Multi-Criteria Decision Analysis (MCDA) Models in Health Technology Assessment of Orphan Drugs-a Systematic Literature Review. Next Steps in Methodology Development?

Aleksandra Baran-Kooiker,Marcin Czech,Coen Kooiker

doi:10.3389/fpubh.2018.00287

Abstract

Background: Multi-criteria decision analysis (MCDA) is a decision-making tool that can take into account multidimensional factors and enables comparison of (medical) technologies by combining individual criteria into one overall appraisal. The MCDA approach has slowly gained traction within Health Technology Assessment (HTA) and its elements are gradually being incorporated into HTA across Europe. Several groups of scientists have proposed MCDA approaches targeted toward orphan drugs and rare diseases by including criteria specific to rare diseases. The goal of this article is to provide an overview of the current state of knowledge and latest developments in the field of MCDA in HTA for orphan drugs, to review existing models, their design characteristics, as well as to identify opportunities for further model improvement.Methods: A systematic literature search was conducted in January 2018 using four databases: MEDLINE (Pubmed), EBSCO HOST, EMBASE, and Web of science to find publications related to use of MCDA in the rare disease field (keywords: MCDA/orphan drug/rare disease and synonyms). Identified MCDA models were analyzed, e.g., structure, criteria, scoring, and weighting methodology.Results: Two hundred and eleven publications were identified, of which 29 were included after removal of duplicates. 9 authors developed own MCDA models, 7 of which based on literature reviews intended to identify the most important and relevant decision criteria in the model. In 13 publications (8 models) weights were assigned to criteria based on stakeholder input. The most commonly chosen criteria for creation of the MCDA models were: comparative effectiveness/efficacy, the need for intervention, and disease severity. Some models have overlapping criteria, especially in the treatment cost and effectiveness areas.Conclusions: A range of MCDA models for HTA have been developed, each with a slightly different approach, focus, and complexity, including several that specifically target rare diseases and orphan drug appraisal. Models have slowly progressed over the years based on pilots, stakeholder input, sharing experiences and scientific publications. However, full consensus on model structure, criteria selection and weighting is still lacking. A simplification of the MCDA model approach may increase its acceptance. A multi-stakeholder discussion on fundamental design and implementation strategies for MCDA models would be beneficial to this end.

Highlights

Health Technology Assessment of Orphan DrugsThere is an ongoing debate if conventional health technology assessment (HTA) methods are still appropriate for orphan medicinal products (OMPs) and other highly specialized, innovative, expensive treatments (1–4)
All publications described the use of Multi-criteria decision analysis (MCDA) for assessment of OMPs in Europe, in 10 European countries: Bulgaria, France, Germany, Italy, the Netherlands, Poland, Russia, Spain, Ukraine and the United Kingdom
In 8 studies weights were assigned to criteria based on stakeholder input [Trip et al (36), Sussex et al (37), GilabertPerramon et al (44), Garau et al (45), Iskrov et al (35), Fedyeva et al, Wagner et al (43) Piniazkho et al (40), and Piniazkho and Nemeth (41)] and in one publication weights were allocated by the authors [Wagner et al (25)]

Summary

Introduction

There is an ongoing debate if conventional health technology assessment (HTA) methods are still appropriate for orphan medicinal products (OMPs) and other highly specialized, innovative, expensive treatments (1–4). The one-size-fits-all approach of traditional cost-effectiveness analysis (expressed as cost per quality-adjusted life-year) no longer seems suitable for many new innovative interventions, leading to difficulties in the reimbursement process and delay in patient access to potentially valuable treatments. The fact that the costs of these specialized interventions are often much higher than treatments for common diseases, makes difficulties during reimbursement assessment nearly inevitable. The goal of this article is to provide an overview of the current state of knowledge and latest developments in the field of MCDA in HTA for orphan drugs, to review existing models, their design characteristics, as well as to identify opportunities for further model improvement

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