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Desarrollo y Acceso de Medicamentos para Enfermedades Raras: ¿Podemos Mejorar?
Carla E M Hollak1,2,3, Noa Rosenberg2, Colinda Post1,2,4
1Department of Endocrinology and Metabolism, Amsterdam UMC, Amsterdam Gastroenterology Endocrinology Metabolism (AGEM) Research Institute, Expertise Center for Inborn Errors of Metabolism, University of Amsterdam, Amsterdam, the Netherlands.
Journal of inherited metabolic disease
|February 17, 2026
Resumen
Los investigadores académicos deben fortalecer su papel en el desarrollo de medicamentos huérfanos para mejorar el acceso y la asequibilidad. Una mayor participación en todas las etapas, la formación regulatoria y los marcos de precios sostenibles son cruciales para las terapias de enfermedades raras.
Área de la Ciencia:
- Biología molecular y genómica; Investigación de enfermedades raras; Desarrollo de medicamentos huérfanos
Sus antecedentes:
- Los avances en genómica mejoran la comprensión de las enfermedades raras y las terapias dirigidas.
- El desarrollo de medicamentos huérfanos enfrenta complejidades en la regulación y la accesibilidad.
- Los altos precios y la eficacia incierta de los tratamientos especializados resaltan las deficiencias sistémicas.
Objetivo del estudio:
- Analizar el papel de los investigadores académicos en el desarrollo de medicamentos huérfanos.
- Identificar los desafíos en el desarrollo, la regulación y la accesibilidad de las terapias para enfermedades raras.
- Proponer estrategias para mejorar la participación académica y la asequibilidad de los medicamentos.
Principales métodos:
- Revisión de literatura y análisis de las contribuciones académicas al desarrollo de medicamentos huérfanos.
- Examen de los modelos actuales de desarrollo impulsados por la industria y las interacciones académico-industria.
- Evaluación de los marcos regulatorios y de acceso al mercado existentes.
Principales resultados:
- Las contribuciones académicas se concentran en la investigación temprana, careciendo de continuidad.
- Existe una limitada experiencia académica en asuntos regulatorios, acceso al mercado, fijación de precios y licencias.
- El desarrollo impulsado por la industria a menudo deja de lado a la academia, lo que lleva a roles reactivos.
Conclusiones:
- El compromiso académico sostenido en todas las etapas de desarrollo es vital.
- La integración de la formación regulatoria y de acceso al mercado es esencial para los investigadores.
- Se recomienda la colaboración académica-industrial independiente y los marcos socialmente responsables para mejorar el acceso y la asequibilidad de las terapias para enfermedades raras.
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