Defining a Clinically Meaningful Benefit in Cancer Clinical Trials: From the Perspectives of the Clinical Trialist, Patient, and Society
In value-based medicine, clinical trials must show both statistically significant and clinically meaningful improvements, such as ASCO's recommendation of a 20% relative improvement in overall survival for metastatic solid tumors. Lawrence et al. found that while many phase III trials met statistical significance, fewer achieved clinically meaningful results, emphasizing the need for ethical trial design and maintaining participant engagement. Quality of life improvements are also crucial, as small survival benefits or reduced toxicity can be significant for patients. Lastly, assessing the value of new therapies involves balancing clinical benefits with costs, which varies by societal and regulatory perspectives.
In an era of value-based medicine, it is increasingly important to design clinical trials of new therapies that not only show statistically significant benefits compared with current standard therapies but also yield an adequate magnitude of benefit to be deemed “clinically meaningful.” In a 2014 recommendation, the American Society of Clinical Oncology (ASCO) indicated that clinical trials studying targeted therapies for metastatic solid tumors need to demonstrate a 20% or more relative improvement in overall survival (OS) (ie, hazard ratio [HR] ≤0.8) to be considered to have a clinically meaningful benefit...
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