Since March 2020 the EMA has been actively following a strategy named “Regulatory Science to 2025” in order to advance its engagement with regulatory science over the next five to ten years. The EMA are aware that massive amounts of data that could support medicines regulation is going unharnessed.
DARWIN EU, The EMA’s Real-World Data interrogation network
Under the guidance of the HMA/EMA Big Data Task Force and HMA/EMA Big Data Steering Group, large datasets the EMA will evaluate and exploring ways to analyse data sets to reveal patterns, trends, and associations. This will include the opportunity to optimize the utility of RWD and RWE to support regulatory decision making for rare conditions, when conducting traditional randomized clinical trials may not always be feasible.
The EMA’s Real-World Data interrogation network, DARWIN EU, is a large part of the strategy to improve the use of RWD for medicines regulation, with the first pilot studies for a number of use cases across the medicine lifecycle starting in 2022.
DARWIN EU will “deliver real-world evidence from across Europe on diseases, populations and the uses and performance of medicines.” The EMA expects that the range of approved healthcare databases enabling distributed data access via DARWIN EU will evolve and expand over time, and by 2025 it’s expected that the network will increase geographical coverage, scope and regulatory uses of healthcare data.
By 2025 or 2026 DARWIN is expected to be fully operational, undergoing annual reviews to meet the evolving needs of regulators and researchers in multiple ways, by:
- Establishing and expanding a catalogue of observational data sources for use in medicines regulation
- Providing a source of high-quality, validated Real-World Data on the uses, safety and efficacy of medicines
- Addressing specific questions by carrying out high-quality, noninterventional studies, including developing scientific protocols, interrogating relevant data sources and interpreting and reporting study results
Across the Atlantic, the US FDA is also enabling pharma to start making better use of RWD by issuing guidance on data standards for drug submissions that contain RWD such as those released in October 2021.
“The US FDA issued four different guidelines at the end of last year,” says Pfizer’s Sonal Bhatia, “and they’re going to be issuing a lot more. They’re shifting and they’re recognising the need for RWE and looking to unlock its value when they’re getting IP dossiers for marketing approval.”
Rare Diseases Whitepaper
“FAIR” data principles
The EMA, amongst other regulators is placing an increasing emphasis on so-called “FAIR” data principles, whereby data should be findable, accessible, interoperable and reusable, in order to increase the efficiency of how observational research and clinical research feeds into the improvement of evidence-based medicine, something that is particularly important to patients and researchers studying rare diseases.
In June 2022 the HMA/EMA Big Data Steering Group published a list of metadata describing Real-World Data sources available in Europe and observational studies. In the future this list will feed into two EU catalogues; a catalogue of data sources, and a catalogue of observational studies performed on those data sources.
The HMA/EMA Big Data Steering Group are working to prioritize the discoverability of Real-World Data sources and studies to help pharmaceutical companies and researchers identify and use RWD when investigating the use, safety and effectiveness of medicines.
In conclusion, in order for RWD and RWE to be accepted and recognised by regulatory authorities, data quality and standards must be standardized and defined. The EMA’s data interrogation network, DARWIN EU has begun pilot studies in 2022 to explore the applications of RWD in clinical research and expects to have established a catalogue of observational data sources that can provide high-quality, validated real world data for the regulations of medicines by 2025.