International experiences in multicriteria decision analysis (MCDA) for evaluating orphan drugs: a scoping review.

International experiences in multicriteria decision analysis (MCDA) for evaluating orphan drugs: a scoping review. Expert Rev Pharmacoecon Outcomes Res. 2019 Jun 18;: Authors: Lasalvia P, Prieto L, Moreno M, Castrillón J, Romano G, Garzón N, Rosselli D Abstract Introduction Orphan diseases are low-prevalence conditions with chronically debilitating or life-threatening consequences. Their treatments are generally called orphan drugs (OD). Health technology assessment processes have traditionally considered cost-effectiveness analysis (CEA), when making reimbursement and pricing decisions for healthcare plans. Valuing OD with standard CEA raises important issues due to uncertain evidence, inability to meet cost-effectiveness thresholds for reimbursement and high budget impact, among others. Multi-criteria decision analysis (MCDA) allows to overcome these issues and improve technical and ethical quality of decisions regarding prioritization, coverage and reimbursement of OD. Areas covered A scoping review was conducted in order to characterize MCDA frameworks for assessing OD and implementation experiences. We reviewed electronic databases (Medline, Embase, Cochrane Library, EBSCO, CINAHL, EconLit, Web of Science, LILACS, Google Scholar) key journals (Orphanet Journal of Rare Diseases and Value in Health) and organization repositories. Expert opinion The theoretical framework for MCDA considers areas related to characteristics of orph...
Source: Expert Review of Pharmacoeconomics and Outcomes Research - Category: Health Management Tags: Expert Rev Pharmacoecon Outcomes Res Source Type: research