000110786 001__ 110786
000110786 005__ 20230519145519.0
000110786 0247_ $$2doi$$a10.1186/s13023-021-01809-1
000110786 0248_ $$2sideral$$a127443
000110786 037__ $$aART-2021-127443
000110786 041__ $$aeng
000110786 100__ $$ade Andrés-Nogales F.
000110786 245__ $$aA multi-stakeholder multicriteria decision analysis for the reimbursement of orphan drugs (FinMHU-MCDA study)
000110786 260__ $$c2021
000110786 5060_ $$aAccess copy available to the general public$$fUnrestricted
000110786 5203_ $$aBackground: 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.
000110786 540__ $$9info:eu-repo/semantics/openAccess$$aby$$uhttp://creativecommons.org/licenses/by/3.0/es/
000110786 590__ $$a4.302$$b2021
000110786 592__ $$a1.105$$b2021
000110786 594__ $$a5.2$$b2021
000110786 591__ $$aMEDICINE, RESEARCH & EXPERIMENTAL$$b68 / 140 = 0.486$$c2021$$dQ2$$eT2
000110786 593__ $$aPharmacology (medical)$$c2021$$dQ1
000110786 591__ $$aGENETICS & HEREDITY$$b61 / 177 = 0.345$$c2021$$dQ2$$eT2
000110786 593__ $$aMedicine (miscellaneous)$$c2021$$dQ1
000110786 655_4 $$ainfo:eu-repo/semantics/article$$vinfo:eu-repo/semantics/publishedVersion
000110786 700__ $$aCruz E.
000110786 700__ $$aCalleja M.Á.
000110786 700__ $$aDelgado O.
000110786 700__ $$aGorgas M.Q.
000110786 700__ $$aEspín J.
000110786 700__ $$aMestre-Ferrándiz J.
000110786 700__ $$aPalau F.
000110786 700__ $$aAncochea A.
000110786 700__ $$aArce R.
000110786 700__ $$aDomínguez-Hernández R.
000110786 700__ $$aCasado M.Á.
000110786 700__ $$aGómez Pajuelo P.
000110786 700__ $$aGorgas Torner M.Q.
000110786 700__ $$aLópez Andrés A.
000110786 700__ $$aLópez Rodríguez M.
000110786 700__ $$0(orcid)0000-0002-7815-8356$$aMarín Ballvé A.$$uUniversidad de Zaragoza
000110786 700__ $$aMartín Herranz M.I.
000110786 700__ $$aMorell Baladrón A.
000110786 700__ $$aSánchez Martínez F.I.
000110786 700__ $$aAncochea A.
000110786 700__ $$aAntoñanzas F.
000110786 700__ $$aBonanad S.
000110786 700__ $$aCruz E.
000110786 700__ $$aCaballero T.
000110786 700__ $$aCabasés J.M.
000110786 700__ $$aCalleja M.Á.
000110786 700__ $$aCruz J.
000110786 700__ $$aDelgado O.
000110786 700__ $$aEspín J.
000110786 700__ $$aGarcía-Goñi M.
000110786 700__ $$aGil R.
000110786 700__ $$aPajuelo P.G.
000110786 700__ $$aTorner M.Q.G.
000110786 700__ $$aAndrés A.L.
000110786 700__ $$aRodríguez M.L.
000110786 700__ $$aBallvé A.M.
000110786 700__ $$aHerranz M.I.M.
000110786 700__ $$aMestre-Ferrándiz J.
000110786 700__ $$aBaladrón A.M.
000110786 700__ $$aMur C.
000110786 700__ $$aPalau F.
000110786 700__ $$aMachado M.P.
000110786 700__ $$aMartínez F.I.S.
000110786 700__ $$aSantos A.R.
000110786 700__ $$aSuárez M.
000110786 700__ $$aTrillo J.L.
000110786 700__ $$athe FinMHU-MCDA Group
000110786 7102_ $$11007$$2610$$aUniversidad de Zaragoza$$bDpto. Medicina, Psiqu. y Derm.$$cArea Medicina
000110786 773__ $$g16, 186 (2021), 1-12$$pOrphanet j. rare dis.$$tOrphanet Journal of Rare Diseases$$x1750-1172
000110786 8564_ $$s1440202$$uhttps://zaguan.unizar.es/record/110786/files/texto_completo.pdf$$yVersión publicada
000110786 8564_ $$s2580791$$uhttps://zaguan.unizar.es/record/110786/files/texto_completo.jpg?subformat=icon$$xicon$$yVersión publicada
000110786 909CO $$ooai:zaguan.unizar.es:110786$$particulos$$pdriver
000110786 951__ $$a2023-05-18-15:21:19
000110786 980__ $$aARTICLE