Congress should protect the intent of the Orphan Drug Act and pass the RARE Act

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Rare disease patients, including children, deserve safe and effective therapies.

By Karin Hoelzer, Director of Policy and Regulatory Affairs at NORD 

This year marks the 40th anniversary of the Orphan Drug Act (ODA), a law that has transformed rare disease drug development.

The ODA provides critical incentives for rare disease research and development, including funding and tax credits for clinical research to help encourage drug companies to undertake basic drug development for rare diseases. Another key ODA incentive is orphan drug exclusivity – which bars FDA from approving another company’s marketing application for the same drug to treat the same orphan disease for 7 years after the first drug is approved. Unfortunately, FDA’s longstanding interpretation of this exclusivity was challenged by a recent court case.

The case, Catalyst Pharms., Inc. v. Becerra, centered on how orphan drug exclusivity should be awarded – specifically, whether it should be based on a drug’s FDA-approved indication or the usually much broader designation. Drug companies obtain designations early in the drug development process to unlock ODA incentives, before the drug has been extensively studied.  Once it has been proven to be safe and effective, it is given an FDA-approved indication. For instance, a drug might be designated for the treatment of a given rare disease. Assume, after conducting clinical studies, the drug company is only able to demonstrate the drug’s safety and effectiveness for a narrower population subgroup, such as only adults with the disease, and so it is FDA approved to only treat adults with the rare disease, not a child.  Under the court ruling, if another drug company studies the drug and shows its effectiveness in children with the diseases, FDA would be barred from approving the second company’s drug for the period of orphan drug exclusivity. Moreover, by broadly blocking competition, the court ruling would disincentivize the first company from further studying the drug.

This court’s decision threatens to undermine 40 years of how the FDA has interpreted this aspect of the Orphan Drug Act and would leave little incentive for manufacturers to continue to study the safety and efficacy of orphan drugs in special populations, like children. More than half of people with rare diseases are children, so the implications of this Court ruling have the potential to be profound. Recognizing how problematic this would be, NORD and 77 other patient organizations sent a letter to the Senate HELP Committee urging them to pass the RARE Act.

In a major victory for the rare disease community, the RARE Act (S. 1214), introduced by Senator Tammy Baldwin of Wisconsin and co-sponsored by Senator Mike Braun of Indiana, clarifies the original intent of the Orphan Drug Act (ODA) and codify the Food and Drug Administration’s (FDA) longstanding interpretation that orphan drug exclusivity is limited to indications that were actually studied and shown to be safe and effective.

Thank you Senators Baldwin and Braun for championing this important issue and for recognizing that all rare disease patients deserve safe and effective treatments!

Join NORD’s National Policy and Advocacy Task Force to lend your voice to the rare disease community and advocate on this and other federal or state policy issues.