Updated on 22 March 2024:
'Artificial Intelligence' section

'Big data' is a widely-used term without a commonly-accepted definition. The HMA-EMA Big Data Task Force 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.

HMA-EMA Big Data Steering Group

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 recommendations in the Big Data Task Force final report.

Priority recommendations of the HMA-EMA joint Big Data Task Force

HMA-EMA Joint Big Data Taskforce Phase II report: Evolving data-driven regulation

The Steering Group began its work in May 2020. It is co-chaired by Jeppe Larsen, Director of Medical Devices Division at the  Danish Medicines Agency and Peter Arlett, Head of Data Analytics and Methods at EMA.

The Steering Group reviews the workplan annually to cover any new emerging topics. It last updated the workplan in July 2023.

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. Implementation of the workplan will be flexible and certain actions may be re-scheduled.

 

Big data workplan 2022-2025

 

For more information, see:

Big Data Steering Group (BDSG): 2023 report

Workplan 2023-2025: HMA-EMA joint Big Data Steering Group

Clusters of Excellence Discussion Paper

Workplan 2022-2025: HMA-EMA joint Big Data Steering Group

Big Data Steering Group (BDSG): 2022 report

Big Data Steering Group (BDSG): 2021 report

Workplan 2021-2023: HMA-EMA joint Big Data Steering Group

Workplan 2020: HMA-EMA joint Big Data Steering Group

Big Data Steering Group (BDSG): 2020 report

Mandate: HMA-EMA joint Big Data Steering Group

Membership list: HMA-EMA joint Big Data Steering Group

Big Data Steering Group meeting minutes

Final Minutes – HMA-EMA joint Big Data Steering Group teleconference - 30 January 2024

Final Minutes – HMA-EMA joint Big Data Steering Group teleconference - 5 December 2023

Final Minutes – HMA-EMA joint Big Data Steering Group teleconference - 30 October 2023

Final Minutes – HMA-EMA joint Big Data Steering Group teleconference - 29 September 2023

Big data training curriculum

The Big data training curriculum is a collection of training modules aimed to help European medicines regulatory network staff develop expertise in integrating big data analysis into decision-making processes related to medicine regulation. 

The first two modules, on real-world evidence and real-world data, are available for eligible users via the EU Network Training Centre platform. The Big data training curriculum also covers biostatistics, data science and other training topics which will be added over time.

EMA's Methodology Working Party oversees its development.

The Big data curriculum is part of the Big data workplan for 2023-2025 and the European medicines agencies network strategy to 2025. For more information, see: 

Workplan 2023-2025: HMA-EMA joint Big Data Steering Group

HMA-EMA catalogues of real-world data sources and studies

Two online catalogues are available from HMA and EMA, one for real-world data sources and one for real-world data studies:

Real-world data are observational data stored for instance in electronic health records and disease registries.

Making greater use of these can improve the evidence base for benefit-risk decisions and bringing medicines to patients.

The catalogues serve to:

  • help regulators, researchers and pharmaceutical companies identify the most suitable data sources to address specific research questions;
  • support the assessment of study protocols and results;
  • promote transparency;
  • encourage the use of good practices;
  • build trust in research based on real-world data. 

They enhance and replace two databases previously maintained by EMA:

CatalogueDiscontinued database 
Real-word data sources European Network of Centres for Pharmacoepidemiology and Pharmacovigilance (ENCePP) catalogue
Real-world data studiesEuropean Union electronic register of post-authorisation studies (EU PAS Register)

The catalogues use an agreed list of metadata to describe and connect data sources to studies, using ‘FAIR’ (Findable, Accessible, Interoperable and Reusable) data principles. 

The HMA-EMA Big Data Steering Group reviews the list of metadata annually, based on feedback from catalogue users. 

A draft good practice guide is also available to help users of the catalogues.

By end of June 2024, EMA intends to publish a revised version addressing feedback from a public consultation. Separate sections will cover using the catalogues to identify and assess the suitability of data sources, and definitions and descriptions of the metadata list.

All European data holders, marketing authorisation holders, networks, researchers and institutions interested in having their data used for medicines regulation, or mandated by policy on non-interventional post-authorisation safety studies (PASS), are encouraged to use these catalogues.

