The Net Treatment Benefit (NTB) is a comprehensive estimator of treatment effect for randomized clinical trials. Unlike traditional statistical assessments, the NTB expresses multiple clinically relevant outcomes integrated hierarchically into a single, holistic assessment.
The Net Treatment Benefit bridges the gap between robust statistic assessments and the complex realities of patient experiences, aligning with the increasing demand for patient-focused research. The methodology fosters a quantitative understanding of treatment benefits and risks, enabling clinical research to reflect the multifaceted nature of patient care.
The Net Treatment Benefit gives a unique opportunity to leverage all clinically relevant information into a single assessment, reducing the sample size required to run phase II and III clinical trials. By working closely with clinicians, patients and patient-advocacy groups, relevant clinical outcome preferences and hierarchy can be integrated into a single framework. One2Treat Voice solution offers an elegant solution to formalize such clinical outcomes preferences.
With One2Treat’s proprietary Insights solution, multiple clinical outcomes can be analyzed simultaneously, providing a comprehensive, multidimensional perspective. This broader approach allows for a deeper understanding of treatment effects, identifying valuable insights that might otherwise remain hidden. By seamlessly processing clinical trial data, we not only inform and optimize future trial designs but also lay the groundwork for smooth regulatory approval, HTA considerations, and market access strategies.
By integrating prespecified analyses into the design phase, One2Treat ensures that the product’s clinical and economic benefits are more effectively communicated to key stakeholders—payers, providers, and patients. This proactive approach strengthens the product’s value proposition, making it more compelling across the entire treatment decision spectrum, from clinical efficacy to market positioning.
The methodology behind the Net Treatment Benefit, Generalized Pairwise Comparisons (GPC) is a generalization of the Wilcoxon-Mann-Whitney test. Marc Buyse contributed to develop this non-parametric method, initially designed for comparing two groups across a continuous variable, to accommodate multiple outcomes of various types. This allows for a more nuanced understanding of treatment effects by considering a broader range of patient-relevant outcomes, enhancing the statistical power and relevance of clinical trials.
In a nutshell, all possible pairs of patients from the experimental and control arms are created. Each pair is first compared on the highest-priority outcome, classified as “favorable to treatment”, “unfavorable to treatment”, or a “tie.” If a tie occurs, the comparison moves to the next priority outcome, continuing until all pairs are classified or all outcomes are assessed.
The NTB, which can be calculated for each outcome or the global test, estimates the treatment effect and has an intuitive interpretation. A NTB of 15% means that any random patient in the experimental arm has a 15% net probability of doing better than any random patient in the control arm.
GPC provides a more detailed and patient-focused analysis, capturing the nuanced differences between treatments and facilitating informed decision-making.