Newsletter June 2024

Dear Subscriber,

Welcome to the latest edition of our newsletter! In this issue, we delve into a patient-focused approach, exploring the most relevant and meaningful topics for those at the core of our work. We also present one of our partners, the European Organization for Professionals and Patients with ALS, Amyotrophic Lateral Sclerosis.

Patient Associations - Empowering Patients in Medical Research and Real-World Data Use

In recent years, patient associations have emerged as pivotal players in medical research, contributing invaluable insights and driving innovations that resonate with patient needs. These organisations, often founded and led by individuals directly affected by specific conditions, bring a unique perspective that traditional research teams may lack.

That’s the case with our partner EUpALS. To facilitate better alignment between patients and the clinical development process of ALS therapies, EUpALS has set up a Patients and Carers Expert Board. This group of patients and carers provides the industry with input on specific study information based on their real-world experiences with ALS. Univocally, their slogan is “Nothing about us without us!”.

By listening to patients and carers, the industry can adopt practical solutions that will enhance the experience of participants in their studies. This will likely have a positive effect on the attractiveness of studies to potential participants. By creating study designs that work for patients and carers, the burden for enrollment in clinical studies will be lowered, and the ability of participants to remain in studies will be increased. By this, sponsors will have less missing data, and participants will more likely continue taking an experimental medicine throughout the disease course.

Real-World Data: Patient Insights for Better Outcomes

The utilization of real-world data (RWD) is revolutionizing the healthcare landscape, providing insights that traditional clinical trials may not capture.
From the patient perspective, the use of RWD can offer the advantage of reflecting the diverse and dynamic nature of everyday clinical settings, capturing variations in patient populations, treatment regimens, and health outcomes. This comprehensive view can lead to more personalized and effective healthcare strategies, tailored to real-world circumstances rather than idealized trial conditions.
However, the use of RWD also raises important considerations about data privacy and consent. Patients are increasingly aware of the need for robust safeguards to protect their personal health information. Transparent communication about how data will be used, who will have access to it, and the measures in place to ensure its security is essential to maintaining trust and participation in RWD initiatives.

For these reasons, the Real4Reg project has several activities designed to empower patients and enhance the quality and utility of real-world data. These activities include:

• Patient-Dedicated Events: One such event is an online workshop entitled Demystifying Real-World Data - Insights for Patients, which will take place later this year.
• Patient Survey: Recently, we conducted a survey to collect the perspectives and opinions of patients regarding the use of RWD/RWE. The results of this survey will be available in the coming months, providing valuable insights into patient views.
• Patient-Dedicated Website Section: Our website features a dedicated section for patients, providing information about the project in lay language to ensure accessibility and understanding.

We will continue working with the patients and for the patients!

Partner Presentation: EUpALS

Aims

EUpALS is the European Organization for Professionals and Patients with ALS. As international non-profit organisation, EUpALS take the interests of all people living with Amyotrophic Lateral Sclerosis (pALS) in Europe to heart.
ALS is a lethal neuromuscular disease characterised by progressive loss of the motor neurons that drive muscle function. It may affect any muscle group with the exception of the autonomously controlled muscles. A person living with ALS dies on average 33 months after diagnosis due to paralysis of respiratory and swallowing muscles.

As an umbrella organisation, EUpALS currently unites 28 national ALS associations from 22 European countries to cooperate better.
EUpALS create equal rights for all the 50.000 people living with ALS in Europe. In particular, by providing them better access to ALS clinical research and information about it.

To assist with scientific advice, the EUpALS Scientific Expert Board consists of Key Opinion Leaders in the domain of ALS clinical research.
As patients are at the heart of the organisation, EUpALS furthermore established the EUpALS Patients and Carers Expert Board. By this, the role of patients and carers in the clinical development process of ALS therapies can be assured.

Role in Real4Reg

The participation of EUpALS in Real4Reg ensures that patients are actively involved in the project, in which ALS is a use case. Within the project consortium, EUpALS mainly focus on accessible communication and dissemination of the project activities and results. 

News

Information for patients in different languages

The Real4Reg website has received an update aimed at improving accessibility for patients across Europe. The Information for Patients section is now available in multiple languages, allowing individuals from various countries to access project information in clear, layman's terms and in their native languages. Our website now offers translations in Danish, German, Finnish, French, Portuguese, and Swedish.

Project Coordinator's Visits to Consortium Partners Enhances Collaboration within Real4Reg

The recent series of visits by our project coordinator Kerstin Pfeifer (BfArM, Germany) to all work package (WP) leaders has significantly reinforced collaboration and innovation within the Real4Reg project. Since she joined Real4Reg ten months into the project, these visits provided an in-depth overview of the partners’ work and their teams. The focus of the meetings was to get to know each other better, to understand what is important to the partners in our project, and to learn more about the progress of the WPs, including challenges, possible solutions and cooperation across WPs.

Upcoming Events

21-25 June 2024, Thessaloniki, Greece - 45th Annual Conference of the International Society for Clinical Biostatistics
read more

26-27 June 2024, Helsinki, Finland - Real4Reg Consortium Meeting

24-28 August 2024 – Berlin, Germany  - 40th Annual Meeting International Society for Pharmacoepidemiology (ISPE).
read more

For more information on additional events in the realms of real-world data, artificial intelligence, and health, please consult our Events page

Follow Real4Reg on Social Media

RealReg is a project funded by the European Union under the Horizon Europe programme –Project No. 101095353. The consortium of ten European institutions aims to promote the use of real-world data to support regulatory decisions about medicines. For media inquiries, please contact: real4reg@infarmed.pt

Views and opinions expressed are however those of the author(s) only and do not necessarily reflect those of the European Union or the European Commission. Neither the European Union nor the granting authority can be held responsible for them.

Imprint

Federal Institute for Drugs and Medical Devices (BfArM)
Represented by the President
Prof. Dr Karl Broich

Headquarters Bonn:
Kurt-Georg-Kiesinger-Allee 3
53175 Bonn
Germany

Headquarters Cologne:
Waisenhausgasse 36-38a
50676 Köln

Phone: +49 (0)228 99 307-0
Fax: +49 (0)228 99 307-5207
E-mail: poststelle@bfarm.de

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