PDUFA V: Washington's biggest bipartisan policy triumph this year.

The FDA's user fee legislation sailed through the Senate last week with enormous bipartisan support (92-4) - although you probably missed it since the vote was drowned out by the Supreme Court's decision on Obamacare two days later. BioCentury's Steve Usdin has a terrific article describing the key provisions of the legislation (subscription required).

The most important aspect of PDUFA V may be that Congress has signaled that the FDA should no longer be hamstrung by the chimera of "perfect safety" as it weighs the risks and benefits of new medicines. Usdin writes that

By enacting the FDA Safety and Innoavtion Act, Congress has explicitly embraced the notion that the appropriate goal of drug regulation is to ensure a positive balance of benefits and risks. It also has implicitly accepted the idea that the risks posed by the lack of therapeutic options must be part of the benefit/risk calculation. [emphasis added]

This is a vital message for the FDA to hear, since Congress has often sent the exact opposite message after post-market safety concerns emerged with drugs like Vioxx and Avandia.

In addition to ploughing $4 billion in user-fees back into the agency over 5 years (2013-2017), the legislation also contains a number of provisions designed to speed up drug development, improve agency transparency and communications with stakeholders, and advance regulatory science.

Among other things, it encourages the FDA to expand the use of Fast Track and Acclerated approval beyond HIV and cancer; elminates caps on conflict of interest waivers for FDA advisory committees; creates a new breakthrough therapies designation that is supposed to allow for expedited development and review of drugs for "serious and life threatening illnesses" that show significant improvement over existing treatments in early stage testing; expands PDUFA review deadlines by 60 days to allow for additional meetings between sponsors and FDA; and requires the FDA to establish a new risk/benefit framework for describing how reviewers are actually evaluating the benefits and risks of new medicines.

How successful will these initiatives be? No one can really say for sure. Skeptics can argue (plausibly) that the FDA already has all of the statutory power it needs to do these things already. But that may be beside the point. The "permission" from Congress may give the FDA's leadership the leverage it needs to push changes down to review staff. It will also fall to Congress to follow through with additional oversight to ensure that FDA meets Congress' goals for advancing innovation and patient access to more effective therapies.

To date, the agency is saying all the right things. "From the FDA's perspective," FDA Commissioner Margaret Hamburg emarked at the recent BIO meeting in Boston, "the critical challenge now becomes implementation...delivering both on expectations and delivering on the real-world opportunities that are presented to us through this agreement and reauthorization legislation."

There are other, broader reasons to be bullish on the future of medical innovation and more flexible regulation. The speed at which the science is advancing in fields like genomics is truly astonishing, and will only continue to accelerate as costs drop (faster than Moore's law).

First, like it or not, the U.S. will find itself in increased regulatory competition with countries in Europe and Asia to commercialize R&D investments and bring them to market as quickly as possible - or risk losing the jobs and tax revenues that come from those R&D investments.

Second, there is the real possibility that the FDA could find itself lagging behind the science of personalized medicine as hospitals and health systems, health IT companies, and drug developers mine new data made available by linking electronic health records with genomic and other data that can translate latent knowledge into improved treatment protocols, promising new drug targets/indications, and more personalized (and thus cost effective) therapies.

Take, for instance, this partnership between Oracle and Aurora Health Care in Wisconsin.

Three or so years ago, Alfred Tector, one of the state's pioneering heart surgeons, contacted Oracle Corp., the database software company, about drawing on Aurora Health Care's electronic health records to find potential candidates for clinical trials of new drugs and medical devices.

Last week, Oracle announced the result of that initial call: the Oracle Health Sciences Network.

The new service will enable drug companies and health systems to cull information, with patients' names and other identifying characteristics removed, from electronic health records and other databases to determine whether a health system has enough patients to participate in a clinical trial.

That initially could enable clinical trials to be done quicker. But, more importantly, if the service is successful, it could become part of the infrastructure needed to develop drugs targeted for patients with a specific genetic makeup and for other medical research, such as comparing the effectiveness of alternate treatments.

Linking the network (as Aurora is already doing) with biospecimens and tissue samples will help make the next logical leap - allowing companies to quickly develop and test hypothesis for emerging biomarkers, recruit patients for clinical trials, and rapidly develop "proof of concept" trials that the treatments work as advertised and have a significant treatment effect. (That is, in itself, a roadmap for developing "breatkthrough therapies.")

In this emerging model, pressures will rapidly build on the FDA - from industry, academic medical centers, patients' groups, and others - for the agency to lead, follow, or just get out of the way. That is a paradigm shift that the FDA isn't in place to grapple with yet, but PDUFA V gives it Congress' imprimatur - and some new tools - for adapting to the new environment.

The challenge of FDA modernization shouldn't be underestimated. But science moves at its own pace. It'll be left for the regulators to catch up.

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