Legislation & Regulation

'21st Century Cures' Isn't a Cure for Everything

The NIH would get a $9 billion shot in the arm, but some fear the FDA provisions will mean lax oversight of drugs and devices.

Richard Mark Kirkner

Something very strange happened in the House of Representatives a few days after the July 4 recess: The chamber passed legislation to increase funding for medical research by almost $9 billion over the next five years—and with overwhelming bipartisan support.

Let me repeat that: The House voted to spend nearly $9 billion more on medical research with overwhelming bipartisan support amid the run-up to next year’s presidential election.

Now that you’ve picked yourself up off the floor, the legislation—titled 21st Century Cures—would give an additional $1.75 billion over each of the next five years to the NIH, but it would also tinker with FDA oversight of drugs and medical devices.

The legislation isn’t a sure thing; the Senate still has to pass a version, and that’s where opponents will focus their attention over the next few months. There’s a chance that the full Senate may not get to the bill until next year.

Pushback on FDA provisions

The drug industry trade group PhRMA has gone all in on 21st Century Cures. In a letter to House leaders, PhRMA President and CEO John Castellani states, “Scientific, regulatory and payment policy reforms included in [the act] are essential to ensuring medical advances continue to be made and are available to the patients who need them.”

Support for the increased NIH funding is almost universal, but enthusiasm for the overall legislation is not. A coalition that includes the National Physicians Alliance (NPA), Consumers Union, and AIDS United sent a letter to House and Senate members expressing their dismay and opposition to the legislation. “Rather than addressing the true scientific bottleneck in drug and device development, the bill includes unnecessary, costly, and potentially harmful regulatory changes and financial incentives for pharmaceutical and medical device companies that would put patient safety at risk and undermine public health,” the letter states.

The critics take issue with several provisions. Among them: allowing the FDA to approve “high-risk” medical devices based on case studies or journal articles alone rather than clinical trials in which the devices are tested in hundreds of live patients; a framework to consider “patient experience” for drug approvals; lower standards for approving antibiotics and antifungals; and incentives for hospitals to use new antibiotics—a provision that “would hasten the rise of resistant superbugs,” the coalition letter states.

Things like “patient experience” and journal articles might seem hazy medical evidence to scientists and clinicians more accustomed to randomized controlled trials. “In talking to other people who like this bill, do they really think we should allow clinical experience and anecdotal evidence to be evidence for drug approvals?” asks Lisa Plymate, MD, a Seattle internist and co-chair of the NPA FDA Task Force.

Lisa Plymate, MD

“We are approving most drugs faster than our European and Canadian counterparts,” points out Lisa Plymate, MD, co-chair of the National Physicians Alliance’s FDA Task Force.

The FDA’s critics in Congress and their powerful allies, the drug and device industries, have complained that the agency is too slow in approving new drugs and devices, but the NPA has evidence to the contrary. “We have fast-track mechanisms for new drug approvals already in place, and it’s not a bottleneck,” Plymate says. From 2002 to 2013, 56% of the drugs approved by the FDA used at least one of the accelerated new-approval pathways, according to Plymate. “We are approving most drugs faster than our European and Canadian counterparts, and most of the reviews are just taking 6 to 10 months,” she says.

Scant evidence

Two studies that looked at cardiac devices found scant evidence of an onerous FDA. A January 2014 report in JAMA found that most implantable cardiac devices were approved through a supplemental process, not a full FDA review. A 2009 JAMA study of cardiac devices concluded that “premarket approval of cardiovascular devices by the FDA is often based on studies that lack adequate strength and may be prone to bias.”

Ameet Sarpatwari, an instructor at Harvard Medical School and member of the Program On Regulation, Therapeutics, and Law at Brigham and Women’s Hospital, explains the importance of randomized controlled trials in the drug-approval process.

“Federal law permits drugs to be approved on the basis of a single adequate and well-controlled investigation,” he says. “The FDA prefers that this investigation be a randomized controlled trial—and for good reason. Such trials are extraordinarily important for evaluating safety and effectiveness because they distribute both measured and unmeasured variables equally between the trial arms, allowing researchers to isolate the effect of the investigational drug.

“If science has taught us anything, it’s that we should have an element of humility regarding what we don’t know or understand. These unmeasured variables can oftentimes be very critical.”

Climbing out of a hole

The concern is that short-circuiting the clinical trial pathway would eventually drive up health care costs. It may be faster and cheaper to get a product on the market, but that may come at the expense of patient safety and public safety, says Sarpatwari. “The last thing we want to be doing is paying for treatments that are unsafe or ineffective,” he says.

Ameet Sarpatwari

“If science has taught us anything, it’s that we should have an element of humility regarding what we don’t know or understand,” says Ameet Sarpatwari, an instructor at Harvard Medical School.

Critics of the FDA provisions say increased funding for the NIH should address many of the concerns about the lack of innovation and FDA inertia, but that’s a deep hole from which to climb out of. Overall NIH funding has dropped 22% over the last 10 years when inflation is factored in, according to Dick Woodruff, vice president of federal affairs for the American Cancer Society Cancer Action Network (ACS CAN), one group that pushed for the additional NIH funding.

“Show me the money!”

In 2013, the chance of a clinical investigator getting an NIH grant reached an all-time low of around 16%, about half of what the rate was at the turn of the century, according to NIH data. “That increases the chances that someone isn’t going to receive a grant to begin with,” says Jon Retzlaff, director of science policy and government affairs for the American Association for Cancer Research.

The 21st Century Cures still faces some hurdles. The House-approved version came out of the Energy and Commerce Committee, to which the health subcommittee reports. “Energy and Commerce has jurisdiction over some of Medicare and all of energy, and they came up with sales of petroleum from the strategic reserve to pay for the NIH Innovation Fund,” says Woodruff.

Bad timing

The handling of the bill in the Senate could get interesting, especially if opponents step up their game. The Senate committee that deals with health issues doesn’t have access to the offsets the House counterpart used.

“It would be difficult for the Senate committee to come up with the budget offsets that would match by any stretch what the House was able to do,” Woodruff says.

Dick Woodruff

NIH funding has dropped 22% over the last 10 years when inflation is factored in, says Dick Woodruff of the American Cancer Society.

Timing could also be a problem. Tennessee Republican Sen. Lamar Alexander, chair of the Senate Health, Education, Labor and Pensions Committee, has said his committee doesn’t plan to report out a 21st Century Cures bill until later this year and possibly next year.

Nonetheless, Woodruff at ACS CAN says that some new NIH funding could get included in an end-of-year budget agreement between the two chambers, even if legislation doesn’t make it to the finish line intact.

That would be the best of both worlds for critics of the bill.

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