Posted by Michael Wonder on 21 Jun 2018
Confirmatory versus explorative endpoint analysis: decision-making on the basis of evidence available from market authorisation and early benefit assessment for oncology drugs
21 June 2018 - The early benefit assessment of pharmaceuticals in Germany and their preceding market authorisation pursue different objectives.
Data from assessments up to July 2015 are used to estimate the impact of explorative in comparison to confirmatory endpoints on market authorisation and early benefit assessment by contrasting the benefit-risk ratio of EMA and the benefit-harm balance of the HTA jurisdiction. Agreement between market authorisation and early benefit assessment is examined by Cohen’s kappa .
21 of 41 assessments were considered in the analysis. Market authorisation is more confirmatory than early benefit assessment because it includes a higher proportion of primary endpoints.
