The treatment of patients in clinical practice relies on the results of randomized clinical trials. The statistical methods currently used to test the treatment effects in such trials take into account a single criterion called “primary endpoint”. This is an incomplete assessment of trial results, especially in oncology and chronic diseases for which quality of life and symptom control are of prime importance. IDDI has contributed to the development of a new statistical method named “generalized pairwise comparisons” (GPC). This new method allows the analysis to take into account all the endpoints at once, whether they represent treatment benefit or harm, as long as these endpoints can be ranked in an order of priority. The method will eventually allow physicians and/or patients to define their own priorities regarding treatment outcomes, thereby leading to “personalized medicine”.
The objective of this collaborative research is to establish proof of the concept that the GPC method can usefully complement traditional methods for the design, analysis and interpretation of clinical-trial results. This objective will be realized after further development of the GPC method and its implementation in dedicated software for the various stakeholders in clinical trials: patients and their families, clinicians, statisticians, health-care organizations, pharmaceutical companies, and regulatory agencies.
This collaborative research will be carried out by a Consortium led by the International Drug Development Institute (IDDI) and comprising Bristol-Myers Squibb, the European Organisation for Research and Treatment of Cancer (EORTC), the Université Catholique de Louvain (UCLouvain) and the University Hospital and Cancer Center of Lyon. The Consortium receives financial support from the Walloon Region, Biowin – the Health Cluster of Wallonia and Innoviris, the Brussels Institute for Research and Innovation. B12 Consulting, based in Louvain-la-Neuve, will support the Consortium in the open source and proprietary software development. The project, called BENEFIT (for Biostatistical Estimation of Net Effects for Individualization of Benefit), will be led by Marc Buyse, IDDI’s Chief Scientific Officer. Damien Tremolet, IDDI’s Chief Executive Officer says: “We believe this project can radically change the analysis of randomized clinical trials in the era of personalized medicine. With this project, IDDI remains true to its commitment of being at the forefront of methodological research in order to develop new therapies in the most reliable, efficient, and patient-relevant manner.”