BETHESDA, Md.—In March, the FDA's Center for Drug Evaluation and Research (CDER) released the long-awaited report "Guidance for Industry: Pharmacogenomic Data Submissions", which offers the regulatory agency's thoughts on voluntary genomic data submission as a component of the drug and biologics review process. The goal of the report is "to facilitate scientific pro-gress in the field of pharmacogenomics and to facilitate the use of pharmacogenomic data in drug development."
According to Dr. Felix Frueh, associate director of genomics at CDER, there has been a lot of uncertainty in the drug industry of what was going to happen when pharmacogenomic data was submitted to the agency. The guidance document, he explains, is designed to provide clarity and to open new avenues of discussion between sponsoring companies and the FDA, without making a regulatory commitment or taking regulatory action.
"You might have exploratory data that was used in development that is of great interest to us so that we can get familiar with that type of information," Dr. Frueh says. "This gives sponsors an opportunity to learn about how the FDA is thinking and facilitate the movement forward of the development process. The advice we're giving is of a scientific nature, not a strategic one.
"If, however, that information is being used by the sponsor as a pharmacogenomic biomarker for stratification purposes in a clinical trial, then the data would become a required submission," he adds. Thus, the guidance describes what kind of genomic information needs to be submitted and when, and drug companies can use one of three decision trees to determine the correct course of action when considering an IND, NDA, or BLA.
The report has been long in coming and industry representatives have repeatedly expressed their dismay at the delays. The final report, however, has met with significant and vocal endorsements from drug development companies, such as Genaissance Pharmaceuticals, which called the document "a significant step forward for the pharmacogenomics industry, and provides a clear path for how to gain marketing approval of drugs targeted by an individual's genetics."
Not surprisingly, pharmacogenomics suppliers and service companies have expressed their pleasure with the document. In a press release issued by DxS, a genotyping service firm, company CEO Dr. Stephen Little states: "Clearly, the publication of this Guidance Paper will not only have a major impact on how pharmaceutical manufacturers and CROs conduct drug development programs and clinical trials, but also the whole future of 'personalized' medicine."
According to Anne Anscomb, a drug development analyst for the market research firm Kalorama Information, the new FDA regulations can be viewed as an endorsement of pharmacogenomics as one way in which companies can hope to develop safer and more effective medicines. Not only will pharmacogenomic data increase the industry's confidence level in pursuing marketing approval of targeted medicines based an individual's genetic makeup, she explains, but also pharmacogenomics will evolve into standard practice.
"I think that pharmacogenomics data provides a tremendous opportunity for the marketing and selling of these drugs because if you have a means to identify responders, you get a better product," Frueh adds. "It makes for much better science and better healthcare and reduces the costs associated with the treatment of adverse events and the withdrawal of drugs from the market."
