The Economics of Rare Diseases: incentives and regulation
AbstractThe objective of this paper is to review theoretical and empirical about the economics of rare diseases and orphan drugs and to analyze their main implications, identifying the magnitude of the problem, its current importance and describe the main regulatory instruments for this market. Rare diseases present a low prevalence in a given population, they are usually degenerative, chronically debilitating, and require long-term treatment, affecting the physical, mental, sensory and behavioral capabilities of the patients. Orphan drugs are used for diagnosis, prevention and treatment of rare diseases. The rarity implies difficulties for proof of their clinical efficacy. The regulatory systems for rare diseases in the United States and European Union will be presented and their influence for development of orphan drugs. It was concluded that the regulatory mechanisms are able to stimulate the development of orphan drugs.
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