ABSTRACT
UNLABELLED: There are more than 6000 rare diseases (defined as affecting <5/10 000 individuals in Europe, <200 000 people in the United States). The rarity can create problems including: difficulties in obtaining timely, accurate diagnoses; lack of experienced healthcare providers; useful, reliable and timely information may be hard to find; research activities are less common; developing new medicines may not be economically feasible; treatments are sometimes very expensive; and in developing countries, the problems are compounded by other resource limitations. Emphasis is required to support appropriate research and development leading to better prevention, diagnosis and treatments of rare diseases. Notably, clinical trials using already existing drugs may result in new, affordable, treatment strategies. Moreover, rare diseases may teach us about common disorders. CONCLUSIONS: Countries are encouraged to implement specific research and development activities within their individual capabilities, so that patients worldwide have equal access to necessary interventions to maximize the potential of every individual.
Subject(s)
Biomedical Research , Global Health , Health Policy , Health Services Accessibility , Rare Diseases , Biomedical Research/ethics , Biomedical Research/legislation & jurisprudence , Clinical Trials as Topic/ethics , Clinical Trials as Topic/legislation & jurisprudence , Health Policy/legislation & jurisprudence , Health Services Accessibility/ethics , Health Services Accessibility/legislation & jurisprudence , Humans , Orphan Drug Production/ethics , Orphan Drug Production/legislation & jurisprudence , Patient Advocacy , Patient Rights , Rare Diseases/diagnosis , Rare Diseases/therapySubject(s)
Orphan Drug Production/legislation & jurisprudence , Rare Diseases/drug therapy , Botulism/drug therapy , Clinical Trials as Topic , Drug Approval , Drug Industry , History, 20th Century , Humans , Immunoglobulins/therapeutic use , Immunoglobulins, Intravenous/therapeutic use , Orphan Drug Production/history , Rare Diseases/history , United States , United States Food and Drug AdministrationABSTRACT
For nearly a quarter of a century the FDA Office of Orphan Products Development has administered the US Orphan Drug Act, which assists in bringing a wide variety of drug and biological (drug) products to treat rare diseases to market. Enthusiasm for rare disease product development has been sustained, seen throughout a wide spectrum of product types and disease conditions, and has resulted in clinically meaningful medical advances. Development of programmes for rare disease treatment worldwide, coupled with the development of drugs for diseases affecting developing countries, attests to the strength of this legislation. The marketing of almost 300 products in the US for rare diseases also testifies to the depth and intensity of scientific endeavour in this area.
ABSTRACT
Disorders resulting from inborn errors of metabolism (IEM) affect very small numbers of individuals. The entire population, however, of patients suffering the results of inherited metabolic disorders is large, and has been of increasing concern to patient groups and health care professionals in the United States as well as other countries throughout the world. The 1983 US Orphan Drug Act (ODA) serves to facilitate the development of drugs to treat rare diseases by providing several economic incentives. The sponsor of a product designated as an orphan by the Food & Drugs Administration (FDA) Office of Orphan Products Development (OPD) qualifies for tax credits on clinical trial expenses, the award of grant funding by FDA, through the OPD, and 7 years of marketing exclusivity for a designated drug, or biological product that receives FDA market approval. Orphan drug legislation in the US has benefited victims of IEM by encouraging development of drugs for metabolic deficiencies affecting populations that otherwise would be ignored. America's solution to the orphan drug problem has had worldwide impact. The success of this legislation was a factor leading to the 1993 orphan drug law in Japan; the 1997 implementation of a process whereby most FDA-approved orphan drugs and biological products will be similarly approved in Australia; and, in 1999, regulation on orphan medicinal products in the European Union (EU). Today, international support for rare disease research is providing stimulus and motivation to overcome the financial barriers and encourage development of treatment for very rare diseases throughout the world.
Subject(s)
Legislation, Drug , Metabolism, Inborn Errors/drug therapy , Orphan Drug Production/legislation & jurisprudence , Rare Diseases/drug therapy , Humans , United States , United States Food and Drug Administration/legislation & jurisprudenceABSTRACT
Over the past 20 years, incentives of the Orphan Drug Act (ODA), the largest single source of extramural clinical grants at the US Food and Drug Administration, have had a substantial impact on public health. ODA incentives have contributed to the development of many innovative biotechnology products, and as our understanding of the human genome evolves, it is anticipated that pharmacogenomics will result in the identification of more 'orphan diseases'.
