Massive amounts of data are generated on a daily basis that could potentially be harnessed to support medicines regulation. The European Medicines Agency (EMA) and Heads of Medicines Agencies (HMA) set up a joint task force to describe the big data landscape from a regulatory perspective and identify practical steps for the European medicines regulatory network to make best use of big data in support of innovation and public health in the European Union (EU).
'Big data' is a widely-used term without a commonly-accepted definition. The HMA/EMA Task Force on Big Data defines big data as ‘extremely large datasets which may be complex, multi-dimensional, unstructured and heterogeneous, which are accumulating rapidly and which may be analysed computationally to reveal patterns, trends, and associations. In general, big data sets require advanced or specialised methods to provide an answer within reliable constraints’.
Medicines regulators will increasingly use insights derived from big data to assess the benefit-risk of medicines across their lifecycle. However, in order to benefit from and make prudent use of the data collected, regulators need a deeper understanding of the data landscape and advanced analytical methods.
The sources of such data include real-world data (such as electronic health records and data from Patient registries), genomics, clinical trials, spontaneous adverse drug reaction reports, social media and wearable devices.
A single dataset may not strictly meet the definition of big data but, when pooled with or linked to other datasets, they become sufficiently large or complex to analyse for the data to assume the characteristics of big data.
The HMA/EMA Task Force on Big Data was established in 2017 to report on the challenges and opportunities posed by big data in medicines regulation, by:
- mapping the sources and characteristics of big data;
- exploring the potential applicability and impact of big data on medicines regulation;
- assessing the need for changes to regulatory guidelines or legislation;
- making recommendations for change to strengthen medicines regulation in support of public health.
It was composed of experienced medicines regulators and data experts appointed by the national competent authorities, EMA and the European Commission (EC).
It carried out its work in two phases. In phase one, the task force:
- reviewed the landscape of big data from a regulatory perspective and identified opportunities for improvements in the operation of medicines regulation;
- performed online surveys of national regulatory agencies and the pharmaceutical industry on perspectives, expertise and challenges. This helped develop an understanding of the challenges and the current state of expertise in the regulatory network.
In phase two, the task force made practical recommendations to inform strategic decision-making and planning by the HMA and EMA and to contribute to the European medicines regulatory network's work on developing a five-year EU Network Strategy to 2025.
For more information, see HMA/EMA Task Force on Big Data.
In January 2020, the HMA/EMA Task Force on Big Data published its final report (phase two) containing practical recommendations on how the European medicines regulatory network could make best use of big data by evolving its approach to data use and evidence generation in support of innovation and public health .
It identifies ten priority actions and practical steps to implement them:
- HMA/EMA Joint Task Force on Big Data - Final (Phase II) report - Evolving data-driven regulation
- Summary of ten priority recommendations
The European medicines regulatory network is now considering how to implement the task force's recommendations, in consultation with the European Commission.
In February 2019, the task force published an interim report (phase one) providing a comprehensive summary of various data sources and setting out recommendations for understanding the acceptability of evidence derived from big data in support of the evaluation and supervision of medicines by regulators:
This takes into account the analyses of the task force's subgroups:
- Clinical trial and imaging subgroup report
- Observational data subgroup report
- Genomics subgroup report
- Spontaneous adverse drug reactions subgroup report
- Data analytics subgroup report
- Bioanalytical -omics subgroup report
- Social media and mobile health data subgroup report
- Survey design and analysis
Update: EMA is preparing a question-and-answer guidance document on the impact of EU data protection legislation on the secondary use of health data in support of the development, evaluation and supervision of medicines.
The aim is to help medicine developers, data providers and research bodies comply with EU data protection rules, and to help patients and consumers understand their rights and the existing safeguards to protect personal data.
Secondary use of data refers to the use of data for a different purpose than the one for which it was originally collected. It typically involves the use of electronic health records, health insurance claims data, registry data or drug consumption data for medicines research and public health purposes.
The guidance will cover various operational scenarios, including the development of medicines, the evaluation of marketing authorisation applications and post-authorisation safety monitoring.
As a first step, EMA will gather input from patients and consumers as data contributors as well as from medicines developers, research-performing and research-supporting infrastructures and other data providers (e.g. payers of healthcare) in the second quarter of 2020.
Based on the input received, EMA will draft the guidance in consultation with the European Commission and the European Data Protection Supervisor.
The Agency will launch a targeted stakeholder consultation on the draft guidance in the fourth quarter of 2020. It aims to publish the finalised guidance in the first half of 2021.
Ensuring that personal data are managed and analysed within a secure and ethical governance framework in compliance with EU data protection legislation is one of the recommended priorities of the HMA/EMA Task Force on Big Data.
EU data protection legislation includes:
In May 2018, the task force held a workshop to inform thinking on big data and related challenges, which fed into its recommendations published for consultation in February 2019:
For more information, see:
- Heads of Medicines Agencies (HMA) / European Medicines Agency (EMA) Joint Big Data Task Force meeting: identifying solutions for big data challenges (04/05/2018)
EMA also held a Workshop on identifying opportunities for 'big data' in medicines development and regulatory science in November 2016.