This guidance is intended to assist sponsors of microdose radiopharmaceutical diagnostic drugs on the nonclinical studies recommended to support human clinical trials and marketing applications. This guidance addresses the Food and Drug Administration’s (FDA’s) current thinking regarding regulation of this class of drugs and provides complementary recommendations to the guidance for industry, investigators, and reviewers Exploratory IND (Investigational New Drug application) Studies (exploratory IND guidance) and the ICH guidance for industry M3(R2) Nonclinical Safety Studies for the Conduct of Human Clinical Trials and Marketing Authorization for Pharmaceuticals (ICH M3(R2)). This guidance discusses how to refine nonclinical study recommendations for this class of drug given its unique characteristics (e.g., microdose, radiolabeled, single (or infrequent) use, clinical use setting, and the Agency’s nonclinical and clinical safety experience with these drugs).
This guidance also is intended to help sponsors facilitate the timely conduct of clinical trials, reduce the use of animals with the 3R (reduce/refine/replace) principles, and reduce the use of drug development resources. While both the exploratory IND and the ICH M3(R2) guidances describe recommended nonclinical studies intended to be conducted early in phase 1 exploratory studies of microdose radiopharmaceutical diagnostic drugs, the guidances do not address what additional nonclinical studies are recommended for marketing approval. This guidance is intended to provide recommendations for a pathway to full drug development (marketing authorization) for microdose radiopharmaceutical diagnostic drugs.
As technology advances, microdose drugs that use new modalities may emerge. Although this guidance describes recommendations for current radiopharmaceutical diagnostic drugs, the general principles discussed could apply to new diagnostic drugs.
This guidance does not apply to radioactive drugs for research that are used in accordance with 21 CFR 361.1. These issues are addressed in the guidance for industry and researchers The Radioactive Drug Research Committee: Human Research Without an Investigational New Drug Application.