WASHINGTON — Sen. Ron Johnson, the author of the federal “right-to-try” law signed by President Trump this week, wants to make one thing clear: His new law is meant to weaken the Food and Drug Administration.

“This law intends to diminish the FDA’s power over people’s lives, not increase it,” he wrote in a letter to Commissioner Scott Gottlieb Thursday.

The Wisconsin Republican wrote to Gottlieb to request a meeting about the implementation of the new law, which sets up a new pathway for patients with life-threatening illnesses to request access to experimental therapies the FDA has not approved.

advertisement

Johnson’s comments give new credence to a longstanding criticism of the law from medical ethicists and several former FDA commissioners, who suggested that a separate pathway with less FDA oversight would undermine the agency’s ability to oversee product safety and efficacy. During the legislative process, supporters often said the legislation was about giving dying patients new hope, not about weakening any agency.

There were hints of that intent, however. The law’s main advocate was the libertarian think tank the Goldwater Institute, and Americans for Prosperity, a group backed by the conservative Koch brothers, also offered advertising and advocacy support.

In his letter, Johnson took particular issue with several comments Gottlieb made to STAT earlier this month, in which the commissioner suggested that the law would require further regulation and guidance to protect patients.

This law “is designed to work within existing FDA regulations, definitions and approval processes. 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,” Johnson wrote.

It is clear Gottlieb anticipated the FDA would have to take some action to implement the law, though it isn’t clear if the implementation process he referenced in a statement after the signing ceremony would require “guidance, rules, or regulation,” as Johnson described.

“At the FDA, we stand ready to implement this legislation in a way that achieves Congress’ intent to promote access and protect patients,” he said.

Even Trump, at the signing ceremony for the bill this week, suggested that Gottlieb should get to work implementing the law.

“Scott, can you start immediately? We don’t want to start tomorrow,” he said to the FDA commissioner. “What time is it now? How about in 15 minutes?”

Newsletters

Sign up for our Morning Rounds newsletter

Please enter a valid email address.

An FDA spokesperson said Wednesday that the FDA is convening an internal group to assess how to effectively and efficiently implement the new law and would report regularly on implementation steps.

In a separate email to staff Wednesday, Janet Woodcock, who directs the agency’s Center for Drug Evaluation and Research, told staff to direct any inquiries about the new law to drug companies.

“We believe that sponsors are in the best position to provide information on the development status of their products,” Woodcock wrote.

After the letter was published Thursday, an FDA spokesperson said the agency “will review the letter and respond directly to the senator.”

Ike Swetlitz contributed reporting.

Leave a Comment

Please enter your name.
Please enter a comment.

  • Huh? That was a confusing article. Don’t know what to think of new law or what everyone’s real motivation is.

  • Listen to one of the few stage iv pt.s to get pre- approval access – my wife Lorraine. She is the first pt. to testify for RTT and her story is on Goldwaters website. The FDA of the early Obama yrs. was ” flexing it’s regulatory muscles ” with Pharma ( that’s from the WSJ, 2011 ) and we pts. were caught in the middle . The FDA paperwork back before RTT was hours and hours , tens of pages long. Since RTT much more streamlined. They richly deserved what they got with RTT. We pts were powerless but we got respect from Goldwater and publicity .

  • It seems confusing to write regulations and guidance related to legislation that essentially bypasses FDA. Ostensibly, FDA has no role if drug sponsors choose to go this route. (They still have the option of using the existing FDA pathway, with it’s ethical oversight and patient protections.)

    BUT, wait a minute! According to the legislation, “The manufacturer or sponsor of an eligible investigational drug shall submit to the Secretary an annual summary of any use of such drug under this section. The summary shall include the number of doses supplied, the number of patients treated, the uses for which the drug was made available, and any known serious adverse events. The Secretary shall specify by regulation the deadline of submission of such annual summary and may amend section 312.33 of title 21, Code of Federal Regulations (or any successor regulations) to require the submission of such annual summary in conjunction with the annual report for an applicable investigational new drug application for such drug.”

    So, regulation IS required by the legislation. There is, however, no apparent enforcement authority for this requirement included in the bill.

    People like Trickett, Frank, Jordan, and Matthew have always had the right to ask drug developers for access to their promising investigational therapies. Drug sponsors have had, for decades, a viable pathway to make those investigational agents available outside of their on-going clinical trials for eligible patients (same eligibility requirements as RtT), if they chose to do so. The new legislation is also based on their willingness to do so.

    Although Johnson says this legislation is in the interest of patients, many well-respected national patient organizations actively opposed this legislation.

    Johnson’s objective seems to be to weaken FDA’s regulatory oversight authority, reducing protections for the patients it ostensibly claims to help.

    Possibly this is the wedge in the door to strip FDA of other, more wider-reaching regulatory authorities, ultimately putting the US drug development process, and all patients, at greater risk of harm, and giving them access to less proven and less effective therapies.

  • I pick: “just cynically making a buck” …a whole new round of Peachpit Enemas on overnight TV and AM radio. Betcha, the next round from these pols is forcing Medicare to pay for it. …because, where do they get their money?

  • this is not about accessing drugs legitimately in development by legitimate biopharma companies – these are already accessed rather routinely.
    the danger in this law is about desperate patients being sold snake oil by physicians and physician-wannabes and manufacturers who have no intent of ever seeking FDA approval for a given treatment. They may be true believers in what they are peddling legitimately works, or just cynically making a buck at the expense of sick patients and families. It is not always easy to separate the 2.
    The FDA conundrum is when they know a treatment to be dangerous and there is no good data proving efficacy. A core mandate of the agency from when it was founded was to protect the public from known dangerous foods and drugs. Senator Johnson seems to be indicating he wants to restrain this.
    This is a very dangerous path, and will enrich a lot of quacks and cynical predators with little – if any – benefit to patients.

Sign up for our Daily Recap newsletter

A roundup of STAT’s top stories of the day in science and medicine

Privacy Policy