Senators Coburn and Burr Advance FDA Reform Legislation
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Earlier this week, U.S. Senators Tom Coburn (R, OK) and Richard Burr (R,NC) introduced the "Promoting Accountability, Transparency, Innovation, Efficiency, and Timeliness at FDA" Act or PATIENTS' FDA Act. A summary of the legislation can be found here. The full text can be found here.

Allow me to sum up the 55 page bill in a single, short sentence.

What gets measured gets done.

Among its key provisions, the Act requires the FDA to identify gaps in regulatory science that may be impeding innovation or slowing new product reviews; review all existing product regulations and guidance to ensure that "the regulatory principles of benefits of regulations and guidance [justify] the costs and the adoptions of the least burdensome approaches to regulation as outlined in President Obama's Executive Order 13563 to improve regulation and regulatory review"; repeals counterproductive conflict of interest restrictions that limit the agencies access to the most authoritative experts; and requires the FDA to (among other things) ensure that its IT capabilities are up to date and meeting the needs of the agency.

It's hard to see how the FDA could strenuously object to many of its provisions. For instance, take Title V, section 601, "Integrated Strategy and Management Plan":

Not later than 1 year after the date of enactment of this Act, the Secretary shall submit to Congress a strategic integrated management plan for the Center for Drug Evaluation and Research, the Center for Biologics Evaluation and Research, and the Center for Devices and Radiological Health. Such strategic management plan shall--

(1) identify strategic institutional goals and priorities for the Center for Drug Evaluation and Research, the Center for Biologics Evaluation and Research, and the Center for Devices and Radiological Health;

(2) describe the actions the Secretary will take to recruit, retain, train, and continue to develop the workforce at the Center for Drug Evaluation and Research, the Center for Biologics Evaluation and Research, and the Center for Devices and Radiological Health to fulfill the public health mission of the Food and Drug Administration;

(3) identify results-oriented, outcome-based measures that the Secretary will use to measure the progress of achieving the strategic goals and priorities identified under paragraph (1) and the effectiveness of the actions identified under paragraph (2), including metrics to ensure that managers and reviewers of the Center for Drug Evaluation and Research, the Center for Biologics Evaluation and Research, and the Center for Devices and Radiological Health are familiar with and appropriately and consistently apply the requirements under the Federal Food, Drug, and Cosmetic Act (21 U.S.C. 301 et seq.), including new requirements under the 2012 user fee agreements.

Developing a strategic plan for workforce management and continuous improvement is something the agency should be doing already - but without the "teeth" of actual Congressional oversight and pressure of reporting requirements, it is something that is likely to fall by the wayside amid the agency's many other responsibilities (like ensuring the safety of the food supply). Indeed, Senators Burr and Coburn note that a 2010 GAO report found that the FDA "does not use established practices for effective strategic planning and management" and that although the FDA agreed with GAO's recommendations, "three years later, many of these recommendations have not been adopted."

Again: what gets measured, gets done. In a competitive marketplace, private companies have powerful incentives to continuously monitor and improve performance of key products and services or risk going out of business. Government agencies can't go out of business, and many, like the FDA, have an absolute monopoly on the "services" they offer (in this case, marketing approval for medical products).

Ironically, the very mechanism that has led to improved funding and performance of the FDA - the Prescription Drug User Fee Act, first passed in 1992, and which gave the agency a much needed infusion of funds to hire new staff while also setting performance goals for the review of new drug applications - has probably diluted pressure for effective Congressional oversight outside of the user fee reauthorization process, which only occurs every five years.

Politico's Sarah Kliff describes PDUFA as the "most important health policy you haven't heard of." Kliff writes that

Over the past two decades, the new funding stream from pharmaceuticals has reduced FDA drug review times by nearly half. The agency added about 200 new staff members. A 2002 Government Accountability Office report found that user fees increased new drug reviewers by 77 percent in the first eight years of the act. The average approval time dropped from 27 months to 14 months over the same period.

Drug companies' user fees have, meanwhile, have become a crucial source of FDA funding: They made up 62 percent of the country's $931 million drug review budget in 2011. ...

But here's the funny thing: Among all the players on PDUFA, there's pretty widespread agreement that this is a fee that pharmaceuticals should be paying. It's a law that nearly everyone, from Republicans to Democrats to industry, thinks is working. ...

The question, now, is whether Congress can keep the law working. PDUFA doesn't make many headlines. But it's the one piece of must-pass health policy legislation in 2012.

Kliff is right that PDUFA is critical "must pass" legislation for patients, companies, and the FDA. But its "must pass" status and infrequent reauthorization results in little ongoing Congressional monitoring of FDA performance short of a full blown crisis (like contaminated Heparin, or drug shortages). But oversight-by-crisis (which frequently produces knee-jerk legislation) is hardly the most effective way for Congress or the FDA to ensure that it is not just "getting by" but actually excelling at its key responsibility of promoting and protecting public health.

In the FDA's defense, Congress continually adds to the agency's responsibilities - tobacco products and biosimilars regulation are among the most recent - without stopping to consider how well it is performing existing responsibilities or whether the agency has the capacity and resources to take on new ones.

This is a recipe for the FDA to fall into a state of dysfunctional disrepair. The PATIENTS' FDA bill is an attempt to remedy the not-so-benign neglect that Congress has inflicted on the agency and to begin to generate the data necessary to improve its performance in a thoughtful way.

Perhaps its most important provision is for the FDA to contract with an independent management company to conduct a top to bottom review of the drug review and approval process. The outcome of that review may or may not be welcome by the FDA - but it would force Congress to pay attention and highlight the FDA's importance as the gateway for medical innovation not just in the U.S., but for the world.

PDUFA must be passed - and the reauthorization negotiated by industry and the FDA (with input from patients' groups) does, in fact, contain several important incremental reforms.

But the PATIENTS' FDA Act is the long overdue start of a discussion on whether America has the FDA that we need in the 21st Century - to battle and eventually defeat life threatening diseases like cancer and Alzheimer's, drive innovation across the life sciences sector, meet emerging challenges like pandemic disease and bioweapons, and secure America's increasingly complex global food and pharmaceutical supply chains.

Do we have the FDA that we need? Let's not another five years to find out.

1 Comment

The issue of the FDA's ability to manage itself has been a serious concern over the increasing user fee agreement for medical devices. At the latest Energy and Commerce House subcommittee hearing this past week, Representative Joe Barton once again questioned the link between FDA's control over resources and review times.

While Barton's comments are, in the proverbial sense, "water under the bridge" at this point in MDUFA III negotiation, the need for the FDA to be as efficient as possible(which this article makes evident)has serious implications for the U.S. as the leading country for medical innovation and technology in the world.
Ultimately, I think Congress needs to adopt the "if you build it, they will come" approach with drug and medical device innovation.

In terms of the biosimilar approval, here's a short piece that highlights this process:

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