One2Treat patented software was used by both clinicians and breast cancer patients’ representatives in Italy and Portugal, enabling to foster more effective, patient-centered discussions. The software provided personalized benefit-risk assessments directly from clinical trial data, prioritizing the most relevant outcomes to each patient. Facilitating this shared decision-making process is instrumental to improve both treatment acceptance and adherence.

One2Treat significantly enhanced a Phase III oncology trial by expanding its design to include analysis of adverse event (AE) severity and duration, beyond mere occurrence. Adapting the trial’s primary endpoint to include these multiple clinically relevant outcomes allowed for a more comprehensive assessment of the treatment effect, in line with the wishes of patients-advocacy group. This innovation led to a 19% reduction in sample size and over 20% faster recruitment, demonstrating our commitment to efficient, patient-centric clinical research. This advancement underscores our capability to streamline clinical development, offering quicker, more cost-effective paths to new treatments.

Initial analysis using conventional statistics focused on the frequency of AE as the single endpoint. One2Treat complemented the analysis of the data by capturing the combined effect of the drug on the frequency, severity, duration, and time to onset of the AE. The One2Treat approach led to increased statistical and clinical significance, offering the FDA a compelling case for the substantial benefits of the drug candidate.

A quantitative assessment was conducted to evaluate the benefit-risk balance of a specific treatment for metastatic pancreatic cancer. This investigation focused on integrating key efficacy outcomes such as overall survival, distant and local tumor progression, with safety outcomes such as occurrences of severe toxicities. The results offered robust empirical evidence, supporting the therapy’s superior efficacy and safety profile compared to competitors. Such research is crucial for informing prescription guidelines and conducting comprehensive cost-effectiveness evaluations.

Investigators needed to lower the dose (to reduce toxicity & improve compliance) without loss of efficacy. Using a conventional trial design to prove non-inferiority had an original sample size of 700 patients.

Using the GPC methodology to include both efficacy and safety criteria to assess the net treatment benefit of the reduced dose, made the trial feasible by reducing the required sample size to only 280 patients.

Results from a phase 3 clinical trial fell short of expectations but exhibited beneficial effects on other outcomes that could have supplemented its primary endpoint. Integrating these other outcomes into a single analysis led to a deeper understanding of the treatment effect and its significant impact on some patient subgroups. This newly discovered potential of the treatment was used as a central point in the design of a new phase 3 trial.

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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