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Critics Worry New Law Will Weaken FDA Regulation


A new law that promises sweeping changes to the Food and Drug Administration was one of the few bipartisan accomplishments for Congress last year. The 21st Century Cures Act will increase funding for the NIH and change the way drugs and medical devices are regulated. Supporters say the FDA will receive a much-needed overhaul and be able to get medicines to patients sooner. Here's testimony from a mother who lost her two sons from a rare form of muscular dystrophy.

UNIDENTIFIED WOMAN: Because Duchenne is 100 percent fatal at a young age, many patients and families are willing to accept higher levels of risk in return for the prospect of potential benefit. Business-as-usual clinical trial designs simply do not hit the mark when working with these populations. Our community needs therapy. And we need them fast.

CHANG: But the Cures Act also has many critics who worry that the new law weakens the FDA and makes patients vulnerable. One of them is Dr. Jerry Avorn. He's a professor of medicine at Harvard Medical School.

JERRY AVORN: It was urged through Congress with the idea that, somehow, Americans are being denied needed drugs because FDA is so slow and has these impossibly high standards. And, actually, the FDA is among the fastest regulatory bodies in the world for drugs. It can turn around an application in as little as six months, which is probably as fast as you want it to go for a treatment that needs to be evaluated very carefully. And it does not have impossibly high standards. It has demanded that companies show that their drugs actually improve patients' clinical status, which seems like a very reasonable request.

CHANG: How does the Cures Act change the way the FDA regulates new medicines and devices?

AVORN: The core of the bill that is the most worrisome is the idea that it authorizes FDA to look into lower standards for approving drugs based on laboratory test changes or other so-called surrogate markers rather than making companies demonstrate that a drug actually helps patients with real clinical outcomes.

CHANG: There were many patient groups who just didn't agree with that. Some patients are denied medications that could've saved their lives. But because of certain inefficiencies in the regulatory process, they couldn't get those medications. What do you tell those patients?

AVORN: It's a little bit of a nuanced argument. But to say, if there was a patient who, you know, was trying to get a treatment for condition X, and it wasn't approved until November, and they had died the preceding August, of course, that's a tragedy. But we also need, when we're thinking about ideal policy, to take into account - if we did have all those drugs available much quicker with much less evidence, how many people would end up not getting a treatment that was helping them or might even have side effects that no one saw coming and got sick from that?

And so it's not as if it's always a better thing to approve drugs quicker and with less standards because we are going to inflict illness on people or deny them needed treatments, like if there's a new cancer drug. And the old cancer drug works pretty darn good. But people flock to the new cancer drug. And then it turns out, as we saw specifically with Avastin for metastatic breast cancer, which the FDA initially approved based on a surrogate measure - and then it turned out that they had to take away that indication when a real study that was done more carefully showed that it did not help women with advanced breast cancer.

All the women who sort of got access to Avastin for that during that intervening period then were not using other treatments that would've helped them because they're all flocking to Avastin, which, when we did the research right, turns out wasn't helping them and was causing side effects and was very expensive.

CHANG: Certainly, everyone can agree that we don't want to carelessly approve drugs that are unsafe or ineffective. But is there room for improvement?

AVORN: Yes. The FDA, since before the AIDS crisis in the 1980s, has been underfunded in the federal budget. They just have not had enough staff to be able to review these very complicated proposals accurately and efficiently. So I think a best - the best solution would be to - not one that we're likely to see in the next couple of years - would be to make sure that Congress funds the FDA adequately so that it cannot be chronically short-staffed and can have enough people to really review these products expeditiously. We are usually the fastest in the world and tend to say yes most of the time. So it's hard to see what the problem there is that this is a proposed solution to.

CHANG: Dr. Jerry Avorn is a professor of medicine at Harvard Medical School and Brigham and Women's Hospital. Thank you for joining us.

AVORN: You're very welcome. Transcript provided by NPR, Copyright NPR.