Finding Hope in the FDA’s New Rare Disease Framework Initiative

By Denise Robinson, Executive Director, EHE Foundation

This week I had the opportunity to attend a public meeting at the U.S. Food and Drug Administration (FDA) to explore the ‘patient voice in rare disease medical product development’, a topic that aligns directly with our mission at EHE Foundation. Your voice - the person living with epithelioid hemangioendothelioma (EHE) - is the one that matters most.

Unexpectedly, just ahead of the meeting, the FDA announced a new broad-reaching initiative to develop a fit-for-purpose regulatory framework tailored to the unique challenges of rare disease drug development. This framework will consider the nuances of rare diseases, patients’ needs, the product type under review, and what data or evidence small patient populations can realistically generate to support the product review. In FDA’s own words, they aim “to reimagine the development of drugs and biologics for rare diseases.”

Why showing up matters for people with EHE

For an ultra-rare cancer like EHE, these opportunities and initiatives can transform how the FDA reviews treatments in development. EHE does not fit the current regulatory framework for drug development, which is built for and well-used for more common cancers.

If you or someone you love is affected by EHE, I encourage you to raise your voice. By documenting that you have the disease and how it affects your life, you can help the FDA realistically explore patients’ voices; their stated goal is to hear you. Shouting isn’t required. This is a breakthrough moment for people with EHE, and at EHE Foundation we’re showing up to let the FDA know that you/we have a voice and it needs to be heard.

Personally, I find a lot of hope in the FDA’s efforts to modernize its rare-disease framework, and I look forward to putting EHE to the test in FDA review.