List of metadata for Real World Data catalogues

Good practice guide for the use of the Metadata Catalogue of Real-World Data Sources

Improving data discoverability is a priority of the HMA-EMA joint Big Data Task Force final report (phase two), reflected in the European medicines agencies network strategy to 2025 and implemented through the joint HMA-EMA Big Data Steering Group workplan. 

Data discoverability refers to the identification and use of real-word data sources and studies.

For more information: 

Data quality framework for EU medicine regulation

The final version of the data quality framework for EU medicines regulation is available below, following a public consultation.

This guideline sets out the criteria for a more consistent and standardised approach to the quality of data used in medicine regulation to support benefit-risk decisions.

Data Quality Framework for EU medicines regulation

It is meant to:

  • help identify, define and further develop data quality assessment procedures and recommendations for current and novel data types;
  • support pharmaceutical companies and other stakeholders in selecting data sources for their studies;
  • ensure the trust of patients and healthcare professionals in data-driven regulatory decision-making.

The data quality framework was co-produced by EMA, the Heads of Medicines Agencies (HMA) and the Joint Action Towards the European Health Data Space (TEHDAS).

Establishing this framework is a key element in the HMA/EMA joint Big Data Steering Group workplan. EMA will work with stakeholders to use the framework’s concepts and develop practical guidelines for assessing the quality level of data. These will initially focus on the domains of real-world data and adverse drug reactions.

Data standardisation strategy

The European medicines regulatory network's data standardisation strategy sets out principles to guide the definition, adoption and implementation of international data standards by the network.

It aims to:

  • enable quicker uptake of international data standards across the EU;
  • improve data quality;
  • enable data linkage and data analysis to support medicine regulation.

The strategy is a key deliverable of the Big Data Steering Group workplan.

EMA and HMA published the strategy in December 2021 and will maintain it over time to reflect any changing priorities or new requirements.

European Medicines Regulatory Network Data Standardisation Strategy

Research projects

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

Project titleChanges in alternative treatments for pain and cough in children after introduction of risk minimisation measures for codeine
Objectives

This project aims to:

It was commissioned under the PRAC strategy on measuring the impact of pharmacovigilance activities, which includes the effectiveness of product specific risk-minimisation.

Timelines
  • Start of project: December 2019
  • Final delivery: July 2021 
Deliverables 

For more information on protocol and study results, please visit EU PAS Register.

The study registration number is EUPAS32021.

FrameworkEuropean Medicines Regulatory Network (EMRN)

Project titleAn European multicentre drug utilisation study of the impact of regulatory measures on prescribing of codeine for pain in children
Objectives

This project aims to:

  • Determine trends of codeine prescribing before and after Article 31 referral to measure the impact of risk minimisation restricting the use of codeine for treatment of pain in children
  • Assess to what extent the data collected can be used for comparisons between Member States 

It was commissioned under the PRAC strategy on measuring the impact of pharmacovigilance activities, which includes the effectiveness of product specific risk-minimisation.

Timelines
  • Start of project: November 2015
  • Final study report: April 2018
Deliverables

For more information on protocol and study results, please visit EU PAS Register.

The study registration number is EUPAS17093.

FrameworkEuropean Medicines Regulatory Network (EMRN)

Project titleImpact of EU label changes and regulatory communication on SARS-CoV-2 adenovirus vector vaccines in context of thrombosis with thrombocytopenia syndrome (TTS): risk awareness and adherence
Objectives

This study aims to determine:

  • Healthcare professional awareness and adherence to revised product information for SARS-CoV-2 adenovirus vector vaccines
  • The extent of change in patient and healthcare professional attitude towards national vaccination campaigns
  • How regulatory actions for TTS have changed national vaccination policy in terms of risk and age group prioritisation, and recommendations for second vaccine dose and other SARS-CoV-2 vaccines

It was commissioned under the PRAC strategy on measuring the impact of pharmacovigilance activities, which includes the effectiveness of product specific risk-minimisation.

Timelines
  • Start of project: November 2021
  • First deliverables: December 2021
  • Final delivery: March 2023
Deliverables

For more information on protocol and study results, please visit EU PAS Register.

The study registration number is EUPAS44970.

