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. 2021 Apr 26;16(1):186.
doi: 10.1186/s13023-021-01809-1.

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

Fernando de Andrés-Nogales  1 , Encarnación Cruz  2 , Miguel Ángel Calleja  3 , Olga Delgado  4 , Maria Queralt Gorgas  5 , Jaime Espín  6   7   8 , Jorge Mestre-Ferrándiz  9   10 , Francesc Palau  11 , Alba Ancochea  12 , Rosabel Arce  13 , Raquel Domínguez-Hernández  14 , Miguel Ángel Casado  14 ; FinMHU-MCDA Group
Collaborators, Affiliations

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

Fernando de Andrés-Nogales et al. Orphanet J Rare Dis. .

Abstract

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.

Keywords: Multicriteria decision analysis; Orphan drugs; Rare diseases; Reimbursement; Spain.

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Conflict of interest statement

FdAN, RDH and MACas are employees of PORIB, a consultancy firm specialized in health technology assessment, which received funding from AELMHU for the development of this project. RA is an employee of AELMHU. EC, MACall, OD, MQG, JE, JMF, FP and AA received fees for the participation in this project.

Figures

Fig. 1
Fig. 1
Results of the literature search for the identification of reimbursement criteria
Fig. 2
Fig. 2
FinMHU-MCDA study: phases of the study
See this image and copyright information in PMC

References

    1. Council Recommendation of 8 June 2009 on an action in the field of rare diseases. Official Journal of the European Union (2009/C 151/02). OJ C 151, 3 July 2009, p. 7–10. Document 32009H.
    1. Regulation (EU) No 536/2014 of the European Parliament and of the Council of 16 April 2014 on clinical trials on medicinal products for human use, and repealing Directive 2001/20/EC. OJ L 158, 27 May 2014, p. 1–76.
    1. Recomendaciones para la búsqueda de soluciones en el ámbito de las enfermedades raras. In: Asociación Española de Laboratorios de Medicamentos Huérfanos y Ultrahuérfanos. 2017. http://www.aelmhu.es/docs/Documento-de-Recomendaciones-EERR-JUNIO2017.pdf. Accessed 11 June 2020.
    1. Regulation (EC) No 141/2000 of the European Parliament and of the Council of 16 December 1999 on orphan medicinal products. OJ L 18, 22 Jan 2000, p. 1–5.
    1. Orphanet. 2020. http://www.orpha.net. Accessed 30 July 2020.

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