Federal Right to Try bill could bypass FDA bottleneck

Right to Try is gaining momentum across the country, yet states worry a cumbersome federal approval process will continue to inhibit patients’ access to experimental drugs.

The Food and Drug Administration allows health-care providers access to experimental drugs and procedures but the process can take months, which frustrates terminally ill patients searching for a cure. Right to Try advocates are hopeful Congress will continue advancing federal legislation backed by the Trump administration.

In North Carolina, House Bill 652 opened the door for terminally ill patients to try experimental drugs or procedures, but only after exhausting all other options. The patient’s doctor must also provide recommendation for the treatment, and patients are required to give “informed consent” before going forward.

The bill, which got bipartisan support, passed unanimously in the House and the Senate. Former Gov. Pat McCrory signed H.B. 652 into law in July 2015.

“There is tons of bipartisan support for this issue,” Starlee Coleman, a senior policy adviser at the Goldwater Institute said. “Red states, blue states, purple states all support this. It is passing unanimously and is signed by Democratic governors and Republican governors. It is truly nonpartisan and is something that has an overwhelming groundswell of grass-roots support.

“The FDA has a process that allows people to ask permission to access investigational medicines, but fewer than 1,000 people a year receive help,” Coleman wrote. “Others die while waiting on their approval.

The expanded access program, sometimes called the compassionate use waiver, is a formal way for terminally ill patients to ask permission from the FDA to use experimental treatments. Going through the process is time consuming. The FDA has a month to determine whether to grant or deny the request, and any questions restarts the entire review process. Finally the Institutional Review Board must decide whether to approve the patient’s application.

“It is the only way a patient who doesn’t qualify for a clinical trial has access to an investigational medication. Only about 1,000 individual patients a year get through this process. We thought, that just can’t be right. That’s just so few people who are helped by this program. It doesn’t sound right but it is.”

Thirty seven states have Right to Try laws, and the other 13 have introduced legislation toward that end. The Goldwater Institute, a conservative and libertarian public policy think tank, has championed Right to Try legislation across the country.

Right to Try laws, such as North Carolina’s, bypass the FDA waiver program and shorten the process to receive treatment.

Advocates for Right to Try laws continue to push for a federal law that would prevent the FDA from superseding the state.  When state law and federal law conflict with each other, it is the federal government that usually wins through the supremacy clause in the U.S. Constitution.

Sen. Ron Johnson, R-Wisconsin, introduced the Trickett Wendler Right to Try Act of 2017 in January, and it passed unanimously in August. A companion bill was filed in the House by Rep. Brian Fitzpatrick, R-Pennsylvania, and Rep. Andy Biggs, R-Arizona.

Among other things, it would insulate doctors from some lawsuits if they prescribed experimental treatments to terminally ill patients.

The White House recently restated its support for a federal Right to Try bill. Vice President Mike Pence signed Indiana’s bill into law in 2015 when he was governor of the Hoosier State.

“We thought there’s just got to be a better way to make sure people who need treatments that are still in clinical trials have access to them,” Coleman explained. “It allows terminally ill patients who have exhausted all of their treatment options and can’t qualify for a clinical trial to access medication that is still in clinical trial, as long as their doctor thinks it might help them, and the pharmaceutical company agrees and is willing to provide the medication.”

Some people may get a false sense of hope, but, says Coleman, the benefits outweigh the risks, and personal choice should trump government bureaucracy.

“If a patient is fully informed of the fact that we don’t know all the answers for this drug, because it is still in testing, that we don’t know if it is going to help them … if the patient understands that and is willing to take the risk anyway, then certainly it shouldn’t be up to the government to tell them no.”