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 health-care 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 the 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 orphan diseases and their technologies' clinical and economic impact. Participation processes are critical in incorporating societal values in weighting different dimensions and constructing decision rules. Local implementation pilots considering different stakeholders are necessary in order to pinpoint specific barriers and opportunities.

中文翻译：

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在评估孤儿药物的多标准决策分析（MCDA）中的国际经验：范围审查。

简介：孤儿疾病是低患病率，具有慢性衰弱或威胁生命的后果。他们的治疗通常称为孤儿药物（OD）。在为医疗保健计划制定费用和定价决策时，传统上将卫生技术评估过程视为成本效益分析（CEA）。由于不确定的证据，无法达到报销的成本效益阈值和较高的预算影响等因素，以标准CEA评估OD会引起重要问题。多标准决策分析（MCDA）可以解决这些问题，并提高有关OD的优先级，覆盖范围和费用报销的决策的技术和道德质量。涵盖领域：为了确定用于评估OD和实施经验的MCDA框架，进行了范围界定审查。我们审查了电子数据库（Medline，Embase，Cochrane图书馆，EBSCO，CINAHL，EconLit，Web of Science，LILACS，Google Scholar）主要期刊（Orphanet罕见病和健康价值期刊）和组织存储库。专家意见：MCDA的理论框架考虑了与孤儿疾病特征及其技术的临床和经济影响有关的领域。参与过程对于在权衡不同维度和构建决策规则中纳入社会价值至关重要。为了确定具体的障碍和机遇，有必要考虑不同利益相关者的地方实施试点。Cochrane图书馆，EBSCO，CINAHL，EconLit，Web of Science，LILACS，Google Scholar），重要期刊（Orphanet罕见病及其健康价值期刊）和组织存储库。专家意见：MCDA的理论框架考虑了与孤儿疾病特征及其技术的临床和经济影响有关的领域。参与过程对于在权衡不同维度和构建决策规则中纳入社会价值至关重要。为了确定具体的障碍和机遇，有必要考虑不同利益相关者的地方实施试点。Cochrane图书馆，EBSCO，CINAHL，EconLit，Web of Science，LILACS，Google Scholar），重要期刊（Orphanet罕见病及其健康价值期刊）和组织存储库。专家意见：MCDA的理论框架考虑了与孤儿疾病特征及其技术的临床和经济影响有关的领域。参与过程对于在权衡不同维度和构建决策规则中纳入社会价值至关重要。为了确定具体的障碍和机遇，有必要考虑不同利益相关者的地方实施试点。MCDA的理论框架考虑了与孤儿疾病特征及其技术的临床和经济影响有关的领域。参与过程对于在权衡不同维度和构建决策规则中纳入社会价值至关重要。为了确定具体的障碍和机遇，有必要考虑不同利益相关者的地方实施试点。MCDA的理论框架考虑了与孤儿疾病特征及其技术的临床和经济影响有关的领域。参与过程对于在权衡不同维度和构建决策规则时纳入社会价值至关重要。为了确定具体的障碍和机遇，有必要考虑不同利益相关者的地方实施试点。