Personalized Medicine: Getting More Out of Clinical Trials

Results of randomized clinical trials can be somewhat limited. Currently used statistical methods to test drug candidates only consider a single criterion—the primary endpoint. This can represent an incomplete assessment of trial results, particularly in oncology and chronic diseases, where quality of life and relief of symptoms are of prime importance.

The International Drug Development Institute (IDDI) has contributed to the development of a new statistical method called “generalized pairwise comparisons” (GPC), which 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 aims to allow physicians and/or patients to define their own priorities regarding treatment outcomes, thereby leading to “personalized medicine”.

IDDI recently initiated collaborative research on a statistical method for individualization of therapy. The goal is to establish proof of the concept that the GPC method can complement traditional methods for the design, analysis and interpretation of clinical trial results.  The research will be carried out by a Consortium led by IDDI and comprising Bristol-Myers Squibb, the European Organization for Research and Treatment of Cancer (EORTC), the Université Catholique de Louvain (UCLouvain) and the University Hospital and Cancer Center of Lyon.

Contract Pharma spoke with Marc Buyse, founder of IDDI, and Everardo Saad, medical director of IDDI, about the goals of the new collaboration, the shortcomings of current methods in clinical trials and how new statistical methods can be used to improve outcomes. –KB