Recognizing the Importance of the Orphan Drug Act's Exclusivity Incentives in Rare Disease Therapy Development

Floor Speech

BREAK IN TRANSCRIPT

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 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.

BREAK IN TRANSCRIPT

Source