Peter L. Saltonstall on PDUFA

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. Drug developers, whether researchers, industry or government, would benefit immensely from understanding better the needs of patients -- what symptoms trouble them most, what is their tolerance for risk, how do their diseases progress and how do patient needs change as a disease progresses.  The issue is especially important for patients with rare diseases, since there often is a lack of information and understanding of rare diseases. At NORD’s recent Rare Diseases and Orphan Products Breakthrough Summit, Janet Woodcock, MD, director of FDA’s Center for Drug Evaluation and Research, set the tone for the entire conference when – in the opening session – she stated that an environment that provides earlier and more frequent patient input into the process would help to accelerate and de-risk orphan product development. Specifically, Dr. Woodcock called for a system that encourages rigorous pre-clinical research and collection of important patient data in registries and natural history studies. NORD agrees with Dr. Woodcock, and we are working with FDA, NIH and our academic and industry partners to facilitate earlier and more frequent patient input throughout the drug development process. One example of this is the new platform NORD has created...
Source: PHRMA - Category: Pharmaceuticals Authors: Source Type: news