A multi-stakeholder multicriteria decision analysis for the reimbursement of orphan drugs (FinMHU-MCDA study)

Background Patient access to orphan medicinal products (OMPs) is limited and varies between countries, reimbursement decisions on OMPs are complex, and there is a need for more transparent processes to know which criteria should be considered to inform these decisions. This study aimed to determine...

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Autores: de andres-Nogales, Fernando, Cruz, Encarnacion, Calleja, Miguel Angel, Delgado Sánchez, Olga, Gorgas, Maria Queralt, Espín, Jaime, Mestre-Ferrandiz, Jorge, Palau, Francesc, Ancochea, Alba, Arce, Rosabel, Dominguez-Hernandez, Raquel, Casado, Miguel Angel, FinMHU-MCDA Grp
Tipo de recurso: artículo
Fecha de publicación:2021
País:España
Institución:Conselleria de Salut i Consum del Govern de les Illes Balears
Repositorio:Docusalut
Idioma:inglés
OAI Identifier:oai:docusalut.com:20.500.13003/19411
Acceso en línea:https://hdl.handle.net/20.500.13003/19411
Access Level:acceso abierto
Palabra clave:Quality of Life
Decision Making
Rare Diseases
Spain
Humans
Cost-Benefit Analysis
Orphan Drug Production
Decision Support Techniques
Técnicas de Apoyo para la Decisión
Humanos
Calidad de Vida
Producción de Medicamentos sin Interés Comercial
Toma de Decisiones
Enfermedades Raras
Análisis Costo-Beneficio
España
Multicriteria decision analysis
Orphan drugs
Rare diseases
Reimbursement
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spelling A multi-stakeholder multicriteria decision analysis for the reimbursement of orphan drugs (FinMHU-MCDA study)de andres-Nogales, FernandoCruz, EncarnacionCalleja, Miguel AngelDelgado Sánchez, OlgaGorgas, Maria QueraltEspín, JaimeMestre-Ferrandiz, JorgePalau, FrancescAncochea, AlbaArce, RosabelDominguez-Hernandez, RaquelCasado, Miguel AngelFinMHU-MCDA GrpQuality of LifeDecision MakingRare DiseasesSpainHumansCost-Benefit AnalysisOrphan Drug ProductionDecision Support TechniquesTécnicas de Apoyo para la DecisiónHumanosCalidad de VidaProducción de Medicamentos sin Interés ComercialToma de DecisionesEnfermedades RarasAnálisis Costo-BeneficioEspañaMulticriteria decision analysisOrphan drugsRare diseasesReimbursementSpainBackground Patient access to orphan medicinal products (OMPs) is limited and varies between countries, reimbursement decisions on OMPs are complex, and there is a need for more transparent processes to know which criteria should be considered to inform these decisions. This study aimed to determine the most relevant criteria for the reimbursement of OMPs in Spain, from a multi-stakeholder perspective, and using multicriteria decision analysis (MCDA). Methods An MCDA was developed in 3 phases and included 28 stakeholders closely related to the field of rare diseases (6 physicians, 5 hospital pharmacists, 7 health economists, 4 patient representatives and 6 members from national and regional health authorities). Initially [phase A], a bibliographic review was conducted to identify the potential reimbursement criteria. Then, a reduced advisory board (8 members) proposed, selected, and defined the final list of criteria that could be relevant for reimbursement. A discrete choice experiment (DCE) [phase B] was developed to determine the relevance and relative importance weight of such criteria according to the stakeholders' preferences by choosing between pairs of hypothetical financing scenarios. A multinomial logit model was fitted to analyze the DCE responses. Finally [phase C], the advisory board review the results using a deliberative process. Results Thirteen criteria were selected, related to 4 dimensions: patient population, disease, treatment, and economic evaluation. Nine criteria were deemed relevant for decision-making and associated with a higher relative importance: Health-related quality of life (HRQL) (23.53%), treatment efficacy (14.64%), availability of treatment alternatives (13.51%), disease severity (12.62%), avoided costs (11.21%), age of target population (7.75%), safety (seriousness