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No. 3: May 24-28, 2010

Marketed drug for cancer treatment

Scenario:

A cancer drug was approved for marketing by FDA several years ago for use in end stage solid tumor cancer after all other treatments have failed. An oncologist at a medical center wants to study this drug for a different type of cancer. He files a research application to the hospital IRB for review and approval. The IRB indicates that an IND should be filed. The oncologist says that his license to practice medicine allows him to use any drug any way he deems appropriate to treat his cancer patients. Therefore, he says he does not need to file an IND to conduct this study using a lawfully marketed drug.

Issue:

If this were clinical practice and not clinical research the oncologist would be correct. The chairman of the IRB has read the FDA’s guidance document on IND exemptions for studies of lawfully marketed drugs for the treatment of cancer. The guidance document describes the types of studies that are IND exempt and those that require the submission of an IND.

Solution?

The oncologist is asked to review this guidance document and provide the necessary documentation to support his position. The guidance document presents many examples to make the IND exemption determination relatively straight forward.

Lessons Learned:

The FDA issues numerous guidance documents on many clinical research topics. Even though guidance documents do not establish legally enforceable responsibilities, they do contain the most current thinking of the FDA on this topic. There are 5 criteria that must be met to exempt a study from the submission of an IND. It is the third criterion that is protocol related and has special meaning in the oncology therapy setting, particularly with respect to doses above the labeled dose, use with other treatments, and use in different populations. The exemption permits deviations from the approved labeling to the extent that such changes are supported by the scientific literature and generally known clinical experience

References: Guidance for Industry: IND exemptions for studies of lawfully marketed drug or biological products for the treatment of cancer. January 2004. http://www.fda.gov/downloads/Drugs/GuidanceComplianceRegulatoryInformation/Guidances/ucm071717.pdf

Comments by Harvey M. Arbit, PharmD, MBA, president of Arbit Consulting, LLC. He specializes in FDA regulatory affairs related to investigator-initiated IND and IDE research.