Twelve years of European cancer drug approval-a systematic investigation of the 'magnitude of clinical benefit'.

Background The magnitude of clinical benefit of solid cancer drugs can be standardly assessed via the Magnitude of Clinical Benefit Scale (MCBS) developed by the European Society for Medical Oncology (ESMO). We applied two ESMO-MCBS versions to the last 12 years of European cancer drug approval and compared two predefined marketing authorisation timeframes to identify potential score changes over time. Material and methods Originator solid cancer drugs and indication extensions that were approved between 1 January 2009 and 31 October 2020 by the European Medicines Agency (EMA) were included in our analyses. To evaluate the clinical benefit of these cancer indications, the original ESMO-MCBS (v 1.1) and a locally adapted ESMO-MCBS version were applied to the study sample. Thus, two ESMO-MCBS versions were compared, and an additional analysis was conducted to identify potential score differences between two approval timeframes 2009-2014 versus 2015-2020. Results A total of 144 cancer indications intended as curative (n = 9) or non-curative (n = 135) treatment options were eligible for an ESMO-MCBS assessment. Solely a minority of the assessed cancer indications met the meaningful clinical benefit (MCB) criteria independent of the applied version of the scale and treatment intention (original: n = 48/144, 33.3% versus adapted: n = 27/144, 18.8%). Comparing the two EMA approval timeframes, a growing number of approved cancer indications could be observed: 2009-2014: n = 9/year versus 2015-2020: n = 14/year. In addition, almost no difference in the proportion of cancer indications that have met the MCB criteria was detectable when comparing the predefined authorisation timeframes (MCB increase original: +4.1% and adapted: +3.9%). Conclusion Applying both versions of the ESMO-MCBS can help to identify potentially beneficial cancer indications, but also those with rather uncertain or low clinical benefit and thus, support the fair allocation of limited health care resources.