Leaders of the National Health Council (NHC) recently applauded the U.S. Food and Drug Administration (FDA) for adding new patient engagement goals, which are now part of the performance goals letter that reauthorizes the Prescription Drug User Fee Act (PDUFA).
The act accounts for more patient engagement, encouraging industries to include patient perspectives in their developments and research. This addition will improve the involvement of patients in the scientific process of developing and approving novel treatments and therapies for diseases.
New provisions include endpoints for biomarkers and surrogates. This means there will be a public meeting in which the FDA will provide guidance for creating biomarkers. The organization will heighten staff capacity to handle biomarkers, develop a public website and discuss new surrogate endpoints.
Other changes apply to rare diseases, real-world evidence (RWE), FDA hiring and retention, and combination product review.
“This agreement contains provisions that will, over time, significantly improve the lives of people with chronic diseases and disabilities and their families,” NHC CEO Marc Boutin said. “These efforts will result in a giant step forward for the FDA’s Patient Focused Drug Development program, which was created by the last reauthorization of PDUFA.”
One of the more significant changes relates to patient engagement.
“The agency has strengthened its commitment to making patient engagement an integral aspect of drug development, and we look forward to working with them to make their goals a reality,” Boutin said. “Other provisions included in the agreement -- such as increased emphasis on biomarkers and surrogate endpoints, advancing development of drugs for rare diseases, streamlining combination product reviews, studying the feasibility of incorporating real-world evidence provided from clinical settings, and improving FDA hiring and retention practices -- will lead to a more efficient FDA that is better equipped to review 21st century treatments in a timely fashion.”
