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Right-to-try author: Law was intended to 'diminish the FDA's power'

The author of a newly enacted law granting Americans the right to bypass the U.S. Food and Drug Administration's process for seeking access to experimental drugs confirmed what many critics had warned about — the Right To Try Act was meant to loosen U.S. regulators' authority.

"This law intends to diminish the FDA's power over people's lives, not increase it," Sen. Ron Johnson, R-Wis., who sponsored the legislation, said in a May 31 letter to FDA Commissioner Scott Gottlieb.

The Trickett Wendler, Frank Mongiello, Jordan McLinn and Matthew Bellina Right to Try Act, or S.204, signed May 30 by President Donald Trump, was designed to work within existing FDA regulations, definitions and approval processes, Johnson wrote.

"It is not meant to grant FDA more power or enable the FDA to write new guidance, rules or regulations that would limit the ability of an individual facing a life-threatening disease from accessing treatments," the Wisconsin Republican said.

Implementing the law

Shortly after Trump signed the bill — adopted by the Senate last year and by the House on May 22 — Gottlieb stated that the FDA stood "ready to implement this legislation in a way that achieves Congress' intent to promote access and protect patients."

The FDA chief had previously made similar statements in public and on Twitter.

In his May 31 letter, Johnson said he wanted to meet with Gottlieb to hear what the commissioner had in mind for implementing the law but also to set the FDA leader straight on the intent of the legislation.

The senator took particular issue with comments Gottlieb had made in a May 17 interview with Stat News, in which the commissioner said the FDA would "have to do a little bit more" work in writing "guidance and perhaps in regulation to achieve" the goals of balancing access to experimental medicines with the appropriate patient protections.

Johnson, however, said the FDA has limited control over the right-to-try process — unlike the agency's compassionate-use program, in which regulators help terminally ill patients obtain and use investigational medicines outside of the clinical trial process.

"As I made clear to my colleagues in the Senate and the House before each body voted on S. 204, this legislation is fundamentally about empowering patients to make decisions in cooperation with their doctors and the developers of potentially life-saving therapies," the lawmaker wrote.

Given Gottlieb's comments, the senator said he wanted to "make this legislation's intent absolutely clear." Johnson noted the law's "life-threatening disease or condition" criteria was broader than the designation Gottlieb had sought.

The commissioner had proposed adding "immediately" in front of "life-threatening" to ensure patients with chronic diseases, like diabetes, were not swept into the criteria and unnecessarily exposed to side effects from unapproved medicines.

Johnson said he wanted to ensure his legislation would specifically be open to patients with Duchenne muscular dystrophy — a condition affecting one of the patients for whom the bill was named, Jordan McLinn.

McLinn's mother, however, told CBS News the child had been enrolled in a clinical trial for more than a year — a status that ensures certain oversight and safety protections by the FDA, which are not afforded to patients seeking experimental medicines under the Right To Try Act.

Under the law, biopharmaceutical manufacturers are protected against liability and cannot be forced to provide their products to patients.

Johnson noted the Right To Try Act forbids the FDA from using a clinical outcome associated with the use of an experimental drug to delay or adversely affect the medicine's review or approval unless those data are critical to determining safety.

"This language is not intended to enable the FDA to expand the scope of existing safety determinations about investigational drugs," said Johnson.

Under the Right to Try Act, any drugs patients request access to must have completed a phase 1 trial. Those studies, however, only check for toxicity, not safety or efficacy.

Who's responsible?

An FDA spokeswoman told S&P Global Market Intelligence in a May 31 statement that the agency was "convening an internal group to assess how to effectively and efficiently implement the new law."

"We will report on our implementation steps regularly," she said.

A day earlier, Janet Woodcock, director of the FDA's Center for Drug Evaluation and Research, told her staff to refer people to the drug's manufacturer if they received inquiries from patients or doctors about specific products, since the company would be in "the best position to provide information on the development status of their products" and whether the drugmaker "intends to make an investigational product available under right to try."

If manufacturers contact the FDA about their obligations under the new law, "we suggest that you refer them to the statute," Woodcock said in a memo to her staff, which was provided to news reporters.

While Trump vowed that "countless American lives will ultimately be saved" from the Right To Try Act, a top White House official said neither Gottlieb nor Health and Human Services Secretary Alex Azar could be held accountable for fulfilling that promise.