Former deputy FDA commissioner Scott Gottlieb left the agency not long ago, returning to the American Enterprise Institute (AEI). Since leaving FDA, the prolific Gottlieb has written a large number of essays and articles for a variety of publications. We’ve got one of them in PDF format below.

ClinPage heard Gottlieb at the recent BIO show in Boston, held during early May. He’s a nimble, lively speaker. There is some journalism on Dr. Gottlieb’s lengthy curriculum vitae (for JAMA, the British Medical Journal and Forbes), but who’s perfect?

FDA speakers reflexively use disclaimers to pre-disavow anything they utter, the better to avoid making policy from the podium. Gottlieb, of course, was under no such constraint. That was refreshing.

Underfunded FDA

Gottlieb bluntly said Congressional funding is insufficient. “The agency’s funding stream is barely enough to keep the lights on,” he said. The absence of adequate funding, he said, is hardly a secret to the physician-scientists the agency would most like to recruit. “It’s extremely detrimental to your ability to recruit good people,” Gottlieb said of the miserly FDA budget. “It’s not going to engage the high caliber scientific minds who want to be leaders in their field.”

Toward Collaborative Decisions

Gottlieb’s remarks provided a peek into some of the intra-office dynamics at the FDA. Inexorably, Gottlieb argued, the FDA will need to move to a more group-based decision-making model.

That will minimize the role of one seasoned, omniscient reviewer.  “More of the things need to be made by collaborative teams, not by individual reviewers. Industry moved to this years ago,” Gottlieb says. “FDA will have to approach that.”

Marginalization

Gottlieb immediately concedes, however, that such an approach may leave some at the FDA feeling ignored. “Sometimes that creates people who feel marginalized,” he said. Such hard feelings are especially likely, Gottlieb said, if the FDA staffer in question is not a physician or a specialist physician. Such sentiments can create problems if such individuals find their way to a hostile congressman or the media. 

Gottlieb gently mocked the performance reviews at the FDA (he said everyone at the FDA receives “star, star, star”). He suggested that more stringent reviews, by scientific criteria, could begin to resolve some of the internal FDA dissent that has begun to leak out of the agency with more regularity. Some dissent will always be unfolding at the FDA, Gottlieb suggests, and some is worrisome. “There [are] always cultural or scientific battles taking place inside the agency. You’re starting to see those percolate out publicly.”

Dealing With Dissent

Of course, the people offering that dissent feel that lives are at stake, and it’s entirely possible that efforts to ease them out of the agency could backfire or run afoul of civil service and whistleblower regulations. 

Gottlieb is also concerned about proposed legislation. The current zeitgeist at the agency, the unstated bias, is against primary care physicians—and for specialist physicians. “FDA is mostly specialists,” he says. “There are not a lot of primary care doctors.”

Primary Care Prejudice

In the case of a drug like Smylin, from Amylin, the constraints placed on physicians effectively barred it from being prescribed by primary care doctors. Why? Partly because of a requirement for a certain type of nurse. Says Gottlieb, an internist: “The practical effect of that risk management plan was to drive that drug out of the hands of primary care physicians.”

That troubles Gottlieb on two fronts. First, as a physician, he’s appalled that many patients without access to specialists will not get access to the medicines they need. Second, as a political matter, he’s dubious of the government making access decisions correctly. “You really start to create obstacles to innovative therapies,” he says.

Expanding Federal Power

In the attached article, published in Health Affairs, Gottlieb writes:

“To ensure and document compliance with distribution restrictions, they would have to create entire audit and compliance departments devoted to policing doctors and pharmacies, and the FDA would have to watch the watchers, which would require it to regulate the medical practice environment and drug distribution channel. Even if drug companies could maintain tight control over the distribution of certain drugs by dispensing them directly or supplying them only through certain pharmacies and by monitoring how doctors prescribe them, such measures would inevitably limit access to drugs.”

It’s not controversial that Gottlieb thinks the nation and the FDA need a better system to monitor drug safety. But he seems to minimize the role of marketing in stimulating demand for drugs from primary care physicians who may not understand them as well as specialists. With luck, we have no primary care physicians who will be reading the site today.

Indeed, the risk management plans in the proposed PDUFA legislation seem to be acceptable to both political parties and to industry, to the extent lawyers can wrap their minds around such nuances at all.

Prescribing Pitfalls

For Gottlieb, of course, one central issue is the medical community’s right of self-governance. Writes Gottlieb in Health Affairs: “There is an inherent fallacy in the belief that federal authorities can reduce medical side effects by promulgating rules and requirements that create very prescriptive scenarios on how drugs get prescribed.”

It seems to border on the ludicrous, Gottlieb says, that the FDA alone is blamed for drug safety issues. In fact, he says, the medical community and medical publications may be able to clarify some of the risks of drugs. So we concede Gottlieb’s point on one key issue. The proposed new policies in PDUFA could be a departure from current practice: they chip away at individual physician autonomy and the existing state-level authority over medical practice.

Here’s an earlier ClinPage story about BIO 2007.