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  3. Development & Approval Process | Drugs
  4. How Drugs are Developed and Approved
  5. Types of Applications
  6. Investigational New Drug (IND) Application
  7. IND Applications for Clinical Investigations: Pharmacology and Toxicology (PT) Information
  1. Investigational New Drug (IND) Application

IND Applications for Clinical Investigations: Pharmacology and Toxicology (PT) Information

This component of an IND application is expected to contain information about pharmacological and toxicological (laboratory animals or in vitro) studies on the basis of which the sponsor of the IND application has concluded that it is reasonably safe to conduct the proposed clinical investigations. The kind, duration, and scope of animal and other studies required in the application will depend on the duration and nature of the proposed clinical investigations. For recommendations regarding study types and duration, refer to Guidance for Industry: M3(R2) Nonclinical Safety Studies for the Conduct of Human Clinical Trials and Marketing Authorization for Pharmaceuticals (PDF - 325KB).

Compliance with Good Laboratory Practice (GLP) is generally expected for pivotal in vitro and in vivo studies submitted in support of an IND application. For each non-clinical laboratory study subject to the GLP regulations, investigators are expected to state in the study report that the study was conducted in compliance with the GLP regulations. If the study was not conducted in compliance with the GLP regulations, investigators should submit a brief statement of the reason for noncompliance.

FDA Guidance documents relevant to Pharmacology and Toxicology information are available and describe ways in which requirements related to conduct of non-clinical toxicology studies may be met. In addition to description of the results of each study, the IND sponsor is required to include the identification and qualifications of the individuals who evaluated the results of each study and concluded that it is reasonably safe to begin the proposed investigations.

The IND sponsor should also provide a statement describing where the non-clinical investigations were conducted and the location of all records available for inspection. As drug development proceeds, the IND sponsor is required to submit Information Amendments, with additional PT information pertinent to safety or other aspects of the IND application.

A brief description of each section related to Pharmacology and Toxicology information is provided below.

 

Pharmacology and Drug Disposition 

This section is expected to include description of the pharmacological effects and the mechanisms of action of the drug in animals and information on the absorption, distribution, metabolism, and excretion of the investigational product, if known.

Toxicology 

This section is expected to include information on the toxicological effects of the drug in animals and in vitro. For detailed explanation of what should be included in this section, refer to the Guidance for Industry: Content and Format of Investigational New Drug Applications (INDs) for Phase 1 Studies of Drugs, Including Well-Characterized, Therapeutic, Biotechnology-derived Products (PDF - 41KB).

Depending on the nature of the drug and the phase of the investigation, the description is expected to include:

  1. the results of acute, subacute, and chronic toxicity tests;

  2. the results of tests of the drug's effects on reproduction and the developing fetus;

  3. any special toxicity test related to the drug's particular mode of administration or conditions of use (e.g., inhalation, dermal, or ocular toxicology); and

  4. any in vitro studies intended to evaluate drug toxicity. 

For each toxicological study that is intended primarily to support the safety of the proposed clinical investigation, a full tabulation of data suitable for detailed review is expected. This should consist of line listings of the individual data points, including laboratory data points for each animal along with appropriate summary tabulations. 

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