of adverse events) (4.72%), quality of evidence (3.82%) and size of target population (3.12%). The remaining criteria had a < 3% relative importance: economic burden of disease (2.50%), cost of treatment (1.73%), cost-effectiveness (0.83%) and safety (frequency of adverse events) (0.03%). Conclusion The reimbursement of OMPs in Spain should be determined by its effect on patient's HRQL, the extent of its therapeutic benefit from efficacy and the availability of other therapeutic options. Furthermore, the severity of the rare disease should also influence the decision along with the potential of the treatment to avoid associated costs.BMC20212021-04-2620212021-04-26research articlehttp://purl.org/coar/resource_type/c_2df8fbb1info:eu-repo/semantics/articleapplication/pdfhttps://hdl.handle.net/20.500.13003/19411reponame:Docusalutinstname:Conselleria de Salut i Consum del Govern de les Illes BalearsInglésengopen accesshttp://purl.org/coar/access_right/c_abf2Attribution 4.0 Internationalhttp://creativecommons.org/licenses/by/4.0/info:eu-repo/semantics/openAccessoai:docusalut.com:20.500.13003/194112026-06-22T12:44:07Z
dc.title.none.fl_str_mv A multi-stakeholder multicriteria decision analysis for the reimbursement of orphan drugs (FinMHU-MCDA study)
title A multi-stakeholder multicriteria decision analysis for the reimbursement of orphan drugs (FinMHU-MCDA study)
spellingShingle A multi-stakeholder multicriteria decision analysis for the reimbursement of orphan drugs (FinMHU-MCDA study)
de andres-Nogales, Fernando
Quality of Life
Decision Making
Rare Diseases
Spain
Humans
Cost-Benefit Analysis
Orphan Drug Production
Decision Support Techniques
Técnicas de Apoyo para la Decisión
Humanos
Calidad de Vida
Producción de Medicamentos sin Interés Comercial
Toma de Decisiones
Enfermedades Raras
Análisis Costo-Beneficio
España
Multicriteria decision analysis
Orphan drugs
Rare diseases
Reimbursement
Spain
title_short A multi-stakeholder multicriteria decision analysis for the reimbursement of orphan drugs (FinMHU-MCDA study)
title_full A multi-stakeholder multicriteria decision analysis for the reimbursement of orphan drugs (FinMHU-MCDA study)
title_fullStr A multi-stakeholder multicriteria decision analysis for the reimbursement of orphan drugs (FinMHU-MCDA study)
title_full_unstemmed A multi-stakeholder multicriteria decision analysis for the reimbursement of orphan drugs (FinMHU-MCDA study)
title_sort A multi-stakeholder multicriteria decision analysis for the reimbursement of orphan drugs (FinMHU-MCDA study)
dc.creator.none.fl_str_mv de andres-Nogales, Fernando
Cruz, Encarnacion
Calleja, Miguel Angel
Delgado Sánchez, Olga
Gorgas, Maria Queralt
Espín, Jaime
Mestre-Ferrandiz, Jorge
Palau, Francesc
Ancochea, Alba
Arce, Rosabel
Dominguez-Hernandez, Raquel
Casado, Miguel Angel
FinMHU-MCDA Grp
author de andres-Nogales, Fernando
author_facet de andres-Nogales, Fernando
Cruz, Encarnacion
Calleja, Miguel Angel
Delgado Sánchez, Olga
Gorgas, Maria Queralt
Espín, Jaime
Mestre-Ferrandiz, Jorge
Palau, Francesc
Ancochea, Alba
Arce, Rosabel
Dominguez-Hernandez, Raquel
Casado, Miguel Angel
FinMHU-MCDA Grp
author_role author
author2 Cruz, Encarnacion
Calleja, Miguel Angel
Delgado Sánchez, Olga
Gorgas, Maria Queralt
Espín, Jaime
Mestre-Ferrandiz, Jorge
Palau, Francesc
Ancochea, Alba
Arce, Rosabel
Dominguez-Hernandez, Raquel
Casado, Miguel Angel
FinMHU-MCDA Grp
author2_role author
author
author
author
author
author
author
author
author
author
author
author
dc.contributor.none.fl_str_mv
dc.subject.none.fl_str_mv Quality of Life
Decision Making
Rare Diseases
Spain
Humans
Cost-Benefit Analysis
Orphan Drug Production
Decision Support Techniques
Técnicas de Apoyo para la Decisión
Humanos
Calidad de Vida
Producción de Medicamentos sin Interés Comercial
Toma de Decisiones
Enfermedades Raras
Análisis Costo-Beneficio
España
Multicriteria decision analysis
Orphan drugs
Rare diseases
Reimbursement
Spain
topic Quality of Life
Decision Making
Rare Diseases
Spain
Humans
Cost-Benefit Analysis
Orphan Drug Production
Decision Support Techniques
