Reported Challenges in Health Technology Assessment of Complex Health Technologies

• Published in: Value in health Vol. 25; no. 6; pp. 992 - 1001
• Main Authors: Hogervorst, Milou A., Vreman, Rick A., Mantel-Teeuwisse, Aukje K., Goettsch, Wim G.
• Format: Journal Article
• Language: English
• Published: United States Elsevier Inc 01.06.2022
• Subjects: challenges; cost-effectiveness assessment; data quality; decision making; health technology assessment; personalized health technologies; relative effectiveness assessment; health technology assessment; data quality; personalized health technologies; challenges; decision making; cost-effectiveness assessment; relative effectiveness assessment
• DOI: 10.1016/j.jval.2021.11.1356
• ISSN: 1098-3015

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. •In the current data-driven world, we know that also in health technology assessment (HTA) the lack of data causes major challenges. Increasingly complex health technologies may bring novel challenges for HTA, which may require new HTA methods. Previous research identified challenges for specific types of technologies, such as gene- and cell-based therapies, in limited sets of countries.•We systematically assessed the challenges experienced by HTA organizations throughout Europe for a broad set of complex health technologies. We found that reported problems expressed in methodological aspects of HTA of these technologies also often find their root in a lack of available data, rather than in the complexity of the technology itself. Health technologies that were perceived as most challenging, that is, advanced therapeutic medicinal products (ATMPs) and histology-independent therapies, inherently create new difficulties in evidence generation.•HTA organizations report many challenges when assessing complex health technologies. Nevertheless, our results show that many of these challenges originate from a lack of data rather than from the complexity of the technology itself. As the development of these complex technologies continues to grow, the need to find a solution for lacking (quality) data increases in importance. Future research that attempts to improve data quality could focus on the alignment of clinical data generation with evidentiary requirements for subsequent stakeholders for future technologies early in the development process.