(R1) Impact of Accelerated Approval: Focus on Medications for Rare Diseases, Oncology, and Alzheimer's Disease

In 1992, the U.S. Food and Drug Administration (FDA) established the Accelerated Approval Program to provide early access to medications for patients with serious and life-threatening conditions that fill an unmet need. The drug approval process for accelerated approval uses surrogate endpoints or intermediate clinical endpoints for approval that may reasonably predict the clinical benefit of the drug. The risk of using surrogate endpoints is that they may not represent a clinically meaningful outcome. This presents a challenge to payers who are managing very high-cost therapies with uncertain clinical effectiveness, especially when looking at medications used to treat rare diseases, cancer, and Alzheimer's Disease.

This session will discuss the accelerated approval process and how the therapeutic agents approved by this pathway may be managed. The session will also focus on three therapeutic areas that are impacted by the accelerated approval pathway – rare diseases, cancer, and Alzheimer's Disease.