Ryan Fischer on PDUFA

Parent Project Muscular Dystrophy has long held the belief that new approaches are required to expedite the development of treatments for rare diseases like Duchenne. Patient advocates have worked tirelessly with Congress, over time through legislation, to increase the engagement of the patient voice within the regulatory review process for therapeutics.  Passed in 2012, The Food and Drug Administration Safety and Innovation Act (FDASIA) was a culmination of those advocacy efforts by patients.  The legislation included a number of provisions under the patient-focused drug development and patient preference headings that aim to more systematically gather patients’ perspectives on their condition and bring the patient voice to the table like never before.  Within the user fee package, FDA agreed to hold 20 disease specific hearings to begin creating a framework for incorporating the patient voice into the review process. Along with over 7,000 other rare diseases, Duchenne was not one of the “chosen” conditions to be taken up by the agency within the five-year authorization. With a Duchenne pipeline full of potential therapies in or heading to clinical testing, we knew we had little choice but to try and come up with novel approaches to inform the FDA about our patient population and the current state of research.  In 2013, PPMD embarked on the first-ever rigorous scientific survey of benefit/risk.  While patient testimonies and stories are critical to educating indus...
Source: PHRMA - Category: Pharmaceuticals Authors: Source Type: news