Does the US drug approval process need an overhaul?

(Reuters)

It takes 12 years, on average, to develop a prescription drug and get it approved for use by the Food and Drug Administration (FDA), according to pharmaceutical company Eli Lilly.

Some experts and lawmakers say that’s too long.

A bill now before Congress, the 21st Century Cures Act, would ease guidelines for certain drug and device approvals. If approved, it would also provide $10 billion over 10 years to the National Institutes of Health (NIH) to accelerate research efforts by providing grants to scientists working on potential new cures.

Voices of support

The bill is currently before the House of Representative, but it has already received a rare showing of bipartisan support. In May it passed the House Energy and Commerce Committee’s preliminary vote 51-0, and it is poised to pass the House, too. Its sponsors, Colorado Democratic Rep. Diana DeGette and Michigan Republican Rep. Fred Upton, want the United States to be the “health care innovation capital of the world,” according to a promotional video, and they say this bill will help us get there.

DeGette says in an email that in recent years, the NIH research budget has eroded. “With the new funding we provide NIH in 21st Century Cures, we seek to reverse those past cuts so that the agency can deliver new discoveries sooner,” she says.

That includes discoveries like precision techniques that help doctors treat patients based on their genetic profiles, new cancer drugs and vaccines for diseases like Ebola. “While we won’t be able to help the 10,000 people who died in last year’s [Ebola] outbreak, we can do something to save untold numbers of lives in the future if NIH is working at full strength,” DeGette says.

Voices of dissent

Opponents of the bill don’t necessarily have a problem with the increased research funding for the NIH. Many of them do, however, object to the way the bill proposes to change approvals processes for many medical devices and drugs, including antibiotics.

Antibiotics are a special case because they become less effective as disease-causing pathogens develop resistances to them. One provision in the bill would provide physicians with rewards from Medicare when they use a new antibiotic.

“That’s exactly the opposite of what Congress should be doing,” says Sarah Sorscher, an attorney with Public Citizen's Health Research Group. Public Citizen is an independent advocacy group that opposes 21st Century Cures. Instead of incentivizing the use of new antibiotics, Sorscher says, “we should be preserving new antibiotics and discouraging their use. Hospitals should be using older antibiotics first.”

Failing to hold some types of antibiotics in reserve runs the risk of creating “superbugs”— bacteria that are resistant to all antibiotics— in the near future. Although Sorscher agrees that new antibiotics are needed, she and Public Citizen disagree with how the bill incentivizes development.

In addition to that provision, a measure that would allow the FDA to approve earlier-stage research is also under scrutiny. Drugs for rare diseases would be approved based on smaller trials, while drugs approved for one cancer would receive accelerated approvals for other, usually rarer, cancers.

Yet another provision would ease regulations on implantable medical devices, like pacemakers and metal knee or hip replacements. Instead of controlled, randomized clinical trials, Sorscher says the bill would allow those devices to be approved based on medical journal articles and case studies, which she says are “essentially clinical anecdotes.” Many experts like Sorscher are concerned that easing requirements will make those devices less safe.

Putting it all together

DeGette isn’t worried about safety— just the opposite. She says the FDA provided collaboration and feedback on the bill, and all parties involved are confident that patient safety won’t be compromised. “The agency’s biggest needs are greater scientific expertise and additional resources,” DeGette says. “The 21st Century Cures Act provides more of both.”

Like most bills, 21st Century Cures will likely face revisions and negotiations before it can become law. Sorscher says some of the more dangerous provisions are likely to come out when that happens, such as the one that provides rewards to doctors for prescribing new antibiotics.

Another provision likely to come out would eliminate the requirement for doctors to disclose when they’ve received money or gifts from pharmaceutical companies. “We think they should have to disclose that,” Sorscher says, speaking on behalf of Public Citizen.

For now, the bill is under close scrutiny in the House. Which of its many provisions will become law is yet to be seen, but no matter which form the bill is passed in, it has the potential to overhaul major areas of the health care industry.