DARWIN EU and the evolution of real-world data regulation in Europe

By Fisentzos Stylianou, PhD

Contact: [email protected]

One of the main healthcare challenges raised by the European Medicines Agency’s (EMA) network strategy to 2025 is the introduction of increasingly complex and targeted therapies to the market1.

With massive amounts of healthcare data being generated, the Heads of Medicines Agencies (HMA) and EMA set up a joint Big Data Task Force (BDTF). The BDTF’s aim is to characterise the big data landscape and outline practical steps for the European medicines regulatory network to optimally use big data, thereby supporting innovation and public health in the European Union (EU)2.

The BDTF recognises that big data, which includes real-world data (RWD) such as electronic health records, registry data, claims data, and pooled clinical trials data, can complement traditional clinical trial data and offer major opportunities to improve evidence-based decision-making for reimbursement and access of novel medicines.

Progress towards evolving data-driven regulation

During phase I of its work, the BDTF identified appropriate sources of big data, expertise across the European regulatory network, the current and future landscape, areas of usability and applicability, and generated a list of 47 core recommendations. Phase II built upon these foundations, condensing the 47 core recommendations into 10 priority recommendations, with a strong emphasis on implementation.

The first priority recommendation on the BDTFs list is to “deliver a sustainable platform to access and analyse healthcare data from across the EU (Data Analysis and Real World Interrogation Network -DARWIN)”, and on February 9th 2022, the EMA announced the establishment of the DARWIN EU project coordination centre. The centre will serve as an infrastructure hub, maintaining a secure EU data platform to develop and manage various data sources, as well as conduct scientific studies requested by medicines regulators, and stakeholders at a later stage too, providing access to and analysis of healthcare data from across the EU3.  

The BDTF’s next steps include guiding the industry’s use of big data, with the goal of establishing an EU framework for data quality and discoverability. Whether it’s data for preauthorisation drug development or on-market performance monitoring, the aim is to point industry and regulators to the best data source to address a specific regulatory use case. The industry may also benefit from the “Big data learnings initiative,” which will track big data submissions and outcomes in order to promote transparency on study methods, as well as feed learnings into reflection papers and guidelines. The full list of BDTF’s priority recommendations can be found in the HMA-EMA Joint Big Data Taskforce Phase II report: ‘Evolving Data-Driven Regulation’.

Opportunities for drug manufacturers

The news of progress towards harmonised RWD standards across the EU will be particularly positive for manufacturers in the rare disease space, where patient populations are often sparse and heterogeneous, making it challenging for traditional clinical trials to convey the benefits of treatment. Payers will have increased confidence in the treatment’s effectiveness, safety, and comparative effectiveness as a result of better understanding real-world treatment patterns and outcomes through RWD. This would potentially allow manufacturers to gain leverage in pricing negotiations, enable managed access agreements, inform the design of value-based contracts, and facilitate labelling revisions.

Potential challenges for drug manufacturers

Andrea Hofmaier, general manager for Novartis Gene Therapy Germany, described the implementation of RWD collection in Germany on Zolgensma as a huge cooperative task. RWD collection for Zolgensma in Germany commenced on February 1st 2022, nearly two years after its launch, with the project encountering various issues due to differences between stakeholders of the G-BA, and G-BA with Novartis. Learning from this experience, Hofmaier advises other manufacturers “to start the dialogue early, have good exchange with the players who have to work together – doctors, patient organisations, health insurers and G-BA”4. Even with harmonised standards across the EU, Hofmaier’s recommendation to engage early with stakeholders will undoubtedly remain relevant, with the BDTF stating that the “key to continual optimisation is effective engagement with stakeholders.” However, given that the process has been considered complex in Germany alone, it will be interesting to see if EU stakeholders can agree on the guidelines for the acceptability of evidence derived from different populations and jurisdictions.

As RWD becomes a driving force for reimbursement, manufacturers will need to think strategically about their long-term market access ambitions. Decisions about the types of investments, infrastructure, technology, personnel, and partnerships required to not only execute, but also sustain, grow, and adapt high-quality RWD collection programmes over a drug’s lifecycle are becoming more crucial and relevant to the commercial success of novel and innovative therapies.

To ensure that RWD is used to its full potential and gain market access, the patient journey in clinical practise should be meticulously mapped and unmet needs assessed. To design optimal clinical trials, pharmaceutical companies should have a deep awareness of key stakeholders and engage them early in the research and design process. Contact P4A to learn more about our services to support with the design of RWD studies, and follow us on LinkedIn to remain up to date on how to seize opportunities in this space.






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