A new peer-reviewed article developed within the ERAMET project has been published in Clinical and Translational Science: “Co-creating a framework to assess the quality and fitness-for-purpose of real-world data for use in rare disease treatment and regulatory decision-making.”
The publication presents a structured framework to evaluate the quality and appropriateness of real-world data (RWD) for use in the context of rare diseases. The framework was co-created through a multi-stakeholder approach involving patients, regulators, clinicians, and data experts, ensuring relevance and applicability across diverse perspectives.
The article outlines:
A transparent methodology for assessing RWD quality and fitness-for-purpose;
Key criteria and dimensions to guide data evaluation;
Practical considerations for integrating RWD into regulatory and clinical decision-making processes.
This work contributes to the broader goals of the ERAMET project by supporting the development of robust, patient-centred approaches to the use of real-world evidence (RWE) in rare disease research and regulatory pathways.
đź”— The full article is available here.
