New law makes doctors 'gatekeepers' of new drug uses

John FrankThe 21st Century Cures Act signed into law late last year will place a greater onus on doctors to monitor and report results for new indications of previously approved drugs, say those who have followed the law’s progress through Congress.

The 996-page document addresses a long laundry list of healthcare issues, from earmarking $1 billion for states to fight opioid abuse to how the Food and Drug Administration (FDA) looks at new drugs and medical devices. Indeed, the broad scope of provisions that were folded into the measure as it wound its way through Congress are credited with getting it the rare bipartisan support it needed to pass and get signed into law.

Among its many provisions are a $4.8 billion increase in funding for the National Institutes of Health over a 10-year period, although those funds are not guaranteed and must be appropriated by Congress each year. That research funding includes $1.8 billion for former Vice President Joe Biden’s cancer “moonshot” research initiative and $1.4 billion for ex-President Obama’s precision medicine initiative, which seeks to use better data analysis to treat and cure diseases.

In terms of provisions for how the FDA approves new drugs and medical devices, the measure includes an approval process that allows smaller clinical trials to be used in testing new antibacterial drugs designed to fight infections resistant to existing drugs. For new medical devices, the measure creates a “breakthrough device” process that will allow expedited review for devices achieving that designation. It also includes $500 million over 10 years in increased funding for the FDA.

Ameet Sarpatwari, JD, PhD, an instructor in medicine at Harvard Medical School, says that while the law doesn’t specifically mandate faster drugs and device approval, that will be its impact. 

“The bottom line for physicians is you’re likely going to have more questionable drugs,” as a result, contends Sarpatwari, who is also an assistant director at Brigham and Women’s Hospital in Boston.

But Peter Pitts, a former FDA associate commissioner, says the law “doesn’t mean the FDA is going to bring new drugs to market faster, it means it’s going to review drugs faster. Those drugs that get expedited review also get review by more senior staff, so the FDA is not lowering the standard by which they approve products.” 

The law requires the FDA to rely more heavily on real-world evidence, as opposed to clinical trials, for approving new indications of previously approved drugs, both supporters and detractors agree, and that means doctors will need to devote more time to reporting such results. 

James Rickert, MD, president of the Society for Patient Centered Orthopedic Surgery in Bloomington, Indiana, worries that doctors may not be able to determine if a new drug is adversely affecting patients because of mitigating circumstances, such as other drugs a patient may be taking or pre-existing medical conditions. 

The law “puts more pressure as sort of a gatekeeper role on physicians,” agrees Sarpatwari. Pitts says that even with new review processes called for in the law, “doctors should have the same faith that they always had in the FDA and hopefully that means a lot of faith” in its decisions to approve new drugs and medical devices.  

John Frank is a freelance healthcare journalist. Do you think the law will help or hurt physicians? Tell us at medec@ubm.com.