Biglia Luiza Vasconcelos, Felippini Arturo, Ribeiro Tatiane Bomfim, Lima Tácio de Mendonça, Aguiar Patricia Melo
Department of Pharmacy, Faculty of Pharmaceutical Sciences, University of São Paulo, São Paulo, Brazil.
Faculty of Public Health, University of São Paulo, São Paulo, Brazil.
Int J Technol Assess Health Care. 2025 Jan 9;41(1):e6. doi: 10.1017/S0266462324004835.
The aim of this study is to propose and validate a value assessment framework for Health Technology Assessment (HTA) for rare diseases drugs in Brazil.
A scoping review was performed to identify criteria used by HTA agencies in countries with public healthcare systems when evaluating orphan drugs. Based on the findings, a criteria framework for rare disease drugs was proposed for Brazil. Content validity was conducted over three rounds using Delphi technique and content validity ratio (CVR) approach was employed to evaluate the ratings from the eighteen stakeholders (experts and patients).
Twenty-nine HTA criteria for rare disease drugs were identified to compose the Brazilian framework. After three Delphi rounds, the final value framework comprised fifteen criteria categorized into four domains: disease-related factors, treatment-related factors, social and political factors, and economic factors. Among the most well-rated criteria by the CVR, considering the relevance attribute, were "relevance of outcomes for a rare disease," "impact on patient's quality of life," "price negotiation," and "adjusted cost-effectiveness threshold." On the other hand, "budget impact threshold," "innovative nature of treatment," and "willingness to accept greater uncertainty in clinical evidence" received negative evaluations and were excluded from the final framework.
A value assessment framework validated by key stakeholders of rare diseases in Brazil could contribute to improve HTA transparency, decision making, and efficiency of the healthcare system, and inspire the development of a local guidance for rare-disease HTA.
本研究旨在提出并验证巴西罕见病药物卫生技术评估（HTA）的价值评估框架。
进行了一项范围综述，以确定公共医疗体系国家的HTA机构在评估孤儿药时使用的标准。基于这些发现，为巴西提出了罕见病药物标准框架。采用德尔菲技术进行三轮内容效度评估，并运用内容效度比率（CVR）方法评估18名利益相关者（专家和患者）的评分。
确定了29条罕见病药物HTA标准以构成巴西框架。经过三轮德尔菲法后，最终的价值框架包括15条标准，分为四个领域：疾病相关因素、治疗相关因素、社会和政治因素以及经济因素。在CVR评分最高的标准中，从相关性属性来看，有“罕见病结局的相关性”“对患者生活质量的影响”“价格谈判”以及“调整后的成本效益阈值”。另一方面，“预算影响阈值”“治疗的创新性”以及“愿意接受临床证据中更大的不确定性”得到负面评价，并被排除在最终框架之外。
一个经巴西罕见病关键利益相关者验证的价值评估框架有助于提高HTA的透明度、决策制定以及医疗体系的效率，并激发制定罕见病HTA的本地指南。
