CRED ERP 25

07/07/2025

● Currently, there is a lack of detailed information on the use of RWE in marketing authorization applications in terms of objectives (for example, to assess safety or efficacy endpoints, use in special populations, or inform risk management planning), data sources, methods, the strengths and weaknesses of these, and the outcome of its assessment. ● Previously published studies on the topic have been dependent on information available in the public domain, for example, European Public Assessment Reports (EPARs), and have therefore based their evaluation on products that complete the authorization process rather than those submitted for authorization,thereby ignoring evidence submitted in withdrawn applications. ● In addition, considering exclusively the evidence leading to a final opinion by the EMA Committee for Medicinal Products for Human Use (CHMP) provides only a partial picture of the usefulness of RWE for marketing authorization. ● An evaluation of the totality of the information, including the nonpublished assessment of products that were withdrawn or for which additional data were requested during the assessment procedure, would support the provision of recommendations for best practice, improve the efficient use of RWE for regulatory applications, and enable assessment of the needs for data validation, expertise, and training.

The Organisation for Professionals in Regulatory Affairs

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REFERENCES

1. https://www.ema.europa.eu/en/homepage

2. Key steps and considerations of the EU centralised procedure | Journal | Regulatory

Rapporteur

3. Orphan designation: Overview | European Medicines Agency (EMA)

4. Marketing Authorisation Applications in a Nutshell - Somerville Development Partners

5. Marketing Authorization Applications Made to the European Medicines Agency in 2018 – 2019: What was the Contribution of Real ‐ World Evidence? - Flynn - 2022 - Clinical

Pharmacology & Therapeutics - Wiley Online Library

The Organisation for Professionals in Regulatory Affairs

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