Rare Diseases and Orphan Products: Accelerating Research and Development

Excerpt

This Institute of Medicine (IOM) study grew out of discussions with the NIH Office of Rare Diseases Research and the FDA Office of Orphan Products Development about opportunities to accelerate rare diseases research and orphan product development. As discussions progressed, the focus expanded from drugs and biologics to include medical devices. In 2009, the IOM appointed a 14-person committee to oversee the study. Consistent with its charge (which is presented in full in Appendix A), the committee examined the epidemiology, impact, and treatment of rare diseases as context for an assessment of research and development; investigated the strengths and limitations of the current development pathways for new drugs, medical devices, and biologics for rare diseases; assessed public policies that may influence research and development decisions involving rare diseases and orphan products; and developed recommendations for an integrated national policy on rare diseases research and orphan product development.

This report presents the committee's conclusions and recommendations. It is written for a broad and diverse audience, including public officials in research and regulatory agencies; advocacy and philanthropic groups that support rare diseases research and orphan product development; companies that develop pharmaceutical, medical device, and biologic products; academic medical centers, research institutes, and researchers engaged in basic and clinical research; and the interested general public.