2017
DOI: 10.1159/000464100
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Reimbursed Price of Orphan Drugs: Current Strategies and Potential Improvements

Abstract: The pricing and reimbursement policies for pharmaceuticals are relevant to balance timely and equitable access for all patients, financial sustainability, and reward for valuable innovation. The proliferation of high-cost specialty medicines is particularly true in rare diseases (RDs) where the pricing mechanism is characterised by a lack of transparency. This work provides an overall picture of current strategies for the definition of the reimbursed prices of orphan drugs (ODs) and highlights some potential i… Show more

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Cited by 17 publications
(16 citation statements)
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References 60 publications
(80 reference statements)
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“…In a context where organisational and economic issues about rare diseases are still a challenge and there is high heterogeneity in guidance and practice as well as lack of transparency or methods, 86 ERN ReCONNET can offer a real opportunity to develop novel solutions to address current challenges and issues related to rCTDs. At the same time, rCTDs could also be the ideal context for testing potentialities and limits of the novel HTA approaches, as a starting point for applications in other contexts and diseases.…”
Section: Discussionmentioning
confidence: 99%
“…In a context where organisational and economic issues about rare diseases are still a challenge and there is high heterogeneity in guidance and practice as well as lack of transparency or methods, 86 ERN ReCONNET can offer a real opportunity to develop novel solutions to address current challenges and issues related to rCTDs. At the same time, rCTDs could also be the ideal context for testing potentialities and limits of the novel HTA approaches, as a starting point for applications in other contexts and diseases.…”
Section: Discussionmentioning
confidence: 99%
“…El ADMC (Análisis de Decisión Multi-Criterio) es uno de los métodos que se proponen para superar algunos de estos escollos. El punto fuerte de estas propuestas es que el ADMC es uno de los métodos disponibles de una taxonomía de herramientas metodológicas para combinar o agregar múltiples criterios en un solo parámetro de valor del medicamento, que puede ayudar a priorizar alternativas y asignar recursos limitados Hughes-Wilson et al, 2012;Hutchings et al, 2012;Ethgen et al, 2012;Fedyaeva et al, 2014;Paulden et al, 2015;Sussex et al, 2013a;Mincarone et al, 2015;Kolasa et al, 2016;Devlin y Sussex, 2011;Garau et al, 2016;Schlander et al, 2014;Annemans et al, 2017;Gutierrez et al, 2015;Solon y Kanavos, 2016;Mincarone et al, 2017;Hughes et al, 2005;Culyer, 2014).…”
Section: íNdice Tablas Figurasunclassified
“…Se esgrimen varios argumentos sobre las limitaciones de la ETS para estimar y tomar decisiones sobre el valor de los MMHH y sobre su cobertura y rembolso. La decisión de incluir este apartado estriba en que, basándose en estos argumentos, unos proponen el ADMC como complemento de la ETS y otros, formulando críticas más severas de las limitaciones señaladas, como candidato a sustituirlas y erigirse en un nuevo paradigma del appraisal Winquist et al, 2012;Sussex, et al, 2013c;Mincarone et al, 2017;Kolasa et al, 2016;Schey y Conolly, 2014;Schlander et al, 2014;Gutierrez et al, 2015;Hughes et al, 2005;Lloyd et al, 2015;Drummond et al, 2013). En aras de la brevedad, y por su relevancia, en la tabla 8.1 se resumen estos argumentos y en un apartado posterior se cote- En el conjunto de estudios revisados se afirma que, por todos los motivos que se resumen a continuación, el amplio abanico de elementos normativos y positivos que subyacen a la lógica extrawelfarista del coste-efectividad para priorizar intervenciones en situaciones de recursos limitados (como criterio de eficiencia asignativa) debe revisarse en profundidad.…”
Section: Justificaciones De La Utilización Del Admc En Las Decisionesunclassified
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