Manchester, UK – Variability in response to treatment (also called heterogeneity of treatment effects) is a common finding in clinical trials. Some variability is hypothesised and expected, such as differences between treatment and placebo groups, across doses, or between comparator drugs. However, heterogeneity can result in non-significant differences between treatment groups. Given the great expense associated with designing new pharmaceuticals and biologics and conducting clinical trials to evaluate their safety and efficacy, it would be beneficial to have analytic methods that can efficiently identify patients who are more responsive to treatment.
Researchers at RTI Health Solutions in Manchester, UK, presented a step-by-step guide to innovative methods for examining and modelling heterogeneity within longitudinal clinical trial data. The report, “Identifying Differential Responders and Their Characteristics in Clinical Trials: Innovative Methods for Analysing Longitudinal Data,” published in Value in Health, was co-authored by Donald E. Stull, PhD, and Katherine Houghton, BSc.
The methods the authors present are extremely flexible and powerful, allowing analysts to examine large datasets relatively quickly, and study multiple outcome variables simultaneously (e.g., clinical and patient-reported outcomes). Donald E. Stull, PhD, the lead author of the study notes, “Identifying differential responders obviously has advantages for understanding treatment effects and may provide clues that can move us closer to personalized medicine. The insights from these analyses, especially when used in early-phase trials, can aid in designing subsequent trials that are more appropriately powered and include the most relevant patients, requiring fewer patients for the same or better effect size. This could save substantial amounts of money on a trial and overall trial program. Furthermore, this can help minimize exposure to potentially toxic treatments for those patients who are likely to be the least responsive.”
Value in Health (ISSN 1098-3015) publishes papers, concepts, and ideas that advance the field of pharmacoeconomics and outcomes research as well as policy papers to help health care leaders make evidence-based decisions. The journal is published bi-monthly and has over 8,000 subscribers (clinicians, decision makers, and researchers worldwide).
International Society for Pharmacoeconomics and Outcomes Research (ISPOR) is a nonprofit, international, educational and scientific organization that strives to increase the efficiency, effectiveness, and fairness of health care resource use to improve health.
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