Department of Pharmacy, Faculty of Pharmaceutical Sciences, University of São Paulo, Av. Prof. Lineu Prestes, 580 - Conj. das Químicas - Bloco 13 - Cidade Universitária Butantã, São Paulo, Brazil.
Department of Pharmacy and Pharmaceutical Administration, Faculty of Pharmacy, Fluminense Federal University, R. Dr. Mario Vianna, 523 - Santa Rosa, Niteroi, Brazil.
Health Policy. 2024 Jun;144:105080. doi: 10.1016/j.healthpol.2024.105080. Epub 2024 May 6.
Access to drugs for rare diseases constitutes a challenge to healthcare systems, especially those with public funding. This study aimed to map and summarize the criteria used by HTA agencies in different healthcare systems to evaluate reimbursement recommendations for orphan drugs. A comprehensive literature search was performed on the databases PubMed, LILACS, Scopus, and Embase and the gray literature (Google Scholar and websites of HTA agencies). Publications addressing the criteria used by HTA agencies in countries with public healthcare systems when evaluating reimbursement recommendations for orphan drugs were included. This scoping review included 23 studies published between 2014 and 2023, mostly consisting of reviews of HTA reports, guidance documents, and original articles. The criteria were mapped from 19 countries and ranked within three models of healthcare systems (National Health System, National Health Insurance, and Social Health Insurance). All models shared concerns about unmet needs and disease nature. In addition, NHS countries (e.g., United Kingdom, Sweden, and Italy) prioritized innovation and system-level impact, while SHI countries (e.g., Germany, France, the Netherlands) usually valued budget impact and employed expedited evaluation processes. This review provides a comprehensive understanding of the general tendencies of each healthcare system model in establishing differentiated criteria to address the challenges posed by the limited evidence and investment in the field of rare diseases.
罕见病药物的可及性对医疗保健系统构成了挑战，尤其是那些有公共资金支持的系统。本研究旨在绘制和总结不同医疗保健系统中的 HTA 机构在评估孤儿药报销建议时使用的标准。我们在 PubMed、LILACS、Scopus 和 Embase 数据库以及灰色文献（Google Scholar 和 HTA 机构网站）上进行了全面的文献检索。纳入了针对有公共医疗保健系统的国家中的 HTA 机构在评估孤儿药报销建议时使用的标准的出版物。本范围综述包括 2014 年至 2023 年间发表的 23 项研究，主要由 HTA 报告、指导文件和原始文章的综述组成。这些标准来自 19 个国家，并在三种医疗保健系统模型（国家卫生系统、国家健康保险和社会健康保险）中进行了排名。所有模型都关注未满足的需求和疾病性质。此外，NHS 国家（如英国、瑞典和意大利）优先考虑创新和系统层面的影响，而 SHI 国家（如德国、法国、荷兰）通常重视预算影响并采用加速评估流程。本综述全面了解了每个医疗保健系统模型在制定有区别的标准以应对罕见病领域证据和投资有限所带来的挑战方面的总体趋势。
