FDA endorses new breakthrough therapies provision in PDUFA

I think it's arguable that the FDA doesn't need explicit authority from Congress to develop more flexible regulations for the most promising therapies early in drug development. The FDA has at least some regulatory flexibility already, under the 1997 Food and Drug Modernization Act.

But let's get to that thought in a moment.

MSNBC reports that the FDA is explicitly endorsing a provision in both the House and Senate versions of pending legislation that reauthorizes FDA user fee programs for drugs, devices, generic drugs, and biosimilars. (And kudos to Senators Bennet, Burr, and Hatch, who introduced the legislation back in late March.)

Experimental drugs that show a big effect early in development for treating serious or life-threatening diseases would get a faster and cheaper path to U.S. approval, under a proposal likely to become law this year.

U.S. drug regulators would be able to label such treatments "breakthrough" therapies, and work with companies to speed up clinical trials, for example by testing the drugs for a shorter time or enrolling fewer patients.

The U.S. Food and Drug Administration has said it supports the proposal, which is included in both versions of an FDA "must-pass" funding bill currently working its way through Congress and set to be passed by the end of the summer. ...

Dr. Janet Woodcock, head of the FDA's drugs center, has said the FDA needs more flexibility to bypass "business as usual" when it sees unexpected effects, or when a new medicine can greatly help patients.

"What happens when you have a breakthrough drug that shows an effect that's never been seen before?" she told reporters in March, discussing the proposal.

"If we'd done business as usual during the AIDS epidemic, we would have never controlled that epidemic," Woodcock said.

This is all to the good, as I noted in another blog post.

On the other hand, it also reinforces an underappreciated reality: the FDA looks over its shoulder at Congress when it reviews and approves new medicines, and develops new drug approval mechanisms.

This is only natural. The FDA is in the news only when there is drug safety problem, at which point it will get savaged by Congressional committees for not predicting and preventing every potential safety problem in advance.

Sadly, the FDA isn't going to be called up to the Hill to be congratulated on new drug approvals, or for streamlining the drug development process in general. So the pressure on the FDA tends to go only in one direction - towards requiring more data, more clinical trials, and more tests. This, in turn, drives up the costs of drug development and leads to delays in patient access to new medicines.

(To be fair, FDA aside, public and private payers in the U.S. and Europe are also demanding more data from companies to justify premium pricing in markets that are increasingly crowded with cheap, relatively safe, and effective generic drugs. The public is also increasingly wary about side effects from medicines for chronic illnesses that patients may take for years or decades. So the agency is only one factor in the development equation, albeit one of the most decisive ones.)

In short, the environment on the Hill often isn't very hospitable to regulatory innovation.

Still, the FDA is empowered to set standards for "adequate and well controlled trials", and Congress has explicitly given the agency the authority (under the 1997 FDA Modernization Act) to approve drugs based on single arm trials, but it still almost always requires two placebo controlled trials for drug approval.

In other words, the FDA has plenty of discretion. What it needs from Congress - and from the public - is permission to exercise that discretion and to do so with confidence that policymakers will not excoriate them when something goes awry. (And something will always go awry eventually, because neither medical science nor human beings are perfect.)

On that front, there's a lot to like in the current FDA user fee reauthorization. On both the House and Senate side, Congress has broadened the accelerated approval pathway and directs the FDA to embrace new technologies like biomarkers.

To be sure, FDA leadership hasn't embraced every proposal for updating its drug development toolkit - it expressly opposed the first version of Senator Hagan's TREAT bill - but its recent support for the breakthrough therapies designation is a very welcome sign that agency leadership knows that it needs Congress' permission to stop doing "business as usual" and help drive a more flexible mindset among its own reviewer staff.

AIDS was a very visible crisis. The challenges we face today are more subtle and longer term - unsustainable health care costs, an aging population that will become increasingly vulnerable to chronic diseases like Alzheimer's and cancer, and a drug development pipeline that is floundering. But they also demand a rethinking of the entire drug development and approval process - starting with the FDA and it's stakeholders.

Since FDA is a regulatory body, Congress must take the lead role in defining the policies that frame and provide effective oversight for the FDA's regulatory functions. The House and Senate should be applauded for for crafting user-fee legislation that can facilitate and acclerate access to more innovative treatments for millions of American patients.

Now, it is up to the FDA to show that it can embrace and create real change.

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