RESUMO
Policy Points The Orphan Drug Act (ODA) was the result of patient advocacy and by many measures has been strikingly successful. However, approximately 95% of the more than 7,000 known rare diseases still have no US Food and Drug Administration-approved treatment. The ODA's success led to sustained criticism of high drug prices, often for products that have orphan drug indications. Critics misconstrue the ODA's intent and propose reducing its incentives instead of pursuing policies focused on addressing broader prescription drug price challenges that exist in both the orphan and nonorphan drug market. Patients and their families will continue to defend the purpose and integrity of the ODA and to drive investments into rare disease research and clinical development.
Assuntos
Aprovação de Drogas , Produção de Droga sem Interesse Comercial , Estados Unidos , Humanos , Doenças Raras/tratamento farmacológico , United States Food and Drug AdministrationAssuntos
Continuidade da Assistência ao Paciente , Atenção à Saúde , Modelos Teóricos , Doenças Raras/terapia , Pesquisa Translacional Biomédica , Criança , Acessibilidade aos Serviços de Saúde , Humanos , Equipe de Assistência ao Paciente , Garantia da Qualidade dos Cuidados de Saúde , Consulta Remota , Estados Unidos , United States Food and Drug AdministrationRESUMO
One of the remarkable and unique aspects of the recent history of rare disease research has been the evolving role of patient advocacy groups and the collaborative partnership that exists among such groups and the scientists who study rare diseases, as well as the government officials charged with overseeing medical research and regulatory processes. This collaboration, which in many respects developed out of necessity on all sides, is unparalleled in other areas of medical research and product development. It has played a significant role over the past 30 years in the adoption of public policies, available research funding and other factors affecting the general climate for research on rare diseases. Specific areas of interest include the adoption of the Orphan Drug Act in the U.S. in 1983 and subsequent similar legislation elsewhere in the world; the relationship of patient advocacy groups with government research funding and regulatory entities; the role of patient advocacy groups in seeking to "de-risk" orphan product development through initiatives such as facilitating patient registries and disease natural histories; the role of advocacy groups in ensuring that patients have access to treatments; and the increasing globalization of patient advocacy initiatives.
