The Economics of Rare Diseases: incentives and regulation


  • Giacomo Balbinotto Neto UFRGS
  • Ramon Wiest UFRGS - FCE
  • Franciele Cipriani PPGF/UFRGS



The 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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Author Biographies

Giacomo Balbinotto Neto, UFRGS

Giácomo Balbinotto Neto

Giácomo Balbinotto Neto - possui graduação em Ciências Econômicas pela Universidade Federal do Rio Grande do Sul (1985), Mestrado em Economia pelo Instituto de Estudos e Pesquisas Econômicas (IEPE, 1990) e doutorado em Economia pela Universidade de São Paulo (2000). Atualmente é professor adjunto da Universidade Federal do Rio Grande do Sul (UFRGS) junto ao Programa de Pós-Graduação em Economia (PPGE).

Ramon Wiest, UFRGS - FCE


Franciele Cipriani, PPGF/UFRGS

Mestre em Farmácia/UFRGS