FDASIA Places Emphasis on Patient-Focused Drug Development

On July 9, President Obama signed into law the US Food and Drug Administration Safety and Innovation Act (FDASIA). The bipartisan legislation is the culmination of more than a year of negotiations and aims to spur the development of lifesaving treatments and therapies for the approximately 30 million Americans who suffer from rare diseases. The act also gives the FDA the authority to collect user fees from industry to fund reviews of innovator drugs, medical devices, generic drugs, and biosimilar biologics.

On July 9, President Obama signed into law the US Food and Drug Administration Safety and Innovation Act (FDASIA). The bipartisan legislation is the culmination of more than a year of negotiations and aims to spur the development of lifesaving treatments and therapies for the approximately 30 million Americans who suffer from rare diseases. The act also gives the FDA the authority to collect user fees from industry to fund reviews of innovator drugs, medical devices, generic drugs, and biosimilar biologics.

Other provisions within the FDASIA are primed to enhance and increase patient participation in medical product regulation. Policy and Medicine notes that “the new law will allow the agency to develop and implement strategies to solicit the views of patients during the medical product development process and consider the perspectives of patients during regulatory discussions.” Just how involved will patients be? Through the end of the summer, the FDA “will develop preliminary list of 20 disease areas for public comment to inform planning for the set of 20 PDUFA V meetings,” in addition to the creation of a “basic roadmap that could be used by patient groups interested in pursuing need for and development of patient-reported outcomes measures in a specific disease area.”

In September of this year, the FDA will post a federal register notice containing the preliminary list of 20 disease states that will be open to public commentary. Following the release of that list, the FDA plans to hold a public meeting in October with the intention of identifying “important but currently unaddressed aspects of their disease experience to potentially be considered in evaluating new therapies.” Perhaps more importantly, the additional input from patients will help improve strategies for identifying important patient outcomes. This increased emphasis on patient perspectives during the medical product development process should help remove communication barriers and increase the flow of important information. In turn, the FDA hopes that this will lead to new drugs, biologics, and medical devices getting to market faster, and to patients sooner.

Additional Resources:

Food and Drug Administration Safety and Innovation Act [FDA Website]

FDA: Food and Drug Administration Safety and Innovation Act (FDASIA) and Patient Focused Drug Development [Policy and Medicine]