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NEJM: Right-to-Try Laws Could Still Undercut FDA Authority, Even if Ineffective

January 22, 2018

While the versions of federal right-to-try legislation pending on Capitol Hill are expected to have a limited impact on access to experimental therapies — and, therefore, limited potential to increase harm to terminal patients — they still represent a broader effort to weaken medical product regulation and FDA oversight, according to a new piece in the New England Journal of Medicine.

The bills target “alleged barriers to early access that aren’t actually rate-limiting — barriers that may even benefit patients — while leaving others in place,” wrote Steven Joffe and Holly Fernandez Lynch, medical ethicists from the University of Pennsylvania’s Perelman School of Medicine.

By cutting the FDA out of the process, they wrote, patients would be denied the benefit of safety recommendations and other protocol changes offered by the agency.

Rep. Morgan Griffith (R-Va.), sponsor of one of the federal bills, disagreed that right-to-try would threaten the agency’s authority.

“The FDA’s regulatory role is meant to protect people from unreasonable risk or harm by medical treatments or products,” Griffith said. “I don’t think their mission is undercut if a person confronting a terminal illness, who understands the risks, consults a doctor and pays for the treatment, decides an experimental treatment is the best course to pursue.”

But many drugs that appear promising in early development are later not proven safe or effective, cautioned the NEJM authors, who also warned about the lack of publicly available information for prescribers. In addition, expanding access to experimental drugs to patients outside clinical trials may delay the generation of data used for approval.

According to FDA Commissioner Scott Gottlieb, the agency makes safety-related changes in approximately 10 percent of expanded access requests — including modifications to dosing amounts, patient monitoring and informed consent — based on access to confidential commercial information unavailable to physicians and the public.

In addition, the FDA grants almost all expanded access applications, Gottlieb told a House health subcommittee during an October 2017 hearing on right-to-try. That same day, the agency also loosened the requirement that physicians receive approval from a full, convened IRB — instead allowing them to proceed with clearance from the chair or a designated board member — as a way to accelerate access. 

Meanwhile, a nationwide campaign led by the Goldwater Institute, a libertarian think tank, has resulted in the passage of 38 state right-to-try bills, with six becoming law in 2017. 

Largely, the legislation aims to bar the FDA from restricting a terminal patient’s access to drugs that have passed Phase I trials, deny the agency from considering any resulting outcomes in their eventual product review and insulate pharmaceutical companies and physicians from legal liability. 

Three different federal bills are pending in the House, one having passed the Senate in August, and the idea has been largely favored by the White House. 

Last week, Vice President Mike Pence said he met with Gottlieb to discuss the importance of passing such a bill, writing on Twitter: “It’s about restoring hope and giving patients with life threatening diseases a fighting chance. Let’s get this DONE.” As governor of Indiana, Pence signed his state’s right-to-try bill into law in 2015.

In a White House meeting with pharmaceutical industry executives in January of last year, President Donald Trump expressed how he was personally disturbed by the idea of terminal patients not being able to access experimental treatments.

“I am glad the concept of right-to-try has gained traction and hope to see a right-to-try bill, whether mine or someone else’s, sent to the President’s desk soon,” said Griffith. 

The House could bring up legislation as early as the last week of this month, according to the sponsor of another version, Rep. Andy Biggs (R-Ariz.). In a recent Washington Examiner editorial, Biggs said he was promised a floor vote on the topic by the end of January; however, the House is not in session the week of Jan. 22, instead returning Jan. 29.

The patchwork of differing state laws can create compliance headaches for pharmaceutical companies wishing to respond to right-to-try requests, and the current federal proposals do not do enough to smooth over the “substantial quirks” that fall along state lines, according to Alison Bateman-House, a bioethicist and assistant professor at NYU Langone Medical Center.

Texas, for example, bars manufacturers from charging for treatment, while other states allow it. Four more states’ laws do not apply to patients being treated in hospitals. Arizona does not allow primary care providers to request investigational treatments, while Oregon’s policy does not apply to patients under 18. Still more states have varying definitions of “terminally ill.”

“Regulatory folks at companies are going to have to know who’s getting the drug, and it’s going to vary what kind of protections or rules are demanded,” said Bateman-House, who co-chairs a NYU working group on expanded access. “And, of course, patients travel from state-to-state to seek specialty care.” 

“I’ve definitely gotten calls from pharmaceutical companies wanting to know what this means for them,” she added. “It’s anyone’s guess. … It’s legally sticky.”

This past week, Janssen, the pharmaceutical arm of Johnson & Johnson, announced that it would not field right-to-try requests as a matter of policy. Instead, physicians can continue to submit requests through traditional expanded access pathways, Janssen said. Neither right-to-try laws nor expanded access programs can compel a manufacturer to grant a request.

The company said it will not evaluate any requests that do not allow for input from the FDA, which Janssen described as an impartial stakeholder — citing the agency’s access to confidential information and oversight as critical to ensuring patient safety. 

 

—by Conor Hale @conorhale