ContractorEU PE&PV Research Network, coordinated by Utrecht University: Pharmacoepidemiology and Clinical Pharmacology - Universiteit Utrecht (uu.nl)

Project titleImpact of EU label changes for systemic diclofenac products: post-referral prescribing trends
Objectives

This study aims to determine:

  • How medicines containing diclofenac are prescribed before and after the Article 31 referral
  • Prescriber compliance with product information warnings about cardiovascular risk factors
  • Prescriber compliance with the product information recommendation to avoid diclofenac in patients with certain cardiovascular diseases
  • Drug use and prescribing patterns for alternative treatments in patients who previously used diclofenac

It was commissioned under the PRAC strategy on measuring the impact of pharmacovigilance activities, which includes the effectiveness of product specific risk-minimisation.

Timelines
  • Start of project: September 2017
  • First deliverables: November 2017
  • Final delivery: March 2019
Deliverables

For more information on protocol and study results, please visit EU PAS Register.

The study registration number is EUPAS24089.

ContractorUniversity of Dundee

Project titleImplementation of EU risk minimisation measures for medicinal products in clinical guidelines
Objectives

This study aims to:

  • Describe key stakeholders, processes, roles and responsibilities for updating clinical guidelines on pharmacological treatment in EU Member States
  • Determine how medicinal product specific risk-minimisation measures for five disease priority areas have been integrated in clinical guidelines
  • Provide recommendations for engagement with responsible bodies to strengthen the role of clinical guidelines for implementing risk-minimisation

It was commissioned under the PRAC strategy on measuring the impact of pharmacovigilance activities, which includes the effectiveness of product specific risk-minimisation.

Timelines
  • Start of project: April 2022
  • First deliverables: June 2022
  • Final delivery: November 2023  
Deliverables

For more information on protocol and study results, please visit EU PAS Register.

The study registration number is EUPAS47588.

ContractorEU PE&PV Research Network, coordinated by Utrecht University: Pharmacoepidemiology and Clinical Pharmacology - Universiteit Utrecht (uu.nl)

Project title

Study of impact of EU label changes for fluoroquinolone-containing medicines for systemic and inhalation use: post-referral prescribing patterns

Objectives

This study aims to determine:

  • How fluoroquinolones are prescribed and used by patients before and after the Article 31 referral.
  • Prescriber compliance with product information warnings about tendinitis, tendon rupture and aortic aneurism/dissection
  • Prescriber compliance with recommendation to avoid concomitant use of corticosteroids and fluoroquinolones in patients at risk of aortic aneurism/dissection
  • Drug use and prescribing patterns for alternative treatments in patients who previously used fluoroquinolones

It was commissioned under the PRAC strategy on measuring the impact of pharmacovigilance activities, which includes the effectiveness of product specific risk-minimisation.

Timelines
  • Start of project: May 2020
  • First deliverables: July 2020
  • Final delivery: November 2021 
Deliverables

For more information on protocol and study results, please visit EU PAS Register.

The study registration number is EUPAS37856.

ContractorIQVIA Ltd

Project titleImpact of EU label changes for hydroxyzine products: post-referral prescribing trends
Objectives

This study aims to determine:

  • How medicines containing hydroxyzine are prescribed and used by patients before and after the Article 31 referral
  • Prescriber compliance with the product information daily dose restriction and warnings about use in the elderly
  • Prescriber compliance with the product information recommendation to avoid hydroxyzine in patients at risk of QT prolongation
  • Use and prescribing patterns for alternative treatments in patients who previously used hydroxyzine

It was commissioned under the PRAC strategy on measuring the impact of pharmacovigilance activities, which includes the effectiveness of product specific risk-minimisation.

Timelines
  • Start of project: March 2018
  • First deliverables: May 2018
  • Final delivery: September 2019
Deliverables

For more information on protocol and study results, please visit EU PAS Register.

The study registration number is EUPAS26363.

ContractorUniversity of Dundee

Project titleImpact of EU label changes for medicinal products containing methotrexate for weekly administration: risk awareness and adherence
Objectives

This study aims to determine the extent of awareness and knowledge among:

  • Prescribers, of the risk of methotrexate dosing errors and adherence to risk minimisation measures following the Article 31 referral
  • Pharmacists, of the patient card and outer packaging reminder
  • Patients, of the risk minimisation measures to avoid incorrect administration schedules

It was commissioned under the PRAC strategy on measuring the impact of pharmacovigilance activities, which includes the effectiveness of product specific risk-minimisation.

