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.
This is not rocket science! Running a molecule through all of the traps is really hard. Understanding what people feel and think and experience with regard to the drugs they take and those they need is simple. In most other areas of our lives, various industries know quite well what features we want on our smartphones, that we suddenly all want Greek yogurt, and that we don’t want to pay to use the bathroom on a plane. In medicine, we have not yet realized the same dynamic and robust methods, tools and studies for understanding the needs and values of the end user. The needs, wants, va
As important stakeholders in the drug development and approval process, patients provide a unique and valuable perspective when considering the benefits and risks of potential new and innovative medicines. Patients can identify areas of unmet medical need, provide critical perspective on the impact of a disease and its manifestations, and uniquely inform the development of outcome measures that are meaningful to patients. A scientific approach to gathering patient input is necessary to realize the shared goal of a patient-centered approach to drug development and regulatory review. PhRMA
The most recent reauthorization of the Prescription Drug User Fee Act (PDUFA) included new efforts by the Food and Drug Administration (FDA) to enable patients and patient groups to become actively involved in FDA evaluations of new medicines. Two programs in particular--the Patient-Focused Drug Development (PFDD) Initiative and Benefit/Risk Assessment--work in tandem to inform the FDA's decisions on whether or not to approve a medicine for patient use.
We at NORD believe that patients need to play a central and much more active role in the drug development process, from start to approval. Until very recently, the process has largely ignored the patient as an active participant.