Hill Day Issue Information

Legislative

Ask #2 Support Rare Disease Appropriations Priorities

Appropriations Requests:

• Support increased funding for rare disease research at both the FDA and NIH:

• Fully fund the Orphan Products Grant Program to help support the critical programs that fund clinical trials and natural history studies to aid in the development of new rare disease therapies.

• Increase funding to help NCATS leverage its robust rare disease research program to accelerate the development of new treatments.

• Support the National Academy of Medicine to facilitate a scientific process around defining ultra-rare diseases and explore the opportunities and implications around such a definition. The process would include a consensus-based approach, convening the rare disease community, regulators, and researchers.

Talking Points:

• Investments into rare disease research benefits everyone.

• A 2022 study showed that the conservatively estimated impact of rare diseases in the U.S. in 2019 was nearly $1 trillion. Discovery of treatments for the more than 95% of rare diseases with no FDA approved therapy – and investments into opportunities to improve health outcomes -- is a societal priority.

• For many rare diseases, treatments are now within reach.

• Advances in the understanding of rare diseases and novel technology platforms have transformed many disease pipelines. Yet we must ensure that incentives and regulatory pathways exist for all rare diseases to make development financially viable for companies.

• Natural history studies are essential for powering rare disease therapeutic development.

• Robust natural history studies and very small population clinical research are critical to development programs, yet funding sources are extremely limited.

• The ODA reshaped the rare disease landscape, yet more remains to be done.

• The incentives within the Orphan Drug Act helped to exponentially increase the number of FDAapproved rare disease therapies.

• Small, rare disease populations (ultra-rare diseases) still face significant challenges in the development of new therapies and may require additional policy and regulatory considerations to address the unique complexities of therapy development.