What This Bill Does
This bill limits when the government can grant exclusive approval or licensing rights for orphan drugs (medications for rare diseases that affect small patient populations). The bill requires drug makers to prove they cannot recover their development costs from U.S. sales within 12 years before receiving these exclusive rights.
Who It Affects
The bill directly affects drug companies that develop orphan drugs and the federal government's Food and Drug Administration (FDA), which approves medications.
Key Provisions
• The government cannot grant exclusive approval or licensing for orphan drugs unless the drug maker proves there is no reasonable expectation they will recover development and marketing costs from U.S. sales within 12 years of first marketing the drug (Sec. 2(f)(1)).
• When calculating expected sales, the government and drug makers must consider sales from all related drugs developed or marketed by the same company or related entities that received the same rare disease designation (Sec. 2(f)(2)).
• A drug can only receive exclusive approval or licensing if it met the definition of an orphan drug on the date it was approved or licensed (Sec. 2(f)(3)).
• If exclusive approval or licensing was already granted before this law, the government shall revoke it if the sponsor cannot meet these new requirements (Sec. 2(f)(1)).
What Changes
If this becomes law, drug companies seeking exclusive orphan drug approval must demonstrate financial hardship by proving they cannot recover costs within 12 years. The government may revoke exclusive rights already granted if companies cannot meet these new standards.
Important Definitions
None defined in the bill text.
I
118TH CONGRESS
1ST SESSION
H. R. 456
To amend the Federal Food, Drug, and Cosmetic Act with respect to limita-
tions on exclusive approval or licensure of orphan drugs, and for other
purposes.
IN THE HOUSE OF REPRESENTATIVES
JANUARY 24, 2023
Mr. CARTER of Georgia 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 with
respect to limitations on exclusive approval or licensure
of orphan drugs, and for other purposes.
Be it enacted by the Senate and House of Representa-
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tives of the United States of America in Congress assembled,
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SECTION 1. SHORT TITLE.
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This Act may be cited as the ‘‘Fairness in Orphan
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Drug Exclusivity Act’’.
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SEC. 2. LIMITATIONS ON EXCLUSIVE APPROVAL OR LICEN-
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SURE OF ORPHAN DRUGS.
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Section 527 of the Federal Food, Drug, and Cosmetic
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Act (21 U.S.C. 360cc) is amended—
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•HR 456 IH
(1) in subsection (a), by striking ‘‘Except as
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provided in subsection (b)’’ and inserting ‘‘Except as
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provided in subsection (b) or (f)’’; and
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(2) by adding at the end the following:
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‘‘(f) LIMITATIONS ON EXCLUSIVE APPROVAL, CER-
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TIFICATION, OR LICENSE.—
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‘‘(1) IN
GENERAL.—For a drug designated
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under section 526 for a rare disease or condition
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pursuant to the criteria set forth in subsection
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(a)(2)(B) of such section, the Secretary shall not
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grant, recognize, or apply exclusive approval or licen-
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sure under subsection (a), and, if such exclusive ap-
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proval or licensure has been granted, recognized, or
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applied, shall revoke such exclusive approval or licen-
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sure, unless the sponsor of the application for such
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drug demonstrates with respect to an application ap-
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proved or a license issued after the date of enact-
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ment of this subsection, upon such approval or
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issuance, that there is no reasonable expectation at
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the time of such approval or issuance that the cost
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of developing and making available in the United
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States such drug for such disease or condition will
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be recovered from sales in the United States of such
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drug, taking into account all sales made or reason-
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•HR 456 IH
ably expected to be made within 12 years of first
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marketing the drug.
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‘‘(2) CONSIDERATIONS.—For purposes of para-
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graph (1), the Secretary and the sponsor of the ap-
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plication for the drug designated for a rare disease
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or condition described in such paragraph shall con-
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sider sales from all drugs that—
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‘‘(A) are developed or marketed by the
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same sponsor or manufacturer of the drug (or
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a licensor, predecessor in interest, or other re-
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lated entity to the sponsor or manufacturer);
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and
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‘‘(B) are covered by the same designation
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under section 526.
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‘‘(3) CRITERIA.—No drug designated under
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section 526 for a rare disease or condition pursuant
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to the criteria set forth in subsection (a)(2)(B) of
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such section shall be eligible for exclusive approval
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or licensure under this section unless it met such
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criteria under such subsection on the date on which
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the drug was approved or licensed.’’.
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Æ
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