With Congress poised to vote this week on a fast-tracked reboot of the 21st Century Cures Act, manufacturers of prescription drugs and medical devices may be about to win long-sought revisions to how the Food and Drug Administration approves and regulates their products.
The bill proposes major changes to the FDA's oversight of drugs, devices and biologics, while also opening new avenues for quicker approval of unique products. It also authorizes $500 million in new FDA funding over the next 10 years to implement the changes, although that money would have to be appropriated in future legislation.
The House was expected to vote on the bill as soon as Wednesday.
Pharmaceutical Research and Manufacturers of America, the nation's major drug lobby, said in a statement that the legislation would “enhance the FDA's ability to adapt to cutting-edge technologies utilized by America's biopharmaceutical companies to bring new medicines to patients and their healthcare professionals.”
When legislation moved through the House under the same name last year, the FDA provisions raised concerns from consumer advocates and some policy experts that patients could be harmed while speeding medical innovation to market.
Dr. Michael Carome, director of Public Citizen's Health Research Group, said the latest version of the Cures legislation poses the same risks.
“What this will do is it will hasten to market drugs and devices where there is not adequate evidence that they are safe and effective for their approved uses, and that's not good for patients,” Carome said.
Many details of the legislation were fluid Tuesday as lawmakers traded concessions to get it passed this week.
But the published provisions have won enthusiastic support from research institutions and patient advocacy groups eager to see more federal funding and regulations supporting their work and causes.
Amanda Jezek, vice president for public policy and government relations at the Infectious Diseases Society of America, said she is glad to see the newest draft include improved methods for getting more antibiotics to the market.
The Cures Act includes an approval pathway allowing smaller clinical trials for antibacterial drugs that can treat infections that are resistant to existing treatments.
Antibiotic resistance levels are continuing to climb and make practicing medicine especially difficult for doctors with patients who have limited ability to fight infections, Jezek said. “Unfortunately, antibiotic development is nowhere near keeping pace with antibiotic resistance.”
That provision has received bipartisan support in previous versions of the legislation and is also supported by numerous industry organizations and patient advocacy groups, she said. Another provision lets the FDA approve antimicrobial drugs based on a limited population if the drug treats a life-threatening infection.
The bill would also create a “breakthrough device” pathway that allows products that achieve the designation to proceed through an expedited development and review process. It also raises the population cap to 8,000 patients from 4,000 on the humanitarian device exemption, which allows devicemakers to get approval for a device intended for a very small population without having to demonstrate its effectiveness in treating the targeted disease.
Scott Whitaker, CEO of the Advanced Medical Technology Association, called the breakthrough device pathway the most significant FDA improvement offered in the bill. Like PhRMA, the device lobby called for Congress to quickly pass the legislation.
The “innovation ecosystem” that drives device development is severely stressed, Whitaker said. “Policy improvements such as those contained in the revised 21st Century Cures legislative package will help strengthen that ecosystem and support the development of life-changing medical technology.”
Some provisions require the FDA to give more weight to patient input and experience data, and they require the agency to lay out guidance for how such data should be collected.
Another part of the bill requires the FDA to consider real-world evidence or data in making approval decisions or evaluating studies required after approval, despite concerns that such data is not necessarily reliable.
Two provisions that Carome of Public Citizen identified as especially alarming would make it easier for pharmaceutical companies to get drugs already on the market approved for additional uses. One would allow the use of real-world evidence instead of randomized clinical trials. The other would allow the use of data summaries instead of individual-level data.
“They substantially undermine the process for approval of drugs,” Carome said.
Sen. Bernie Sanders (I-Vt.) who railed against drug prices during his upstart campaign for the Democratic presidential nomination, noted in a statement Tuesday that the Cures bill does nothing to address that issue—which was also frequently raised during the campaign by President-elect Donald Trump.
“The greed of the pharmaceutical industry has no limit, and this bill includes numerous corporate giveaways that will make drug companies even richer,” Sanders said.
