FDA wants to be judged on its overall impact on public health - not on drug approvals - Commissioner Margaret Hamburg and Principal Deputy Joshua Sharfstein say in an ¹ editorial published in the New England Journal of Medicine May 26.

"The ultimate measure of FDA's success should reflect its fundamental goals and go beyond such intermediate measures as the number of facilities inspected or drugs approved," they contend.

In other words, the agency's new leadership team says, public health goals come before user fee goals.

For biopharma companies desperate for a rebound in chronically low new drug approvals, those may sound like discouraging words.

However, there is more than a glimmer of hope in the discussion of product reviews included in the editorial.

"Too Fast" or "Too Slow"?

The section begins with an even-handed discussion of the perception that "FDA has just two speeds of approval - too fast and too slow."

"Critics concerned about haste point out, accurately, that drugs and other products are generally approved on the basis of relatively small studies and that safety problems often emerge when large populations are exposed to the products," the editorial states. "Those worried about delay note, correctly, that people with life-threatening diseases have no time to wait."

"A public health approach recognizes that the potential good of a new medical product or policy must be balanced against the potential harm," Hamburg and Sharfstein continue.

"Some benefits are not worth the risk; some risks are worth taking."

The even-handed tone may frustrate some in industry, who find the notion that FDA ever reviews drugs "too fast" to be laughable.

However, in the context of the criticism registered against the agency and the user fee system - prominently in the New England Journal of Medicine itself - those neutral-sounding statements read more like quiet assertions that taking risks on drug approvals is necessary and important.

Already De Facto Policy

For one thing, the idea that FDA will be putting a broader public health mission ahead of the review deadlines and other goals set by the Prescription Drug User Fee Act is nothing new: that has been the operating policy of the Center for Drug Evaluation & Research for the past 18 months (" ² The New User Fee Rules ," The RPM Report, March 2008).

So sponsors may want to focus on the glass-half-full aspects of the editorial as an indication that the new leadership embraces the idea that moving too slowly on reviews can be bad for the public health.

And, most importantly, the editorial strongly suggests that FDA is ready to take more risk on approval decisions - something made much easier in a climate where FDA's leadership is aligned with the agency's congressional overseers as closely as is the case with Hamburg and Sharfstein.

"FDA must make difficult decisions in the absence of ideal information," the editorial states.

Hamburg and Sharfstein, though, make it clear that not every new product deserves the benefit of the doubt.

"Key considerations are the severity of the illness at issue, the availability of alternative treatments or preventive interventions, and the current state of knowledge about individual responses," they say.

FDAAA Makes Approvals Easier

But they also highlight the new FDA Amendments Act authorities as a source of greater confidence to allow more approvals.

The new regulatory tools "allow FDA opportunities to change the regulatory oversight of products as they move from limited use in clinical trials to adoption in the medical system," they write. "The ability to detect and act on safety signals quickly can give an additional layer of confidence to support earlier approval of important medications."

The editorial also includes a tantalizing hint that FDA under Hamburg will define its mission as doing more than just approving important new therapies, but also encouraging their uptake in the marketplace.

"To make important new treatments available to patients, the FDA should collaborate with the Centers for Medicare & Medicaid Services ... as well as with industry and patient and consumer groups to explore ways of shortening the time from approval to reimbursement," they say.

That sentence comes amid a broader discussion of the importance of collaboration and reads almost like an afterthought to more fleshed out ideas for working with NIH and CDC (see ³ Also see "Hamburg/Sharfstein Agenda For FDA Emphasizes Collaboration" - Pink Sheet, 1 June, 2009.).

The call for closer coordination between FDA and CMS has been a recurring theme for more than a decade - and one that sometimes sounds threatening to industry.

However, the goal outlined in the Hamburg/ Sharfstein editorial suggests that revival of that idea may present an opportunity to simplify the launch process for new technologies.

And it gives industry another reason to be optimistic that the public health focus of the new FDA leadership can be compatible with commercial success.

- Michael McCaughan (m.mccaughan@elsevier.com)

Public Health Trumps User Fee Goals, FDA Leaders Say – But That May Be Good News For Sponsors

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