Will Health Technology Assessment (HTA) Bodies in Europe Accept Evidence from an External Control Arm to Supplement Evidence from Clinical Trials for Chronic Diseases?
Author(s)
Mangla K1, Kolovos S2, Lisica A3, Schlueter M3, Nielsen N4
1Novo Nordisk A/S, Søborg, 84, Denmark, 2IQVIA, Athens, Greece, 3IQVIA, London, UK, 4Novo Nordisk A/S, Bagsværd, 84, Denmark
Presentation Documents
OBJECTIVES: As HTA bodies become increasingly familiar with real-world evidence (RWE), and as indicated in the NICE RWE framework, there is a greater recognition of its value in supplementing the clinical evidence package, primarily from randomized-controlled trials (RCTs). External control arms (ECAs) use historical RCT or RW data, allowing for comparison against relevant therapeutic options. This study examined the acceptance of ECA in HTA for chronic diseases. METHODS: HTA guidelines for RWE from NICE, HAS, and IQWiG were reviewed to examine the detail of guidance for ECA analysis. Furthermore, six double-blinded payer expert interviews were conducted to explore the acceptance of ECA in the United Kingdom (UK), Germany, France, and Italy. RESULTS: NICE RWE framework provides guidance on ECA, however insights from HAS and IQWiG are scarce. In the UK, NICE is more open to accept evidence that support the findings from RCTs, whereas in Germany there is a stronger preference for RCT alone. In France and Italy there is an increased interest in ECA, but its acceptance remains low. Acceptance of ECA is higher in the absence of relevant comparator in the RCT, due to evolving treatment landscape or ethical reasons, and in rare diseases and oncology rather than chronic diseases. Overall, ECA is more likely to be accepted in HTA if a strong rationale supporting its use is provided and if data sources are suitable. Moreover, appropriate methodology should be applied to ensure unbiased analysis, especially when ECA is used to show clinical efficacy. CONCLUSIONS: HTA acceptance of ECA remains low, with UK being the exception. A detailed rationale regarding why ECA is relevant, in combination with the use of appropriate data sources and analytical methods can improve acceptance across therapy areas, ultimately leading to an enriched evidence package and reduced uncertainty.
Conference/Value in Health Info
2023-11, ISPOR Europe 2023, Copenhagen, Denmark
Value in Health, Volume 26, Issue 11, S2 (December 2023)
Code
PT30
Topic
Health Technology Assessment
Topic Subcategory
Decision & Deliberative Processes
Disease
No Additional Disease & Conditions/Specialized Treatment Areas