04 February 2020 - Authored by:Benjamin Scrace
A joint task force of the European Medicines Agency (EMA) and Heads of Medicines Agencies (HMA) has proposed a new approach to the use of “big data” in EU medicines regulation.
The report, from the Big Data Task Force (BDTF) (accessed here), contributes to the EMA and HMA’s “EU Network Strategy” to 2025 and shows a willingness of – or perhaps a growing need for – EU regulatory bodies to develop a clear approach to big data analytics in clinical trials.
Acknowledging the EU regulatory network’s limited expertise in big data and the need for quality data sources, the report calls on the EMA and HMA to develop greater capacity and capability in response to analytical technologies using big data, and encourages greater stakeholder engagement between patients, healthcare professionals, industry, regulators and EU agencies.
The BDTF is composed of EU medicines regulators, national competent authorities, and the European Commission. The recent report follows an initial review of the use of big data in medicines regulation published in February 2019 (accessed here).
The report identifies ten priority recommendations (further outlined here):
- Deliver a sustainable platform (DARWIN) to access and analyse healthcare data from across the EU.
- Establish an EU framework for data quality and representativeness.
- Enable data discoverability.
- Develop EU regulatory skills in big data.
- Strengthen EU regulatory processes for big data submissions.
- Build EU regulatory capability to analyse big data.
- Modernise the delivery of expert advice.
- Ensure data are managed and analysed within a secure and ethical governance framework.
- Collaborate with international initiatives on big data.
- Create an EU big data “stakeholder implementation forum”.
The most ambitious recommendation, and the top priority, is the proposal for a “Data Analysis and Real World Interrogation Network” (or DARWIN) – an EU platform to provide the European medicines regulatory network with access to real-world EU healthcare data.
Noting the increase in digital capture of healthcare data, BDTF recommends the creation of a network of EU databases holding real-world healthcare records that could be accessed and analysed by EU regulators. Analysis of this data would be used to inform regulatory decision-making, for example, to support product development and the authorisation of new medicines, or to monitor the effects and performance of medicines on the market (pharmacovigilance).
The next step is to build stakeholder support and a business case in order to launch the platform by 2023. As a result, the BDTF report does not provide practical, operational or technical details for DARWIN – all of which are likely to present regulatory as well as financial challenges.
The preliminary outline in the report acknowledges that (in addition to initial funding of EUR 30-50million) DARWIN would require annual funding of approximately EUR 10-20million. The report suggests this could be covered via a new dedicated EMA fee, though this would require a change to current EMA fees regulation.
Data quality & discoverability
Several of the recommendations acknowledge the need for EU regulators to access, and assess the quality of, appropriate data sources.
The BDTF therefore proposes new guidelines and data quality metrics for EU regulators to assess the extent to which they can rely on novel data sources when making regulatory decisions. The guidelines envisage the expansion of qualification advice when establishing database certification, and the expansion of criteria to assess the reliability of device-based diagnostic or in vitro diagnostics. The BDTF hopes that new guidelines would promote the uptake of electronic health records, electronic registries, and geonomics.
The BDTF also calls for the expansion of scope and utility of the ENCePP database (the European Network of Centres for Pharmacoepidemiology and Pharmacovigilance), and further engagement by EU regulators with EU and international initiatives (such as the Genomic Alliance for Genomic Health) to improve the discoverability of healthcare data sources for regulatory use.
Big data expertise
The recommendations acknowledge the EU regulators’ lack of expertise in the analytical approaches enabled by big data. The BDTF therefore proposes high-level training on big data for EU regulators and encourages external placements for regulators as well as placements for researchers within regulatory science centres.
Ethical governance & stakeholder engagement
The report recognises that the use of big data should be subject to a secure and ethical governance framework, built through active dialogue with stakeholders. The BDTF proposes an ethical advisory committee to advise on the ethical aspects of the regulatory use of big data, and a “stakeholder implementation forum”, to obtain patient representation and encourage communication and discussion between patients, healthcare professionals, industry, device regulators and technology companies.
The report and the implementation of its recommendations are being considered by the European Commission.