Skip to main page content
U.S. flag

An official website of the United States government

Dot gov

The .gov means it’s official.
Federal government websites often end in .gov or .mil. Before sharing sensitive information, make sure you’re on a federal government site.

Https

The site is secure.
The https:// ensures that you are connecting to the official website and that any information you provide is encrypted and transmitted securely.

Access keys NCBI Homepage MyNCBI Homepage Main Content Main Navigation
Review
. 2025 Apr 22;41(1):e25.
doi: 10.1017/S0266462324004720.

The role and value of real-world evidence in health technology decision-making in France, Germany, Italy, Spain, and the UK: insights on external control arms

Alexa Vidalis  1 , Oscar Dumoulin  1 , Mrunmayee Godbole  2 , Catia C Proenca  2
Affiliations
Review

The role and value of real-world evidence in health technology decision-making in France, Germany, Italy, Spain, and the UK: insights on external control arms

Alexa Vidalis et al. Int J Technol Assess Health Care. .

Abstract

Real-world evidence (RWE) is increasingly used and accepted by health technology assessment (HTA) bodies as supportive evidence to inform the approval of new technologies. However, the criteria driving RWE acceptance are often unclear.This study aims to improve understanding of the role and value of RWE in HTA decision-making and outline the best practices in building real-world external control arms (ECAs).A mixed approach of a targeted literature review and HTA expert interviews was applied. The HTA reports of ten selected technologies and the expert interviews from France, Germany, Italy, Spain, and the UK informed the criteria driving the acceptance of RWE. Overall, the UK and Spanish HTA bodies are more receptive to accepting RWE, whereas the French and German are the least accepting. When RWE is used to substantiate efficacy claims, the level of scrutiny from regulators and HTA bodies is considerably higher than when RWE has different intended uses. Representativeness of the data source, overall transparency in the study and robust methodologies are the key criteria driving RWE acceptance across markets.

Keywords: Europe; decision-making; external control arms; health technology assessment; real-world evidence.

PubMed Disclaimer

Conflict of interest statement

Oscar Dumoulin, Alexa Vidalis, Catia C Proenca, and Mrunmayee Godbole were all employed by Alira Health while contributing to this project. Alira Health is a healthcare consulting company providing services to pharmaceutical and MedTech companies. The work conducted in this project was fully funded by Alira Health and was not associated with any external organization.

Figures

Figure 1.
Figure 1.
Overview of the RWE acceptance status and reimbursement decision among the selected 12 technologies.
See this image and copyright information in PMC

References

    1. Arlett P, Kjaer J, Broich K, Cooke E. Real-world evidence in EU medicines regulation: enabling use and establishing value. Clin Pharmacol Ther. 2022;111(1):21–23. - PMC - PubMed
    1. EMA. Guideline on registry-based studies. 2021. Available from: https://www.ema.europa.eu/en/guideline-registry-based-studies-0.
    1. FDA. Framework for FDA’s real-world evidence program. December 2018. Available from: https://www.fda.gov/media/120060/download.
    1. EMA. Data analytics and real world interrogation network (DARWIN EU). Available from: https://www.ema.europa.eu/en/about-us/how-we-work/big-data/data-analysis....
    1. Flynn R, Plueschke K, Quinten C, et al. Marketing authorization applications made to the European Medicines Agency in 2018–2019: what was the contribution of real-world evidence? Clin Pharmacol Ther. 2022;111(1):90–97. - PMC - PubMed

MeSH terms

LinkOut - more resources