gms | German Medical Science

The challenge of modern drug regulation is the correct risk benefit estimation in different patient groups over the complete life cycle of a drug. Regulators need to weigh their decisions on best current knowledge on patient risk factors leading to rational stratification of patient populations according to their estimated benefit-risk ratio. Research gaps and the accelerated turnaround of knowledge in modern medicine brings up the necessity for regulatory motivated research on molecular and clinical patient risk factors for drug safety and efficacy to support stratified risk-benefit assessments. At the BfArM, a regulatory research division was founded in 2012 to address these specific topics. We are performing studies on patient-specific risk factors for drug toxicity and efficacy. Such studies on molecular and genetic patient risk factors may support regulatory work in three ways: first, it provides data on the prognostic versus predictive value of molecular patient profiles which is important for regulatory advices. Second, it enables the recognition of safety signals associated with specific patient risk profiles, which is important for pharmacovigilance, and, third, it supports the validation of utility to include biomarkers and companion diagnostics into drug therapy.