ECRIN'S Director General Provides a Report on ICTD 2016

ECRIN's Director General, Jacques Demotes, provides a brief report on this year's celebration of International Clinical Trials Day (ICTD). 

The ICTD 2016 conference on “Clinical trials in the era of personalised medicine", held in the Senate of the Parliament in Prague, Czech Republic on May 20th, 2016 first addressed the unclear definition of personalised medicine, a phrase used in three distinct situations:

• individualized medicine, where the treatment is designed and manufactured for a given patient
• targeted therapy, where the treatment decision is based on the molecular / genomic abnormality characteristic for the patient
• stratified medicine, where the healthcare strategy is based on refined phenotyping techniques and disease taxonomy

This approach therefore refers to healthcare strategies where the treatment option depends on decision trees involving identification of genomic biomarkers (in the case of tumors, both somatic and tumoral genome), of epigenetic factors, of phenotypic characteristics (including imaging markers, metabolomic and other biomarkers), and of psychological, social and environmental determinants. Personalised medicine however appears as a quantitative rather than qualitative change in medical practice : treatment decision is based on a more complex and comprehensive diagnostic procedure, more closely related to the molecular and cellular mechanisms underpinning the disease.

Such increased complexity is expected to result in improved accuracy of treatment, enhancing safety and/or efficacy. However, targeting a protein or signaling pathway may not necessarily lead to the expected therapeutic action, because of cross-talks in transduction pathways, of redundancies, and of partial lack of specificity of therapeutic agents. Despite thorough understanding of the mechanism of disease, convincing evidence regarding their mechanisms of action, and elegant biomarkers to define responsive patients, these treatments still have to be tested in interventional trials to demonstrate their superiority in terms of efficacy and safety when compared to the standard of care. This raises many issues regarding the sample size, multiple analyses and trial design: for instance, should we randomise broad patient populations (to compare the standard treatment vs. the whole personalized medicine strategy - diagnostic and therapeutic procedure), or only the subgroup of potentially responder patients (to compare the ‘personalised’ treatment vs. standard of care in this subgroup)?

Access to patients and to medical expertise is anyway critical to provide evidence for the efficacy and safety of such healthcare strategies. Therefore international cooperation, and access to multinational funding sources, is crucial for the development of this therapeutic approach.