FDA’s Recent Guidance on Accelerated Approval and Implications for Rare Diseases

Lexology February 28, 2025
Morgan Lewis & Bockius LLP

In honor of Rare Disease Day on February 28, 2025, we will publish a series of posts throughout the month on As Prescribed and Health Law Scan, focusing on issues impacting the rare disease community.

The US Food and Drug Administration (FDA) recently published two new draft guidances to implement provisions of the Food and Drug Omnibus Reform Act (FDORA) aimed at refining the accelerated approval pathway. These guidances seek to address regulatory challenges and enhance oversight of drug products granted accelerated approval. Additionally, a recent report from the Office of the Inspector General (OIG) underscores FDA’s efforts in refining the accelerated approval pathway. These efforts raise important implications for the rare disease community.

FDA’s accelerated approval authority, described in...

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