Expedited drug and device approvals spark quality concerns for providers

As policymakers and the drug and device industry look for new ways to bring treatments for life-threatening conditions to the market sooner, providers are concerned that expedited approvals could compromise patient care.

The U.S. Food and Drug Administration and lawmakers on Capitol Hill have proposed fast-tracking drug and device reviews and allowing some "breakthrough" products that treat cancer and drug-resistant infections to undergo smaller clinical trials. But critics say those accelerated reviews could compromise quality and significantly burden providers that would spend more on replacing failed devices or changing treatments.

If the FDA accelerates approval of a drug, that is often at the price of rigorous study, and providers may not know if the new product is actually going to provide significant benefits, said Erin Fox, who directs the Drug Information Center at the University of Utah.

"This makes using those products risky because you could end up providing a medication that may cause more side effects, or may cause significant financial toxicity without additional benefit," she said.

Recent studies bolster those concerns. One study published in JAMA on Wednesday determined that post-market trials on drugs that received expedited approval don't evaluate the products' benefits and risks reliably and use surrogate metrics that indirectly measure potential outcomes to predict clinical benefit.

Researchers compared preapproval and confirmatory clinical trials for 22 fast-tracked drugs that had 24 applicable uses, mostly for cancer treatment. They found that only 10 of those 24 relevant uses (42%) had randomized clinical trials and demonstrated efficacy.

In another JAMA study, researchers found issues with evaluations on modified, high-risk devices like cardiac stents. Most of the trials did not require random, blind or controlled studies and lacked thoroughness, they said.

In an accompanying editorial, Duke University professor of medicine Dr. Robert Califf, expressed concern that the regulatory system has become "too permissive" by not mandating randomized control trials after drugs that receive accelerated approvals hit the market. The current regulatory system does not always impose the "most rigorous standard of clinical evidence," he continued.

But using a placebo or randomized trials that add complexity and time to studies doesn't make sense for patients with potentially fatal illnesses, said Dr. David Pearce, executive vice president of innovation and research at Sanford Health.

"As long as the drug is safe and there is efficacy in the majority of patients studied, we should get the drugs as soon as possible," he said. "Why give a child diagnosed with a fatal disease an empty virus in a gene therapy trial as a placebo when they can die from their disease? Are you compromising rigor to that study? Yes, but you are bringing treatment to a child."

Supporters of expedited reviews say surrogate metrics are "reasonably likely" to predict clinical benefit, and the method saves time and money and allows patients to benefit from critical treatments sooner.

Providers have to balance making sure drugs are safe and getting products to patients, said Dr. Scott Knoer, chief pharmacy officer of Cleveland Clinic.

"I lean toward preserving the rigor of the FDA related to the safety and efficacy of clinical trials," he said. "Everyone wants things to come to market faster, but I have a concern that we can go too far and get things too fast that aren't ready."

The more risk providers take on as they move toward population health management and new payment models, the more impact a shift in treatment strategy would have on providers' financials, Knoer added.