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Utilización de experimentos de elección discreta para explorar las preferencias sociales en la valoración de nuevos
Constanza Vargas1, Stephen Goodall2, Deborah J Street2
1Centre for Health Economics and Evaluation, University of Technology Sydney, Building 20, 100 Broadway, Chippendale, Sydney, NSW, 2008, Australia. constanza.vargas@uts.edu.au.
Orphanet journal of rare diseases
|December 30, 2025
Resumen
Las preferencias sociales para la financiación de fármacos para enfermedades raras priorizan la supervivencia y la certeza de la evidencia. Abordar la incertidumbre en la evidencia es un desafío clave para la evaluación y el reembolso de fármacos para enfermedades raras.
Área de la Ciencia:
- Economía de la Salud
- Política de Salud
- Enfermedades Raras
Sus antecedentes:
- Las enfermedades raras afectan colectivamente a una población importante, lo que plantea desafíos únicos para la financiación pública debido a la incertidumbre de la evidencia y los impactos más amplios.
- Las decisiones de reembolso para tratamientos de enfermedades raras son complejas, influenciadas por opciones de tratamiento limitadas y la necesidad de equilibrar diversos valores sociales.
Objetivo del estudio:
- Explorar las preferencias sociales para los factores que influyen en las decisiones de subsidio público para fármacos para enfermedades raras.
- Comprender la importancia relativa de la incertidumbre de la evidencia, el beneficio clínico, la calidad de vida y el costo en las evaluaciones de financiación.
Principales métodos:
- Un experimento de elección discreta que involucró a 1099 australianos simulando roles de asesores gubernamentales.
- Se evaluaron atributos que incluyen incertidumbre de la evidencia, necesidad insatisfecha, beneficio clínico, calidad de vida y costo.
- Análisis utilizando modelos logit mixtos y de clases latentes para identificar la heterogeneidad de las preferencias y estimar la disposición a pagar.
Principales resultados:
- Las preferencias sociales favorecieron los fármacos que mejoraron la supervivencia, demostraron alta confianza en la efectividad y mejoraron el funcionamiento diario de los pacientes.
- El análisis de clases latentes reveló grupos distintos: fuerte preferencia por la supervivencia (58%), confianza en la evidencia (21%) y aumento del gasto público (21%).
Conclusiones:
- Los hallazgos se alinean con estudios previos que enfatizan las mejoras en la supervivencia y la calidad de vida.
- Reducir la incertidumbre de la evidencia es un desafío metodológico y de política fundamental para evaluar y financiar fármacos para enfermedades raras.
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