By Gary Evans, Medical Writer

On May 30, President Trump signed into law the Right to Try Act, which seeks to give patients a new pathway to experimental drugs. The law, however, has been dogged by implementation questions and remains in regulatory limbo.

Given the circumstances, the prevailing thought is that the ongoing “expanded access” pathway to experimental drugs currently in place at the FDA will continue to be the preferred method.

“My prediction is that all reputable players — be they doctors, institutions, or companies — are going to decide to stick with expanded access,” says Alison Bateman-House, PhD, MPH, MA, a professor in the division of medical ethics at New York University Langone Medical Center.

David Borasky, MPH, CIP, vice president of IRB compliance at the WIRB-Copernicus Group in Cary, NC, says his organization is taking a “wait and see” approach to the new law.

“We have always as a company done our best to help anybody that needed expanded access,” he says. “We are very comfortable and used to processing the IRB review requirements for the expanded access pathway. In the short-term, we are going to continue to work with anybody [under] the FDA expanded access program for unapproved drugs and devices.”

The Right to Try law is aimed at increasing access to investigational products for those with life-threatening illness who have no other medical options and are unable to join a clinical trial. To be eligible, the investigational drug cannot be approved for any FDA use, but Phase I of a clinical trial must be completed. Under these conditions, FDA and IRB approval would not be required, although institutions and drug companies could seek IRB guidance.

After the law was passed, FDA Commissioner Scott Gottlieb, MD, said the agency’s expanded access program would continue to operate alongside Right to Try. Gottlieb said drug companies will still prefer the FDA oversight under expanded access. (For more information, see the related story in the July 2018 issue of IRB Advisor.) Under expanded access, prior IRB approval is waived in emergency situations, but the IRB must be notified within five days of approval.

“I think a legitimate company that has a plan to develop a drug and bring it to market — and has to be accountable to a board, shareholders, and everyone else — will continue to use expanded access. So far, that’s what we are seeing,” says Bateman-House.

Informed Consent Requirements Unclear

Advocates of the law said one of the primary goals was to bypass FDA regulations and oversight. As a practical matter, the law lacks implementation guidance, particularly on the issue of informed consent.

The law requires that a patient give the treating physician written informed consent and specifies that the FDA’s informed consent regulations do not apply, explains Patricia J. Zettler, JD, associate professor at Georgia State University College of Law in Atlanta.

“The law basically says that the patient must give informed consent,” she says. “That informed consent does not have to conform to the FDA regulations, but then the law gives no additional guidance about what informed consent means. It is not necessarily clear what information physicians should be providing to Right to Try patients. It is unknown at this time.”

Thus, bypassing the FDA on this issue is problematic, as the agency would appear to be the most logical entity to clarify informed consent requirements, Bateman-House says.

“Somebody has to turn a vague law into rules or else it’s not going to work and no one will be able to use it,” she says. “There doesn’t seem to be any other reasonable actor — it has to be the FDA. If [politicians] want to hamstring the FDA and say ‘you’re not going to do it,’ then the law is effectively dead.”

Still, she reminds that the FDA is not completely out of the loop, as Right to Try requires the agency to submit an annual report on patients granted drug access under the new law.

“The FDA is removed from reviewing each individual request, but it is not removed from having some role to play overall,” Bateman-House says.

Climate of Uncertainty

In the current climate of uncertainty, some institutions are adopting policies saying they will continue with expanded access and not consider Right to Try requests, Bateman-House says.

“I’m hearing lots of backchannel conversations of institutions putting in policies saying that they will only allow these things via expanded access,” she says. “If your institution has no such policies, then theoretically a doctor could treat a patient with an investigational product without it ever going past the IRB.”

Given liability concerns, institutions may adopt a policy clarifying that the IRB must approve any experimental drug use, regardless of pathway.

“That is speculation. I am not aware of anyone doing that, but I could see an institution saying that just for liability purposes,” Bateman-House says.

“They may say as part of the agreement you have to have an IRB review because otherwise who is going to say there is valid informed consent?”

A particular concern to Bateman-House is how the Right to Try Act affects similar laws already on the books in 40 states.

“Lawyers tell me that when a federal law and the state law deal with the same topic, the federal law is paramount,” she says. “That is what I am worried about because my understanding is that the things that are not mentioned in the federal law are still valid, on-the-book laws in those states. I think that adds to the confusion.”

Some of the state requirements that did not make it into the federal bill are “patient-hostile provisions,” she said. These provisions include potentially denying patients home healthcare, hospice, or other insurance if they try an investigational drug.

“There are reasons they didn’t make it into the federal law because they are highly punitive, and it would not have passed,” she says. “But they are still law in some of those states.”