OBJECTIVES: In oncology research, two main endpoints are typically measured – progression free survival (PFS) and overall survival (OS). Minimal residual disease (MRD) testing has been found to be impactful in both the diagnostic and treatment pathways for patients with cancer. This study aimed to better understand the use of MRD as an endpoint in the drug approval process.

METHODS: A targeted literature review was conducted using GoogleScholar to identify publicly available resources between Jan 2020 and Apr 2024 covering the regulatory approval status of MRD as an endpoint for drug approvals in the European Union (EU) and United States (US). 30 sources were screened and 14 were included.

RESULTS: In the EU, a reflection paper from the European Medicines Agency recommends a case-by-case assessment considering the uncertainty of MRD as a surrogate endpoint for progression-free survival (PFS)/overall survival (OS). Scientific advice will be needed when negative MRD results are proposed as a primary endpoint for regulatory approval purposes.

CONCLUSIONS: This review indicates an opportunity for MRD to be used as a surrogate endpoint in oncology research if the relationship between MRD results and clinical outcomes is strong. The access and availability of MRD testing continues to be subject to the dynamics of the diagnostics’ regulatory environment in both EU and US.