On May 21st 2012, the National Organization for Rare Disorders (NORD) hosted a forum in Washington DC to assess the rare-disease community's performance when campaigning on the next revision of PDUFA (the Prescription Drug User Fee Act), which must be renewed by Congress every five years. Originally enacted in 1992, the PDUFA authorizes the Food and Drug Administration (FDA) to collect fees from companies seeking to have their products reviewed. The Act funds the FDA, and allows it to review new drugs quickly. The current legislative authority for the PDUFA IV, affirmed in 2007 by the FDA Amendments Act, is due to expire in September 2012 (after which it is set to be renewed). US patient organizations have been lobbying for Congress to allow the FDA to be better funded in their specific disease areas. Among the most successful has been NORD. Steven Grossman, Deputy Executive Director of the Alliance for a Stronger FDA, believes that the rare-disease community has gained several concessions for the patients with a rare disease—not least an increase in staffing for rare disease programs in the FDA's Center for Drug Evaluation and Research (CDER) and Center for Biologics Evaluation and Research (CBER); increased opportunities for interaction between reviewers and rare-disease patient representatives, as well as industry; FDA staff training on rare-disease issues and orphan products. Vice President for Public Policy at NORD, Diane Dorman, noted at the May 21st forum that new legal provisions should also help in the development of desperately needed medical devices for small patient populations. Dr Gayatri Rao, Acting Director of the FDA's Office of Orphan Products Development (OOPD), said that the FDA is forming a new Rare Disease Council, to increase communication among all FDA centres on rare-disease and orphan products. CommentsLeave a Reply |