Complementing randomized clinical trials with real-world evidence: Target trial emulation, data linkage, and individualized treatment effects using the example of dual antiplatelet therapy in acute coronary syndrome

• Applicant: Dr. Nils Krüger
• Subject Area: Cardiology, Angiology
• Term: since 2026
• Project identifier: Deutsche Forschungsgemeinschaft (DFG) - Project number 579619668

The analysis of routine clinical data offers the opportunity to evaluate the treatment effects of existing and new drugs beyond randomized controlled trials (RCTs). Such real-world evidence (RWE) makes it possible to specifically examine patient groups that are often underrepresented in cardiovascular RCTs and to investigate heterogeneous therapeutic effects in broad patient populations. Although RWE is increasingly being used to supplement results from RCTs in order to gain a more comprehensive understanding of the Although RWE is increasingly being used to complement results from RCTs to gain a more comprehensive understanding of the efficacy of drugs, its credibility is sometimes called into question by factors such as non-transparent and questionable study designs. However, RWE studies that use proven methods of epidemiology and statistics for causal interpretations have proven to be increasingly robust in cardiovascular medicine, when the data sources are suitable for the purpose of a specific study. Physicians, guideline committees, and regulatory agencies are seeking predictably robust approaches to generating RWE that build on existing RCT evidence while incorporating tools to assess the potential for residual bias. Both the U.S. Food and Drug Administration (FDA) and the European Medicine Agency (EMA) are pursuing strategic programs to integrate RWE into the approval process for new and existing drugs. In cardiovascular medicine, this has led to promising approaches in which RWE can expand our understanding of treatment effects beyond RCTs. Acute coronary syndrome (ACS) serves as an excellent methodological use case in this project. With over 7 million patients affected each year, ACS is one of the most common cardiovascular emergencies worldwide. Dual antiplatelet therapy is the established treatment option in secondary prevention after percutaneous coronary intervention. Dual antiplatelet therapy is the established treatment option in secondary prevention after percutaneous coronary intervention. With its clearly defined endpoints and treatment strategies established over many years, the ACS offers a particularly suitable model for developing innovative methodological approaches to validating and further developing RWE analyses. The aim of this project is to anchor RWE results in RCT results and, in addition, to generate new evidence for underrepresented patient groups and for care in clinically heterogeneous everyday practice.

• DFG Programme: WBP Fellowship
• International Connection: USA
• Host: Professor Dr. Sebastian Schneeweiss