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Orphan Drug Act's Impact on Rare Disease Patients

Two Zebras in center and rare disease awareness ribbon on left

February 21, 2024

Rare diseases, though individually uncommon, collectively affect a significant portion of the population worldwide. Rare disease impacts an estimated 300 million people worldwide and 30 million in the US alone. Children account for 50% of those diagnosed with rare diseases. Behind the statistics and medical terminology are real families facing unimaginable challenges. The burden of caring for a loved one with a rare disease goes beyond the physical and emotional toll—it often includes financial strain, limited treatment options, and a sense of isolation. 

Families dealing with rare diseases navigate a complex healthcare system that may not prioritize their needs. The Orphan Drug Act (ODA), enacted in 1983 to incentivize the development of treatments for rare diseases, has made significant strides in bringing therapies to market. However, with advancements in medical research and evolving healthcare landscapes, there is a growing need to update and strengthen this legislation.

In June 2017 FDA introduced the Orphan Drug Modernization Act, with the focus to eliminate the backlog and respond to all new applications within 90 days of receipt. These measures have direct impact on research and development timelines resulting in approximately 5% of rare diseases have an FDA approved treatment and another 15% have promising treatments in development. 

In 2020, the Catalyst Pharms., Inc. v. Becerra case made waves in the industry. The U.S. Court of Appeals for the Eleventh Circuit clarified the scope of orphan-drug exclusivity. Under the Catalyst decision, the first company to gain approval for any use of a drug designated for a rare disease will typically have seven years of exclusivity for the entire rare disease. This means that if a drug is approved for a narrower use or indication, it will still block approval of another company’s application for the same drug for any indication within that rare disease, regardless of whether the drug was initially approved only for a specific subset of patients.

This decision countered the previous FDA interpretation in which the Orphan Drug Exclusivity would only block approval of the same drug for the same approved use or indication, allowing drug companies to continue studying and seeking approval for other uses of the drug within the same disease or condition.

The Catalyst decision impacted research and development of medical products for rare diseases by binding the exclusivity to the disease or condition for which the drug was designated as an orphan drug. 

Exclusivity applied to an entire disease, rather that the previously accepted exclusivity for specific use, blocks competition and delays generic competition which directly impacts patients access to these life changing treatments. Not only does this block competition but the court decision disincentivizes the initial research and development company from further studying the drug. Often during research, the patient population is narrowed, genetic mutations are identified, populations are selected for treatment or certain populations are eliminated from receiving treatment. This court decision threatens to undermine the original purpose of the Orphan Drug Act, to help protect and encourage drug companies to research and develop drugs to treat rare diseases.

Considering the Orphan Drug Act as it currently stands, there are many ways to improve ODA and turn the focus back to the families affected by rare diseases. By not only enhancing incentives but also closing loopholes like the practice of “piggybacking” on an older drug to extend market exclusivity for a new version of the same drug, updating tax credits for R&D, passing bills such as the Retaining Access and Restoring Exclusivity Act (RARE) of 2022, and looking for ways to adjust exclusivity not only for the drug developer’s benefit but also based on the specific needs of those diagnosed with rare disease. could better support these vulnerable populations.

Shadow of a family standing in front of a sunset

This is a call to action for all those in the drug development industry. In our pursuit to get drugs to market, we cannot dismiss our mission is rooted in empathy for patients and families. 

Find ways to drive change through National Organization of Rare Disorders, Take Action - National Organization for Rare Disorders ( Together, we can raise awareness, advocate for change, and empower individuals & families impacted by rare diseases. United in our efforts we can ensure the Orphan Drug Act gets the updates needed. 



Chandra, A. (2021, February 8). Reforming the orphan drug act - 1% steps for health care reform - policy briefs. 1% Steps for Health Care Reform.

GAFFNEY, A. (2022, April 20). New bill approaches on Orphan Drug Development. AgencyIQ by POLITICO.

Liu, A. (2020, November 19). Newly passed House bill could close orphan drug loophole that evergreens exclusivity. Fierce Pharma.

Nord applauds congressional efforts to restore intent of the Orphan Drug Act. National Organization for Rare Disorders. (2022, May 12).

Hoelzer, K. (2023, May 11). Congress should protect the intent of the Orphan Drug Act and pass the Rare Act. National Organization for Rare Disorders.

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