PNS153 The Role of Real World Evidence in Health Technology Assessment over Time: A Research on the French Situation


      Real-World Evidence (RWE) is more and more recognized as a valuable source of evidence and has been a growing focus of Health Technology Assessment (HTA) in the past few years. This ongoing research aims at describing the role of post-registration studies in the HTA process and the evolution of those studies in France.


      Using the list published by the French HTA body (April 2020) on the commissioned and completed post-registration studies, the corresponding Transparency Committee recommendations were retrieved from the French HTA body website. A systematic review of these recommendations is underway. The types of outcomes expected from the post-registration studies, as well as the methodologies, are being studied. Where post-registration studies have been provided, the evolution of two indicators, the Actual Clinical Benefit (ACB) and Clinical Added Value (CAV), used to determine the reimbursement rate and price, is monitored.


      253 Transparency Committee recommendations were retrieved, 122 concerning ongoing requests and 131 concerning completed studies. Studies were commissioned between 1997 and 2020. The most represented therapeutic areas for completed studies were cardiovascular diseases and rheumatology, while for ongoing studies they were oncology and endocrinology. Assessing effectiveness and Quality of Life were one of the objectives of 10% and 6% of completed studies, respectively, whereas this was the case for 84% and 25% of ongoing studies. Understanding the conditions of prescriptions or use of the medicines remained the most important study objective (>90%). First results show that the impact on ACB and CAV was limited. CONCLUSION: RWE is increasingly requested for HTA in France, while the shape of these studies has deeply evolved over the years. The lack of standardized methodologies to develop RWE has undercut its impact on regulators’ decision-making. It is now crucial to further define how collecting, analyzing, and reporting real-world data should be done.