Several oncology societies have developed tools to quantify the magnitude of clinical benefit of drugs for the treatment of solid tumors. These include the American Society of Clinical Oncology Value Framework (ASCO-VF), the European Society for Medical Oncology Magnitude of Clinical Benefit Scale (ESMO-MCBS), the National Comprehensive Cancer Network (NCCN) Evidence Blocks, and the ASCO Cancer Research Committee criteria (ASCO-CRC).

The magnitude of clinical benefit does not influence regulatory approval of cancer drugs. Approval by regulatory agencies such as the US Food and Drug Administration (FDA) requires substantial evidence of safety and efficacy from adequate and well-controlled trials irrespective of the magnitude of such benefit. Advances in the understanding of the molecular basis of cancer have led to rapid development of new drugs and an increasing number of cancer drug approvals based on non-randomized trials. Despite this, randomized controlled trials (RCTs) remain the gold standard to evaluate the benefits and risks of new cancer therapies.

However, RCTs have limitations, including the increasing use of intermediate endpoints which have not been validated as true surrogates for definitive outcomes, and the choice of control group therapy which may not always reflect contemporary standard of care.

While there is extensive literature exploring the associations between the magnitude of clinical benefit and characteristics of new drugs,