Accelerated approval (FDA)

Accelerated approval is a pathway established by the United States Food and Drug Administration (FDA) to provide earlier access to beneficial new drugs and biological products for the treatment of serious or life-threatening illnesses where there is an unmet medical need. This pathway is based on the use of a surrogate endpoint or an intermediate clinical endpoint.

Key Concepts

• Serious or Life-Threatening Illnesses: Accelerated approval is reserved for diseases or conditions where the potential for significant morbidity or mortality is high.

• Unmet Medical Need: This indicates that there are no existing treatments, or current treatments are not adequately effective, safe, or tolerable.

• Surrogate Endpoint: A surrogate endpoint is a laboratory measurement, radiographic image, physical sign, or other measure that is thought to predict clinical benefit but is not itself a measure of clinical benefit. It is used because measuring the actual clinical benefit (e.g., increased survival, improved quality of life) may take a very long time, delaying access to potentially life-saving treatments.

• Intermediate Clinical Endpoint: This is a measurement of a therapeutic effect that is considered reasonably likely to predict clinical benefit. Like a surrogate endpoint, it allows for earlier approval than waiting for definitive clinical outcome data.

Post-Approval Requirements

A key component of accelerated approval is the requirement that the applicant conduct post-approval studies to verify and describe the anticipated clinical benefit. These studies are often referred to as "confirmatory trials."

• Confirmatory Trials: These trials are designed to demonstrate that the surrogate or intermediate endpoint used for approval actually translates into a clinically meaningful benefit for patients.

Consequences of Failure

If the confirmatory trials fail to verify the anticipated clinical benefit, the FDA may withdraw the accelerated approval.

Mechanism

The legal basis for accelerated approval is found in the FDA's regulations implementing the Federal Food, Drug, and Cosmetic Act. The pathway is intended to balance the need for timely access to potentially life-saving treatments with the need to ensure that those treatments are ultimately effective and safe.

Purpose

The purpose of accelerated approval is to speed up the availability of treatments for serious conditions while still maintaining appropriate standards of safety and efficacy. It is a risk-benefit assessment that allows patients earlier access to potentially beneficial drugs, with the understanding that further research will be conducted to confirm those benefits.