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FDA Preparing Draft Document to Help Guide Microarray Data Consideration


The Food and Drug Administration expects to release within 60 days a draft guidance document on how to bring microarray data closer to the drug approval process.

“The agency wants to create an alternative path for this data, to remove the uncertainty, and encourage companies to explore it,” Lawrence Lesko, director of the FDA Office of Clinical Pharmacology and Biopharmaceutics, told BioArray News in a telephone interview.

The OCPB supports the FDA's Center for Drug Evaluation and Research's review programs and serves as a scientific advisor on policy issues.

The proposed document will detail a process wherein genomic data derived from microarray analysis can be shared and evaluated by the agency in a track outside of the traditional drug approval process.

Last week, at the Lehman Brothers Healthcare Conference in Miami Beach, Steven Galson, the deputy director of the FDA Center for Drug Evaluation and Research, made the agency’s first mention of progress in this arena in a keynote to the pinstripes of the financial industry.

“We want to make sure the data wouldn’t affect the drug review process unless the sponsor and this review group would agree that the data would in some way affect the review,” Galson said. “If after looking at it for a few months, if everybody agrees on what the data means, then we can apply it to helping with a disease claim or a safety claim.”

This safe harbor idea was first floated in May at a two-day workshop held at the University of Maryland, sponsored by the FDA and the Pharmaceutical Research and Manufacturers of America (PhRMA). The workshop was oversubscribed and some registrants had to be turned away, illustrating the pointed interest in this topic among those in the pharmaceutical and microarray industries.

That fact that it has taken almost a year for the FDA to get to the point of a draft document illustrates the pace of regulatory action and some of the pressures that industry, and the FDA, are feeling for shortening the approval process, without creating additional risk. A Tufts Center study released this week said mean approval time for biopharmaceutical products, from 2000-2002, was 19.7 months

Industry is concerned that without some sort of institutional guidelines for handling microarray data and recognizing its experimental nature, this data might cause the agency to order new clinical trials on a drug, or even stop a late-stage trial — both very expensive propositions.

There are other questions too, such as: Who will review the data in the agency? And, will reviewers have expertise in the field? Then, once data is in this safe harbor, could it then go into the review process, and how?

“We haven’t addressed those questions very clearly yet, that’s what we have to address in the next couple of weeks,” said Lesko. Once a guidance document is released, it will be made available for public comment, which will be followed by another industry workshop. “Our plan is not to formalize or complete any guidance without a public workshop.”

“I think everyone believes that (genomics data) has a huge potential,” said Lesko. “My feeling is that the technology to enable genetic and genomic approaches in drug development is here.”

Already, Lesko said the agency is seeing some microarray data being submitted under Investigational New Drug (IND) applications.

“There hasn’t been a huge amount of information coming in since last year, but what has come in has not been very helpful in terms of the review of any submission,” he said. “We have a long ways to go toward standardization, both in terms of the methodology of platforms, and also in the statistical methodology.”

The guidance document will target the pharmaceutical industry, and create an alternative venue for information outside the traditional FDA decision-making process, which now requires any research done under an IND to be reported to the agency.

“Safe harbor data is intended to facilitate the technology and its use in drug development,” said Lesko. This type of data, derived from microarray analysis, or single-gene SNP data, may not be intended by the sponsor for use in the IND process, as the data is exploratory, therefore not necessarily validated, quality controlled or conducted under the FDA’s standards for good laboratory practices.

Lesko said the agency needs to see more data to learn how to apply it in the process.

“It’s a chicken-and-egg thing,” Lesko said. “Until we begin to see the data, we are not going to be in [a] position to develop policies related to it. We hope safe harbor removes that barrier of uncertainty.”

Lesko said he first became aware of microarrays 10 years ago, but was not working at the FDA then and, “honestly, didn’t pay any attention to them.”

Lesko joined the FDA in 1992, leaving PharmaKinetics Laboratories of Baltimore where he was chief science officer. He holds BS and PhD degrees in pharmaceutics from Temple University

Today, he works with an intra-agency group that is considering the safe harbor guidance, and acts as chairman of another group that is working on genomics-genetics issues involved in drug regulation. There are almost monthly meetings.

If the road to getting microarray-based data considered officially in the FDA’s drug evaluation process is likened to Interstate 95, the highway that stretches from Florida north to the Canadian border, a traveler as of last year would be skirting the suburbs of Richmond, Va.

This year, that same traveler has made it to Washington, DC., but the final destination is Baltimore, another 50 miles up the pike, Lesko said.


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