RESUMEN
Although high upfront costs for the high value of gene therapy have resulted in concerns about sufficient reimbursement to allow patient access to these therapies, the significant benefits of gene therapies will not be realized unless patients have access to them. Stakeholders are discussing these issues, and the payment models being developed for the newly approved gene therapies provide an early indication of the flexibility that will be needed from treatment manufacturers, payers, and policy makers to optimize patient access. Maximizing patient access to effective gene therapies is one integral part of the overall mission of the American Society of Gene and Cell Therapy, along with maximizing the quality of therapies and minimizing their costs.
Asunto(s)
Terapia Genética , Accesibilidad a los Servicios de Salud , Mejoramiento de la Calidad , Animales , Costo de Enfermedad , Análisis Costo-Beneficio , Terapia Genética/efectos adversos , Terapia Genética/economía , Terapia Genética/métodos , Terapia Genética/tendencias , Costos de la Atención en Salud , Humanos , Evaluación de Resultado en la Atención de Salud , Mecanismo de ReembolsoAsunto(s)
Tratamiento Basado en Trasplante de Células y Tejidos/estadística & datos numéricos , Ensayos Clínicos como Asunto/organización & administración , Terapia Genética/estadística & datos numéricos , Investigación Biomédica/ética , Investigación Biomédica/legislación & jurisprudencia , Investigación Biomédica/organización & administración , Tratamiento Basado en Trasplante de Células y Tejidos/tendencias , Terapia Genética/tendencias , Humanos , Colaboración Intersectorial , Legislación de Medicamentos , Sociedades MédicasRESUMEN
The rapid expansion of the gene therapy pipeline in recent years offers significant potential to treat diseases with great unmet medical need. However, the unique nature of these therapies poses challenges to regulating them within traditional frameworks, even when developing in a single country. Various factors exacerbate the issues in commercializing products across regions, including the lack of established regulatory frameworks for developing gene therapy products in many jurisdictions. While some countries have established separate regulatory frameworks for advanced therapies/regenerative medicine products, differences exist between them. Recommended solutions to overcome these hurdles include fostering convergence among countries with separate regulatory frameworks for these products and utilizing reliance and recognition for countries without such frameworks. Additionally, regulators who choose to establish new dedicated frameworks for regulating gene therapies should consider the inclusion of key elements such as expedited regulatory pathways that offer early engagement with regulators, innovative clinical trial design, and adequate post-market confirmatory studies. Increasing the alignment of regulatory pathways across countries will be crucial to facilitating the development of, and access to, gene therapies on a global scale.