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A Economia das Doenças Raras: Incentivos e Regulação

DOI: http://dx.doi.org/10.18836/2178-0587/ealr.v5n1p69-98

http://portalrevistas.ucb.br/index.php/EALR/index 

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Ramon Wiest1, Giácomo Balbinotto Neto2 & Franciele Cipriani3

 

Resumo: O objetivo deste artigo é realizar uma revisão teórica e empírica da economia das doenças raras e das drogas órfãs bem como analisar as implicações e o impacto na saúde pública. Pretende-se identificar a magnitude do problema, sua importância atual e descrever os principais instrumentos regulatórios destinados a este mercado. As doenças raras apresentam baixa prevalência em uma determinada população, sendo em geral degenerativas, cronicamente debilitantes, necessitam de tratamento contínuo, afetam capacidades físicas, mentais, sensoriais e comportamentais do paciente. Drogas órfãs são medicamentos usados para o diagnóstico, prevenção e tratamento de doenças raras. A raridade destas doenças gera dificuldades para a comprovação da eficácia clínica de medicamentos desenvolvidos para tratá-las. Serão apresentados os sistemas de regulação dos Estados Unidos e da União Europeia e sua influência sobre o desenvolvimento de medicamentos órfãos. Concluiu-se que os mecanismos regulatórios são capazes de estimular o desenvolvimento de drogas órfãs.

Palavras-chave: Doença Rara; Droga Órfã; Regulação; Saúde.

 

Abstract: 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.

Key words: Rare Disease; Orphan Drug; Regulation; Health.

 

1 PPGE/UFRGS. E-mail: ramon.wiest@ufrgs.br.
2 IATS/UFRGS. E-mail: giacomo.balbinotto@ufrgs.br.
3 Ciências Farmacêuticas/UFRGS. E-mail: francielecipriani@yahoo.com.br.

 

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