HAS (France) analyzed the use of real-world clinical practice data in clinical and economic studies of innovative technologies
The French agency for health technology assessment, the Haute Autorité de Santé (HAS), has published the results of an analysis of the use of real-world data (RWD) in cost-effectiveness analyses (CEAs) submitted for review between 2016 and 2023.
The analysis included 147 CEA studies, 88% of which used RWD. A total of 331 real-world data applications were recorded, based on 527 individual studies, reflecting the systematic inclusion of RWD in the assessment of innovative medical technologies in France.
The main areas of RWD use are:
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Characteristics of the analyzed patient population — 35%;
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External validation of clinical-economic models — 19%;
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Justification of the comparison strategy — 16%.
RWD was used much less frequently for:
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Cost assessment — 10%;
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Quality of life parameters — 7%
The most common sources of RWD were retrospective cohort studies, patient registries, and administrative databases. The largest number of clinical economic studies using RWD were in oncology.
Orphan drugs accounted for 24% of all clinical economic studies. 94% of these assessments used real-world clinical practice data, primarily to form external controls in single-arm clinical trials.
However, HAS noted increased methodological risks in this group, including:
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insufficient control for confounding factors;
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problems with missing data;
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limited validity of quality-of-life parameters.
In 8% of cases, clinical economic studies using RWD were considered methodologically unacceptable.
Key finding of the HAS: Further development should focus not on quantitative expansion of RWD use, but on improving the quality of its collection and analysis.
Priorities include:
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standardization of approaches to RWD generation;
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increasing the transparency of analytical methods;
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stricter control of systematic biases and confounding factors;
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Targeted prospective collection of real-world data to reduce uncertainty in the assessment of innovative and orphan technologies and subsequent review of access and reimbursement decisions.
HAS document (https://drive.google.com/file/d/1SrgOWm3YOD6XC9TqiXi0B6qstdTteMEM/view?usp=sharing)
