Part 1 FDA Expedited Programs Series: Accelerated Approval

Objective

Allows drugs for serious conditions that fill an unmet medical need to be approved based on a surrogate or intermediate clinical endpoint. Using a surrogate endpoint enabled the FDA to approve these drugs faster.

Criteria

A surrogate endpoint is a marker thought to predict clinical benefit and can include:

·         Laboratory measurement

·         Radiographic image

·         Physical sign or other measure

An intermediate clinical endpoint is a measure of a therapeutic effect that is considered reasonably likely to predict the clinical benefit of a drug, such as an effect on irreversible morbidity and mortality (IMM).

FDA bases its decision on whether to accept the proposed surrogate or intermediate clinical endpoint on the scientific support for that endpoint. Studies that demonstrate a drug’s effect on a surrogate or intermediate clinical endpoint must be “adequate and well controlled” as required by the FD&C Act.

The drug company will still need to conduct studies to confirm that the surrogate endpoint actually predicts the anticipated clinical benefit. These studies are known as phase 4 confirmatory trials. Approval of a drug may be withdrawn or its label indication may be changed if these fail to verify clinical benefit or if the benefit is not sufficient to justify the risks associated with the administration of the drug.

Potential Benefits

·         Saves valuable time in the drug clinical evaluation process

Accelerated Approval Process

·         Sponsors should communicate early in development the potential eligibility of a drug for accelerated approval.

·         Conditions of Accelerated Approval include:

o   Preapproval of promotional materials

o   Conduct confirmatory studies

 

·         Particulars are found in Guidance for Industry: Expedited Programs for Serious Conditions – Drugs and Biologics (2014)