Can the EVIDEM Framework Tackle Issues Raised by Evaluating Treatments for Rare Diseases: Analysis of Issues and Policies, and Context-Specific Adaptation

Abstract

BackgroundThe multiplicity of issues, including uncertainty and ethical dilemmas, and policies involved in appraising interventions for rare diseases suggests that multicriteria decision analysis (MCDA) based on a holistic definition of value is uniquely suited for this purpose. The objective of this study was to analyze and further develop a comprehensive MCDA framework (EVIDEM) to address rare disease issues and policies, while maintaining its applicability across disease areas.MethodsSpecific issues and policies for rare diseases were identified through literature review. Ethical and methodological foundations of the EVIDEM framework v3.0 were systematically analyzed from the perspective of these issues, and policies and modifications of the framework were performed accordingly to ensure their integration.ResultsAnalysis showed that the framework integrates ethical dilemmas and issues inherent to appraising interventions for rare diseases but required further integration of specific aspects. Modification thus included the addition of subcriteria to further differentiate disease severity, disease-specific treatment outcomes, and economic consequences of interventions for rare diseases. Scoring scales were further developed to include negative scales for all comparative criteria. A methodology was established to incorporate context-specific population priorities and policies, such as those for rare diseases, into the quantitative part of the framework. This design allows making more explicit trade-offs between competing ethical positions of fairness (prioritization of those who are worst off), the goal of benefiting as many people as possible, the imperative to help, and wise use of knowledge and resources. It also allows addressing variability in institutional policies regarding prioritization of specific disease areas, in addition to existing uncertainty analysis available from EVIDEM.ConclusionThe adapted framework measures value in its widest sense, while being responsive to rare disease issues and policies. It provides an operationalizable platform to integrate values, competing ethical dilemmas, and uncertainty in appraising healthcare interventions.Electronic supplementary materialThe online version of this article (doi:10.1007/s40273-015-0340-5) contains supplementary material, which is available to authorized users.

A Review of Rare Disease Policies and Orphan Drug Reimbursement Systems in 12 Eurasian Countries

“…Researchers in the rare disease area are also looking into the use of MCDA, which has resulted in a list of scientific publications and MCDA model designs, but full consensus on MCDA is still lacking and further research is needed to support implementation in (rare disease) HTA (94,(101)(102)(103)(104)(105)(106)(107)(108)(109)(110)(111)(112)(113)(114)(115)(116).…”

Comment on: “Can the EVIDEM Framework Tackle Issues Raised by Evaluating Treatments for Rare Diseases: Analysis of Issues and Policies, and Context-Specific Adaptation”

“…The article by Wagner et al [1] suggests the EVIDEM (Evidence and Value Impact on DEcisionMaking) framework to measure the value of treatments for rare diseases. EVIDEM assesses the value of treatments based on a set of predefined criteria.…”

“…Using multi-criteria decision analysis (MCDA), each criterion obtains a specific weight that enables calculation of an aggregate rating score for each treatment. Criteria suggested by Wagner et al [1] include disease severity, treatment health gain and population size. They are justified by major ethical theories such as utilitarianism, deontology and Rawls's Theory of Justice [2][3][4].…”

“…They are justified by major ethical theories such as utilitarianism, deontology and Rawls's Theory of Justice [2][3][4]. Thus, ''explicit trade-offs between competing ethical positions of fairness'' [1] are made. Based on the additive multi-attribute utility function underlying MCDA in the hypothetical example, EVIDEM could be described, in my opinion, as calculating a 'preference-weighted average of common ethical positions' or, to put it even more simply, as voting for different ethical positions.…”

Más allá Del análisis Coste-Efectividad. Precios Basados en el Valor y Financiación Orientada a Resultados Como Vía de Sostenibilidad Para el SNS en España

“…Las primeras propuestas, más ambiguas, proponían la incorporación de ponderaciones sociales en el análisis (50), mientras que posteriormente os nuevos enfoques se orientaron más hacia métodos de evaluación multi-criterio, que ofrecen la oportunidad de incorporar todos los elementos relevantes de un medicamento en la decisión de financiación de una manera estructurada, transparente y consistente. Este tipo de enfoque es especialmente útil en el caso de los medicamentos huérfanos, campo en el que empiezan exister numerosas propuestas prácticas para su implementación (49,(51)(52)(53)(54)(55).…”