Beware of Asking FDA to Change Itself

Every five years, Congress must reauthorize a piece of legislation called the Prescription Drug User Fee Act (PDUFA), which gives the Food and Drug Administration authority to charge pharmaceutical companies substantial fees for regulating their products. Every time a manufacturer submits an approval application for a new medicine, a new use for an already approved medicine, a generic copy of an already approved medicine, or any number of other things, it has to pay a “user fee” that ranges roughly from $100 thousand to $2 million dollars, depending on the particular application.

These fees now account for about a quarter of FDA’s total budget, and close to two-thirds of the drug and biologics regulation budget, which means that Congress views reauthorization as a “must pass” bill. So, every five years since PDUFA was first enacted in 1992, Congress has seen reauthorization as an opportunity to force through certain other FDA regulatory measures that may or may not be able to pass on their own.

Usually, Congress just lards on loads of additional regulatory hurdles. But, sometimes, such as in 1997’s FDA Modernization Act, some of these changes are actually moderately positive. With the House of Representatives now under Republican control, there has been discussion of using the 2012 PDUFA reauthorization to make some positive regulatory reforms. But reforming the FDA drug and medical device approval process doesn’t sit well with FDA’s career staff, who would rather prefer to be left to their own devices. At a public meeting on rare diseases two weeks ago, Director of the FDA’s Center for Drug Evaluation and Research Janet Woodcock warned against Republican reform efforts.

“It’s not that I’m against legislation. … I’m not sure in this political environment, and I’ve said this repeatedly, that there’s enough rationale (sic) deliberation that could result in the kind of legislation that we need if we plan to change the legislative framework. I’m afraid people are in a blowing up mood and often beware of what you ask for because you may get it.”

Instead, Woodcock suggested that FDA has all the authority it needs to reform the development and approval process for rare disease treatments on its own. Essentially, “Move along. There’s nothing to see here.” But Steve Walker, a co-founder of the Abigail Alliance for Better Access to Developmental Drugs, begs to differ. In an article for The RPM Report, Walker explains why real reform is needed, and why the modest, incremental steps that are being proposed now are not nearly enough. It’s worth excerpting at length:

“This scripted and oft-used defense plan is rolled out by the FDA every time Congress starts talking about reform legislation, unless of course Congress is crafting legislation FDA has asked for – which always involves granting new powers and funding to the FDA to allow it to regulate more things in more stringent ways.  FDA has never asked for legislation that would allow it to streamline its processes, or to increase its flexibility, and it has certainly never asked for legislation that would require it to do those things. … We have reached a point where the large majority of real stakeholders (patients, industry, investors and others) who directly feel the mounting negative effects of FDA’s scientific and regulatory stagnation have arrived at a conclusion that the only way to get FDA moving forward is through comprehensive reform legislation. …

The FDA has long been focused on trying to be a ‘perfect’ regulator, a concept taken to an irrational extreme in recent years. This has always meant that the agency doesn’t want to approve any ‘unsafe’ drugs, which has rendered the agency fearful of approving anything, even drugs for rare diseases in which the large clinical trials FDA prefers to cover itself are virtually impossible to run. … The need for dramatic changes at FDA has been obvious for years, but the agency, with Dr. Woodcock often in the lead, have waged an aggressive and largely successful holding action against change. …

Instead of the progress we could have made in the last decade, the number of new drugs making it to market dropped precipitously. FDA blames the drug companies – as it always does – but a close look at what the agency has been doing over the last ten years on a drug by drug basis tells a different story. The progress was pushing up against an agency that was very aggressively pushing back. … So here’s a twist on Dr. Woodcock’s favorite cliché regarding change. ‘We should beware of asking FDA to change itself, because we will always end up with the same FDA we were trying to change in the first place’.”

Go get ’em, Steve.