Pharma & drug development

Cure or curse?

The 21st Century Cures Act is moving rapidly through the US legislative process, but critics remain sceptical of its benefits. Abi Millar explores both sides of the story. 

On July 10th, the US House of Representatives passed the nonpartisan 21st Century Cures Act by a vote of 344 to 77. This legislation, if enacted, could have major ramifications for the US pharmaceutical and medical device industry, as well as the American public. Supporters claim it will promote innovation, streamline research, and reduce bureaucratic obstacles to drug and device approval, moving regulatory systems squarely into the 21st century.

The bill has been carefully tailored to appeal to both Democrats and Republicans, having earlier passed through the Committee on a 51-0 vote. One of its authors, the Democrat Rep. Diana DeGette, has claimed: “with billions in support for our premier research and development institutions and comprehensive reform of our systems, 21st Century Cures will make a real difference in the lives of patients and their families.”

For her fellow author, the Republican Rep. Fred Upton, the legislation boils down to a simple, universally shared goal: “more time with those we love”.

Even aside from this kind of rhetoric, it is easy to see why the bill has been so widely acclaimed. The National Institutes of Health (NIH) budget has long been stagnant, with a considerable knock-on effect on innovation. As well as increasing the annual budget by 3%, the bill would create a $2bn a year ‘Innovation Fund’, encouraging young scientists to carry out novel research. It would make research collaborations easier, promote further exploration into personalised medicine and encourage adaptive trial designs.

Ameet Sarpatwari, an instructor in medicine at Harvard Medical School, describes these provisions as a ‘no-brainer’.

“The big thing that’s gaining support is the funding that would come with the bill to NIH and FDA – we’re talking over $9bn in total,” he says. “The research shows that innovation really happens through basic science research and clinical translational research funded by the NIH, whether it be at the NIH or at academic medical centres. The real hub of innovation is not happening in the pharmaceutical industry.”

Look before you leap

All this said, a growing number of commentators are counselling caution. On July 17th, the New York Times ran an op-ed slamming the legislation. Its authors, two prominent cardiologists, called the title of the Act ‘disingenuous’ and claimed that it would weaken “the FDA’s already ineffective regulatory scheme for medical devices”, potentially putting the public’s health at risk.

The authors were particularly troubled by the bill’s creation of a breakthrough pathway for medical devices, which would allow high-risk devices to be approved more quickly and less rigorously. This, they suggested, would “subject millions of Americas to unsafe or untested medical devices”.

With the bill now moving steadily towards the Senate, the debate looks set to rumble on. Is the 21st Century Cures Act a revolutionary piece of legislation that will speed up the passage of groundbreaking new medicines towards those in need? Or will upending existing regulatory structures do more harm than good?

Transparency concerns

According to Sarpatwari, while the bill does contain some enormously positive elements, these mask some more troublesome features. He flags up up three provisions as a particular concern.

For starters, the bill would create an additional six months of exclusive marketing rights for drugs with an ‘orphan designation’ (i.e. an indication for a rare condition). This is intended to spur on manufacturers to develop drugs for uncommon but deadly diseases.

However, Sarpatwari points out that under the terms of the Orphan Drug Act, manufacturers already have a strong incentive to develop such medicines. He feels that all the provision will do is push up costs for the taxpayer, with no real innovation gains.

The bill may also lead to problems with transparency, effectively rolling back the clock on the Physician Sunshine Payments Act. This law, enacted in 2010 to shed light on the financial relationships between healthcare providers and pharma companies, has been instrumental in uncovering potential conflicts of interest. Should the 21st Century Cures Act be passed in its present form, such openness would come under threat.

“What the Act would do is to exempt medical education payments from that reporting,” says Sarpatwari. “That’s a problem because I think it’s important for patients and consumers to know what influences are at stake when it comes to their care.”

Lowering the bar

His biggest issue with the Act, however, is the way it ‘lowers the bar’ for demonstrations of safety and effectiveness. It would mean high-risk devices are approved based purely on case studies or journal articles, without needing to undergo a randomised controlled trial. Meanwhile, antibiotic and antifungal drugs would be approved on the basis of small, early-stage clinical trials, and would be exempted from the need to provide phase III data.

“What it’s basically doing is making it easier for the manufacturers to push their products through to market, and yes that will result in lower costs in pre-approval testing, but it will also result in a greater number of products on the market that are either safe or ineffective,” he says.

“I think there’s a better way to promote innovation, part of which is contained in the Act itself – such as the funding aspect, and removing some of the bureaucracy in terms of hiring senior scientists in the FDA. But any time you lower the bar it can be dangerous.”

Sarah Sorscher, an attorney at Public Citizen’s Health Research Group, agrees. She feels the bill contains a number of ill-advised provisions, not least the fact that medical device manufacturers would no longer need to notify the FDA about any changes to their devices. Then there is the host of changes to drug regulation, such as the fact that when approving new indications, the FDA would need to consider evidence from nonrandomised trials.

“The final provision we are worried about is something that allows Medicare to make additional payments to hospitals when they use new antibiotics,” she says. “That’s troubling because the hospital may be allowed to keep some of that payment, and if the system isn’t set up properly they would have a strong incentive to prescribe antibiotics every time there could be an infection.’

“With antibiotics, the more you use them the less effective they become, because the pathogens adapt, so we think Congress should be focusing on the opposite type of measure if they want to prevent the development of multiple drug-resistant superbugs.”

Collaboration is needed

Evidently, objections of this nature are far from trivial. But with support mounting for the bill among politicians and patient groups alike, it remains to be seen what the Senate will make of the Act in its present form.

“We’ve been working hard to try to make the Senate aware of these concerns, but the objectors are still in the minority,” says Sorscher.

Sarpatwari adds that, while a great deal of thought has undoubtedly gone into the Act, certain aspects ‘send out the wrong kind of message’. He hopes that the Senate, being somewhat insulated from popular pressure, will be able to take a clear-headed look at its scope to promote medical innovation, ultimately taking the good while rejecting the bad.

“I think there’s huge momentum for the Act, and it will depend a little bit on how and when the Senate actually picks it up,” he says. “What we have now is a little bit of a cooling off period, which will hopefully prompt some critical thinking about certain elements of the Act. The more critical thinking that has gone on, the better the chance of seeing some of these more problematic provisions be changed.”

This article appears in the September 2015 edition of Pharma Technology Focus

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