Helping Experts Accelerate Rare Treatments Act of 2022
This bill requires studies on processes for approving drugs that treat rare diseases and conditions.
The Government Accountability Office (GAO) must contract with an appropriate entity to study the European Union's process for evaluating the safety and efficacy of such drugs, including any flexibilities or mechanisms that are specific to rare diseases or conditions. The GAO must also conduct a study about the Food and Drug Administration's mechanisms for ensuring that patient and physician perspectives are considered when evaluating and approving drugs for rare diseases or conditions.
The GAO must report to Congress the results of these studies.
[Congressional Bills 117th Congress]
[From the U.S. Government Publishing Office]
[H.R. 6888 Introduced in House (IH)]
<DOC>
117th CONGRESS
2d Session
H. R. 6888
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
March 1, 2022
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 Representatives of the
United States of America in Congress assembled,
SECTION 1. SHORT TITLE.
This Act may be cited as the ``Helping Experts Accelerate Rare
Treatments Act of 2022''.
SEC. 2. IMPROVING THE TREATMENT OF RARE DISEASES AND CONDITIONS.
(a) Annual Report on Orphan Drug Program.--Subchapter B of chapter
V of the Federal Food, Drug, and Cosmetic Act (21 U.S.C. 360aa et seq.)
is amended by adding at the end the following new section:
``SEC. 529B. ANNUAL REPORT ON ORPHAN DRUG PROGRAM.
``(a) In General.--Not later than the end of each of the 4 years
following the date of enactment of the Helping Experts Accelerate Rare
Treatments Act of 2022, the Secretary shall submit to the Congress a
report summarizing the activities of the Food and Drug Administration
related to designating drugs under section 526 for a rare disease or
condition and approving such drugs under section 505 of this Act or
licensing such drugs under section 351 of the Public Health Service
Act, including--
``(1) the number of applications for such drugs under
section 505 of this Act and section 351 of the Public Health
Service Act received by the Food and Drug Administration, the
number of such applications accepted and rejected for filing,
and the number of such applications pending, approved, and
disapproved by the Food and Drug Administration, arrayed by the
review division assigned to the application; and
``(2) assess the extent to which the Food and Drug
Administration is consulting with external experts pursuant to
section 569(a)(2) on topics pertaining to drugs for a rare
disease or condition, including how and when any such
consultation is occurring.
``(b) Public Availability.--The Secretary shall make each report
under subsection (a) available to the public, including by posting the
report on the website of the Food and Drug Administration.
``(c) Definition.--In this section, the term `rare disease or
condition' means a disease or condition affecting fewer than 200,000
persons in the United States.''.
(b) Study on European Union Safety and Efficacy Reviews of Drugs
for Rare Diseases and Conditions.--
(1) In general.--The Comptroller General of the United
States shall enter into a contract with an appropriate entity
to conduct a study on the European Union process for evaluating
the safety and efficacy of drugs for rare diseases or
conditions, including--
(A) any flexibilities, authorities, or mechanisms
available in the European Union specific to rare
diseases or conditions; and
(B) consideration and use of supplemental data
submitted during the review process, including data
associated with open label extension studies and
expanded access programs specific to rare diseases or
conditions.
(2) Consultation.--The contract under paragraph (1) shall
provide for consultation with relevant stakeholders,
including--
(A) rare disease or condition patients; and
(B) patient groups that--
(i) represent rare disease or condition
patients; and
(ii) have international patient outreach.
(3) Report.--The contract under paragraph (1) shall provide
for--
(A) not later than 2 years after the date of
enactment of this Act--
(i) the completion of the study under
paragraph (1); and
(ii) the submission of a report on the
results of such study to the Congress; and
(B) the inclusion in the report under subparagraph
(A)(ii) of recommendations for changes to the processes
and authorities of the Food and Drug Administration to
facilitate development of, and access to, treatments
for rare diseases or conditions.
(4) Public availability.--The contract under paragraph (1)
shall provide for the appropriate entity referred to in
paragraph (1) to make the report under paragraph (3) available
to the public, including by posting the report on the website
of the appropriate entity.
