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Mapping benefits, risks, and challenges of expedited drug approval programs
Mapeo de los beneficios, riesgos y desafíos de los programas de aprobación acelerada de medicamentos
Mapeando os benefícios, riscos e desafios dos programas de aprovação acelerada de medicamentos
DOI:
https://doi.org/10.15446/rcciquifa.v55n3.123946Palabras clave:
Drug approval, expedited approval, accelerated approval, early access, neoplasms, antineoplastic agents. (en)Aprobación de medicamentos, aprobación acelerada, acceso temprano, Neoplasias de la mama , agentes antineoplásicos (es)
Aprovação de medicamentos, aprovação acelerada, acesso precoce, Neoplasias, agentes antineoplásicos (pt)
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Objective: This scoping review identified the benefits, risks, and challenges associated with expedited drug approval programs implemented by regulatory agencies. Methods: A systematic search was conducted in Embase, Scopus, PubMed, Lilacs, Cochrane, and Google Scholar, complemented by gray literature, to identify reviews published in English, Spanish, or Portuguese. Data on regulatory agencies, drugs, and approval programs were extracted. Results: Ten regulatory agencies were identified, and their expedited programs were grouped into regulated programs and exceptional-use programs. Regulated programs included priority review, flexible efficacy and safety requirements, accelerated development, collaborative review, and reliance on other agencies’ decisions. Exceptional-use programs addressed high unmet medical needs, public health emergencies, and negative impacts on healthcare coverage. The main benefit was the reduction in approval times (e.g., FDA: average 6.9 months). Among the risks, notable issues included uncertainty stemming from the use of surrogate endpoints, a higher incidence of problems in pivotal trial design (4.06 times more than in regular approvals), noncompliance with post-marketing study requirements after a median of 7.25 years, and the conversion of accelerated approvals into regular approvals without confirmed clinical benefit (79% in the case of the FDA). Challenges included persistent clinical uncertainty, confusion between “early approval” and “timely access”, high drug prices, poor cost–effectiveness ratios, conceptual high opportunity costs, and reliance on FDA/EMA decisions—an especially critical issue for middle- and low-income countries. Conclusion: Regulatory frameworks should optimize expedited approval processes to ensure genuine clinical benefit while safeguarding healthcare systems sustainability.
Objetivo: Esta revisión de alcance identificó los beneficios, riesgos y desafíos de los programas de aprobación acelerada de medicamentos implementados por agencias reguladoras. Métodos: Se realizó una búsqueda sistemática en Embase, Scopus, PubMed, Lilacs, Cochrane y Google Scholar, complementada con literatura gris, de revisiones en inglés, español o portugués. Se extrajeron datos sobre las agencias reguladoras, medicamentos analizados y programas acelerados. Resultados: Se identificaron diez agencias reguladoras y sus programas acelerados implementados, que se agruparon en dos categorías: regulados y de uso excepcional. Los primeros incluyeron programas que priorizan revisiones, flexibilizan requisitos de eficacia y seguridad, aceleran desarrollo, promueven revisiones colaborativas o aplican reliance. Los segundos abordan necesidades médicas insatisfechas, emergencias sanitarias o mitigan impactos negativos en cobertura. El beneficio principal fue la reducción de tiempos de aprobación (Ej. FDA: media 6.9 meses). Entre los riesgos se destacaron la incertidumbre derivada del uso de medidas subrogadas, mayor número de problemas en el diseño de los ensayos fundamentales (4.06 veces más que en aprobaciones regulares), incumplimiento de estudios postcomercialización tras una mediana de 7.25 años y conversión de aprobaciones aceleradas en regulares sin corroborar beneficio clínico (79% en la FDA). Los desafíos incluyeron incertidumbre clínica persistente, confusión conceptual entre “aprobación temprana” y “acceso oportuno”, precios elevados, baja relación costo-beneficio, altos costos de oportunidad y dependencia de decisiones de FDA/EMA, especialmente crítica para países de ingresos medios y bajos. Conclusión: Los marcos regulatorios deben optimizar estos programas para asegurar beneficios clínicos reales y preservar la sostenibilidad de los sistemas de salud.
Objetivo: Esta revisão de escopo identificou os benefícios, riscos e desafios associados aos programas de aprovação acelerada de medicamentos implementados por agências reguladoras. Métodos: Foi realizada uma busca sistemática nas bases de dados Embase, Scopus, PubMed, Lilacs, Cochrane e Google Scholar, complementada por literatura cinzenta, para identificar revisões publicadas em inglês, espanhol ou português. Foram extraídos dados sobre agências reguladoras, medicamentos e programas de aprovação. Resultados: Dez agências reguladoras foram identificadas, e seus programas de aprovação acelerada foram agrupados em programas regulamentados e programas de uso excepcional. Os programas regulamentados incluíam revisão prioritária, requisitos flexíveis de eficácia e segurança, desenvolvimento acelerado, revisão colaborativa e dependência das decisões de outras agências. Os programas de uso excepcional abordavam necessidades médicas não atendidas, emergências de saúde pública e impactos negativos na cobertura de saúde. O principal benefício foi a redução no tempo de aprovação (por exemplo, FDA: média de 6,9 meses). Entre os riscos, os problemas mais notáveis incluíram a incerteza decorrente do uso de desfechos substitutos, uma maior incidência de problemas no desenho de estudos pivotais (4,06 vezes mais do que em aprovações regulares), o não cumprimento dos requisitos de estudos pós-comercialização após uma mediana de 7,25 anos e a conversão de aprovações aceleradas em aprovações regulares sem benefício clínico comprovado (79% no caso do FDA). Os desafios incluíram incerteza clínica persistente, confusão entre “aprovação antecipada” e “acesso oportuno”, preços elevados dos medicamentos, baixas relações custo-efetividade, altos custos de oportunidade conceituais e dependência das decisões do FDA/EMA — uma questão especialmente crítica para países de baixa e média renda. Conclusão: Os marcos regulatórios devem otimizar os processos de aprovação acelerada para garantir um benefício clínico genuíno, ao mesmo tempo que salvaguardam a sustentabilidade dos sistemas de saúde.
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