Timelines
  • Start of project: May 2021
  • First deliverables: June 2021
  • Final delivery: November 2022  
Deliverables 

For more information on protocol and study results, please visit EU PAS Register.

The study registration number is EUPAS44827.

ContractorIQVIA Ltd

Project titleStudy of exposure and use patterns of alternatives to ranitidine-containing medicines in patients treated with ranitidine
Objectives

This study has the following objectives:

  • Determine how ranitidine-containing medicines have been prescribed and used by patients before and after the Article 31 referral
  • Describe patterns of patients switching from ranitidine-containing medicines to alternative treatments or stopping these medicines without switching to alternative treatments
  • Describe use patterns in new patients starting with ranitidine prior to its suspension in the EU

It was commissioned under the PRAC strategy on measuring the impact of pharmacovigilance activities, which includes the effectiveness of product specific risk-minimisation.

Timelines
  • Start of project: October 2020
  • First deliverables: January 2021
  • Final delivery: April 2023 
Deliverables 

For more information on protocol and study results, please visit EU PAS Register.

The study registration number is EUPAS44548.

ContractorIQVIA Ltd

Project titleStudy of impact of EU label changes and revised pregnancy prevention programme (PPP) for oral retinoid-containing medicines: risk awareness and adherence
Objectives

This study aims to determine:

  • The extent of awareness of the PPP revised with Article 31 referral and the risk of teratogenic effects in women of childbearing potential and pregnant women exposed to oral retinoid-containing medicines
  • The impact of recommendations from regulatory authorities on knowledge, attitudes and practices of patients, prescribers, midwives and pharmacists
  • The extent of adherence to the revised PPP and risk minimisation measures for oral retinoid-containing medicines for use in women of childbearing potential

It was commissioned under the PRAC strategy on measuring the impact of pharmacovigilance activities, which includes the effectiveness of product specific risk-minimisation.

Timelines
  • Start of project: February 2019
  • First deliverables: April 2019
  • Final delivery: February 2021 
Deliverables

For more information on protocol and study results, please visit EU PAS Register.

The study registration number is EUPAS32408.

ContractorEU PE&PV Research Network, coordinated by Utrecht University: Pharmacoepidemiology and Clinical Pharmacology - Universiteit Utrecht (uu.nl)

Project titleStudy of impact of EU label changes and revised pregnancy prevention programme for oral retinoid-containing medicines: use and prescribing patterns
Objectives

This study aims to determine:

  • How oral retinoid-containing medicines are prescribed and used in women of childbearing potential before and after Article 31 referral
  • Whether significant changes in prescribing patterns have occurred
  • Prescriber compliance with recommendations included in the product information for oral retinoids
  • Patient use of effective contraception in compliance with the revised product information
  • Drug prescription and use patterns in women where oral retinoids were or would have been previously prescribed
  • The effectiveness of the 2018 risk minimisation measures

It was commissioned under the PRAC strategy on measuring the impact of pharmacovigilance activities, which includes the effectiveness of product specific risk-minimisation.

Timelines
  • Start of project: February 2019
  • First deliverables: April 2019
  • Final delivery: June 2022 
Deliverables

For more information on protocol and study results, please visit EU PAS Register.

The study registration number is EUPAS31095.

ContractorEU PE&PV Research Network, coordinated by Utrecht University: Pharmacoepidemiology and Clinical Pharmacology - Universiteit Utrecht (uu.nl)

Project titleSingle-arm studies with historical controls for cancer drug development
Objectives
  • Simulate single-arm trials using completed randomised-controlled clinical trials as a basis, to construct historical comparisons between patient groups using different data sources such as clinical trials and patient registries
  • Assess the impact of non-randomised trials in a clinical development programme
  • Develop recommendations on when and how to use historical controls to help understand the natural history of the disease
Timelines
  • Start of project: November 2020
  • First deliverables: February 2021
  • Final delivery:  October 2021 
Deliverables 

For more information on protocol and study results, please visit EU PAS Register.

