Today's announcement by the U.S. Food and Drug Administration (FDA) of guidance for exploratory investigational new drug (IND) studies will help streamline the earliest phases of clinical research in the development of life-saving medical interventions for cancer patients. This novel concept has been a major focus for the joint National Cancer Institute (NCI) - FDA Interagency Oncology Task Force (IOTF), which was established in 2003 to enhance and accelerate the overall process of developing new cancer diagnostics and therapeutics. The ability to study very small doses of experimental agents will allow researchers to test promising drugs to get information about their distribution and activity in the human body. This guidance will not only benefit the development of new cancer interventions, but has implications across drug and biologics development for all diseases. NCI is part of the National Institutes of Health.

"Today's FDA exploratory IND guidance documents are important steps in fulfilling our goals to help streamline cancer drug development by identifying the gaps and barriers that can be addressed through new approaches and new applications of scientific evidence," said NCI Deputy Director Anna D. Barker, Ph.D. "I am pleased that working through the Interagency Oncology Task Force, we were able to crystallize opportunities to accelerate progress in the cancer drug development process that will be enabled by this new FDA guidance."

Cancer drug and biologics development begins with discovery and moves through a series of well-defined pre-clinical steps and clinical trials, culminating in submission for regulatory review. The guidance documents announced today are designed to increase the number of agents that can be tested in microdoses in small numbers of patients, which will allow many more agents to be evaluated before entering into a Phase I clinical trial.

The guidance will enable researchers to conduct early proof-of-principle evaluations of compounds and target effects to identify the most promising drugs for more extensive testing. This new approach will allow a better understanding of the metabolism and pharmacology of potential new agents, resulting in drugs that are safer and more effective. The companion GMP (good manufacturing practices) Guidance document issued today will allow researchers who are interested in evaluating investigational drugs to do so without the time and expense required to demonstrate capacity to produce the drug in large scale. This should significantly accelerate drug development and facilitate the selection of safer and more effective agents to enter Phase I clinical trials. Cancer patients should benefit from these paradigm-shifting changes.

The initial request for comment on today's guidance was published in the April 14, 2005, Federal Register. In this request, the FDA clarified submission requirements for early Phase I exploratory studies under an IND application. According to the FDA, such applications often contain more data than required by regulations.

"Through the IOTF, and input from many other communities, the FDA has responded to a critical need in drug development," said James Doroshow, M.D., director of NCI's Division of Cancer Therapeutics and Diagnosis. "It is now vital that cancer researchers in academia and the private sector take full advantage of these new opportunities to perform these types of exploratory studies. In fact, through the NCI intramural program, the NCI has set up a special new unit to do these types of trials on promising candidate drugs with a goal of speeding safer and more effective cancer interventions to patients."

For more information about cancer, please visit the NCI Web site at http://www.cancer.gov or call NCI's Cancer Information Service at 1-800-4 CANCER (1-800-422-6237).

For the final FDA guidance on Exploratory IND Studies, please go to http://www.fda.gov/cder/guidance/index.htm.

For more information about the FDA, please visit http://www.fda.gov/.

For information on the IOTF Joint Fellowship Training Program, please go to http://iotftraining.nci.nih.gov/.