Guidance intended to ensure that evolving policies are based on the best science; provide public confidence in this new field
The Food and Drug Administration (FDA) today issued a new document -- Draft Guidance for Industry: Pharmacogenomic Data Submissions -- that encourages drug and biologic developers to conduct pharmacogenomic tests during drug development and clarifies how FDA will evaluate the resulting data.
"Pharmacogenomics holds great promise to shed scientific light on the often risky and costly process of drug development, and to provide greater confidence about the risks and benefits of drugs in specific populations," said FDA Commissioner Mark B. McClellan, M.D., Ph.D. "Pharmacogenomics is a new field, but we intend to do all we can to use it to promote the development of medicines. By providing practical guidance on how to turn the explosion of pharmacogenomic information into real evidence on new drugs, we are taking an important step toward that goal."
Pharmacogenomics deals with the small genetic differences that help explain why some people respond positively to a drug, while others don’t respond, or may experience a side effect. Genetic differences also can predict variations in drug metabolism—how quickly or slowly a drug is eliminated from the body. In the draft guidance, FDA said that the promise of pharmacogenomics lies in its potential ability to individualize therapy by predicting which individuals have a greater chance of benefit or risk -- thus helping to maximize the effectiveness and safety of drugs. FDA believes that pharmacogenomic testing can be smoothly integrated into drug development processes.
This is FDA’s first step towards integration of this new field into the process of demonstrating that new drugs are safe and effective, and thus the regulatory guidance is intended to facilitate this integration. This guidance is intended to ensure that evolving regulatory policies and study designs are based on the best science; provide public confidence in this new field where scientifically appropriate; facilitate the use of such tests during drug development; and clarify for industry what types of pharmacogenomic data to submit to FDA.
"Using genomic testing to guide drug therapy will constitute a significant shift from the current practice of population-based treatment towards "fine-tuning" individual therapy," said Janet Woodcock, FDA’s Director of the Center for Drug Evaluation and Research.
Currently, scientific understanding of phamacogenomics is most advanced in the drug metabolism area, and early results are expected in this field. However, FDA anticipates rapid evolution of additional uses. For example, it is hoped that pharmacogenomic testing will help identify cancers that have a high probability of responding to a particular medication or regimen. Pharmacogenomics may also be used to help track down the cause of certain rare, serious drug side effects.
The guidance provides specific criteria and recommendations on submission of pharmacogenomic data to investigational new drug applications (INDs) and New Drug Applications (NDAs) and Biological License Applications (BLAs). This includes information on what data is needed, and how FDA will or will not use such data in regulatory decisions.
Because there is a need for scientific exchange, the agency is asking for voluntary submissions of research information. This data will help FDA gain experience as the field evolves. In these cases, FDA advises sponsors to clearly label voluntary submissions; and the agency advises that it will not use information from voluntary reports for regulatory decisions. If a sponsor subsequently develops additional data that meet the criteria for submission for regulatory purposes, the Agency advises sponsors that such data should be submitted as explained in the guidance.
FDA’s Science Board recently (April 2003) endorsed FDA proposals to move forward with this guidance on assisting industry in deciding whether a submission is needed. In addition, FDA held public meetings and workshops in which the key issues for drug development were identified.
FDA plans to issue further guidance on co-development of pharmacogenomic tests and pharmaceuticals in the near future. The draft guidance on display today is available on FDA’s website at: http://www.fda.gov/cder/guidance/5900dft.pdf.
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Message posted by: Frank S. Zollmann
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