The Neuromuscular Disease Foundation (NDF) is proud to be among the eighty five advocacy organizations representing people living with rare diseases that recently came together in support of the rare disease community to urge the Senate to include the S. 4185, the RARE Act, in the FDA Safety and Landmark Advancements Act ( FDASALA). This legislation has significant repercussions for the rare disease community, and together, we made our voices heard. Our final sign-on letter in support of S. 4185, the RARE Act, as introduced by Senators Baldwin and Cassidy, will be included in the FDASALA manager’s amendment.

We are proposing that the RARE Act should clarify the original intent of the Orphan Drug Act (ODA) and codify the Food and Drug Administration’s (FDA) long-standing interpretation of that landmark law, to protect and ensure continued progress in rare disease drug development. The NDF and 84 other organizations stepped in, as we feel that it is our duty as advocates of rare disease patients to preclude the possibility of the Government leaving some rare disease patients, including children, or those with less common variations of a rare disease, without access to an FDA approved treatment that has been proven safe and effective for their specific circumstances and/or condition.

The reasons why the NDF feels impelled to act alongside peer organizations in support of the RARE Act being included in the developing legislation are many, including:

  • The RARE Act is necessary because a recent court case held that the rare disease “designated” at the outset of the drug development process dictates the scope of the orphan drug exclusivity, not the approved “use or indication.” In most cases, the orphan designation is intentionally broader than the use ultimately approved.
  • The RARE Act would maintain the original intent of the Orphan Drug Act, making clear that orphan drug exclusivity is tied to the approved indication, while ensuring proper incentives remain in place to foster robust rare disease drug development.
  • The RARE Act clarification for the scope of orphan drug exclusivity would be applied retroactively and prospectively, which in this specific instance, would actually be maintaining the status quo, since the FDA’s decades-long interpretation of the Orphan Drug Act is that the exclusivity protects the “use or indication” ultimately approved.
  • Any efforts to make the RARE Act provisions only prospectively apply would create a very confusing and challenging regulatory environment for rare disease drug development and leave little incentive to continue to study the safety and efficacy of already approved drugs in special populations, like children.

We are proud of the efforts and progress that the rare disease community has made thus far on the RARE Act, but there is still more to do!


If you would like to help: If you have relationships with Democrats or Republicans on the Senate HELP Committee, and you would be willing to reach out to share your support for Senator Baldwin’s amendment, that would be incredibly helpful toward ensuring that the RARE Act will remain in effect in the final version of the FDA Safety and Landmark Advancements Act (FDASALA).

Thank you to everyone for joining us and taking action!