title: New and evolving rare diseases research programs at the National Institutes of Health.
abstract: Research emphasis on rare diseases and orphan products remains a major focus of the research Institutes and Centers of National Institutes of Health (NIH). NIH provides more than USD 31 billion annually in biomedical research and research support. This research is the basis of many of the health advances in rare and common diseases. Numerous efforts and a major emphasis by the public and private sector initiatives have resulted in an increase of interventions and diagnostics for rare diseases. Newer translational research programs provide a more systematic and coordinated approach to rare diseases research and orphan products development. The approach that is offered requires extensive public-private partnerships with the pharmaceutical industry, contract research organizations, philanthropic foundations, medical and scientific advisory boards, patient advocacy groups, the academic research community, research and regulatory scientists, government funding agencies, and the public. Each program is unique and requires lengthy planning and collaborative efforts to reach programmatic goals.
authors: Office of Rare Diseases Research, National Center for Advancing Translational Sciences, National Institutes of Health, Bethesda, Md., USA.
authors: S C
authors: SC
authors: Groft
authors: Y R
authors: YR
authors: Rubinstein
mesh_terms: D035843:Biomedical Research / Q000639:trends*; D003299:Cooperative Behavior; D016208:Databases, Factual; D004345:Drug Industry; D006801:Humans; D009316:National Institutes of Health (U.S.); D055818:Public-Private Sector Partnerships; D035583:Rare Diseases / Q000175:diagnosis* / Q000628:therapy*; D012042:Registries; D012109:Research Support as Topic; D057170:Translational Research, Biomedical; D014481:United States