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medical researchFDA Launches Rare Disease Framework Initiative: What It Could Mean for Our Community Highlights
A Cure In Sight
Oct 18 · Raleigh
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Aug 12 · Raleigh
medical researchNew Immunotherapy Research May Help Strengthen Future Cancer Treatments
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medical research28th Annual V Foundation for Cancer Research Wine Celebration Raises Nearly $7 Million to Accelerate Breakthrough Cancer Research
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medical researchHope Beyond a Rare Diagnosis: One Father’s Uveal Melanoma Journey Shows the Power of Clinical Trials
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The V Foundation for Cancer Research Announces Recipients for the 2026 Breaking Barriers in Cancer Research Grant Award
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Jul 20 · Cary
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Jul 18 · Raleigh
medical researchGlioblastoma Foundation Marks 10 Years with New Push to Accelerate Personalized Treatment
Glioblastoma Foundation
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medical researchThe V Foundation for Cancer Research and ESPN Announce Return of Annual ESPYS Week Charity Radio Auction
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medical researchWNBA Legends Unite Sports, Culture and Community in Support of the V Foundation for Cancer Research
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Why Glioblastoma Treatment is Becoming More Personalized - Glioblastoma Treatment & Research
Glioblastoma Foundation
Apr 8 · Durham
medical researchPhase 1/2a study on Lisavanbulin Published in Journal Cell Reports Medicine - Glioblastoma Treatment & Research
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Glioblastoma Foundation
Apr 11 · Durham
medical researchGenomic Testing and 2021 World Health Organization Glioblastoma Classification - Glioblastoma Treatment & Research
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Jan 14 · Durham
medical researchGlioblastoma Foundation® Acquires Drug Lisavanbulin - Glioblastoma Treatment & Research
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Oct 24 · Durham

FDA Launches Rare Disease Framework Initiative: What It Could Mean for Our Community Highlights
If you’re living with a rare disease, or caring for someone who is, you know how frustrating it can be to wait for treatments that seem to take forever to arrive. That’s why a new announcement from the U.S. Food and Drug Administration caught our attention, and we wanted to share it with our community.
On October 14, 2026, the FDA launched the Rare Disease Framework Initiative, an effort to rethink how treatments for rare diseases are developed and reviewed. As a patient-founded, patient-led community, we were especially glad to see patients placed at the center of this work.
According to the FDA’s white paper, about 30 million Americans, roughly one in ten, live with a rare disease. In the U.S., a disease is considered rare when it affects fewer than 200,000 people, and many affect far fewer. Yet only about 5% of recognized rare diseases have an FDA-approved treatment. The FDA also notes that many patients end up using treatments approved for other conditions because nothing has been proven for their specific disease.
The white paper is honest about the challenges. Patient groups are small and often spread out geographically, which makes clinical trial enrollment slow. Information about how a disease naturally progresses is often limited, and the tools commonly used in larger studies may simply not be available. The FDA says rare diseases need an approach based on the evidence that can realistically be gathered, the type of treatment, and what patients need, and that this approach should be used consistently across its review teams.
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