A patient-centered approach

April 15, 2012

Patients can serve as checkpoints to guide drug development down the most efficient path.

The increasing value of patient perspectives suggests the evolving outlook on the drug approval process. Patients are now seen as invaluable resources in expressing the reality of a drug. They weigh risks and benefits, and then offer a perspective that is beyond the vision of drug developers. This has encouraged patients to be the voice of those who can't speak and to compel decision-makers to listen. What was once thought to be a rigidly organized approval process now has avenues through which it seeks and integrates public comments at a greater level.

Patients voice their opinions

FDA's Center for Drug Evaluation and Research and Center for Biologics Evaluation and Research offer patients with serious illnesses an opportunity to contribute their thoughts, including during meetings between FDA and pharmaceutical manufacturers in the DDPCP. These patients offer feedback concerning clinical, legal, and regulatory matters. And the PRP allows patients who have personal experience managing a condition to express their issues and concerns.

The role of patient reps

Patient representatives are responsible for providing FDA with this unique perspective on serious or life-threatening conditions.

Patient reps serve as voting or non-voting members on advisory committees while actively communicating concerns related to the product under review. The goal is to aid FDA in making reliable assessments regarding the safety, effectiveness, and appropriate use of the product.

Active involvement in advisory committees encourages public interaction with FDA as it arrives at conclusions. President Obama's dedication to allowing public access to the government spurred the U.S. Department of Health and Human Services to open its proceedings to the public. As a result, FDA Commissioner Margaret Hamburg, MD, introduced a Transparency Initiative in June 2009. Part of the initiative focuses on assimilating public feedback to modifiy the agency's mechanism. The structured patient advocacy programs are taking FDA closer to achieving its transparency goal.