Rare Oncology & The FDA: Taking The Guesswork Out Of Expedited Pathways

By Vanessa Atayde

According to reports, rare cancers contribute to 25-30% of new cancer diagnoses and 25% of cancer-related deaths. Their complex nature, including poorly understood natural histories, phenotypic heterogeneity, and diverse clinical presentations, presents obstacles to drug development and creates a substantial unmet need in the field of oncology.

Given the scarcity of treatment options, researchers, clinicians, and patients are actively exploring ways to expedite the development and approval of innovative therapies. On the regulatory side of things, the U.S. Federal Drug Administration initially outlined its approach to expediting the availability of promising new therapies in Title 21 of the Code of Federal Regulations, specifically in Section 312, Subpart E. These regulations aim to fast-track the accessibility of new therapies for patients with critical conditions, particularly when there are no satisfactory alternative treatments available. However, the FDA ensures that appropriate standards for safety and efficacy are upheld throughout the process.

Review this collection of available regulatory programs used to accelerate treatments for rare disorders and serious diseases.