Presentation (CTI)

OBJECTIVES: For several years, French health authorities have made Real-World Data (RWD) and its use an ambition to better understand and consider common French practice in decision-making. Then, a study was conducted to understand the use of RWD by the Transparency Commission (CT) of the Haute Autorité de Santé (French HTA body), assess the impact of the use of RWD on the CT opinions and finally generate learnings.
METHODS: We analyzed CT opinions on drugs indicated in obesity, oncology-pneumology (O&P) or immunology-inflammation (I&I) from January 2023 to December 2025. RWD related to the following keywords were quantified then analyzed for their impact of CT assessments: medical need/disease burden, clinically relevant comparator, place in the therapeutic strategy, efficacy, quality of life, tolerance, target population, ongoing or future studies, use/sales. Contribution of patient associations was also investigated.
RESULTS: During this period, the CT conducted 6, 14, and 37 assessments for obesity, O&P and I&I drugs, respectively. Of these, 17 were for primo-inscription, 23 for indication extension, and 17 for reassessment. RWD was most used in medical need/disease burden (85%) - with disease burden data mainly from France, target population (81%) and tolerance (62%). In contrast, quality of life (7.2%), efficacy (8.7%), and therapeutic strategy (11.1%) saw limited RWD usage. Patient associations contributed to the assessments for 17%, 57% and 41% of obesity drugs, O&P and I&I, respectively.
CONCLUSIONS: RWD are commonly used in CT evaluations, mostly to define pathology disease burden, describe epidemiology and estimate target populations. They often support the tolerance section, relying primarily on French data, with some international sources as supplements sometimes. Quality of life is rarely documented in real-world studies within CT opinions, despite its expected relevance. CT frequently notes the lack of RWD in dossiers assessed.