Newsletter April 2024

Dear Subscriber,
Welcome to the latest edition of our newsletter. In this issue, we delve into Real-World Evidence (RWE) and its significance within regulatory contexts. Additionally, we are pleased to introduce you to our esteemed project coordinator, BfArM - the Federal Institute for Drugs and Medical Devices. Looking ahead, please anticipate our next edition, which will have a focus on patients. We invite you to explore the latest developments in our field with us.

Use of RWE for regulatory purposes

Randomized controlled trials (RCTs) are the gold standard for generating evidence for regulatory decision-making in the product lifecycle of drugs, i.e. for legal decisions concerning a drug before and after it comes onto the market. Nevertheless, RCTs have limitations, some of which may be addressed by the use of real-world evidence (RWE). RWE is information derived from the analysis of real-world data (RWD), i.e., health information from registries, health insurance, health apps etc. While the results of an RCT are highly valid within the context of that trial, the applicability of the findings to the real world are compromised because RCT and the real world differ with regard to selection of population, length of treatment, and adherence to treatment. Effectiveness and safety of drugs in routine care may differ from the results of the underlying RCT. The use of RWD can address these challenges.

Moreover, RWD can help reduce the burden of study participants and fasten the processes of drug development. Besides conducting studies with RWD only, RWD can supplement clinical studies, e.g., with external or historical control groups, or clinical trials can be nested within existing cohorts (TwiCs).

Partner Presentation: BfArM

The Federal Institute for Drugs and Medical Devices (Bundesinstitut für Arzneimittel und Medizinprodukte, BfArM) is an independent federal higher authority within the portfolio of the German Federal Ministry of Health. The BfArM's core tasks include the licensing and registration of medicinal products, recording and assessment of risks related to medical devices, monitoring of the legal traffic in narcotics and precursors, as well as the publication of medical coding systems for healthcare sectors.

Aims

The primary goal of the BfArM’s activities is to increase the safety of medicinal products and thus that of the patients. Throughout the EU and internationally, the BfArM contributes significantly to the supply with safe and effective medicines.
The BfArM works on improving the information management on international developments throughout the European Union and internationally. It is represented at European and international levels in cross-functional EU bodies (HMA (Heads of Medicines Agencies), CHMP (Committee for Medicinal Products for Human Use)) and working groups of the European Medicines Agency and at several committees responsible for the scientific evaluation of applications for Orphan Drug Status, of paediatric investigation plans, and co-ordination of issues related to licensing and pharmacovigilance of medicinal products with (planned) national marketing authorisation in more than one Member State of the European Union/the European Economic Area. Moreover, the BfArM provides various advice procedures for the development of new medicinal products and medical devices, it assesses Digital Health Applications to be prescribed, it hosts the Health Data Lab (which makes pseudonymised statutory health insurance data available for research purposes with the goal of improving healthcare for all) and the German Clinical Trials Register.
The BfArM is also one of the federal government's department research institutions and conducts independent scientific research in order to fulfil its tasks (the department “Research” includes the areas pharmacogenomics, pharmacoepidemiology, drug allergies, biostatistics, neurophsychopharmacology, special indications). Further duties include legal mandates and the ordinances related to these as well as individual tasks assigned by the Federal Ministry of Health.

Role in Real4Reg

The BfArM is responsible for the coordination and project management of Real4Reg, and also for contextualisation and implementation of the results obtained. The BfArM also provides data from the German health system to the project and is involved in data analyses.

News

First 2024 Consortium Meeting

The first Consortium meeting of the second year of the Real4Reg project took place virtually on the 27th of February. The meeting encompassed updates on progress, lessons learned, preliminary results and outlining forthcoming tasks.

Upcoming Events

14-16 April 2024, Orlando, USA - ISPE 2024 Mid-Year Meeting. The theme of the 2024 International Society for Pharmacoepidemiology Mid-Year Meeting is Expanding Pharmacoepidemiology to meet Emerging Global Challenges.
read more

2-4 May 2024, Valencia, Spain - The 27th International Conference on Artificial Intelligence and Statistics (AISTATS). AISTATS is an interdisciplinary gathering of researchers at the intersection of computer science, artificial intelligence, machine learning, statistics, and related areas.
read more

14-15 May 2024, Utrecht, The Netherlands - The GetReal Institute Annual Conference 2024 - A Growing Convergence: RWE for Regulatory & HTA Decision Making.
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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