The study registration number is EUPAS43709.

ContractorIQVIA Ltd

Project titleStrengthening use of real-world data in medicines development - metadata for data discoverability and study replicability
Objectives
  • Define a set of metadata describing key characteristics of real-world data sources that can support regulatory needs
  • Conduct a stakeholder’ consultation on the metadata to be collected
  • Define criteria to identify real-world data sources to include in the project
  • Collect metadata from at least ten data sources using a pre-defined process
  • Deliver a proof-of-concept tool to access and visualise the metadata
  • Draft a good practice guide on using the defined metadata to identify suitable real-world data sources for a specific study and to assess the evidentiary value of study results
Timelines
  • Start of project: November 2020
  • First deliverables: December 2020
  • Final delivery: December 2021 
Deliverables

For more information on protocol and study results, please visit EU PAS Register.

The study registration number is EUPAS39322.

ContractorResearch Triangle Institute d/b/a RTI Health Solutions

Project titleTrade-offs between benefits and harms of drugs - a stated preference study with adult cancer patients in Europe
Objectives
  • Identify and describe the preferences of cancer patients about benefits and harms of cancer drugs, and explore patient characteristics associated with certain preferences
  • Establish a database containing the results of the study to contribute to the knowledge of cancer patient preferences
Timelines
  • Start of project: November 2020
  • First deliverables: February 2021
  • Final delivery: May 2022 
Deliverables 

For more information on protocol and study results, please visit EU PAS Register.

The study registration number is EUPAS41433.

ContractorIQVIA Ltd

Project titleStudy of impact of EU label changes and pregnancy prevention programme for valproate-containing medicines and related substances: risk awareness and adherence
Objectives

This study aims to determine the extent of:

  • Awareness of the risk of adverse effects and neurodevelopmental disorders to children of women exposed to valproate and related substances during pregnancy, following the introduction of the pregnancy prevention programme (PPP) with the Article 31 referral
  • Influence of recommendations from regulatory authorities on knowledge, attitudes and practices of patients, prescribers, midwives and pharmacists
  • Knowledge and adherence to the PPP and risk-minimisation measures for valproate-containing medicines and related substances for use in women of childbearing potential

It was commissioned under the PRAC strategy on measuring the impact of pharmacovigilance activities, which includes the effectiveness of product specific risk-minimisation.

Timelines
  • Start of project: February 2019
  • First deliverables: April 2019
  • Final delivery: February 2021  
Deliverables

For more information on protocol and study results, please visit EU PAS Register.

The study registration number is EUPAS32405.

ContractorEU PE&PV Research Network, coordinated by Utrecht University: Pharmacoepidemiology and Clinical Pharmacology - Universiteit Utrecht (uu.nl)

Project titleStudy of impact of EU label changes and pregnancy prevention programme for valproate-containing medicines and related substances: post-referral use and prescribing patterns
Objectives

This study aims to determine:

  • How valproate-containing medicines and related substances are prescribed and used in women of childbearing potential before and after the Article 31 referral
  • Prescriber compliance with the pregnancy prevention programme (PPP) and recommendations for patient counseling and pregnancy testing included in the revised product information for valproate-containing medicines
  • Patient use of effective contraception in compliance with the PPP and revised product information
  • Drug prescription and use patterns for alternative medicines in women where valproate was or would have been previously prescribed
  • The effectiveness of the 2018 risk minimisation measures and PPP

It was commissioned under the PRAC strategy on measuring the impact of pharmacovigilance activities, which includes the effectiveness of product specific risk-minimisation.

Timelines
  • Start of project: February 2019
  • First deliverables: April 2019
  • Final delivery: June 2022  
Deliverables

For more information on protocol and study results, please visit EU PAS Register.

The study registration number is EUPAS31001.

ContractorEU PE&PV Research Network, coordinated by Utrecht University: Pharmacoepidemiology and Clinical Pharmacology - Universiteit Utrecht (uu.nl)

Darwin EU

EMA is establishing a coordination centre to provide timely and reliable evidence on the use, safety and effectiveness of medicines for human use, including vaccines, from real world healthcare databases across the EU. 

