Exploratory Investigational New Drug (IND) Studies in Humans

Abstract

In recent years drug development costs have escalated despite the fact that new technology is evolving and the development process has accelerated. This is due in part to the extremely high failure rate of drugs at early phases of drug development. Efforts are underway within the pharmaceutical industry to shift this attrition rate to early stages of drug development rather than at late stages. To this end, the exploratory IND approach is considered as one of the tools in a new drug development “tool box,” to distinguish earlier in the process those candidates that hold promise from those that do not. The phrase exploratory IND study is intended to describe a clinical trial that is conducted early in phase 1, involves very limited human exposure, has no therapeutic or diagnostic intent (e.g., screening studies, microdose studies), and is NOT intended to establish a maximally tolerated dose (MTD). In this article, authors attempt to summarize the goals of eIND, a brief description of eIND requirements, and limitations of an eIND.

Notes

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3. Guidance for Industry, Investigators, and Reviewers Exploratory IND Studies, CDER, FDA, January 2006.

4. Guidance for industry Content and Format of Investigational New Drug Applications for Phase 1 Studies of Drugs, Including Well-Characterized, Therapeutic, Biotechnology-Derived Products, CDER, FDA, 1995.

5. International Conference on Harmonization (ICH) guidance for industry M3 Nonclinical Safety Studies for the Conduct of Human Clinical Trials for Pharmaceuticals, 1997.

6. International Conference on Harmonization (ICH) guidance for industry S7A Safety Pharmacology Studies for Human Pharmaceuticals, 2005.

7. European Medicines Agency (EMEA), Evaluation of Medicines for Human Use, Position Paper on Non-Clinical Safety Studies to Support Clinical Trials with a Single Microdose. CPMP/SWP/2599/02Rev 1, 23 June 2004.