A standardised, generic, validated approach to stratify the magnitude of clinical benefit that can be anticipated from anti-cancer therapies: the European Society for Medical Oncology Magnitude of Clinical Benefit Scale (ESMO-MCBS)

Abstract

The value of any new therapeutic strategy or treatment is determined by the magnitude of its clinical benefit balanced against its cost. Evidence for clinical benefit from new treatment options is derived from clinical research, in particular phase III randomised trials, which generate unbiased data regarding the efficacy, benefit and safety of new therapeutic approaches. To date, there is no standard tool for grading the magnitude of clinical benefit of cancer therapies, which may range from trivial (median progression-free survival advantage of only a few weeks) to substantial (improved long-term survival). Indeed, in the absence of a standardised approach for grading the magnitude of clinical benefit, conclusions and recommendations derived from studies are often hotly disputed and very modest incremental advances have often been presented, discussed and promoted as major advances or 'breakthroughs'. Recognising the importance of presenting clear and unbiased statements regarding the magnitude of the clinical benefit from new therapeutic approaches derived from high-quality clinical trials, the European Society for Medical Oncology (ESMO) has developed a validated and reproducible tool to assess the magnitude of clinical benefit for cancer medicines, the ESMO Magnitude of Clinical Benefit Scale (ESMO-MCBS). This tool uses a rational, structured and consistent approach to derive a relative ranking of the magnitude of clinically meaningful benefit that can be expected from a new anti-cancer treatment. The ESMO-MCBS is an important first step to the critical public policy issue of value in cancer care, helping to frame the appropriate use of limited public and personal resources to deliver cost-effective and affordable cancer care. The ESMO-MCBS will be a dynamic tool and its criteria will be revised on a regular basis.

Availability of evidence of benefits on overall survival and quality of life of cancer drugs approved by European Medicines Agency: retrospective cohort study of drug approvals 2009-13

“…Firstly, only 18 (26%) indications for use in our cohort were supported by trials in which extension of life was the primary outcome, and trials that evaluated survival as a secondary endpoint might not have been powered to detect differences between groups with and without the experimental treatment. Secondly, crossover from control to experimental arm after disease progression (or to similar drugs after the study) is a commonly cited reason for the lack of survival benefits in clinical trials of oncology products 38. It should be noted, however, that for some indications for use in our cohort there were no survival gains even in the absence of crossover 7…”

“…For those drugs associated with an overall survival gain at the time of approval or in the postmarketing period, we used the European Society for Medical Oncology’s (ESMO) Magnitude of Clinical Benefit Scale (ESMO-MCBS) to assess the clinical value of these gains as reported in published studies 38. The ESMO-MCBS scale is a tool for evaluating the clinical benefit of new treatments for solid tumours to facilitate the presentation of clear and unbiased statements regarding the relative clinical benefit of new treatments.…”

“…Our scoring was limited to drugs for solid tumours as the ESMO scales are not intended for evaluation of drugs to treat haematological malignancies 38. Two investigators (AA and CD) independently graded individual trials based on published papers that reported a significant difference in overall survival (as a prespecified primary or secondary study endpoint) using the most recent version of the ESMO-MCBS evaluation forms 39.…”

Price Transparency for Whom? In Search of Out-of-Pocket Cost Estimates to Facilitate Cost Communication in Cancer Care

“…Professional societies, such as ASCO, National Comprehensive Cancer Network (NCCN), or European Society for Medical Oncology, have established their own homegrown version of a 'value framework' or 'evidence block', as in the case of the NCCN [17][18][19][20]. Only the ASCO value framework acknowledges the role of out-of-pocket costs in its value assessment criteria.…”

Did It Matter That the Cancer Drugs Fund Was Not NICE? A Retrospective Review

“…There is no provision in the prioritisation tool to distinguish between measures of survival in curative and non-curative settings, unlike the overall distinction made between curative and non-curative therapies in the European Society for Medical Oncology Magnitude of Clinical Benefit Scale (ESMO-MCBS) (22), or the distinction between advanced and potentially curative in the ASCO (American Society Of Clinical Oncology) framework (23). The use of PFS, and similar measures other than OS, are not reliable surrogates for OS in general (24,25) (23) and specifically in noncurative settings (22).…”