CONFIRMATORY VERSUS EXPLORATIVE ENDPOINT ANALYSIS- DECISION-MAKING UNDER UNCERTAINTY ON THE BASIS OF EVIDENCE AVAILABLE FROM MARKET AUTHORIZATION AND EARLY BENEFIT ASSESSMENT IN GERMANY
Author(s)
Niehaus I1, Dintsios C2
1Cologne Institute for Health Economics and Clinical Epidemiology, Cologne, Germany, 2Heinrich Heine University of Düsseldorf, Düsseldorf, Germany
OBJECTIVES: To compare the use and importance of confirmatory endpoints in German early benefit assessment (EBA) versus market authorization (MA) for oncology drugs with both evaluations relying on the same evidence. The EBA of pharmaceuticals in Germany and their preceding MA pursue different objectives resulting in divergent decision-making strategies. This is reflected inter alia by the inclusion of varying confirmatory endpoints within the evaluation of oncology drugs. METHODS: Identification of endpoints is based on a specifically developed guide. Data from completed assessments up to July 2015 are used to estimate the impact of explorative endpoints in comparison to confirmatory endpoints on MA and EBA by contrasting the benefit-risk ratio of EMA and the benefit-harm balance of the HTA jurisdiction. Agreement between MA and EBA is examined by Cohen’s kappa (k). RESULTS: CONCLUSIONS: The comparability of the two evaluation processes based on endpoints is only possible with some limitations when using the evidence contributed by clinical trials. To warrant a broader confirmatory basis for decisions supported by HTA, closer inter-institutional cooperation of approval authorities and national HTA jurisdictions would be favorable. A reduction of the uncertainty surrounding decisions can be achieved by means of joint advice for manufacturers regarding endpoint definition and hierarchy.
Conference/Value in Health Info
2017-11, ISPOR Europe 2017, Glasgow, Scotland
Value in Health, Vol. 20, No. 9 (October 2017)
Code
PHP254
Topic
Health Technology Assessment
Topic Subcategory
Decision & Deliberative Processes
Disease
Multiple Diseases