Clinical Trial 2.0: Can Health 2.0 Transform the FDA Drug Approval Process?

Abstract

Historically, patient activism has played a great part in shaping the drug approval process. Today, aided by developing web technologies, patients are once again seeking increased involvement in their medical care. Their desire to be involved is manifesting in the development of patient-run clinical trials, where patients, with the aid of online health-oriented social networks, are testing the safety and effectiveness of new drugs. While these trials are not currently recognized as valid, scientifically rigorous endeavors and are not accepted by the FDA as evidence of the safety and effectiveness of new drugs, small changes in trial structure can make these patient initiatives a meaningful part of the drug development process. This paper examines the structure and challenges of patient-run clinical trials in the context of the drug development and approval process and suggests an point-of-care design alternative as a way to address the concerns aroused by such patient-led studies. Ultimately, the paper concludes that the enormous social value of such studies should not be ignored, and that the medical, scientific, and regulatory communities should work with patients in helping them designing reliable studies responsive to their needs that can become a meaningful part of the drug development and approval process.