The new 21st Century Cures law has proven to be divisive, with both advocates and critics getting hyperbolic on the law’s potential impact on the drug and medical device industries, as well as on the US Food and Drug Administration (FDA).
Critics have said, among other things, that the law will forever tarnish FDA’s ability to keep dangerous new drugs and devices from hitting the market, while advocates believe it will open the US to new cures, particularly for rare diseases, that would have never been marketed because of the once onerous regulations.
The real impact of the law – as it covers so many different medical products, from pharmaceuticals to devices to combo products to medical countermeasures to cell and gene therapies, and involves all aspects of drug development, priority review vouchers, biomarkers, as well as not just FDA but its foundation and the National Institutes of Health – will not be easy to evaluate.
In addition to what’s covered in the law’s text, there might also be a more subtle shift in the way medical product sponsors and FDA interact and negotiate over what’s necessary to win approval.
James O’Reilly, a professor of law and public health at the University of Cincinnati and once quoted by the US Supreme Court as an expert on FDA, told Focus in a phone interview that provisions in Cures “will change the balance of negotiating power between the [FDA] reviewing divisions and the sponsor.”
In the past, FDA has had the upper hand on sponsors in terms of holding up a drug or device’s approval until reviewers were satisfied with the clinical and safety evidence provided by a sponsor, O’Reilly explained.
But this new law calls on FDA to create a pathway in the next two years that allows companies to submit ongoing safety surveillance, observational studies, registries, claims and other patient-centered outcomes research data to support the approval of a new indication of a drug.
Innocuous as that might seem, O’Reilly said it “dramatically alters that balance by forcing FDA to accept information that typically had not been a sufficient basis for approval … The balance of power has shifted to companies going forward that might say look: Congress requires you to accept data other than controlled clinical trials, now accept it.
“The fact that Congress would reach into the approval process and make such a change to what data is to be accepted is a radical change compared to what we’ve had for 60 years,” he added.
Other areas that may see some of the biggest shifts thanks to the new Cures law include: software that probably would have been classified as a medical device but might not be now, how much new funding FDA really will see from the law and the fact that excipient manufacturers will no longer be inspected.
On the software as a medical device front, O’Reilly told us: “Congress has run away from wanting FDA to be in the software regulatory business. FDA will still regulate software and other applications that directly affect a patient, but they will have no control over a lot of stuff that’s now borderline.”
On the FDA funding front, O’Reilly said he’s skeptical because the law authorizes money for FDA, rather than appropriating it, noting, “Behind the headlines, what does FDA actually get in this fiscal year? It’s extremely small.”
And perhaps the most vexing part of the whole bill is buried on page 320 of its text, where it changes the language governing FDA’s inspections of establishments “by striking ‘an active ingredient of a drug, a finished drug product, or an excipient of a drug’ and inserting ‘an active ingredient of a drug or a finished drug product.’”
O’Reilly told us: “I have no idea why excipient plants would no longer be inspected. You’re going to get raw materials with active ingredient but the fluids and liquids are not being studied … no more inspections of excipient plants?”
In explaining the importance of ensuring excipients are safe, O’Reilly pointed to a story from the mid-1990s when more than a hundred children died in Haiti from a cough syrup that included an excipient called glycerin that was contaminated.
“I don’t have a dog in this fight, but it’s a random amendment that people will wonder why,” he added.