FDA Guide for Non-clinical Studies of Oncological Therapeutic Radiopharmaceuticals

In early August, the FDA published a final guideline for radiopharmaceuticals for cancer treatment with a focus on non-clinical studies and product labelling. As a rule, such therapeutic radiopharmaceuticals are administered intravenously and are designed to selectively deliver cytotoxic radiation levels to tumor sites.

This guideline therefore deals specifically with radiopharmaceutical products that are administered systemically and contain a radionuclide and that are used to treat or to relieve tumour-related symptoms. The document is intended to complement and provide guidance and recommendations in areas not covered by other FDA and ICH guidelines, such as non-clinical studies in support of first-in-human (FIH) studies and approvals. It also provides additional information that goes beyond the guidance on non-clinical assessment of late radiation toxicity of therapeutic radiopharmaceuticals (November 2011). In principle, this guidance refers to products without prior clinical phase experience. However, it may also be the case that there are already studies on safety and/or efficacy in cancer treatment, e.g. for ligands such as antibodies or the like. In such cases, the non-clinical program may be reduced or the FIH dose determined from clinical data.


  • This guide contains concepts for
  • Assessment of the toxicity of the ligand
  • Evaluation of radiotoxicity
  • Information on product labelling in relation to reproductive toxicity, genotoxicity, carcinogenicity, prevention and use in breastfeeding women

Not covered by this guide:

  • Therapeutic radiopharmaceuticals with local route of administration (e.g. intratumoral, intrathecal)
    external radiotherapy,
  • Radio-labelled vaccines
  • Diagnostic radiopharmaceuticals or radioactive medicaments for research purposes as defined in 21 CFR 361.1

However, non-clinical studies conducted as part of or to support product development of diagnostic radiopharmaceuticals may meet the requirements for studies for a therapeutic radiopharmaceutical.

Quality issues such as impurities, specifications or product stability are also not covered by this guideline, although the entire radioactive decay cascade should be considered in the biodistribution and dosimetry studies to estimate radiation activity in organs and absorbed radiation doses.

For further details please refer directly to the guideline "Oncology Therapeutic Radiopharmaceuticals: Nonclinical Studies and Labeling Recommendations".

Please note in this context the newly created course of the ECA Academy "GMP and Quality for Radiopharmaceuticals".

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