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 defined 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’.
A single dataset may not strictly meet the definition of big data but, when pooled or linked with other datasets, they become sufficiently large or complex to analyse to assume the characteristics of big data. Sources include real-world data (such as electronic health records, insurance claims data and data from patient registries), genomics, clinical trials, spontaneous adverse drug reaction reports, social media and wearable devices.
Medicines regulators will increasingly use insights derived from big data to assess the benefit-risk of medicines across their lifecycle.
The joint HMA/EMA Big Data Steering Group advises the EMA Management Board and HMA on prioritisation and planning of actions to implement the ten priority recommendations in the Big Data Task Force final report (phase two).
The Steering Group began its work in May 2020. Its workplan sets out actions for delivery in 2020 and 2021:
The workplan aims to increase the utility of big data in regulation, from data quality through study methods to assessment and decision-making. It is patient-focused and guided by advances in science and technology.
Stakeholders discussed the workplan and its implementation at a virtual multi-stakeholder forum in late 2020.
|Implementation of the workplan will be flexible and certain actions may be re-scheduled, since the European medicines regulatory network has to prioritise the unprecedented public health challenge of the Coronavirus disease (COVID-19) pandemic.|
The Big Data Steering Group is co-chaired by Nikolai Constantin Brun, Director of Medical Evaluation & Biostatistics at the Danish Medicines Agency and Peter Arlett, Head of Data Analytics and Methods at EMA.
For more details, see:
EMA has contracted several institutions to conduct research projects collecting and analysing real-world data from clinical practice to help monitor the safety and effectiveness of medicines.
For research projects related to COVID-19, see Treatments and vaccines for COVID-19: post-authorisation
The HMA/EMA Task Force on Big Data operated from 2017 until December 2019 to report on the challenges and opportunities posed by big data in medicines regulation. 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.
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.
EMA is preparing dedicated guidance 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.
By July 2020 EMA had gathered 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 September 2020, stakeholders discussed with EMA the key questions concerning the application of the General Data Protection Regulation (GDPR) in the health sector and the secondary use of health data for medicines and public health purposes:
- Workshop on the application of the General Data Protection Regulation (GDPR) in the area of health and Secondary Use of Data for Medicines and Public Health Purposes
- Workshop on the General Data Protection Regulation (GDPR) and secondary use of data for medicines and public health purposes
EMA aims to finalise the guidance in consultation with the European Commission and the European Data Protection Supervisor (EDPS) in the last quarter of 2021. It will take into account stakeholder input and guidance from the EDPS on the processing of health data for research.
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: