From frustration to innovation: Marc Buyse's mission with One2Treat

One2Treat was born out of my frustration as a biostatistician with conventional approaches to clinical trial research. Over the years, I've witnessed the limitations of relying solely on a single primary criterion for evaluating new treatments. This narrow focus often overlooks valuable data and potential therapeutic benefits.

In many clinical trials, vast amounts of patient data are collected, yet the analysis tends to focus narrowly on one principal endpoint, with just a few secondary endpoints considered. This means that a significant portion of collected data does not influence regulatory decisions. Treatments that might have shown promise under a more comprehensive evaluation end up being disregarded.

One memorable example is the addition of oxaliplatin to 5-fluorouracil for metastatic colorectal cancer. Initially, the treatment failed to gain FDA approval when its primary endpoint, Overall Survival (OS), missed statistical significance. However, it exhibited significant benefits in progression-free survival. This discrepancy underscores the complexity of evaluating treatments solely on OS, especially when subsequent lines of therapy can influence survival outcomes. Oxaliplatin was ultimately approved a few years later and remains a standard of care today, reflecting its proven efficacy. Still, I often wondered how many patients could have benefited from this treatment within the few extra years it took to get approved.

Another case is the addition of erlotinib to gemcitabine for metastatic pancreatic cancer. This combination was approved based on reaching its primary endpoint, which demonstrated a mild yet statistically significant improvement in progression-free survival. However, this evaluation did not consider important factors such as toxicities and a decrease in quality of life, failing to capture the drug's overall benefit-risk profile. Although erlotinib was approved, its use in this indication was extremely limited.

The need for a more inclusive and patient-focused trial design became even clearer when one of my relatives shared his challenging experiences participating in a clinical trial. This story sheds light on a widespread issue: participants frequently receive scant details about the trial's purpose, the drug being tested, or the significance of their involvement, leaving them feeling overlooked. Such a mismatch between trial procedures and patient engagement highlights the urgent need for change. It emphasizes the necessity of a paradigm shift that would place patient insight sand experiences at the heart of clinical research.

To bridge these gaps, I've dedicated myself to developing the Generalized Pairwise Comparisons (GPC) statistical method. This innovative approach enables the comprehensive analysis of treatment effects across multiple clinically relevant criteria, offering a fuller picture of a treatment's potential benefits. One2Treat was founded to broaden the use of this methodology through the creation of software focused on comprehensive benefit-risk analysis. Our aim is to democratize access to advanced statistical analysis, making it accessible to a wide range of users irrespective of their programming or statistical expertise. By emphasizing simplicity and efficiency, One2Treat aims to make advanced statistical analysis a standard part of clinical research, empowering more informed treatment decisions.

One2Treat is dedicated to transforming clinical research by making trials more patient-centric and data-informed. By leveraging our software to consider a broader range of patient-relevant outcomes, we are not only improving the precision of clinical studies but also accelerating the path to market for promising treatments. As we move forward, One2Treat is committed to enhancing drug development processes, ensuring that every piece of patient data is valued, and every potential treatment is thoroughly assessed, incorporating all key patient needs.

Join us in this mission to revolutionize clinical trials and improve patient care.