This capability is called the Data Analysis and Real World Interrogation Network (DARWIN EU®). For more information, see:

Pilot on using raw data in medicine evaluation

Through a proof-of-concept pilot, selected applicants can submit 'raw data' to EMA as part of their initial and post-authorisation marketing authorisation applications. 

Information about the raw data proof-of-concept pilot for industry

Application of EMA’s transparency principles to the raw data proof-of-concept pilot

Raw data refers to individual patient data from clinical trials. These include:

  • clinical laboratory results;
  • imaging data;
  • patient medical charts.

Currently, applicants are submitting data in an aggregated format as clinical summaries or as individual patient data in PDF listings. This can hinder data analysis and slow down the evaluation process.

In contrast, raw data are stored in electronic structured format. This enables regulators to more easily visualise and analyse the data if needed.

The pilot aims to assess whether using raw data can help speed up and improve the medicine-evaluation process. The goal of this is to allow patients faster and better informed access to innovative medicines.

EMA launched the pilot in July 2022.

The pilot currently accepts expressions of interest for procedures with a submission date up to Q1 2024.

It will run for up to two years and include approximately ten regulatory procedures submitted to EMA from September 2022.

For any queries and to apply to take part in the pilot, write to rawdatapilot@ema.europa.eu

The pilot is a key activity under the priority recommendations of the HMA-EMA Big Data Task Force. It refers to the priority of building network capability to analyse data.

For more information, see: 

Presentation - 5.2 Pilot on raw data analysis - E Psarelli (EMA) and A Bradshaw (AE)

Support for pharmaceutical companies

Further information is available to support pharmaceutical companies with their participation in EMA's raw data pilot. 

The documents include:

  • a questions and answers document on the raw data pilot;
  • a participation letter to confirm pilot participation for a specific regulatory procedure;
  • a cover letter for pilot participants to attach to their data packages.

Questions and Answers about the raw data proof-of-concept pilot for industry

Pilot participation letter

Raw data submission cover letter template

Data protection

For information on data protection in the raw data proof-of-concept pilot, see:

European Medicines Agency’s data protection notice for the Raw data proof-of-concept pilot

Records of data processing activity for the raw data proof-of-concept pilot

Big Data Highlights

banner_image_big_data_newsletter.png

Big Data Highlights provides a quarterly update on the implementation of the HMA-EMA Big Data Steering Group workplan.

EMA publishes Big Data Highlights every three months. 

Issues from June 2023 (Issue 6) onwards are available at the link below: 

Previous issues from 2022 are available on EMA's website in PDF format: 

Use the link below to receive Big Data Highlights by email: 

Work of the former HMA-EMA Big Data Task Force

The HMA-EMA Big Data Task Force 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.

The HMA/EMA Task Force on Big Data published its final report in January 2020. It contained 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 identified ten priority recommendations and practical steps to implement them:

HMA-EMA Joint Big Data Taskforce Phase II report: Evolving data-driven regulation

Priority recommendations of the HMA-EMA joint Big Data Task Force

    The task force published an interim report in February 2019. It provided a comprehensive summary of various data sources and set out recommendations for understanding the acceptability of evidence derived from big data in support of the evaluation and supervision of medicines by regulators:

    HMA-EMA Joint Task Force on big data: Summary report

     

    This took into account the analyses of the task force's subgroups:

    Clinical Trial and Imaging - Subgroup report

    Observational data (Real World Data) - Subgroup report

    Genomics, Genetics, Transcriptomics and Epigenetics - Subgroup report

    Spontaneous Adverse Drug Reactions - Subgroup report

    Data Analytics - Subgroup report

    Bioanalytical Omics - Subgroup report

    Social Media and M-Health Data - Subgroup report

    HMA/EMA Joint Task Force on big data – Surveys results

    The task force was composed of experienced medicines regulators and data experts appointed by the national competent authorities, EMA and the European Commission (EC). For more information, see HMA-EMA Big Data Task Force

    Meetings and workshops

    For information on related meetings and workshops, see:

    Veterinary big data

    EMA and HMA established the veterinary big data initiative to explore the use of new digital technologies in key veterinary regulatory activities.

    It takes account of the increasing amount of data generated via new digital systems put in place to implement the Veterinary Medicinal Products Regulation.

