It’s official: pharmaceutical companies can finally submit genomic data with their drug applications and not have to worry that it will be used against them.
In a move that was welcomed by pharmaceutical and biotech companies, the US Food and Drug Administration released for public comment last November its draft guidance on the submission of pharmacogenomic data.
The guidance document is also good news for informatics firms who plan to bridge the genomics knowledge gap between pharma and the FDA. Just as an entire cottage industry of contract research organizations sprang up to assist pharma with the clinical trials process, some informatics companies are positioning themselves to act as analytical go-betweens once the pharmacogenomics data submissions come pouring — or even trickling — in.
The draft guidance lays out the fundamental conditions for submission of such data derived from gene expression or SNP-genotyping experiments as part of the regulatory process. More importantly from the perspective of reluctant pharmas, the guidance explicitly spells out the conditions for voluntary submission of pharmacogenomics data.
Seeking “to be prepared to appropriately evaluate the anticipated future submissions,” the FDA wants to test out as much experimental data as possible. To encourage submissions, the agency established the new VGDS category — Voluntary Genomic Data Submissions — and created a cross-center Interdisciplinary Pharmacogenomic Review Group to review it, work on ongoing policy development, and advise decisions regarding pharmacogenomics data.
“The FDA will not use information submitted through the voluntary process for regulatory decision-making on INDs or NDAs,” the guidance document states. Instead, the agency “intends to gain experience and to develop an aggregate genomic knowledge database from multiple VGDSs that could be used to rationally facilitate the use of pharmacogenomics in drug development and to share what general knowledge is learned from the data repositories.”
The draft is a “great start,” according to Les Browne, COO of Iconix Pharmaceuticals. “It’s really a document they’re putting out there to stimulate discussion … and it starts the process of getting genomic data into the drug evaluation and approval process.”
The comment period for the draft guidance closes February 2.
Giving Birth to the Genomics CRO
Doug Dolginow, senior vice president of pharmacogenomics at Gene Logic, says the guidance should encourage pharmaceutical and biotech firms “to immediately begin to generate data in the preclinical and clinical areas using genomic technology … without having a negative regulatory or unexpected regulatory impact on that.” The upshot, he says, is “quite enabling for companies like ours.”
Gene Logic acquired CRO Ther- Immune earlier this year in anticipation of genomic data being fed into the regulatory process. The FDA’s blessing “makes it a little bit easier to conduct our business,” Dolginow says, especially “now, as a CRO-based company where we’re providing submissions to the FDA and working with our customer base to do that.”
Enodar BioLogic is also staking a claim in the territory between pharmaceutical firms and the FDA, and already calls itself a “GCRO,” or genomics CRO. Lue Ping Zhao, Enodar’s CEO, says he gives the guidance document “high marks” for striking a balance between “regulation and flexibility.”
No Standards Required?
But Kurt Jarnagin, vice president of biological sciences and chemical genomics at Iconix Pharmaceuticals, questions whether the FDA’s avoidance of formatting or standards issues would discourage submitters.
According to the guidance document, the FDA is not offering recommendations for specific formats for the VGDS because “consensus standards do not exist for presenting and exchanging genomic data.” While Jarnagin agrees that the standards issue is a tricky one, he notes that with the decision to avoid the issue, “the agency is taking on the onus of accepting the entire variety of formats that might arise.”
Jarnagin would like a “standard input tool” — along the lines of Sequin for Genbank submissions — that would make it easier for drug companies to submit their data. Nevertheless, he says, “We’re delighted to see it. Any progress is better than none.”
An expanded version of this article originally appeared in the Nov. 10, 2003, edition of BioInform.
Bernadette Toner is editor of BioInform, a weekly newsletter from GenomeWeb at www. bioinform.com. She can be reached at btoner @genomeweb.com.