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QA: FDA Says No New Regs in Sight


No matter how the market flourishes or how promising a technology’s outlook, there’s still one thing that can bring even the most high-flying genomics execs to their knees. It’s the US FDA, headed up by Bernard Schwetz, acting principal deputy commissioner. His years of toxicology research experience have prepared him for what may be the coming onslaught of genomics-based drugs. He recently agreed to an e-mail interview with GT’s Meredith Salisbury.

If a genomics-based drug came out right now, would the FDA be ready to look at it?

Schwetz: The FDA understands well the issues surrounding gene expression and the genomic basis for new drugs. Over the years it has reviewed applications related to gene therapy and drugs based on genetically engineered proteins.

What kind of regulations would govern this kind of drug, and how long would it take to get approval?

Schwetz: The current regulations would suffice in governing genomic-based drug applications. However, because this is an evolving area, the FDA may find that issues emerge that are either not explicitly stated in the regulations or will require guidances for industry to explain the current regulatory thinking on these issues.

NDAs [New Drug Applications] for genomic-based drugs could go through the accelerated approval process, or be deemed a priority application. Conventional review times would apply, as they would for traditional assessments of NDAs. The time to complete the review is related to the quality of the data in the submission.

How do you expect the regulations to evolve?

Schwetz: Current FDA regulations exist to allow for genetically defined subgroups of patients. In the past, INDs [Investigational New Drug applications] and NDAs have been submitted where the target patient populations were defined on the basis of gender, race, or gene expression levels. We don’t think new regulations are needed, although as new issues emerge, clarification of the regulations may be necessary via domestic or international guidances. Some examples of need might be to clearly define the attributes and validation of an ideal genetic/genomic probe, and to what degree it is expected that genetics/genomics predicts the variability in clinical response to drugs.

People in the genomics industry worry that the FDA would require approval of technology much further upstream than they’re prepared to account for. Just how far upstream will FDA go?

Schwetz: Sponsors are responsible for the quality, integrity, and credibility of their data. The standards for genetic and/or genomic tests that are prerequisite to determining the strength of evidence to support regulatory approval are emerging and may need further discussion with sponsors. FDA generally will advise a sponsor on their drug development plans to impart learned information or to avoid unnecessary clinical trials. Sponsors are responsible for validating bioinformatic information that may be obtained from a contract facility.

What’s your advice to companies in the genomics field that are considering going into discovery? What about to pharma companies developing drugs based on data from these companies?

Schwetz: FDA acknowledges that genetics/genomics is an important new area that may positively affect public health. Thus, the agency encourages sponsors to explore this technology systematically and with rigor to assure quality data. This is an important new area: pursue it, build in quality processes and oversight procedures, and assure the validity of information. Early and frequent communication between the FDA and the sponsor will be important to identify new or unique issues related to drug development.

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