Big Data Steering Group’s 2023 report unveils eleven recommendations for real-world evidence integration

The Big Data Steering Group (BDSG) advises the European Medicines Agency (EMA) and the Heads of Medicines Agencies (HMA) on implementing recommendations from the Big Data Task Force, addressing matters related to both human and veterinary medicines. The recently-published BDSG 2023 report provides an overview of the key activities and achievements of the BDSG in 2023 and outlines 11 priority recommendations to advance the integration of real-world evidence (RWE) and big data into regulatory decision-making. The report emphasizes the value of RWE and the integration of big data into regulatory decision-making to support the development of innovative treatments.

The highlights from 2023 and recommendations made in the report include:

1. DARWIN EU®: The DARWIN EU® coordination center, formed in February 2022, has advanced RWE use with 20 data partners providing access to millions of patients across 12 EU member states.
   • Recommendation: Real-world data (RWD) analysis complements randomized controlled trials, ensuring timely and reliable evidence for medicine development, authorization, and supervision.
2. Data quality and representativeness: The EU Data Quality Framework, developed in 2023, sets criteria for a consistent and standardized approach to data quality in medicine regulation. This framework document was informed by a multi-stakeholder workshop, co-organized with TEHDAS.
   • Recommendation: Enhancing data quality informs data selection, aids in interpreting study results, and allows for evaluating the evidentiary value of studies.
3. Data discoverability: In 2022, the first metadata list for real-world data (RWD) sources and studies was published. HMA and EMA plan to roll out catalogues of RWD sources and non-interventional studies in Q1 2024, replacing current databases.
   • Recommendation: Metadata standardization enhances data discoverability, including through a public catalog of real-world datasets, and improves the ability to assess the evidentiary value of non-interventional studies and RWD sources.
4. EU Network skills in Big Data: Launched in December 2023, the Big Data training curriculum includes initial modules on Pharmacoepidemiology and RWE. More modules are expected in the next two years, covering biostatistics and data science.
   • Recommendation: Training contributes to building a skilled workforce capable of providing guidance and interpreting large-scale data.
5. EU Network processes: In 2023, the EMA’s report highlighted the increasing value of RWE, with 58 studies requested or offered. The report emphasized RWE’s role in regulatory decision-making, complementing clinical trials.
   • Recommendation: Insights gained from pilots guide process enhancements, inform guidance development, and establish evidentiary value. This enables rapid, high-quality assessments of medicines, improving decision-making throughout their lifecycle and strengthening readiness for health crises.
6. EU Network capability to analyse Big Data: In 2022, CHMP launched a proof-of-concept (PoC) pilot to assess clinical trial raw data’s benefits. The pilot, covering diverse regulatory procedures, is at the midway point, with a summary due in early 2024. EMA discussed a non-clinical raw data exchange PoC study. BDSG co-developed an AI reflection paper and 2028 workplan. Several agencies collaborated on Clusters of Excellence, and BDSG discussed and learned from EU Commission projects on advanced analytics and RWD use in regulatory decisions and health technology assessment. Scientific Explorer, an AI tool for EU regulators, will enhance knowledge management for core regulatory processes, beginning with Scientific Advice in 2024.
   • Recommendation: Demonstrating raw data’s value and enhancing EU network knowledge are crucial for improving medicine assessment quality and speed.
7. Delivery of expert advice: Established in 2022, the Methodology Working Party (MWP) initiated a 3-year workplan and created the European Specialist Expert Community (ESEC) with 180+ experts. MWP is developing a reflection paper on RWD for non-interventional studies and crafting an RWE guidance roadmap.
   • Recommendation: Expert guidance, covering advanced analytics, RWE, and omics, enhances robust assessment and decision-making within regulatory committees.
8. Governance framework: In 2023,EMRN concluded a review of data governance, endorsing revised mandates for Network Data Board and BDSG, enhancing their roles and addressing evolving policy environments, including EHDS and the Pharmaceutical Strategy.
   • Recommendation: Ethical data governance is essential for secondary healthcare data use. BDSG’s work aims to assist stakeholders in navigating and complying with these principles.
9. International initiatives: International collaboration on RWD/RWE advanced in 2023 with key initiatives, including ICH’s endorsement of a public consultation on harmonizing terminology and principles. The ICMRA COVID-19 RWE Working Group now focuses on RWE in public health emergencies.
   • Recommendation: Collaborating with global partners on standards and guidelines optimizes expertise and reduces stakeholder burden.
10. Stakeholder engagement: Collaborative efforts with external stakeholders persisted throughout 2023, including workshops on novel methodologies and RWD quality. Stakeholder forums, Methodology Working Party meetings, BDSG industry engagements, and the EMA Big Data newsletter promoted ongoing communication.
    • Recommendation: Engaging stakeholders and utilizing their contributions enhance the shift toward data-driven regulation, fostering trust through transparency and effective communication.
11. Veterinary recommendations: The BDSG also provided recommendations related to veterinary aspects.
    • Recommendation: The shared use of data between human and veterinary domains can drive transformative initiatives.