[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

                                 ______
                                 

                         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.

                          ____________________