Building Primary Evidence Differently

It is estimated that more than half of patients with a chronic disease have chronic multi-morbidities (i.e., at least two chronic diseases). We developed a new concept, the “burden of treatment” defined as the “work of being a patient” using innovative internet-based qualitative methods. Particularly, our team was the first to propose a comprehensive view of the components and consequences of the burden of treatment across multiple countries, settings, and treatment contexts. We showed that the workload entailed by strict adherence to all clinical practice guidelines’ recommendations by multimorbid patients was not sustainable. Finally, we recently set up the ComPaRe e-cohort (“Communauté de Patients pour la Recherche”) aimed at including patients with chronic diseases for 10 years. ComPaRe was a breakthrough in its construction: it was not built to answer a specific question but to analyze a wide range of problems and perform nested interventional studies; participants are enrolled through specific cohorts on the basis of their conditions, treatments, or any other characteristic (e.g., participants with multimorbidities); they are characterized with broad clinical variables and followed up according to non–disease-specific outcomes; their data are enriched via their link to large administrative databases (e.g., French National Reimbursement databases).

Our team is internationally recognized for its work on non-pharmacologic treatments [NPTs]. During this mandate, we updated the CONSORT extension for NPTs. We tackled issues raised by the assessment of NPTs in observational studies. We showed a huge increase of observational studies using propensity score analyses in surgery, but methods and reporting raised important concerns. We compared treatment effects between observational studies with propensity scores and RCTs and found no statistically significant difference, on average. Finally, we explored methodological issues when assessing fecal microbiota transplantation, a novel treatment option for chronic diseases associated with altered gut microbiota.

Precision medicine mainly relies on choosing the treatment based on the presence or absence of predictive biomarkers. To explore how precision medicine is achieved in practice, we investigated the presence of a biomarker in drug labels and questioned the evidence supporting the approval of oncology drugs with a predictive biomarker based mainly on trials restricted to biomarker-positive patients. Finally, we showed that only a few new cancer drugs provided meaningful clinical benefit.