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AIDS Drugs Were the Start. Let’s See More FDA Accelerated Approvals

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the laser-focused motivation they once had to complete clinical trials which might end up reflecting badly on their cash cows. The FDA hasn’t often exercised authority to revoke accelerated approvals. But commissioner Margaret Hamburg did make one of those tough calls last November when Roche’s bevacizumab (Avastin) lost its clearance as a breast cancer treatment.

Essentially, there’s a risk here that the FDA could end up letting a lot of sketchy drugs onto the market, forcing it to later make painful and embarrassing recalls when the evidence becomes more clear-cut. And while enthusiasts of new biomarkers may be right about their value some of the time, there are bound to cases where some supposedly compelling biomarker turns out to mean little. Researchers have seen plenty of cases, for example, of cancer drugs that appear to shrink tumors which don’t end up helping people live longer.

The bill, known as House Bill 4132, which I took time to read in all its boring glory, doesn’t include any specifics about what kinds of data will be necessary to satisfy “accelerated approval” standards at the FDA. That’s wise, because those decisions will have to be made on a case-by-case basis, by the FDA, companies, and researchers—not Congress. If this bill is passed, the details—and the details are really all that matter—will be hashed out in a million little decisions made between the FDA and companies over the years to come.

Still, Maraganore says passage of the bill would make a big difference in one key respect—it would boost the confidence of investors in biotech. And he says the FDA has been willing to work on a variety of reform proposals, including this one. “They have gone a long way to work with industry to think about the proposals that are now in front of Congress,” Maraganore says. “There is a solid working relationship between industry and the FDA on thinking about what’s the right thing for patients.”

There are a number of smallish ideas tucked into the bill that also make some sense. In the Senate version of the FDA reform bill, known as TREAT, there’s language that would call for the FDA to hire a “chief innovation officer” who would be charged with identifying “promising new scientific and regulatory approaches” to help speed up drug development and regulatory reviews.

Every year, lots of bills get introduced that go nowhere, but this year there’s an incentive to get things done, as the Prescription Drug User Fee Act (PDUFA) V—a key piece of legislation that provides the FDA with industry user fees it needs to function—expires at the end of September. It’s entirely possible that the ideas contained in the FAST and TREAT bills could get rolled into the broader PDUFA reauthorization bill, which has to pass to essentially keep the FDA operating.

I’ve already gone on my soapbox with “Five Things Industry Can Do to Support True FDA Reform and Restore Public Confidence.” I don’t think these bills are going to come close to solving all that ails the drug development industrial complex, but they are reasonable. I’d love to hear your thoughts about what kind of impact this legislation could have.

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  • The TREAT and FAST bills are a start. If passed, we will likely see a modest shift in the way FDA regulates development and approval of some of the most needed new medicines. However, if Congress fails to actively oversee FDA’s performance in implementing the new law, and make adjustments with new legislation on an annnual basis, we will see little change. Broad use of Accelerated Approval was supposed to happen after passage of the Food and Drug Administration Modernization Act of 1997. It was used extensively in HIV/AIDS, and has been used occasionally in cancer (although often in name only for oncology drugs), and not much in any other disease area. FDA will neutralize these bills as well, which some FDA review division directors will do automatically, unless Congress makes it clear that its direction is to be followed.

  • Steve–I’m not so sure the FDA will neutralize these bills, it sounds more and more to me like the FDA is willing to embrace some new ideas for improving its processes. In theory, Congress could do some good by passing these bills and holding the FDA feet to the fire to make sure it happens, but I’m not optimistic Congress will continue to pay close attention. It’s always been easier for members of Congress to say to the voters, ‘Look at that cozy regulator approving dangerous drugs’ when something clearly goes wrong than to say ‘Look at that overly cautious regulator that’s delaying lifesaving drugs from reaching the market because of process technicalities.’ Sadly, I don’t see anyone in Congress taking on this issue and popularizing it.

  • CMCguy

    FAST/Treat sound good conceptually yet wonder if will do much in the end if are passed. Since inception “Accelerated Approval” most inside industry viewed has offered a feasible path to gain quicker access to new drugs through use of surrogate markers. FDA probably has been “guilty” of neutralizing this an option for many things however see has more been lack of resources with recruiting and retaining qualified staff (amount of info to review remains huge and complex but time to look at compressed) and mission focus and responsibilities growth that diverts efforts. Not sure FDA alone totally at fault as I see those as a more result of Congress (general) that has not always provided sufficient and consistent budgets plus Congress (as individuals) either adding increased mandates or getting caught up in hot-topic issues (hearings). So as you state Luke when Congress focuses elsewhere will the FDA lose momentum (or have to deal with immediacy of Congress target interest).