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DRUG EVALUATION AND DECISION MAKING IN CATALONIA: DEVELOPMENT AND VALIDATION OF A METHODOLOGICAL FRAMEWORK BASED ON MULTI-CRITERIA DECISION ANALYSIS (MCDA) FOR ORPHAN DRUGS

Published online by Cambridge University Press:  24 April 2017

Antoni Gilabert-Perramon ,
Josep Torrent-Farnell ,
Arancha Catalan ,
Alba Prat ,
Manel Fontanet ,
Ruth Puig-Peiró ,
Sandra Merino-Montero ,
Hanane Khoury ,
Mireille M. Goetghebeur  and
Xavier Badia
Antoni Gilabert-Perramon
Affiliation:
Gerència de Farmàcia i del Medicament; Servei Català de la Salut (CatSalut)
Josep Torrent-Farnell
Affiliation:
Hospital de la Santa Creu i Sant Pau
Arancha Catalan
Affiliation:
Agència de Qualitat i Avaluació Sanitàries de Catalunya (AQuAS)
Alba Prat
Affiliation:
Gerència de Farmàcia i del Medicament; Servei Català de la Salut (CatSalut)
Manel Fontanet
Affiliation:
Hospital de la Santa Creu i Sant Pau
Ruth Puig-Peiró
Affiliation:
Gerència de Farmàcia i del Medicament. Servei Català de la Salut (CatSalut)
Sandra Merino-Montero
Affiliation:
Omakase Consulting S.L.
Hanane Khoury
Affiliation:
LASER Analytica
Mireille M. Goetghebeur
Affiliation:
LASER Analytica and School of Public Health, University of Montreal (Canada)
Xavier Badia
Affiliation:
Omakase Consulting S.L.xbadia@omakaseconsulting.com
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Abstract

Objectives: The aim of this study was to adapt and assess the value of a Multi-Criteria Decision Analysis (MCDA) framework (EVIDEM) for the evaluation of Orphan drugs in Catalonia (Catalan Health Service).

Methods: The standard evaluation and decision-making procedures of CatSalut were compared with the EVIDEM methodology and contents. The EVIDEM framework was adapted to the Catalan context, focusing on the evaluation of Orphan drugs (PASFTAC program), during a Workshop with sixteen PASFTAC members. The criteria weighting was done using two different techniques (nonhierarchical and hierarchical). Reliability was assessed by re-test.

Results: The EVIDEM framework and methodology was found useful and feasible for Orphan drugs evaluation and decision making in Catalonia. All the criteria considered for the development of the CatSalut Technical Reports and decision making were considered in the framework. Nevertheless, the framework could improve the reporting of some of these criteria (i.e., “unmet needs” or “nonmedical costs”). Some Contextual criteria were removed (i.e., “Mandate and scope of healthcare system”, “Environmental impact”) or adapted (“population priorities and access”) for CatSalut purposes. Independently of the weighting technique considered, the most important evaluation criteria identified for orphan drugs were: “disease severity”, “unmet needs” and “comparative effectiveness”, while the “size of the population” had the lowest relevance for decision making. Test–retest analysis showed weight consistency among techniques, supporting reliability overtime.

Conclusions: MCDA (EVIDEM framework) could be a useful tool to complement the current evaluation methods of CatSalut, contributing to standardization and pragmatism, providing a method to tackle ethical dilemmas and facilitating discussions related to decision making.

Keywords

EVIDEMMulti-criteria decision analysisHealthcareDecision making
Type
Policies
Information
International Journal of Technology Assessment in Health Care , Volume 33 , Issue 1 , 2017 , pp. 111 - 120
Copyright
Copyright © Cambridge University Press 2017 

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