Robert Metcalf on PFDD

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. There’s no doubt that the feedback gained through the PFDD Initiative gives the FDA deeper insight into patient preferences, especially regarding the benefits and risks of already-approved medicines. However, in order for it to have a meaningful impact on drug development review, the Agency now needs to articulate how this feedback will be incorporated into the benefit/risk assessments of new individual therapies being actively reviewed. So far, the FDA has held PFDD meetings with representatives from 11 disease states, including fibromyalgia, lung cancer, and sickle cell disease. These meetings have illuminated some of the day-to-day difficulties of living with a particular condition and offered critical perspective into what some individual patients think about the benefits and risks of available treatment options. The FDA is a science-driven organization, and also needs mechanisms to efficiently obtain input from large, targeted, and representative cohorts of patients in order to more effectively use patient data in infor...
Source: PHRMA - Category: Pharmaceuticals Authors: Source Type: news