Is FDA's senior leadership paralyzed by internal fights over the science and process of new product approvals? You wouldn't have thought so in 2011 after the agency approved 35 new medicines, the highest since 2009. In 2012, the agency also managed to adroitly shepherd a new user fee agreement through Congress, fending off provisions from industry and advocacy groups that it didn't like.
But there are other worrying signs that all is not well at White Oak - at least in the medical devices division. Recent revelations that the FDA had been monitoring the emails of self-described "whistleblowers" who leaked confidential company documents to the New York Times call into question the ability of senior staff to enforce agency approval decisions - at least without a media firestorm.
This concern is explicitly raised today by Jon Entine in a Forbes article dissecting the whistleblower suits:
The [New York Times] and many activist groups are now portraying the self-proclaimed whistleblowers as beleaguered heroes and victims of an agency "enemies list" designed to muzzle public minded employees. They hint at a corrosive and corrupt culture inside the FDA that is captured by big business and limits the agency from encouraging dissent.
But now, as the backstory is coming into sharper focus, it appears that culture is far more nuanced. The FDA appears not so much closed as split between various factions, with a minority of junior scientists determined to push an ultra-aggressive regulation strategy even after higher level science reviews consider but reject their input. This grueling internal battle appears to have left the CDRH, the FDA office empowered to oversee innovative medical technology devices, including genetic tests, in disarray. ...
The latest FDA black eye highlights an internal battle inside an agency struggling to balance a commitment to science with an intensely politicized decision-making process. As Steve Usdin reports in a superb deconstruction of the scandal in BioCentury, the origin of the dispute seems to reflect internal conflicts that often occur in agencies with many employees holding differing scientific and ideological agendas. ...
"If junior staff can always go around senior management when there is disagreement, and successfully use politicians and the media to have decisions overturned, then junior staff are running the organization by default," [Usdin] writes.
To be fair, the problem is that the FDA is tasked by Congress with inherently conflicting roles, both setting the standards that define how new products are evaluated as safe and effective, and then reviewing and approving the data submitted by companies that purports to meet FDA criteria.
Both roles require nuanced judgments and trade-offs in a rapidly evolving scientific environment that don't lend themselves to an all-or-nothing regulatory approval process - especially in today's hyper-partisan, 24-hour media environment, when the losing party at the agency can go to Congress or the media to plead their case.
Scott Gottlieb, a former FDA Deputy Commissioner, looks at the same phenomenon from the perspective of drugs rather than devices in a recent National Affairs article.
Conflicts are inevitable, Gottlieb believes, when "the same individuals simultaneously [play] the roles of detective, judge, and jury when it comes to considering new drugs for approval." Given the conflicting demands placed on FDA staff, the natural tendency is to delay product approval in favor of more data not just on the product's safety, but also its efficacy and utility, i.e., how it can best be used in clinical practice.
More data will protect the FDA against charges of favoritism from its critics on the left and the right, or at least the agency hopes that it will. Gottlieb thinks this ill serves the interest of patients, who have to wait longer for new products to come to market. It also doesn't seem to insulate the agency from criticism - or quell its own rancorous internal disagreements.
Gottlieb suggests that one way to cut this Gordian knot would be to separate the collection of and review of data on product safety and efficacy and let an internal committee of FDA's senior staff or scientists make the final call on product approval - thus embracing the reality that balancing the risks and benefits (and uncertainties) associated with new technologies is a policy decision, not a black-and-white exercise in number crunching where differing opinions can be dismissed as the product of industry influence or outright corruption. Gottlieb believes that this change would allow the agency's reviewers to become more flexible and innovative, since the final decision for product approval or denials wouldn't rest with them.
I think this makes eminent sense, but it's also worth noting that the agency's schizophrenia about medical products is reflected broadly in popular culture and politics. Drug and device companies are simultaneously lauded as the saviors (seeking the next cure for cancer) and villains (driving up health care costs and hawking unproven products). In short, Congress and the FDA take their cues from a public that wants ever more and better health but is unwilling to tolerate any risks from medical innovation.
That's a recipe for dysfunction.
Contrast this with the rapid innovation in consumer products (particularly software and IT) where Americans have broadly made a choice for surrendering more privacy - to Google, Facebook, et al - in return for a better suite of products and services from industry. Concerns about industry's potential abuse of privacy have (largely) failed to materialize since companies recognize that abusing that trust would be tantamount to financial suicide.
There may also be other lessons to be learned from the IT sector. The wired world offers a potential paradigm for how health care companies can offer improved services and tailored products to consumers, and a new venue for testing and evaluating competing product claims in close to real time through carefully designed electronic medical records. In this world, patients and physicians don't need and shouldn't expect certainty - but they should expect better information and more informed choice.
That role - as America's "chief information officer" for health care - is one that the FDA could embrace, and Alex Tabarrok offered one vision of this approach in an earlier post on MPT.
But first we'd have to recognize that the uncertainty we abhor lies not with our officials or our institutions, but in the very nature of the endeavor to tailor treatments to vastly diverse patients under many different circumstances. This may be one of the few ways to end the schizophrenia that we've imposed on the FDA - and on ourselves.