On October 14, the Real4Reg 2024 workshops provided an engaging platform for discussing the perspectives and insights surrounding real-world data (RWD) and real-world evidence (RWE). These sessions focused on the views and expectations of both professionals and patients, offering valuable input on the evolving role of RWD in healthcare.
Workshop 1: Insights into Stakeholder Needs
This workshop targeted professionals from various fields, including regulatory agencies, health technology assessment bodies, academia, industry, payers, healthcare institutions, patient associations, among others.
Luca Giraldi from the European Medicines Agency (EMA) delivered a keynote discussing the EMA’s vision on RWE, highlighting the challenges regulators face. He also introduced the Big Data Training Curriculum, a program designed to help regulators develop the skills needed to work effectively with RWD.
Lucas Depner from Real4Reg and the German Center for Neurodegenerative Diseases (DZNE) presented the results of the Real4Reg survey, which explored stakeholders’ views and perspectives on key themes such as RWD usage, current challenges, AI/ML adoption, guidance utilisation, interest in further training, and preferred training topics.
The presentation was followed by a multi-stakeholder panel discussion moderated by Vera Ehrenstein (Aarhus University/Real4Reg), with participants including Luca Giraldi (EMA), Niklas Hedberg (Heads of HTA Agencies Group), Gianmario Candore (Bayer), Carla Torre (Faculty of Pharmacy, University of Lisbon), and Paulo Gonçalves (RD-Portugal). The participants expressed significant interest in the survey findings, engaging in a lively discussion on various key themes, including:
- Stakeholder perspectives on the survey results
- Enhancing academic collaboration with other fields
- Training strategies and stakeholder needs in real-world evidence
- The value of RWD
- Involving patients and patient organisations in the development of training programs
Recordings and presentations
Real-world evidence & training needs: a regulatory perspective
Real4Reg: Insights into Stakeholder Needs / Luca Giraldi – European Medicines Agency
Real4Reg survey results
Lucas Depner, German Centre for Neurodegenerative Diseases (DZNE)
Key takeaways:
- Keywords: Communication, education, trust, involvement, and cooperation.
- There is a clear need to bridge the existing gaps between academia and regulatory agencies, as well as between academia and the general public. This could be achieved through collaborative training initiatives.
- Joint training sessions should be implemented to explore the different viewpoints of various stakeholders on the same topics and to identify which types of data are suitable for addressing their specific questions.
- Greater transparency is needed to provide deeper insights into the activities of different stakeholders, which can help identify areas of common ground.
- Incorporating real-world use cases can aid in understanding the diverse perspectives of stakeholders.
Workshop 2: Demystifying Real-World Data for Patients
This session aimed to simplify the concepts of RWD and RWE, providing patients and the general public with a clearer understanding of the field while addressing common questions related to data privacy and ethics. A diverse group of participants attended, including representatives from universities, healthcare institutions, pharmaceutical R&D organizations, regulatory agencies, and private companies.
Nicolas Thurin (University of Bordeaux) presented on RWD and its uses, covering key topics such as the definitions of RWD and RWE, the pyramid of evidence, and a comparison between RWE and clinical trials. He discussed the generalisability of results from RWE studies and provided practical examples.
The session was followed by a presentation by Britta Haenisch from BfArM – Federal Institute for Drugs and Medical Devices and Real4Reg Principal Investigator. Haenisch introduced the Real4Reg project and its methods, including the use cases, datasets, and work packages that form the core of the initiative. She shared the first results of the project.
In a session focused on the ethical implications of RWD, Chloé Antoine from the University of Namur explored data privacy, security, and ethical considerations in the use of RWD. She outlined the journey of RWD from a data protection perspective, presented how RWD can be collected and the safeguards that must be in place to ensure compliance with the General Data Protection Regulation (GDPR). Antoine also informed attendees about patients’ rights when it comes to the use of their data.
The event concluded with a roundtable session, moderated by Real4Reg partner Rebecca Moore (EIWH – European Institute of Women’s Health), which featured insights from Antonella Cardore (Cancer Patients Europe), Elsa Frazão Mateus (EUPATI – European Patients’ Academy on Therapeutic Innovation), Ywan Dierick (EUpALS – European Organisation for Professionals and People with ALS), and Chloé Antoine (University of Namur). The discussion centred around key points such as:
- Patients as a diverse group, with varied opinions on the use of their data and differing concerns about privacy.
- From the Real4Reg survey, it was highlighted that privacy and security are major concerns for patients regarding the use of RWD/E.
Recordings and presentations
Understanding Real-World Data and its potential
Nicolas Thurin – Université de Bordeaux
The Real4Reg project – work in progress
Demystifying Real-World Data – Insights for Patients, 14 October 2024 / Prof. Britta Hänisch, BfArm, Principal Investigator
Demystifying Real-World Data – Insights for Patients
Data Privacy, Security and Ethical Considerations / Chloé Antoine – Legal Researcher at CRIDS (University of Namur)
Key takeaways:
- Key Concepts: Trust and transparency are fundamental values, central to both RWD initiatives and Real4Reg’s objectives.
- Patient Involvement: Two patient associations are actively involved as partners in Real4Reg, ensuring direct insights from the patient perspective.
- Patients as Individuals: RWD mitigates selection bias by including a broad spectrum of patients, making analyses more representative.
- Clear Language: Using accessible, clear language is essential to fostering patients’ willingness to share their data.
- Communication Efforts: Real4Reg is committed to improving communication and increasing interaction with patients, aiming for greater collaboration and understanding.
Looking Ahead: Building Bridges in RWD and RWE
The Real4Reg 2024 workshops highlighted the critical need for collaboration among various stakeholders to unlock the full potential of RWD and RWE. By addressing the distinct needs of different audiences, the workshops fostered productive discussions that set the stage for future Real4Reg work, driving initiatives that focus on tackling training needs, patient involvement, and the effective use of RWD in healthcare decision-making.
Disclaimer:
The information presented in the Real4Reg slides is proprietary and unpublished. It is intended solely for consultation purposes. Any use, reproduction, or publication of the content is prohibited without explicit permission.
As the operator of the website “www.real4reg.eu”, Federal Institute for Drugs and Medical Devices (BfArM) has agreed to make the presentations of the speakers available for download on this page with their explicit consent. Should you notice that the presentation slides contain incorrect information, copyrighted material, or any other content that violates the law, please notify real4reg@infarmed.pt. We will promptly remove the relevant content.