Benefit-risk analyses in cancer treatment

Significant challenges persist in the realm of advanced cancer treatment, particularly for types resistant to standard immune checkpoint blockers (ICBs). These blockers, which have revolutionized first-line therapies over the past decade, often see patients developing resistance and relapsing, leaving a gap in effective subsequent treatments.

One strategy to address this unmet medical need involves modulating mechanisms that help tumor cells evade the immune system. These innovative approaches offer significant potential in cancer treatment, representing a shift that may overcome the limitations of more traditional therapeutic strategies.

However, conventional statistical tests used to assess treatment effects can fail to capture the full impact of these emerging therapies. This is because they focus solely on overall patient survival, neglecting other critical aspects of these therapies such as varying toxicity profiles and quality of life improvements.

To illustrate the limitations of standard statistical assessments of treatment effect, consider the scenario of two patients in a clinical trial. Both have similar survival times but experience vastly different levels of toxicity. Traditional analyses might treat these patients as equivalent, failing to differentiate between the treatments due to the similar survival times. However, the two patients’ experiences suggest a stark contrast in their treatment experience, underscoring the necessity for a more holistic approach.

One2treat has successfully applied its methodology* to provide a more holistic view on the overall effects of a novel therapy. By listening to patients’ voices, multiple outcomes were identified, including both benefits and risks of the treatment, and then prioritized in collaboration with clinicians. Leveraging data from the Phase II clinical trial, One2Treat informed the design of the future Phase III trial.

Incorporating multiple patient-relevant outcomes into a single Net Treatment Benefit metric allowed to reflect the actual overall benefits of the treatment to patients. Moreover, the specific contribution of each outcome is transparently displayed. This approach not only highlighted the clinical relevance of the treatment’s effects in this comprehensive manner, but also allowed for a large sample size reduction. This robust statistical analysis successfully convinced regulatory authorities, more accurately reflecting the preferences and needs of patients.

As we advance in cancer research, it's crucial to adopt more comprehensive evaluation methods that consider the full spectrum of patient outcomes. This approach marks a substantial advancement in our commitment to transforming cancer care through more patient-centered research and treatment analysis.

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*One2Treat is leveraging the robust and innovative Generalized Pairwise Comparisons methodology, already approved by the regulatory agencies in multiple clinical trials led by various pharma sponsors.

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