    A European veterinary big data strategy sets out how the European medicines regulatory network intends to implement this initiative:

    European veterinary big data strategy 2022-2027

    For more information:

    Data protection

    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:

    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 Big Data Task Force.

    EU data protection legislation includes:

    • Regulation (EU) 2016/679, known as the General Data Protection Regulation (GDPR), which applies to private and public entities in the Member States;
    • Regulation (EU) 2018/1725, known as the EU Data Protection Regulation (EUDPR), which applies to all EU institutions and bodies.

    Artificial Intelligence (AI)

    An EMA draft reflection paper on the use of artificial intelligence (AI) in the medicinal product lifecycle is available, to support the regulation of human and veterinary medicines in a world where pharmaceutical companies increasingly use AI-powered tools in the research, development and monitoring of medicines.

    The reflection paper outlines the scientific principles relevant for regulatory evaluation of medicines developed or monitored with the help of AI.

    An AI workplan for the European medicines regulatory network is also available, to guide the use of AI in medicines regulation in Europe to 2028.

    Multi-annual artificial intelligence workplan 2023-2028: HMA-EMA joint Big Data Steering Group

    It identifies actions for regulators in four key areas:

    • Guidance, policy and product support - delivering guidance on the use of AI throughout the medicine lifecycle
    • AI tools and technology - providing frameworks for the use of AI tools
    • Collaboration and training - developing capacity for the use of AI technology
    • Experimentation - ensuring a structured and coordinated approach

    The Big Data Steering Group developed the plan endorsed by HMA in November 2023, and subsequently by EMA's Management Board in December 2023, and maintains it over time.

    AI-enabled knowledge mining tool

    In March 2024, EMA introduced an AI-enabled knowledge mining tool called Scientific Explorer for EU regulators.

    The tool enables easy, focused and precise search of regulatory scientific information from network's sources to support decision-making and simplify processes.

    The first release focuses on scientific advice procedures for human medicines.

    For more information, see the document below with answers to frequently asked questions about the tool:

    Scientific Explorer - Frequently Asked Questions

    Findings include:

    • Real-world evidence can support decision-making in various regulatory contexts
    • Regulators need access to additional data sources such as secondary care databases, claims databases and registries from across Europe
    • Research needs should be identified as early as possible so that studies can be conducted in time for regulatory decisions
    • More information on data source characteristics is needed to facilitate the interpretation of real-world evidence derived from these sources
    • There is a need to build more capability and capacity for real-world evidence generation

    This report informs the development of the Data Analysis and Real World Interrogation Network (DARWIN EU).

    For more information, see below the report document (also accessible by clicking on the thumbnail image in this section) and the list of research topics and use cases:

    Real-world evidence framework to support EU regulatory decision-making: Report on the experience gained with regulator-led studies from September 2021 to February 2023

    Annex 1: List of EMA RWD study requests

    Related information materials

    International collaboration on real-world evidence

    Within the International Coalition of Medicines Regulatory Authorities (ICMRA), EMA works to help integrate real-world evidence into regulatory decision-making across the world. 

    ICMRA held a workshop in June 2022 for regulators to share experience in obtaining and using real-world evidence for the assessment of medicines, and issued a pledge in July 2022 to foster global efforts in this area. 

    For more information:

    Reflection paper

    A reflection paper was available for public consultation aiming to harmonise real-world evidence terminology and enable the convergence of general principles for planning and reporting studies using real-world data to support regulatory decision-making. 

    The document builds on the 2022 statement from the International Coalition of Medicines Regulatory Authorities (ICMRA), offering a strategic approach for future ICH guidelines on the assessment of real-world data and real-world evidence.  

    EMA encouraged medicine regulators, academic researchers, the pharmaceutical industry and other stakeholders to comment. 

    The public consultation was open until 30 September 2023.

    The reflection paper was released by the Council for Harmonisation of Technical Requirements for Pharmaceuticals for Human Use (ICH), and co-sponsored by EMA, the United States of America's (USA) Food and Drug Administration (FDA), and Health Canada.

    More information:

    ICH reflection paper on proposed international harmonisation of real-world evidence terminology and convergence of general principles regarding planning and reporting of studies using real-world data, with a focus on effectiveness of medicines

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