Speeding Therapy Access Today Act of 2021
This bill requires and authorizes various actions to accelerate the development of therapies for rare diseases.
The Food and Drug Administration (FDA) shall establish the Intercenter Institute on Rare Diseases and Conditions. The institute shall (1) coordinate engagement with relevant stakeholders, (2) build the FDA's expertise in the review of medical products to treat rare diseases, (3) coordinate regulatory science initiatives related to rare diseases, (4) establish and implement a program to make recommendations to address challenges associated with developing medical products to treat rare diseases in an individual or in very small populations, (5) convene a stakeholder meeting to consider potential amendments to labels for medical products to treat rare diseases, and (6) establish and carry out a program to facilitate voluntary communication between the sponsors of such medical products and third-party payers (e.g., insurance companies).
The bill also authorizes the FDA to make grants to assist in developing practices related to the development and production of individualized therapies or therapies to treat very small populations.
The bill also establishes an advisory committee to advise the FDA on issues related to the development of therapies to treat rare diseases.
[Congressional Bills 117th Congress]
[From the U.S. Government Publishing Office]
[H.R. 1730 Introduced in House (IH)]
<DOC>
117th CONGRESS
1st Session
H. R. 1730
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 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 Representatives of the
United States of America in Congress assembled,
SECTION 1. SHORT TITLE.
This Act may be cited as the ``Speeding Therapy Access 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.
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 Cosmetic 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 sentence.
(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 Access Today Act of 2021, the
Secretary shall establish, in accordance with this section and section
529B, an Institute 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:
``SEC. 529B. INTERCENTER INSTITUTE ON RARE DISEASES AND CONDITIONS.
``(a) Responsibilities.--In addition to carrying out activities
listed in section 1014(a), the Intercenter Institute on Rare Diseases
and Conditions shall--
``(1) serve as the Food and Drug Administration'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 Administration,
knowledge and understanding associated with the review of
medical products to treat rare diseases and conditions,
including advancements in trial design, statistical analysis,
regulatory science, product 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-specific
regulatory science initiatives;
``(5) facilitate stakeholder engagement to the external
community and international regulatory agencies on rare disease
and condition product development;
``(6) establish and implement the Accelerating Lifesavings
Therapies in Treating Ultra-rare Disease Entities Program under
subsection (b); and
``(7) establish and carry out the rare disease and
condition third-party payor program under subsection (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 current and emerging regulatory science and public
policy 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 therapies
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;
``(D) regulatory science, chemistry, manufacturing,
and other needs associated with developing 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, including 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 diseases or conditions demonstrate
clinical benefits and reflect relevant scientific data
including surrogate endpoints.
``(d) Rare Disease and Condition Third-Party Payor Program.--
``(1) In general.--The Intercenter Institute shall
establish and carry out a voluntary rare disease and condition
early third-party payor feedback program--
``(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 representation from--
``(i) the Center for Medicare; and
``(ii) the Center for Medicaid and CHIP
Services.
``(3) Annual report.--The Intercenter Institute shall--
``(A) on an annual basis, submit a report to that
Congress on--
``(i) the participation within the program
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 Secretary shall issue a bulletin to State
Medicaid directors containing information to help inform
coverage decisions on the product by State Medicaid and
Children's Health Insurance programs.
``(e) Definition.--In this section, the terms `Intercenter
Institute on Rare Diseases and Conditions' and `Intercenter Institute'
refer to the Intercenter Institute on Rare Diseases and Conditions
established pursuant to section 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:
``SEC. 529C. RARE DISEASE AND CONDITION DRUG ADVISORY COMMITTEE.
``(a) In General.--The Secretary shall establish and maintain a
committee, to be known as the Rare Disease and Condition Drug Advisory
Committee (in this section referred to as the `Advisory Committee').
``(b) Duty of Committee.--The Advisory Committee shall advise the
Secretary on issues associated with development of therapies to treat
rare diseases or conditions.
``(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 development 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 approval
of a therapy to treat a rare disease or condition; and
``(B) with respect to which a review division 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 employees.--Members of the
Advisory Committee appointed pursuant to paragraph
(1)(A) shall serve as special Government employees (as
defined in section 202(a) of title 18, United States
Code).
``(B) Eligibility.--To be eligible for appointment
pursuant to paragraph (1)(A), an individual shall--
``(i) be eligible to serve as special
Government employee (as defined in section
202(a) of title 18, United States Code); and
``(ii) have expertise in the fields of
public policy, law, regulatory policy,
economics, patient-focused product development,
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 include at least
one expert in each of--
``(I) rare disease product
development;
``(II) conducting clinical trials
with respect to rare diseases and
conditions, including with respect to
very small patient populations;
``(III) rare disease and condition
natural history and related studies;
``(IV) health economics pertaining
to the development of medical products
for rare diseases or conditions;
``(V) manufacturing and related
needs associated with medical products
for rare diseases or conditions; and
``(VI) patient experience data
collection; 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
person diagnosed with a rare disease or
condition.
``(3) Nonvoting ex officio members.--The nonvoting ex
officio members of the Advisory Committee under paragraph
(1)(B) shall consist of the following:
``(A) The Secretary (or the Secretary's designee).
``(B) The Director of the Intercenter Institute on
Rare Diseases and Conditions.
``(C) The Director of the Center for Biologics
Evaluation and Research (or the Director's designee).
``(D) The Director of the Center for Drug
Evaluation and Research (or the Director's designee).
``(E) The Director of the Center for Devices and
Radiological Health (or the Director's designee).
``(F) The Director of the National Center for the
Advancing Translational Sciences of the National
Institutes of Health (or the Director's designee).
``(G) The Administrator of the Centers for Medicare
& Medicaid Services (or the Administrator's designee).
``(H) Any additional officers or employees of the
Department of Health and Human Services as the
Secretary determines necessary for the Advisory
Committee to effectively carry out its functions.
``(4) Chair.--The Chair of the Advisory Committee shall be
the Director of the Intercenter Institute for Rare Diseases and
Conditions.
``(5) Terms.--
``(A) Members.--
``(i) In general.--The term of a member of
the Advisory Committee appointed pursuant to
paragraph (1)(A) shall be 4 years, except that
any member appointed to fill a vacancy in an
unexpired term shall be appointed for the
remainder of that term.
``(ii) Continued service.--A member
appointed pursuant to paragraph (1)(A) may
continue serving as a member of the Advisory
Committee for up to 180 days after the
expiration of that member's term if a successor
has not been appointed.
``(B) Reappointment.--A member of the Advisory
Committee who has been appointed pursuant to paragraph
(1)(A) for a term of 4 years may not be reappointed to
serve as a member of the Advisory Committee before the
date that is 2 years after the date of expiration of
that member's term.
``(e) Quorum.--A majority of the appointed members 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 CONDITIONS.
(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 chemistry,
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 regarding
the Accelerating Lifesavings Therapies in Treating 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--
(1) identify the regulatory science and related challenges
and needs associated with developing individualized therapies
or therapies to treat very small patient populations; and
(2) support research to address such challenges.
<all>
Introduced in House
Introduced in House
Referred to the House Committee on Energy and Commerce.
Referred to the Subcommittee on Health.
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