Right now, a few severely ill patients at the University of Michigan’s hospital are waiting in suspense to hear if they’ll get a chance to try some of the world’s most cutting-edge experimental drugs or medical devices.

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They’re hoping that these unproven treatments might have some effect on their terminal cancers, severe infectious diseases, rare genetic conditions or life-threatening injuries.

They also know the treatments might not help at all.

But patients are willing to take that chance, and, at U-M and other institutions, care teams are willing to offer them these last-ditch options because other types of care haven’t helped, or have stopped working.

Such patients are the kind members of Congress had in mind when they introduced recent federal “Right-to-Try” legislation. Earlier this month, the Senate unanimously passed the bill, and the House might vote soon. President Trump has signaled support for it.

If enacted, the bill will allow terminally ill patients nationwide access to unproven therapies outside of the existing U.S. Food and Drug Administration clinical trials framework. The legislation would extend “right to try” beyond the 37 states — including Michigan — that already have such laws in place, and provide a uniform framework for access.

But in fact, such a system already exists.

And more than 40 patients being treated at Michigan Medicine, U-M’s academic medical center, gained access to investigational products outside of clinical trials in 2016 alone, says Kevin Weatherwax, who leads the U-M team that helps make such access happen.

Expanded Access at Michigan

Weatherwax and his U-M colleagues use the well-established Expanded Access program offered by the FDA to provide treatment to patients outside of clinical trials.

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The program allows physicians to apply on behalf of patients for access to drugs and devices that aren’t yet ready for clinical trials, or that are in trials that the patients don’t qualify for.

“We’ve been supporting treating clinicians with expanded access requests for many years now, and last year was a banner year,” says Weatherwax, who heads a group in the Michigan Institute for Clinical & Health Research that prepares the applications.

“Most of these are life-and-death cases where patients have exhausted all available options, including rare diseases and extraordinary cases. And the FDA has always shown a willingness to review what we bring forward, approving all the cases U-M has requested in a timely way,” he says.

Helping patients and doctors navigate

Weatherwax’s team, funded by U-M and the NIH’s National Center for Advancing Translational Science, makes sense at a research university and advanced clinical care provider such as U-M.

For instance, the team worked with U-M engineers and pediatric surgeons to obtain Expanded Access approval for a 3-D-printed tracheal splint that it customizes to hold open the airways of toddlers born with malformed ones.

In the past three years, families have traveled hundreds of miles to U-M’s C.S. Mott Children’s Hospital to have the splint inserted in their children’s airways. Researchers are tracking the children’s progress.

The research team is working toward opening a clinical trial that will study the impact of the splints in a group of future patients born with the same condition. But the FDA approval for Expanded Access to the splint on a case-by-case basis has allowed them to save young lives during the pendency of a clinical trial.

And the legislation, both in Michigan and the bill passed in the Senate, doesn’t address medical devices, just drugs, says Weatherwax. So children who have benefited from the 3-D-printed splints would not have been aided by “right to try.”

More important, he says, such bills in general don’t offer the same safety and cost protections that the Expanded Access program does.

Expanded Access requires review by a local Institutional Review Board, which ensures protections are in place for patient safety, including a mechanism to ensure that the language in the informed consent documents sufficiently and accurately conveys potential risks to the patient.

The FDA also sets out specific requirements about how companies providing products under Expanded Access should set costs. Generally, right-to-try bills haven’t laid out the same kinds of controls, nor have they required FDA review.