110786 20230519145519.0 doi 10.1186/s13023-021-01809-1 sideral 127443 ART-2021-127443 eng de Andrés-Nogales F. A multi-stakeholder multicriteria decision analysis for the reimbursement of orphan drugs (FinMHU-MCDA study) 2021 Access copy available to the general public Unrestricted 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. info:eu-repo/semantics/openAccess by http://creativecommons.org/licenses/by/3.0/es/ 4.302 2021 MEDICINE, RESEARCH & EXPERIMENTAL 68 / 140 = 0.486 2021 Q2 T2 GENETICS & HEREDITY 61 / 177 = 0.345 2021 Q2 T2 1.105 2021 Pharmacology (medical) 2021 Q1 Medicine (miscellaneous) 2021 Q1 5.2 2021 info:eu-repo/semantics/article info:eu-repo/semantics/publishedVersion Cruz E. Calleja M.Á. Delgado O. Gorgas M.Q. Espín J. Mestre-Ferrándiz J. Palau F. Ancochea A. Arce R. Domínguez-Hernández R. Casado M.Á. Gómez Pajuelo P. Gorgas Torner M.Q. López Andrés A. López Rodríguez M. Marín Ballvé A. Universidad de Zaragoza (orcid)0000-0002-7815-8356 Martín Herranz M.I. Morell Baladrón A. Sánchez Martínez F.I. Ancochea A. Antoñanzas F. Bonanad S. Cruz E. Caballero T. Cabasés J.M. Calleja M.Á. Cruz J. Delgado O. Espín J. García-Goñi M. Gil R. Pajuelo P.G. Torner M.Q.G. Andrés A.L. Rodríguez M.L. Ballvé A.M. Herranz M.I.M. Mestre-Ferrándiz J. Baladrón A.M. Mur C. Palau F. Machado M.P. Martínez F.I.S. Santos A.R. Suárez M. Trillo J.L. the FinMHU-MCDA Group 1007 610 Universidad de Zaragoza Dpto. Medicina, Psiqu. y Derm. Area Medicina 16, 186 (2021), 1-12 Orphanet j. rare dis. Orphanet Journal of Rare Diseases 1750-1172 1440202 http://zaguan.unizar.es/record/110786/files/texto_completo.pdf Versión publicada 2580791 http://zaguan.unizar.es/record/110786/files/texto_completo.jpg?subformat=icon icon Versión publicada oai:zaguan.unizar.es:110786 articulos driver 2023-05-18-15:21:19 ARTICLE