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TOPRA Symposium 2023: Big data and regulatory decision-making

Posted on the 6th December 2023

TOPRA Big Data

Rachael O’Kane from G&L continues her discussion of the big topics being explored at the recent TOPRA Symposium.

Part two: Real-world evidence: what role does big data play in regulatory decision-making?

Andrej Segec, the European Medicines Agency (EMA)’s Scientific Administrator, initiated the session with a discussion on the scale-up of real-world evidence (RWE) generation for regulators in Europe.

RWE relates to information derived from the analysis of real-world data (RWD), while the EMA defines RWD as routinely collected data relating to patient health status or the delivery of healthcare from sources other than traditional clinical trials.

A key element of the European medicines regulatory network (EMRN) strategy is the Data Analysis and Real-World Interrogation Network (DARWIN EU®).

Launched in 2022, DARWIN EU® is a federated network of data, expertise and services that supports better decision-making throughout the product lifecycle by generating reliable evidence from real-world healthcare data1.

It is anticipated that the value of RWD will have been established across the spectrum of regulatory use cases by 2025, with DARWIN EU® being fully operational by 2025 and delivering 150 RWE studies per year.

To achieve this deadline, DARWIN EU® is currently focusing on its scale-up of data partners, studies and pilot use cases, and the development of standard analytical pipelines.

Dr Kimberly Smith, the U.S. Food and Drug Administration (FDA)’s Medical Officer, provided the US perspective.

FDA real-world definitions from the 2018 framework are as follows:

  • Real-world data (RWD) are data relating to patient health status and/or the delivery of health care routinely collected from a variety of sources
  • Real-world evidence (RWE) is the clinical evidence about the usage and potential benefits or risks of a medical product derived from analysis of RWD

The FDA created a programme for evaluating the potential use of RWE in supporting the approval of new indications for drugs already approved under section 505(c) of the FD&C Act, and in supporting post-approval study requirements.

Further to this, the FDA issued a draft framework in December 2018 outlining sources of RWE, as well as associated challenges and opportunities. This framework consists of guidance for industry on RWD/RWE topics published and in development, and the US effectiveness standard remains unchanged2.

Key considerations of the 2018 framework include:

  • Whether the RWD are fit for use
  • Whether the trial or study design used to generate RWE can provide adequate scientific evidence to answer, or help answer, the regulatory question
  • Whether the study conduct meets FDA regulatory requirements

As outlined in the 2018 framework, the FDA’s RWE programme is advancing. The FDA is also issuing guidance and supporting demonstration projects to help sponsors and other stakeholders better understand how RWE can support regulatory decision-making.



Rachael O’Kane is a Manager, Regulatory Affairs at G&L.

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