Técnicas de Apoyo para la Decisión
Humanos
Calidad de Vida
Producción de Medicamentos sin Interés Comercial
Toma de Decisiones
Enfermedades Raras
Análisis Costo-Beneficio
España
Multicriteria decision analysis
Orphan drugs
Rare diseases
Reimbursement
Spain
description Background Patient access to orphan medicinal products (OMPs) is limited and varies between countries, reimbursement decisions on OMPs are complex, and there is a need for more transparent processes to know which criteria should be considered to inform these decisions. This study aimed to determine the most relevant criteria for the reimbursement of OMPs in Spain, from a multi-stakeholder perspective, and using multicriteria decision analysis (MCDA). Methods An MCDA was developed in 3 phases and included 28 stakeholders closely related to the field of rare diseases (6 physicians, 5 hospital pharmacists, 7 health economists, 4 patient representatives and 6 members from national and regional health authorities). Initially [phase A], a bibliographic review was conducted to identify the potential reimbursement criteria. Then, a reduced advisory board (8 members) proposed, selected, and defined the final list of criteria that could be relevant for reimbursement. A discrete choice experiment (DCE) [phase B] was developed to determine the relevance and relative importance weight of such criteria according to the stakeholders' preferences by choosing between pairs of hypothetical financing scenarios. A multinomial logit model was fitted to analyze the DCE responses. Finally [phase C], the advisory board review the results using a deliberative process. Results Thirteen criteria were selected, related to 4 dimensions: patient population, disease, treatment, and economic evaluation. Nine criteria were deemed relevant for decision-making and associated with a higher relative importance: Health-related quality of life (HRQL) (23.53%), treatment efficacy (14.64%), availability of treatment alternatives (13.51%), disease severity (12.62%), avoided costs (11.21%), age of target population (7.75%), safety (seriousness of adverse events) (4.72%), quality of evidence (3.82%) and size of target population (3.12%). The remaining criteria had a < 3% relative importance: economic burden of disease (2.50%), cost of treatment (1.73%), cost-effectiveness (0.83%) and safety (frequency of adverse events) (0.03%). Conclusion The reimbursement of OMPs in Spain should be determined by its effect on patient's HRQL, the extent of its therapeutic benefit from efficacy and the availability of other therapeutic options. Furthermore, the severity of the rare disease should also influence the decision along with the potential of the treatment to avoid associated costs.
publishDate 2021
dc.date.none.fl_str_mv 2021
2021-04-26
2021
2021-04-26
dc.type.none.fl_str_mv research article
http://purl.org/coar/resource_type/c_2df8fbb1
dc.type.openaire.fl_str_mv info:eu-repo/semantics/article
format article
dc.identifier.none.fl_str_mv https://hdl.handle.net/20.500.13003/19411
url https://hdl.handle.net/20.500.13003/19411
dc.language.none.fl_str_mv Inglés
eng
language_invalid_str_mv Inglés
language eng
dc.rights.none.fl_str_mv open access
http://purl.org/coar/access_right/c_abf2
Attribution 4.0 International
http://creativecommons.org/licenses/by/4.0/
dc.rights.openaire.fl_str_mv info:eu-repo/semantics/openAccess
rights_invalid_str_mv open access
http://purl.org/coar/access_right/c_abf2
Attribution 4.0 International
http://creativecommons.org/licenses/by/4.0/
eu_rights_str_mv openAccess
dc.format.none.fl_str_mv application/pdf
dc.publisher.none.fl_str_mv BMC
publisher.none.fl_str_mv BMC
dc.source.none.fl_str_mv reponame:Docusalut
instname:Conselleria de Salut i Consum del Govern de les Illes Balears
instname_str Conselleria de Salut i Consum del Govern de les Illes Balears
reponame_str Docusalut
collection Docusalut
repository.name.fl_str_mv
repository.mail.fl_str_mv
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score 15,811543

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