) to inform clinical trial design, patient 
engagement, and other data collections. 
‘‘(2) PROGRAM REQUIREMENTS.—The program 
under paragraph (1) shall— 
‘‘(A) facilitate voluntary communication 
between sponsors of medical products to treat 
rare diseases and conditions and third-party 
payors; and 
‘‘(B) require participation of the Centers 
for Medicare & Medicaid Services with rep-
resentation from— 
‘‘(i) the Center for Medicare; and 
‘‘(ii) the Center for Medicaid and 
CHIP Services. 
‘‘(3) ANNUAL REPORT.—The Intercenter Insti-
tute shall— 
‘‘(A) on an annual basis, submit a report 
to that Congress on— 
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‘‘(i) the participation within the pro-
gram under paragraph (1); and 
‘‘(ii) the impacts of the program 
under paragraph (1); and 
‘‘(B) post each such report on the public 
website of the Intercenter Institute. 
‘‘(4) BULLETIN
TO
MEDICAID
DIRECTORS.— 
Following the approval, clearance, or authorization 
by the Food and Drug Administration of a medical 
product to treat a rare disease or condition, the Sec-
retary shall issue a bulletin to State Medicaid direc-
tors containing information to help inform coverage 
decisions on the product by State Medicaid and Chil-
dren’s Health Insurance programs. 
‘‘(e) DEFINITION.—In this section, the terms ‘Inter-
center Institute on Rare Diseases and Conditions’ and 
‘Intercenter Institute’ refer to the Intercenter Institute on 
Rare Diseases and Conditions established pursuant to sec-
tion 1014.’’. 
SEC. 4. RARE DISEASE AND CONDITION DRUG ADVISORY 
COMMITTEE. 
Subchapter B of chapter V of the Federal Food, 
Drug, and Cosmetic Act is further amended by inserting 
after section 529B of such Act, as inserted by section 3, 
the following new section: 
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‘‘SEC. 529C. RARE DISEASE AND CONDITION DRUG ADVI-
SORY COMMITTEE. 
‘‘(a) IN GENERAL.—The Secretary shall establish 
and maintain a committee, to be known as the Rare Dis-
ease and Condition Drug Advisory Committee (in this sec-
tion referred to as the ‘Advisory Committee’). 
‘‘(b) DUTY
OF COMMITTEE.—The Advisory Com-
mittee shall advise the Secretary on issues associated with 
development of therapies to treat rare diseases or condi-
tions. 
‘‘(c) SPECIFIC ISSUES.—In advising the Secretary, 
the Advisory Committee may address issues including— 
‘‘(1) modified or new regulatory pathways to 
support review of therapies; 
‘‘(2) clinical trial design needs, including devel-
opment of innovative approaches to clinical trials; 
‘‘(3) qualifications of biomarkers or other drug 
development tools for use in reviews; 
‘‘(4) modified or new standards to support the 
review of already marketed drugs being evaluated 
for repurposing to treat a rare disease or condition; 
and 
‘‘(5) issues— 
‘‘(A) that pertain to an application for ap-
proval of a therapy to treat a rare disease or 
condition; and 
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‘‘(B) with respect to which a review divi-
sion has requested that the Advisory Committee 
provide advice. 
‘‘(d) MEMBERSHIP.— 
‘‘(1) IN
GENERAL.—The Advisory Committee 
shall consist of— 
‘‘(A) not more than 15 members appointed 
by the Secretary in accordance with paragraph 
(2); and 
‘‘(B) the nonvoting ex officio members 
under paragraph (3). 
‘‘(2) APPOINTED MEMBERS.— 
‘‘(A) 
SPECIAL
GOVERNMENT
EMPLOY-
EES.—Members of the Advisory Committee ap-
pointed pursuant to paragraph (1)(A) shall 
serve as special Government employees (as de-
fined in section 202(a) of title 18, United 
States Code). 
‘‘(B) ELIGIBILITY.—To be eligible for ap-
pointment pursuant to paragraph (1)(A), an in-
dividual shall— 
‘‘(i) be eligible to serve as special Gov-
ernment employee (as defined in section 
202(a) of title 18, United States Code); 
and 
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‘‘(ii) have expertise in the fields of 
public policy, law, regulatory policy, eco-
nomics, patient-focused product develop-
ment, or patient advocacy. 
‘‘(C) COMPOSITION.—Of the members of 
the Advisory Committee appointed pursuant to 
paragraph (1)(A)— 
‘‘(i) up to 10 shall be selected from 
among experts in the disciplines relevant to 
the activities of the Intercenter Institute 
on Rare Diseases and Conditions, to in-
clude at least one expert in each of— 
‘‘(I) rare disease product develop-
ment; 
‘‘(II) conducting clinical trials 
with respect to rare diseases and con-
ditions, including with respect to very 
small patient populations; 
‘‘(III) rare disease and condition 
natural history and related studies; 
‘‘(IV) 
health 
economics 
per-
taining to the development of medical 
products for rare diseases or condi-
tions; 
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‘‘(V) manufacturing and related 
needs associated with medical prod-
ucts for rare diseases or conditions; 
and 
‘‘(VI) patient experience data col-
lection; and 
‘‘(ii) up to 5 shall be selected from the 
public, to include— 
‘‘(I) at least 4 individuals who 
are representatives of the rare disease 
patient community; 
‘‘(II) at least one individual who 
is directly impacted by a rare disease 
or condition; and 
‘‘(III) at least one person who 
serves as a family caregiver to a per-
son diagnosed with a rare disease or 
condition. 
‘‘(3) NONVOTING EX OFFICIO MEMBERS.—The 
nonvoting ex officio members of the Advisory Com-
mittee under paragraph (1)(B) shall consist of the 
following: 
‘‘(A) The Secretary (or the Secretary’s 
designee). 
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‘‘(e) QUORUM.—A majority of the appointed mem-
bers of the Advisory Committee shall constitute a quorum 
for the conduct of business.’’. 
SEC. 5. GRANTS AND CONTRACTS FOR DEVELOPMENT OF 
DRUGS FOR RARE DISEASES AND CONDI-
TIONS. 
(a) AUTHORITY OF SECRETARY.—Section 5(a) of the 
Orphan Drug Act (21 U.S.C. 360ee(a)) is amended— 
(1) in paragraph (2), by striking ‘‘and’’ at the 
end; and 
(2) by inserting before the period at the end ‘‘, 
and (4) developing practices pertaining to the chem-
istry, manufacturing, regulatory approval of, and 
controls of individualized therapies or therapies to 
treat very small populations’’. 
(b) ALTITUDE PROGRAM.—In supporting grants 
and contracts under section 5(a)(4) of the Orphan Drug 
Act, as added by subsection (a), the Secretary of Health 
and Human Services shall consult with the Director of the 
Intercenter Institute on Rare Diseases and Conditions re-
garding the Accelerating Lifesavings Therapies in Treat-
ing Ultra-rare Disease Entities Program established under 
section 529B(b) of the Federal Food, Drug, and Cosmetic 
Act, as added by section 3(c) of this Act, to— 
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(1) identify the regulatory science and related 
challenges and needs associated with developing indi-
vidualized therapies or therapies to treat very small 
patient populations; and 
(2) support research to address such challenges. 
Æ 
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