Challenges in Research and Health Technology Assessment of Rare Disease Technologies: Report of the ISPOR Rare Disease Special Interest Group
Challenges in Research and Health Technology Assessment of Rare Disease Technologies: Report of the ISPOR Rare Disease Special Interest Group
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DOI：
10.1016/j.jval.2018.03.004
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发表时间：
2018-05-01
期刊：
影响因子：
4.5
通讯作者：
Redekop, William
中科院分区：
文献类型：
--
作者：
Nestler-Parr, Sandra;Korchagina, Daria;Redekop, William
背景：罕见疾病新疗法(RDS)的成功开发及其患者的可持续获取需要克服与研究和卫生技术评估(HTA)相关的一系列挑战。这些障碍可能是RDS独有的，也可能适用于常见疾病，但在RDS中尤其相关，这些障碍多种多样，相互关联。目的：首次建立RD研究和HTA的主要障碍目录，并描述个体挑战的原因和结果。方法：通过一个由22人组成的国际专家工作组确定挑战，并与具有相关专业知识的同事进行定性接触。代表了广泛的利益相关者的观点。结果草案在欧洲和北美国际药物经济学和结果研究学会(ISPOR)的年度大会上提交，385人的ISPOR罕见疾病审查小组从两轮审查中收到了书面意见。通过有针对性的文献搜索，研究结果得到了提炼和确认。结果：与RDS低患病率相关的研究挑战被归类为与疾病识别和诊断、治疗效果评估和临床研究患者招募有关的挑战。与HTA相关的挑战被归类为与缺乏针对RD治疗的定制HTA方法以及HTA机构和医疗保健支付者的不确定性有关的问题。结论：识别和强调RD研究和HTA中不同但相互关联的关键挑战是开发可实施和可持续解决方案的重要第一步。需要多方利益相关者的协作努力，使安全、有效和适当的新研发疗法能够更快、更低成本地开发，从而提供物有所值的治疗方法。
Background: Successful development of new treatments for rare diseases (RDs) and their sustainable patient access require overcoming a series of challenges related to research and health technology assessment (HTA). These impediments, which may be unique to RDs or also apply to common diseases but are particularly pertinent in RDs, are diverse and interrelated. Objective: To develop for the first time a catalog of primary impediments to RD research and HTA, and to describe the cause and effect of individual challenges. Methods: Challenges were identified by an international 22-person expert working group and qualitative outreach to colleagues with relevant expertise. A broad range of stakeholder perspectives is represented. Draft results were presented at annual European and North American International Society for Pharmacoeconomics and Outcomes Research (ISPOR) congresses, and written comments were received by the 385-strong ISPOR Rare Disease Review Group from two rounds of review. Findings were refined and confirmed via targeted literature search. Results: Research-related challenges linked to the low prevalence of RDs were categorized into those pertaining to disease recognition and diagnosis, evaluation of treatment effect, and patient recruitment for clinical research. HTA-related challenges were classified into issues relating to the lack of a tailored HTA method for RD treatments and uncertainty for HTA agencies and health care payers. Conclusions: Identifying and highlighting diverse, but interrelated, key challenges in RD research and HTA is an essential first step toward developing implementable and sustainable solutions. A collaborative multistakeholder effort is required to enable faster and less costly development of safe, efficacious, and appropriate new RD therapies that offer value for money.