Division of Pharmacoepidemiology and Clinical Pharmacology, Utrecht Institute for Pharmaceutical Sciences, Utrecht University, Utrecht, The Netherlands; National Health Care Institute, Diemen, The Netherlands.
Division of Pharmacoepidemiology and Clinical Pharmacology, Utrecht Institute for Pharmaceutical Sciences, Utrecht University, Utrecht, The Netherlands.
Value Health. 2022 Jun;25(6):992-1001. doi: 10.1016/j.jval.2021.11.1356. Epub 2021 Dec 23.
With complex health technologies entering the market, methods for health technology assessment (HTA) may require changes. This study aimed to identify challenges in HTA of complex health technologies.
A survey was sent to European HTA organizations participating in European Network for HTA (EUnetHTA). The survey contained open questions and used predefined potentially complex health technologies and 7 case studies to identify types of complex health technologies and challenges faced during HTA. The survey was validated, tested for reliability by an expert panel, and pilot tested before dissemination.
A total of 22 HTA organizations completed the survey (67%). Advanced therapeutic medicinal products (ATMPs) and histology-independent therapies were considered most challenging based on the predefined complex health technologies and case studies. For the case studies, more than half of the reported challenges were "methodological," equal in relative effectiveness assessments as in cost-effectiveness assessments. Through the open questions, we found that most of these challenges actually rooted in data unavailability. Data were reported as "absent," "insufficient," "immature," or "low quality" by 18 of 20 organizations (90%), in particular data on quality of life. Policy and organizational challenges and challenges because of societal or political pressure were reported by 8 (40%) and 4 organizations (20%), respectively. Modeling issues were reported least often (n = 2, 4%).
Most challenges in HTA of complex health technologies root in data insufficiencies rather than in the complexity of health technologies itself. As the number of complex technologies grows, the urgency for new methods and policies to guide HTA decision making increases.
随着复杂的医疗技术进入市场，健康技术评估（HTA）的方法可能需要改变。本研究旨在确定复杂医疗技术 HTA 中的挑战。
向参与欧洲健康技术评估网络（EUnetHTA）的欧洲 HTA 组织发送了一份调查。该调查包含开放式问题，并使用预先确定的潜在复杂医疗技术和 7 个案例研究来确定复杂医疗技术的类型和 HTA 中面临的挑战。该调查经过验证，由专家小组进行可靠性测试，并在分发前进行试点测试。
共有 22 个 HTA 组织完成了调查（67%）。根据预先确定的复杂医疗技术和案例研究，先进治疗性药物产品（ATMP）和组织学独立疗法被认为最具挑战性。对于案例研究，超过一半的报告挑战是“方法学”的，在相对有效性评估中与成本效益评估相等。通过开放式问题，我们发现这些挑战实际上主要源于数据不可用。18 个组织中的 20 个（90%）报告数据“不存在”、“不足”、“不成熟”或“质量低”，特别是关于生活质量的数据。8 个组织（40%）和 4 个组织（20%）分别报告了政策和组织挑战以及由于社会或政治压力而产生的挑战。建模问题报告的频率最低（n=2，4%）。
复杂医疗技术 HTA 中的大多数挑战源于数据不足，而不是医疗技术本身的复杂性。随着复杂技术数量的增加，需要新的方法和政策来指导 HTA 决策的紧迫性也在增加。
