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Tissue Agnostic Drug Development in Oncology Draft Guidance for Industry October 2022

Draft Level 1 Guidance

Not for implementation. Contains non-binding recommendations.

Docket Number:
Issued by:
Guidance Issuing Office
Oncology Center of Excellence
Center for Drug Evaluation and Research
Center for Biologics Evaluation and Research

This guidance provides recommendations to sponsors regarding considerations for tissue agnostic drug development in oncology. For the purpose of this guidance, the term tissue agnostic oncology drug refers to a drug that targets a specific molecular alteration(s) (a kind of biomarker) across multiple cancer types as defined, for example by organ, tissue, or tumor type. A tissue agnostic oncology drug can therefore be used to treat multiple types of cancer (e.g., colorectal, thyroid, and breast cancers) with the targeted molecular alteration (e.g., either the same targeted molecular alteration or targeted molecular alterations affecting a single pathway). Although applications for a tissue agnostic oncology drug are reviewed for safety and effectiveness under the same legal and regulatory standard as drugs indicated for a tissue specific cancer, the development of a tissue agnostic oncology drug raises issues that generally do not arise in more traditional development approaches. This guidance describes the development of tissue agnostic drugs, scientific considerations in determining when tissue agnostic oncology drug development may be appropriate, and, if appropriate, issues to be addressed during such development.

This guidance does not address the development of drugs intended to prevent or decrease the incidence of cancer and does not address the treatment of cancer in the adjuvant or neoadjuvant setting.

Submit Comments

You can submit online or written comments on any guidance at any time (see 21 CFR 10.115(g)(5))

If unable to submit comments online, please mail written comments to:

Dockets Management
Food and Drug Administration
5630 Fishers Lane, Rm 1061
Rockville, MD 20852

All written comments should be identified with this document's docket number: FDA-2022-D-0286 .

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