Describing the current regulatory environment as a “unique window of opportunity” for diagnostic-therapeutic companion products, Steven Gutman, director of the US Food and Drug Administration’s Office of In Vitro Diagnostics, said an upcoming draft guidance on these so-called theranostics is a “high priority” at the agency.
Gutman also said that his group, which is writing the draft with the FDA’s Center for Drug Evaluation and Research, also is “really quite willing” to work together with CDER in cases when two corporate partners wish to bring a theranostic to market.
However, Gutman, speaking with SNPtech Pharmacogenomics Reporter last week, declined to say when he thinks the draft will be released. Janet Woodcock, director of CDER, said in January the draft would become available in the fall [see 1/15/04 issue].
Following on the heals of the FDA’s draft guide on the submission of pharmacogenomics data and one on developing and marketing multi-analyte-based diagnostic tests, the theranostic document under development is aimed at meeting head-on the rise of these companion products. The draft is also another tool that may help the agency clear a regulatory path to pharmacogenomics.
Indeed, Woodcock had described the document as the logical next step in the FDA’s expanding grasp of how pharmaceutical companies are using pharmacogenomics technologies in drug discovery and development.
Woodcock said the new document will focus on “joint development” issues behind theranostic models. “We kind of promised it in [the November pharmacogenomics] draft,” she said.
For example, the draft will seek to answer “What do you actually do if you are developing a test and a drug in parallel, and you want them to come out even at [the regulatory] end … [where] the drug label says, ‘Use this test,’” Woodcock said. “So we want to explain how that would work, what you should do, and what studies you should do ... so that people have a clear path,” Woodcock added.
According to Gutman, one “really important” objective of the draft is to “raise sensitivity to the fact that if in the course of developing a new therapy, if a new diagnostic is actually going to move from the discovery stage to actually be used to select patients or to dose patients, then it would be better to have upfront attention to both issues — the therapeutic and diagnostic issues — in a sort of parallel manner,” he said.
“So a good thing to do” would be for companies developing both diagnostic and therapeutic components “to approach both centers,” said Gutman, referring to CDER for therapeutics and the Center for Devices and Radiological Health, which oversees the OIVD, for diagnostics. Gutman also said if two companies are collaborating on the theranostic, they should be aware that CDRH and CDER “are really quite willing and able to coordinate reviews, to coordinate panel meetings, to share science.
“There is a unique window of opportunity to address … single studies or very closely linked parallel studies ... to bring both products to market,” he added.
Gutman said the companion products he and Woodcock envision would not include a combination of a therapeutic and an analyte-specific reagent — say, a therapeutic that would be marketed alongside an ASR that helps to determine the way in which the patient would metabolize the drug.
“The ASR has a different twist,” he said. Today, “an ASR would not require FDA pre-market approval — it’s sold as a home brew — which assumes there is no interest in commercially marketing a device.” This contradicts what Woodcock said in January, that “any kind of pharmacogenomic diagnostic” will be allowed. However, it was not known whether the discrepancy was intentional; Woodcock was not immediately available for comment.
Gutman maintained that a “good but imperfect example” of a typical theranostic would resemble the Herceptin/HER test model. Gutman said that FDA panel meetings reviewing Herceptin and its companion HER test were held two days apart “with some members cross-participating … to make sure the diagnostic and therapeutic device were appropriately linked. That’s an example of what we’d like to see more of,” he said. (This goal will likely be a boon for Roche Diagnostics’ new Synergysdx division, which was designed to use the company’s Herceptin experience to create additional theranostic products for itself as well as for drug makers without access to in-house diagnostics R&D infrastructure [see 6/13/03 SNPtech Pharmacogenomics Reporter.)
However, he stressed, this method of “in parallel” filing is not meant to officially supplant existing methods. “You don’t have to do it this way; you don’t have to coordinate your studies,” he said. “We do have products where the therapeutic comes first, and where they then put diagnostics after the fact.
“There’s nothing mandatory about having highly efficient and coordinated studies,” he quipped. “You’re allowed to do this in whatever [way] makes scientific or business sense. It just makes sense to do it this way.”
The new theranostic guidance will also become the latest stepping stone in the agency’s goal of regulating the collection and analysis of microarray data as an increasing number of drug makers begin using the technology more earnestly in their drug discovery and development research.
“We’re working closely with (OIVD on the composition of the draft molecular diagnostics guidance), and what we’re going to do is work on a joint (draft) on drug development and pharmacogenomics tests together,” Woodcock told SNPtech Pharmacogenomics Reporter in January.
She said the agency has not determined when the new draft might be released for comment, but said a final version might be ready in the fall. “We started … the process, but it’s going to be a while,” Woodcock said. “It’s going to be much simpler than” the pharmacogenomics draft guidance.
Woodcock said the new document will focus on “joint development” issues behind theranostic models. “We kind of promised it in [the November pharmacogenomics] draft,” she said. For example, the draft will seek to answer “‘What do you actually do if you are developing a test and a drug in parallel,’ and you want them to come out even at [the regulatory] end … [where] the drug label says, ‘Use this test,’” Woodcock said. “So we want to explain how that would work, what you should do, and what studies you should do ... so that people have a clear path,” Woodcock said.