[Congressional Record Volume 171, Number 32 (Tuesday, February 18, 2025)]
[Extensions of Remarks]
[Pages E135-E136]
From the Congressional Record Online through the Government Publishing Office [www.gpo.gov]
RECOGNIZING THE IMPORTANCE OF THE ORPHAN DRUG ACT'S EXCLUSIVITY
INCENTIVES IN RARE DISEASE THERAPY DEVELOPMENT
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HON. GUS M. BILIRAKIS
of florida
in the house of representatives
Tuesday, February 18, 2025
Mr. BILIRAKIS. Mr. Speaker, in honor of February marking Rate Disease
Month, I rise today to reaffirm the critical importance of the Orphan
Drug Act's exclusivity incentive to the development of life-changing
therapies for patients with rare diseases.
Congress designed the Orphan Drug Act to provide meaningful
incentives, including a seven-year period of market exclusivity, to
encourage biopharmaceutical companies to take on the significant risks
and costs associated with developing therapies for rare diseases that
otherwise would not be developed and approved. Orphan exclusive
approval generally prohibits FDA from approving the ``same'' drug for
the same use during this exclusivity period to ensure the innovator
receives adequate protection to justify the many years of investment in
research and development of a product intended for a small population.
This protection is especially critical in cases where there previously
had been no FDA-approved therapy for the rare disease--or condition. In
these instances, the unmet medical needs and the scientific challenges
in addressing these needs are the greatest.
Without the exclusivity incentive in the Orphan Drug Act,
biopharmaceutical companies would never have invested the resources to
develop and commercialize first ever treatments for AADC deficiency,
achondroplasia, Batten disease, epidermolysis bullosa, Friedreich's
ataxia, metachromatic leukodystrophy, mucopolysaccharidoses,
phenylketonuria, and Pompe disease, among many others. The innovators
that pioneered these therapies took substantial risks investing in
these unserved markets and then therefore should receive the full value
of the seven years of exclusive approval.
Despite this, FDA may approve the ``same'' drug for the same use
during an exclusivity period following a demonstration of ``clinical
superiority'' by the sponsor of the follow-on product. Originally
established in 1992 through regulation, FDA intended this condition to
balance the need to ``protect the primary incentive that Congress
created in the Orphan Drug Act'' with ``the development of safer and
more effective orphan drugs.'' In 2017, following two court decisions
that concluded FDA lacked the
[[Page E136]]
authority to establish the clinical superiority pathway, Congress
enacted the FDA Reauthorization Act of 2017 (P.L. 115-52), which
clarified FDA has such authority, and expressly enumerated the three
ways a sponsor could demonstrate clinical superiority for the ``same''
drug for the same use. Specifically, the subsequent product must
demonstrate a ``significant therapeutic advantage'' relative to the
previously approved drug ``in terms of greater efficacy, greater
safety, or by providing a major contribution to patient care.''
Congress intended for this standard to be rigorous, particularly for
``major contribution to patient care'' (which is only considered when
neither greater safety nor greater effectiveness has been shown), to
ensure it is reserved for truly transformative advancements in treating
the underlying rare disorder Congress did not specifically define
``major contribution to patient care,'' intentionally allowing FDA to
exercise its judgment with the understanding that this threshold would
remain high. In fact, FDA's orphan drug regulation itself describes
``major contribution to patient care'' as a ``narrow category'' to be
used in ``unusual cases.'' In the unique circumstance when FDA applies
``major contribution to patient care'' to allow a follow-on product to
break the exclusivity of a previously approved orphan drug, the
subsequent drug must fundamentally elevate the standard of care for the
rare disease or condition by providing a significant measurable
clinical benefit relative to the previously approved drug. Without
maintaining a rigorous threshold, the incentive underpinning the Orphan
Drug Act risks being diluted and diminished, threatening the progress
made in care disease drug development and undermining future
innovation.
As a co-chair of the Rare Disease Congressional Caucus, I am
committed to ensuring that FDA's authority to evaluate whether a
follow-on treatment provides a ``major contribution to patient care''
is exercised judiciously, in a manner that does not diminish the value
of orphan exclusive approval. This incentive is fundamental to driving
new investments into rare disease research and development, leading to
new treatment options and ensuring that patients with significant unmet
medical needs continue to see meaningful innovation. With 95 percent of
the more than 10,000 rare disorders lacking an FDA-approved therapy,
FDA must be especially diligent in protecting the exclusivity period
for innovators that took the initial substantial risk to bring a first-
ever FDA-approved therapy to a rare disease community.
The intent of Congress is clear--orphan drug exclusivity must be
preserved as a powerful incentive for race disease drug development,
and the criteria for demonstrating clinical superiority must not
diminish that value. The Orphan Drug Act has provided tremendous
benefits for patients with rare diseases, and its continued success
depends on the preservation of strong exclusivity incentives for
innovator therapies. Congress expects FDA to apply the clinical
superiority framework as originally intended, maintaining the high
standard requited to protect the value of orphan exclusivity,
especially for those orphan drugs that were the first FDA-approved
therapy for a rare disease or condition. The success of the Orphan Drug
Act and the continued development of rare disease therapies depends on
the integrity of orphan exclusive approval. I urge FDA to remain
steadfast in maintaining an appropriately high bar in its application
of ``major contribution to patient care'' of the clinical superiority
threshold. As we look to Rare Disease Day 2025, recognized on February
28th, the mission of the Orphan Drug Act remains as important as ever.
I urge my fellow members to work with me and with FDA to keep these
incentives strong in order to get new treatments and cures to rare
disease patients in need.
____________________