Abstract
ObjectivesWith 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. MethodsA 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. ResultsA 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%). ConclusionsMost 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.
Highlights
Health technology assessment (HTA) is “a multidisciplinary process that uses explicit methods to determine the value of a health technology at different points in its lifecycle.”[1]. Given that the determination of the technology’s value is related to the value of other health technologies, the process often includes a “relative effectiveness assessment” (REA), which is sometimes complemented with a cost element, the “cost-effectiveness assessment” (CEA).[2]
Analysis of the follow-up questions demonstrated that the overlap in challenges among the various health technologies lies with the data insufficiencies at time of assessment, despite the challenges being expressed in different domains of the HTA process
When we look at published cost-effectiveness analyses, previous systematic reviews on the economic evaluations of genetic testing, Advanced therapeutic medicinal products (ATMPs), and sequences of treatment with disease-modifying antirheumatic drugs investigated the quality and approaches of economic evaluations of these challenging health technologies
Summary
Health technology assessment (HTA) is “a multidisciplinary process that uses explicit methods to determine the value of a health technology at different points in its lifecycle.”[1] Given that the determination of the technology’s value is related to the value of other health technologies, the process often includes a “relative effectiveness assessment” (REA), which is sometimes complemented with a cost element, the “cost-effectiveness assessment” (CEA).[2] Over the past decades, the role and importance of HTA have developed gradually in response to greater emphasis on evidence-based decision making in healthcare. The treatment of patients has advanced over recent years, because of the development of increasingly tailored health technologies, including combinations of health technologies—consisting of pharmaceuticals, diagnostics, devices, digital tools, and interventions such as wearables and applications—personalized treatments, and treatment pathways, that is, “complex health technologies.”[3] These developments happened alongside increasing pressure on financing and delivery of healthcare because of demographic and other changes.[4]. We know that these complex health technologies come with new challenges to the existing reimbursement framework.[5,6,7,8,9,10,11] Orphan drugs and the trend toward personalized or individualized treatments do not always allow for large randomized controlled trials.[8,9] Increasingly, new trial designs with single arms are used for small populations or interim data are all that is available upon approval.[5,6,7] The claimed lifelong effects of cell and gene therapies are not feasible to capture in clinical trials before initial market approval and reimbursement decisions.[10,11] A recent example of therapies with challenges to the existing HTA framework are the histology-independent therapies, for example, larotrectinib.[12,13,14] The prevalence of the mutations targeted by these therapies is rare and spread over various tumor
Sign-in/Register to view highlights & summary Sign-in/Register to access full text options 
Free) 
Talk to us
Join us for a 30 min session where you can share your feedback and ask us any queries you have
Schedule a call
