Use of Real-World Data and Real-World Evidence for regulatory decision-making

October 2020

Objective: With the present paper, EuropaBio would like to support the generation of EMA/HMA guidance(s) on RWD/RWE aligned with international regulatory requirements already used by other Regulatory Authorities.

Health data which are generated in clinical practice and are increasingly in day-to-day life by novel technologies provide a large data pool that should be more broadly utilized to support regulatory decision-making.

Fit-for-purpose Real-World Data (RWD) and subsequent Real-World Evidence (RWE) should become more broadly acceptable for regulatory decision-making beyond the already established use of reactive post-approval safety changes based on real-world market surveillance data. EuropaBio members, who develop treatments for the recognised unmet needs relying on evolving science for biotherapeutics, call for the development of guidance on RWE to pave the way for its use in support of regulatory decisions for sponsor-desired post-approval changes, as well as of initial filings where Randomized Controlled Trials are not appropriate.

In particular, EuropaBio, asks the EU regulators to foster collaborations between relevant stakeholders including HTA bodies and develop a RWE framework and guidelines in alignment with international stakeholders, establish real-world data standard and a common data model, as well as for facilitation of RWE pilots. EuropaBio will advocate use of high-quality and fit-for-purpose RWD and RWE in the decision-making process in the context of new approvals, where clinical trials are not feasible, or post-marketing authorisation labelling changes. We will also proactively involve in guideline commenting and multi-stakeholder activities related to RWD/RWE for regulatory purpose.

Download the file below to read the full Position Paper.