OBJECTIVES

: Multiple Criteria Decision Analysis (MCDA) is becoming increasingly important as a tool for the evaluation of orphan drugs (ODs), due to limitations in the availability of randomized controlled efficacy studies for rare diseases. In this review, we aimed to identify real-world implementation of MCDA for the assessment of ODs in Europe.

METHODS

: A targeted literature search was conducted using EMBASE to identify research articles published between 2010-2020. Searches were focused using “rare diseases”, “orphan drugs” and “MCDA” or “HTA” (and related synonyms) to identify relevant articles. Two rounds of independent abstract review were conducted to yield a final selection of 10 full text articles for review.

RESULTS

: A total of 57 articles were identified through the literature search, with 10 studies selected for full text review. Of the selected articles, only 2 explored the real-world implementation of an MCDA framework for ODs at a national level. These studies were based in Spain and Poland. In Spain, MCDA the framework was developed following EVIDEM methodology and 15 criteria were selected, weighted by relative importance on a 5-point scale. In Poland, A Simple Linear Additive Model (SLAM) ranking and Analytic Hierarchy Process (AHP) were adopted to rank and weight the 15 criteria selected for inclusion. Common criteria included Efficacy, Safety, Unmet Needs, Disease Severity, Therapy Cost, and Type/Quality of Evidence. In Poland, criteria regarding affordability, system capacity and access to treatment were also selected for inclusion.

CONCLUSIONS

: Two real-world applications demonstrated MCDA as a valuable tool for revealing decision maker preferences in the Assessment of ODs in Europe. MCDA incorporates aspects of equity and social values usually ignored by traditional Health Technology Assessments. If MCDA can be characterized and defined at a European level, with country-specific adaptations of the framework, it could improve access to ODs in various EU countries.