Real-World Data (RWD): a major turning point in clinical evaluation
Real-World Data (RWD) and their use in the form of Real-World Evidence (RWE) now play a central role in the evaluation of health technologies.
Long regarded as a simple complement to randomized controlled trials, RWD are now recognised as a structuring decision-making tool for regulatory, clinical and economic assessments, for both medicines and medical devices.
This evolution reflects several shared observations by health authorities:
limitations of controlled clinical trials, the need for long-term data, improved representativeness of patients treated in routine clinical practice, and increasingly stringent post-marketing surveillance requirements.
Real-World Data and medicines: a key lever after marketing authorisation
A now explicit methodological framework in France
In France, the Haute Autorité de Santé (HAS) has clearly formalised the role of real-world studies in the evaluation of medicines.
RWD may be used to complement clinical trials, document real-world effectiveness and safety, and support reassessments of SMR (actual benefit) and ASMR (added clinical value) after marketing authorisation.
HAS methodological guide – Real-world studies for the evaluation of medicines and medical devices
https://www.has-sante.fr/jcms/p_3274960/fr/guide-methodologique-etudes-en-vie-reelle-pour-l-evaluation-des-medicaments-et-dispositifs-medicaux
This institutional recognition marks a paradigm shift: clinical evidence is no longer static, but becomes evolving, built throughout the entire lifecycle of a medicinal product.
Increasing use of RWD in pricing and reimbursement decisions
Real-World Data have also become a strategic issue for the CEPS (Economic Committee for Health Products), particularly in the context of:
- pricing agreements,
- price revision clauses,
- post-reimbursement follow-up.
While the CEPS has not published a specific methodological guide on RWE, the CEPS / Leem framework agreement constitutes the official reference governing the use of post-authorisation data in economic negotiations.
CEPS / Leem framework agreement
https://sante.gouv.fr/IMG/pdf/accord_cadre_21-24_signe.pdf
In this context, RWE helps document the real-world value of a medicine, beyond results observed in highly selected clinical trial populations.
A European strategy structured around registries and databases
At European level, the European Medicines Agency (EMA) explicitly recognises the value of data derived from patient registries and healthcare databases, notably through its guideline on registry-based studies:
EMA – Guideline on registry-based studies
https://www.ema.europa.eu/en/documents/scientific-guideline/guideline-registry-based-studies_en.pdf
This approach is further reinforced by the deployment of DARWIN EU, a European programme designed to generate real-world evidence to support regulatory decision-making.
Real-World Data and medical devices: a permanent regulatory requirement
MDR: RWD at the core of continuous clinical evaluation
For medical devices, Real-World Data play an even more structural role.
Regulation (EU) 2017/745 (MDR) imposes a continuous clinical demonstration based in particular on:
- Post-Market Clinical Follow-up (PMCF),
- market surveillance activities,
- regular updating of the benefit–risk assessment.
👉 Regulation (EU) 2017/745 – MDR
https://eur-lex.europa.eu/legal-content/FR/TXT/PDF/?uri=CELEX:02017R0745-20250110
Unlike medicines, the collection of real-world data is not optional for medical devices: it is a core pillar of regulatory compliance.
MDCG guidelines: clarifying regulatory expectations
The Medical Device Coordination Group (MDCG) has clarified expectations regarding clinical and post-market data through several reference documents:
MDCG 2020-6 – Guidance on sufficient clinical evidence
https://health.ec.europa.eu/system/files/2020-09/md_mdcg_2020_6_guidance_sufficient_clinical_evidence_en_0.pdf
MDCG 2020-7 – PMCF plan template and evaluation report
https://health.ec.europa.eu/system/files/2020-09/md_mdcg_2020_7_guidance_pmcf_plan_template_en_0.pdf
These documents confirm that data derived from real-world use, when methodologically robust, are fully acceptable to demonstrate the performance and safety of medical devices.
Recognition also supported by the HAS
In France, the HAS also recognises the value of observational data for the clinical evaluation of medical devices, particularly innovative or connected devices, when randomised trials are difficult to conduct.
HAS – Guide on clinical evaluation of connected medical devices (PDF)
https://www.has-sante.fr/upload/docs/application/pdf/2019-02/guide_sur_les_specificites_devaluation_clinique_dun_dmc_en_vue_de_son_acces_au_remboursement.pdf
Medicines and medical devices: convergence under specific conditions
While medicines and medical devices are converging toward greater use of Real-World Data, their regulatory logics remain distinct:
- for medicines, RWD are mainly used post-marketing authorisation, as a complement to clinical trials;
- for medical devices, RWD are embedded in a continuous lifecycle approach, integrated into regulatory compliance.
In both cases, authorities emphasise one fundamental principle: methodological quality.
Data governance, source traceability, bias control and analytical transparency are key conditions for regulatory acceptability.
Conclusion: toward a new culture of clinical evidence
Real-World Data do not replace clinical trials, but they profoundly redefine their role.
They reflect a transition toward dynamic clinical evidence, built over time and under real conditions of use.
For manufacturers and sponsors, the challenge is no longer whether Real-World Data will be used, but how to anticipate and structure them early, as part of development strategies and post-market follow-up, to support informed regulatory decisions and patient safety.
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See other articles on this subject : Real-world data: what role do they play in clinical research today?; Patient Registries and Medical Device Registries: An Underused Resource for Clinical Research?
