I 
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 Representa-
tives of the United States of America in Congress assembled, 
SECTION 1. SHORT TITLE. 
This Act may be cited as the ‘‘Helping Experts Accel-
erate Rare Treatments Act of 2022’’. 
SEC. 2. IMPROVING THE TREATMENT OF RARE DISEASES 
AND CONDITIONS. 
(a) ANNUAL REPORT
ON ORPHAN DRUG PRO-
GRAM.—Subchapter B of chapter V of the Federal Food, 
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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 Help-
ing Experts Accelerate Rare Treatments Act of 2022, the 
Secretary shall submit to the Congress a report summa-
rizing 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 sec-
tion 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 applica-
tions accepted and rejected for filing, and the num-
ber of such applications pending, approved, and dis-
approved by the Food and Drug Administration, 
arrayed by the review division assigned to the appli-
cation; and 
‘‘(2) assess the extent to which the Food and 
Drug Administration is consulting with external ex-
perts pursuant to section 569(a)(2) on topics per-
taining to drugs for a rare disease or condition, in-
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cluding how and when any such consultation is oc-
curring. 
‘‘(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 affect-
ing fewer than 200,000 persons in the United States.’’. 
(b) STUDY ON EUROPEAN UNION SAFETY AND EFFI-
CACY REVIEWS OF DRUGS FOR RARE DISEASES AND CON-
DITIONS.— 
(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 Euro-
pean Union process for evaluating the safety and ef-
ficacy of drugs for rare diseases or conditions, in-
cluding— 
(A) any flexibilities, authorities, or mecha-
nisms available in the European Union specific 
to rare diseases or conditions; and 
(B) consideration and use of supplemental 
data submitted during the review process, in-
cluding data associated with open label exten-
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