With the launch of a new website, the U.S. Food and Drug Administration took the first step in implementing a recently enacted law that lets Americans seek experimental treatments without going through the agency's compassionate-use process.

Critics of the law said it would expose desperately ill patients to "snake oil" salesmen peddling false hopes.

Congress passed the law, known as the Right to Try Act, in May, but left the chore of figuring out how to execute it in the FDA's hands.

A day after it was enacted, however, the legislation's author, Sen. Ron Johnson, R-Wis., explicitly told the agency's commissioner, Scott Gottlieb, that the new law was intended to "diminish the FDA's power over people's lives, not increase it."

In his May 31 letter, Johnson insisted the FDA had limited control over the right-to-try process, unlike the agency's expanded-access program — also called compassionate use — in which regulators help terminally ill patients obtain and use investigational medicines outside of the clinical trial process.

Johnson warned Gottlieb about writing any new guidelines, rules or regulations that would limit the ability of patients with life-threatening diseases from accessing experimental treatments. Gottlieb had previously criticized the Wisconsin senator's legislation before later saying he had grown comfortable with it.

Three weeks later, Johnson said Gottlieb had given his word to the senator that the FDA would implement the law "in a way that maximizes access consistent with its intent."

In late May, the FDA revealed that it was putting together an internal work group to assess the best ways to effectively and efficiently implement the new law.

FDA spokeswoman Amanda Turney told S&P Global Market Intelligence on Nov. 8 that the work group's first deliverable was the creation of the Right to Try Act website, though she was not able to provide any other details about how often the internal panel met and what was on its agenda, which has not been made public.

'Skimpy' website

The new website is intended to help patients understand the right-to-try pathway, Gottlieb said in a Nov. 8 statement.

The site lays out details of what the FDA considers the eligibility criteria for patients and experimental drugs under the Right to Try Act. It also includes some contact information for the FDA if patients have questions.

But the site leaves out some important information, most notably, the fact that drugmakers are not required to provide their experimental medicines under the Right to Try Act to patients and the FDA cannot force the companies to make the drugs available.

The site is "pretty skimpy," Holly Fernandez Lynch, an assistant professor in medical ethics and health policy at the University of Pennsylvania, wrote on Twitter.

It should have a "flashing banner that says 'Expanded access is the better option for patients, always,'" Fernandez Lynch said, noting there was nothing in the new law that precluded such a statement.

But Patricia Zettler, an associate professor of law at Georgia State University, said that, to be fair, the FDA "can't say much else in a webpage at this point."

"It's thinking on how the law should be interpreted should be provided in, at least, guidance not a webpage," Zettler tweeted.

Ambiguities need clarifying

It would be helpful to patients if the FDA included a side-by-side comparison on its new website displaying the requirements under the Right to Try Act versus the agency's expanded-access program, Fernandez Lynch said.

She said such a chart could be similar to what she, Zettler and Ameet Sarpatwari of Brigham and Women's Hospital in Boston and Harvard Medical School included in their Sept. 4 commentary in the Journal of the American Medical Association about the new law.

In that article, the three authors provided recommendations to the FDA for clarifying ambiguities in the Right to Try Act about the eligible patients and drugs, reporting requirements and where the new law intersects with the agency's compassionate-use program.

Given some of the concerns raised about right to try, the FDA should take steps to facilitate the expanded-access program as the preferred pathway for accessing investigational drugs outside clinical trials, they said.

Expanded access remains primary avenue

But on Twitter, Fernandez Lynch praised Gottlieb for putting the greater emphasis in his Nov. 8 statement on the expanded-access program, whose own website has undergone a transformation.

In that statement, Gottlieb pledged to ensure the expanded-access website was regularly updated.

"We are deeply committed to continuing to enhance this program going forward," he stated, calling expanded access the "FDA's primary avenue" for facilitating patients' access to experimental treatments.

Gottlieb noted that over the past five years, the FDA has authorized more than 9,000 compassionate-use applications across drugs, biologics and devices — granting 99% of the requests the agency has received.

Many of the updates made to the expanded-access website were based on public feedback and the findings of a recent independent assessment report, Gottlieb said.

He said the FDA plans to formally establish an agency-wide program to help patients and providers navigate the expanded-access process.

It has also established an agencywide expanded access coordinating committee intended to facilitate communication across the FDA's centers and promote consistency and best practices.

"FDA staff is deeply committed to this program and ensuring that it works quickly and effectively for patients and their physicians," Gottlieb said.