Description
This guidance provides sponsors who wish to submit an Investigational New Drug application (IND) for a therapeutic cancer vaccine with recommendations on critical clinical considerations for investigational studies of these products. This guidance will discuss considerations common to phase 1 and phase 2 clinical trials (collectively referred to as “early phase clinical trials”) and phase 3 clinical trials (referred to as “late phase clinical trials”), as well as considerations that are unique to specific stages of clinical development of therapeutic cancer vaccines. This guidance provides recommendations for the design of clinical trials for cancer vaccines conducted under an IND (Title 21 Code of Federal Regulations (21 CFR) Part 312) to support a subsequent biologics license application (BLA) for marketing approval. This guidance finalizes the draft guidance of the same title dated September 2009.
Scope & Applicability
Product Classes
5Guidance provides clinical considerations for these products intended to result in specific responses to a tumor antigen.; Guidance document focusing on clinical considerations for these products; Guidance document focus on clinical considerations for these products
Specific considerations for autologous vaccine trials.; Design of studies using autologous vaccine products derived from subjects' own tumors
Guidance does not apply to these products such as T cell or NK cell products.
Products combining drug, device, or biological constituents; Generally recommended for Enhanced Documentation; Requires 14971-based framework incorporating ICH Q9; A drug-device combination where the device constituent part detects ingestion.
A type of ATMP involving recombinant nucleic acids or viral vectors.
Stakeholders
2Assist sponsors in the nonclinical evaluation
Entity responsible for submitting applications under section 524B
Regulatory Context
Attributes
4Dose selection criterion for carcinogenicity studies; criteria for adequacy of the rat study; Virtually Safe Dose Based on the Maximum Tolerated Dose.; Based on the Maximum Tolerated Dose for Rodent Bioassays.
Determined during the planning of a research study
Dose level to guide initiation of clinical dosing
Assessment required for therapeutic protein products.
Related CFR Sections (6)
- 21CFR601.43§ 601.43 Withdrawal procedures.
(a) For biological products approved under § 601.41 or § 601.42 , FDA may withdraw approval, following a hearing as provided in part 15 of this chapter , as modified by this section, if:Read full regulation →
- 21CFR601.41§ 601.41 Approval based on a surrogate endpoint or on an effect on a clinical endpoint other than survival or irreversible morbidity.
FDA may grant marketing approval for a biological product on the basis of adequate and well-controlled clinical trials establishing that the biological product has an effect on a surrogate endpoint that is reasonably likely, based on epidemiologic, therapeutic, pathophysiologic, or other evidence, tRead full regulation →
- 21CFR314.510§ 314.510 Approval based on a surrogate endpoint or on an effect on a clinical endpoint other than survival or irreversible morbidity.
FDA may grant marketing approval for a new drug product on the basis of adequate and well-controlled clinical trials establishing that the drug product has an effect on a surrogate endpoint that is reasonably likely, based on epidemiologic, therapeutic, pathophysiologic, or other evidence, to predicRead full regulation →
- 21CFR610.15§ 610.15 Constituent materials.
(a) Ingredients, preservatives, diluents, adjuvants. All ingredients used in a licensed product, and any diluent provided as an aid in the administration of the product, shall meet generally accepted standards of purity and quality. Any preservative used shall be sufficiently nontoxic so that the amRead full regulation →
- 21CFR50.25§ 50.25 Elements of informed consent.
(a) Basic elements of informed consent. In seeking informed consent, the following information shall be provided to each subject:Read full regulation →
- 21CFR3.2§ 3.2 Definitions.
For the purpose of this part:Read full regulation →
Related Warning Letters (10)
- 2025-12-23
In Vivo Bioavailability-Bioequivalence Studies – Clinical
Maria A. Carballosa, M.D.
- 2025-12-09
Unapproved New Drug/Unlicensed Biological Product/Biologics License Application (BLA)
Celularity, Inc
- 2025-11-18
Sponsor/Investigator
Verdure Sciences, Inc.
- 2025-09-30
Clinical Investigator (Sponsor)
Pamela K. Den Besten, DDS, MS
- 2025-09-23
Clinical Investigator/Sponsor
Ralph A. DeFronzo, M.D.
- 2025-09-09
Clinical Investigator
Shirish M. Gadgeel, M.D.
- 2025-07-15
Clinical Investigator
Mark J. Savant, M.D
- 2025-07-01
Clinical Investigator
Peter Michael, M.D.
- 2025-06-10
Clinical Investigator (Sponsor)
American Behavioral Research Institute, LLC
- 2025-06-03
Bioresearch Monitoring Program
Amy Lightner, MD
See Also (8)
- Labeling for Human Prescription Drug and Biological Products Approved Under the Accelerated Approval Regulatory Pathway (Status: Final)
- Clinical Data Needed to Support the Licensure of Seasonal Inactivated Influenza Vaccines: Guidance for Industry (Status: Final)
- Clinical Data Needed to Support the Licensure of Pandemic Influenza Vaccines: Guidance for Industry (Status: Final)
- Postmarketing Studies and Clinical Trials—Implementation of Section 505(o)(3) of the Federal Food, Drug, and Cosmetic Act Guidance for Industry (Status: Final)
- General Principles for the Development of Vaccines to Protect Against Global Infectious Diseases: Guidance for Industry (Status: Final)
- Expedited Programs for Serious Conditions | Drugs and Biologics (Status: Final)
- Indications and Usage Section of Labeling for Human Prescription Drug and Biological Products — Content and Format Guidance for Industry (Status: Draft)
- Expedited Programs for Regenerative Medicine Therapies for Serious Conditions: Guidance for Industry (Status: Final)