MEB-seminarium: "Real World Evidences”: do they matter for health regulatory authorities?
Speaker: Marc Bardou, Professor, Coordinating Physician Clinical Research Unit INSERM 1432 & gastroenterology and liver department, Dijon, France
In the process of drug review by health regulatory authorities (HRA), randomized controlled trials (RCTs) have been considered the “gold standard”, as they are assumed to provide unbiased evaluation of the risk to benefit ratio. Nevertheless the paradigm is being questioned both for efficacy and safety assessment, as the benefit of many medications has been recently challenged.
In France, as in many European countries, drug reimbursement has long been based on public health considerations, the so-called “unmet medical need” and “public health interest”, than on proven clinical benefit for the patient. At a time of cost constrains, it seems rational to cover for medication that have been shown to significantly impact either disease course or clinically meaningful endpoints.
Additionally as cost is increasingly being considered as a driver of the decision making process, real world data are needed to assess real costs, and conduct valid cos-effectiveness analyses. For many reasons, RCTs are sometimes difficult to conduct or cannot properly assess long-term efficacy or safety, or detect rare but severe events. Besides that, when a drug has been approved and comes to the “real world” it will meet real physician and real patients.
In that extent Health regulatory authorities are looking for data from the Real World, to understand how a drug is being prescribed by physicians and used by patients in order to turn efficacy into effectiveness. This talk will first present market approval and market access schemes in France, and then explain why we are in need for new way to assess medication benefit. It will then, based on a few examples, show how real world data can be reviewed by HRA, and what are the limitations of such data. We will then try to suggest some perspective to ensure a proper evaluation a drug risk to benefit ratio.Contact person: Isabelle Le Ray