(c) Public Meeting.--
(1) In general.--Not later than December 31, 2023, the
Secretary of Health and Human Services, acting through the
Commissioner of Food and Drugs, shall convene one or more
public meetings to solicit input from stakeholders regarding
the approaches described in paragraph (2).
(2) Approaches.--The public meeting or meetings under
paragraph (1) shall address approaches to increasing and
improving engagement with rare disease or condition patients,
groups representing such patients, rare disease or condition
experts, and experts on small population studies, in order to
improve the understanding with respect to rare diseases or
conditions of--
(A) patient burden;
(B) treatment options; and
(C) side effects of treatments, including--
(i) comparing the side effects of
treatments; and
(ii) understanding the risks of side
effects relative to the health status of the
patient and the progression of the disease or
condition.
(3) Public docket.--The Secretary of Health and Human
Services shall establish a public docket to receive written
comments related to the approaches addressed during each public
meeting under paragraph (1). Such public docket shall remain
open for 60 days following the date of each such public
meeting.
(4) Reports.--Not later than 180 days after each public
meeting under paragraph (1), the Commissioner of Food and Drugs
shall develop and publish on the website of the Food and Drug
Administration a report on--
(A) the approaches discussed at the public meeting;
and
(B) any related recommendations.
(d) Consultation on the Science of Small Population Studies.--
Section 569(a)(2) of the Federal Food, Drug, and Cosmetic Act (21
U.S.C. 360bbb-8(b)) is amended by adding at the end the following:
``(C) Small population studies.--The external
experts on the list maintained pursuant to subparagraph
(A) may include experts on the science of small
population studies.''.
(e) Study on Sufficiency and Use of FDA Mechanisms for
Incorporating the Patient and Clinician Perspective in FDA Processes
Related to Applications Concerning Drugs for Rare Diseases or
Conditions.--
(1) In general.--The Comptroller General of the United
States shall conduct a study on the use of Food and Drug
Administration mechanisms and tools to ensure that patient and
physician perspectives are considered and incorporated
throughout the processes of the Food and Drug Administration--
(A) for approving or licensing under section 505 of
the Federal Food, Drug, or Cosmetic Act (21 U.S.C. 355)
or section 351 of the Public Health Service Act (42
U.S.C. 262) a drug designated as a drug for a rare
disease or condition under section 526 of the Federal
Food, Drug, and Cosmetic Act (21 U.S.C. 360bb); and
(B) in making any determination related to such a
drug's approval, including assessment of the drug's--
(i) safety or effectiveness; or
(ii) postapproval safety monitoring.
(2) Topics.--The study under paragraph (1) shall--
(A) identify and compare the processes that the
Food and Drug Administration has formally put in place
and utilized to gather external expertise (including
patients, patient groups, and physicians) on specific
applications for diseases or conditions affecting
20,000 or fewer patients in the United States and
specific applications for diseases or conditions
affecting 200,000 or fewer patients in the United
States;
(B) examine tools or mechanisms to improve efforts
and initiatives of the Food and Drug Administration to
collect and consider such external expertise with
respect to applications for diseases or conditions
affecting 20,000 or fewer patients in the United States
compared to applications for diseases or conditions
affecting 200,000 or fewer patients in the United
States throughout the application review and approval
or licensure processes, including within internal
benefit-risk assessments, advisory committee processes,
and postapproval safety monitoring; and
(C) examine processes or alternatives to address or
resolve conflicts of interest that impede the Food and
Drug Administration in gaining external expert input on
rare diseases or conditions with a limited set of
clinical and research experts.
(3) Report.--Not later than 2 years after the date of
enactment of this Act, the Comptroller General of the United
States shall--
(A) complete the study under paragraph (1);
(B) submit a report on the results of such study to
the Congress; and
(C) include in such report recommendations, if
appropriate, for changes to the processes and
authorities of the Food and Drug Administration to
improve the collection and consideration of external
expert opinions of patients, patient groups, and
physicians with expertise in rare diseases or
conditions, including any specific recommendations for
diseases or conditions affecting 20,000 or fewer
patients in the United States.
(f) Definition.--In this section, the term ``rare disease or
condition'' means a disease or condition affecting fewer than 200,000
persons in the United States.
<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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