Over a hundred public companies, dozens of clinicians, and several regulators participated in investment firm Leerink Swann’s 2012 Healthcare Conference in mid-February. Press was barred from the event, but John Sullivan, Leerink’s director of research, gives PharmExec a few of the take-aways.
Everyone knows that Dr. Janet Woodcock, CDER’s director, tends to stress the importance of safety as FDA’s top mission when speaking to a public audience. During a keynote address at the Leerink Swann Healthcare Conference in February, she also spoke about the importance of transparency and communication at FDA, specifically with respect to smaller biotech companies.
John Sullivan, Leerink Swann’s director of research, was intrigued by Woodcock’s comments. With an increase in the number of smaller companies, including small biotechs, appearing before the FDA as drug sponsors, Woodcock acknowledged that a different method of interaction, with different protocols, is necessary. “When your customers are big drug companies, they’re more likely to know the language…you have career regulatory officers on board internally, and a lot more shots on goal,” says Sullivan. “You can send them away with a previously unclear piece of guidance that hasn’t been clarified, and they’ll go do more work, confident in the knowledge that they’ll be back in front of the agency with that candidate or another one. They have a certain way of interacting that’s long-term in nature.”
With smaller companies, like emerging biotechs, however, the drug candidate up for review may be a company’s only product, or one of a very few. “There’s a heightened level of urgency that I think the FDA is now recognizing,” says Sullivan. “It sounds like they are taking specific steps to make themselves more available to smaller drug sponsors, to give them more guidance.” Hopefully, this will help to minimize the risk of a company going out and conducting new trials, only to reappear two or three years later to find out that the trial wasn’t what the FDA wanted, in terms of design, says Sullivan. “I took [Woodcock’s comments] as a positive at the margin. That doesn’t mean that this class of emerging biotechs will get more approvals out of FDA, but hopefully they’ll get the right answers, perhaps a bit sooner and for a bit less money, than what they might have anticipated.”
Woodcock has also been an outspoken proponent of drug and diagnostic combinations, and in addition to panels on new prostate cancer treatments, duel eligible and healthcare exchanges, biosimilars, rheumatology and other topics, Leerink also convened a panel on clinical DNA sequencing. The panel focused on the migration of sequencing toward the clinic. The panel consensus, according to Sullivan, was that DNA sequencing would inevitably come to the clinic, but not before two things happen: “The price has to come down, and the utility of the DNA sequence information, to the clinician and his or her patient, has to go up.” Per Sullivan, more links between genetic presentation and predisposition for disease, or response to therapy, need to be made. The platforms by which cancer diagnosis and treatment are used today are “relatively low-density…they can do maybe ten genes or 50 genes,” says Sullivan. With full genome sequencing, researchers can do tens of thousands of genes at once, but “most clinicians don’t need, nor do they know what to do with that volume of information.” Panelists said that as clinicians learn more about genetic information, and the price of a full scan comes down, patients will start to be scanned more often.
“I think there was broad agreement that DNA sequencing, especially single-molecule sequencing – where you don’t have the risk of mistakes as you amplify the amount of genetic material for analysis – really holds the promise of being more accurate than other methods of interrogating DNA and RNA, so it’s attractive from the standpoint of physicians, who want to make sure they’re giving the right answer. DNA sequencing, especially single molecule sequencing, increases those odds,” says Sullivan.
On the issue of price, Illumina and Life Technologies announced in the respective launches of two platforms that can sequence an entire genome for $1,000, in one day’s time. Sullivan called Illumina, which is currently subject to a hostile takeover attempt by Roche (the bid is currently $5.7 billion), a “leader in research-based DNA sequencing today.” Ion Torrent, acquired in 2010 by Life Technologies (which was itself formed through the merger of Invitrogen Corp and Applied Biosystems, in 2008) released a mock-up photograph of a clinical DNA sequencing instrument small enough to fit in the hand of a physician. While still a ways off, the photo suggests DNA sequencing in the clinic will require a set of tools that differs from what’s currently used in the lab, by researchers, says Sullivan. “Clinicians and clinical lab staffers have very different needs than research lab PhDs,” he says. “As far as the drug industry is concerned, they’ll continue to get good ideas for new products from the genetic research studies that are going on in a million academic research labs all over the world, and they will increasingly look to pair their drugs – whether genetically derived or not – with diagnostics.”