FDA Back in the News Sans Guidances

After a drought in terms of guidances and other regulatory documents, the FDA is back in the news in a big way in the week of May 8, although there are no guidances or other regulatory documents at play. It’s tempting to try to read a lot of things into some of these developments, but other developments seem to hint at a need for more investment for not necessarily more return.

Gottlieb; same old thing or something different?

Former FDAer Scott Gottlieb is the back at the FDA, this time in the commissioner’s chair, thanks to his successful navigation of the Senate minefield. The opposition to Gottlieb included allegations that he will run roughshod over the drug and device review processes, and that he is “a doctor with ties to the drug industry.” Of course, Robert Califf was also a doctor with ties to the drug industry, but try finding a doctor who doesn’t have those ties. FDA advisory committees wrestle madly trying to find such a unicorn so they don’t have to go through the irksome vetting process.

On the other hand, there are those who hail Gottlieb as a breath of fresh air and a much-needed source of level-headedness at the agency. The trade associations issue the usual bland statements such as “we look forward to working with” Gottlieb, but they are not publicly predicting any radical changes at the agency under him, and they shouldn’t.

Anyone who hopes Gottlieb will effect an immediate transformation of the agency’s outlook on product reviews or on commercial speech would do well to remember how much difficulty the agency’s managers have had with the rank-and-file in times gone by. We might recall the letters from device reviewers to the Obama administration and a couple of members of Congress back in 2009, which stirred the political pot quite vigorously. However, the accompanying allegations of regulatory misconduct never quite stuck, and those letters – replete as they were with trade secrets – cost more than one FDA employee their job.

Nonetheless, it took quite some time for the agency to root out those problems, so there are many reasons to be skeptical about an impending change of “culture.” Jeff Shuren, director of the FDA device center, pointed to this cultural difficulty in a recent hearing regarding the user fee agreement, and he should know. He took over the directorship of the Center for Devices and Radiological Health shortly after the FDA renegades started stirring up controversy eight years ago, and despite industry’s misgivings about him in the first couple of years, Shuren was in no mood for the misadventures of these malcontents. Still, it took some time before he was able to give anyone the heave-ho.

User fee bills passed

The Senate HELP Committee put the user fee legislation in a nice, tidy bundle for consideration of the full Senate thanks to a May 11 markup, but there is one provision therein that might have some interesting effects on clinical trials in the years to come.

Sen. Orrin Hatch of Utah proposed an amendment that would require that the FDA mandate that clinical studies both offer greater access to several demographics, including infants and children, while also stipulating that clinical study enrollment more accurately reflect the population that would receive the treatment in question. This amendment, which passed on a voice vote, will also streamline IRB review of individual petitions for access to an investigational product.

It might be argued that there’s a tension of sorts between broader access and ensuring that enrollees reflect real-world usage, but either way you cut it, enrollment in clinical studies seems certain to increase. Needless to say, this could substantially increase the cost of a clinical study, which in the case of some medical devices can easily exceed $50,000 per enrollee.

It’s not clear how this pays off for industry, however, given that the additional enrollment required by the FDA might be of patient subgroups that exhibit a differential response. Supposing this subgroup experiences a lower therapeutic effect than the patient population as a whole, but that the sponsor cannot enroll enough members of this sub-group to allow for a separate analysis?

As for access by individuals, one has to wonder whether IRBs will be swamped with individuals applying to take part in a study for which they might not be a natural fit. Patients aren’t exactly bashful these days, after all, and it does not stretch the imagination to think that IRBs end up with a lot more work to do. Will the additional workload interfere with an IRB’s review of a drug or device study proposal?

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