Assessing the Value of Real-World Evidence for HTA-Decision Making

OBJECTIVES: This study aims to assess the value of Real-World Evidence (RWE) for Health Technology Assessment (HTA) using the French Haute Autorité de santé (HAS) as a case study.

METHODS: Technology appraisal documents published by the French HTA (Haute Autorité de Santé – HAS) between May 1, 2023 and May 31, 2024 were automatically retrieved using a computer script developed in R. A total of 118 documents were retrieved. Subsequently, an AI-assisted tool was used to select documents containing key-words related with RWE studies such as observational study, or Early-Access Programs, resulting in 67 documents. Using an AI-assisted program developed in Python, the documents were manually reviewed. Documents deemed irrelevant were excluded, whereas those including RWE were evaluated. Data was extracted using the AI-assisted program in a semi-automated manner. The same methodology is being applied to additional HTAs including NICE and AEMPS.

RESULTS: RWE was submitted for a total of 30 technologies. Most of these submissions constituted re-evaluations of previously approved technologies (17), four for indication extension and the remaining were a first submission. Half (14) were targeting rare diseases. Most RWE was submitted in the form of drug utilization, such as from Early Access programs, whereas seven submissions leveraged RWE to support the demonstration of efficacy, six for safety/tolerance and three as descriptive data. Two examples leveraged RWE to support an indication extension, one for the pediatric population of patients with primary and secondary immune deficiencies, a rare disease, and another for the treatment of post-partum hemorrhages, which is a common condition.

CONCLUSIONS: RWE plays an important role for the re-evaluation of technologies by the French Transparency Committee and can in some cases support the demonstration of efficacy. The value of RWE is not restricted to rare diseases. Additional data will inform how other HTAs leverage observational research (retrospective of prospective) for decision-making.