A multi-stakeholder multicriteria decision analysis for the reimbursement of orphan drugs (FinMHU-MCDA study).
Por:
de Andrés-Nogales F, Cruz E, Calleja MÁ, Delgado O, Gorgas MQ, Espín J, Mestre-Ferrándiz J, Palau F, Ancochea A, Arce R, Domínguez-Hernández R, Casado MÁ and FinMHU-MCDA Group
Publicada:
26 abr 2021
Ahead of Print:
26 abr 2021
Resumen:
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.
Filiaciones:
de Andrés-Nogales F:
Pharmacoeconomics & Outcomes Research Iberia (PORIB), Calle Paseo Joaquín Rodrigo, 4I. 28224, Pozuelo de Alarcón, Madrid, Spain.
Cruz E:
Asociación Española de Medicamentos Biosimilares, Madrid, Spain
Calleja MÁ:
Servicio de Farmacia, Hospital Universitario Virgen Macarena, Sevilla, Spain
Delgado O:
Servicio de Farmacia, Hospital Universitario Son Espases, Palma de Mallorca, Spain
Gorgas MQ:
Servicio de Farmacia, Hospital Universitari Vall D'Hebron, Barcelona, Spain
Espín J:
Escuela Andaluza de Salud Pública, Granada, Spain
Instituto de Investigación Biosanitaria (IBS), Granada, Spain
CIBER de Epidemiología y Salud Pública (CIBERESP), Madrid, Spain
Mestre-Ferrándiz J:
Consultor Económico Independiente, Madrid, Spain
, Madrid, Spain
Palau F:
Servicio de Medicina Genética y CIBERER, Hospital Universitari Sant Joan de Déu, Hospital Clínic y Universitat de Barcelona, Barcelona, Spain
Ancochea A:
Federación Española de Enfermedades Raras (FEDER), Madrid, Spain
Arce R:
Asociación Española de Laboratorios de Medicamentos Huérfanos y Ultrahuérfanos (AELMHU), Barcelona, Spain
Domínguez-Hernández R:
Pharmacoeconomics & Outcomes Research Iberia (PORIB), Calle Paseo Joaquín Rodrigo, 4I. 28224, Pozuelo de Alarcón, Madrid, Spain
Casado MÁ:
Pharmacoeconomics & Outcomes Research Iberia (PORIB), Calle Paseo Joaquín Rodrigo, 4I. 28224, Pozuelo de Alarcón, Madrid, Spain
Green Published, gold
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