Legislation & Regulation

Can Congress Get ‘Right to Try’ Right?

A federal bill would expand access to experimental treatments, but critics say right to try would take away FDA oversight and create a ‘Wild West.’ Meanwhile, most states’ right-to-try laws have gone unused.

Richard Mark Kirkner
Contributing Editor

In the world of “right to try”—the concept that seriously ill people should have access to experimental drug treatments—Ebrahim Delpassand, MD, stands tall. A radiation oncologist in Houston, Delpassand is the one physician the Goldwater Institute, which has pushed right-to-try to the cusp of being federal law, offers as a success story for the movement it has spawned.

Since Texas passed its right-to-try law in 2015, Delpassand has treated about 100 patients with neuroendocrine cancer in a right-to-try protocol using lutetium Lu 177 dotatate, a drug the FDA just approved in January. Texas is one of 39 states that have adopted right-to-try laws, but it’s the only state where it has been used, thanks in large part to Delpassand.

It’s those largely unused state laws that got the Goldwater Institute, a libertarian think tank, to thinking that uncertainty about FDA jurisdiction over these state laws made them untouchable for physicians and drug companies. So, Goldwater took its idea to Congress to clear up that confusion.

Federal right-to-try legislation has been the target of last-ditch efforts to block it. Scores of patient advocacy groups have weighed in against it. So have four former FDA commissioners. The current commissioner, Scott Gottlieb, MD, has expressed misgivings, as has PhRMA, although officially the drugmakers’ group hasn’t taken a position on the legislation. President Trump has said he would sign it.

Seriously ill people can currently access investigational drugs through an existing FDA program called compassionate use. It involves an application and review process that critics say can be cumbersome, along with agency oversight of the treatment, and it holds drug companies accountable for adverse events. The FDA says it approves 99% of the 1,000 compassionate use requests it gets a year. Right to try would essentially parallel compassionate use but without the regulatory hurdles. Depending on your perspective, it effectively eliminates FDA oversight of treatments or gets rid of a lot of red tape for people who don’t have much time to live.

The debate over right to try has been vigorous. While proponents say it would make it much easier for the terminally ill to get investigative treatments and clarify risks for treating physicians and drug companies, critics say it’s part of the right-wing conspiracy to dismantle federal regulations, would offer false hope to dying patients and their families, and probably wouldn’t make much difference in getting experimental treatments to terminally ill people anyway.

Collecting cobwebs

Starlee Coleman, the Goldwater senior policy adviser who’s guiding the right-to-try initiative, says doctors and patients haven’t taken advantage of right-to-try laws in states other than in Texas because FDA oversight discourages drug companies from participating. “State laws can do everything we need except dictate any kind of action by the FDA,” she says. “Only the federal government can do that.” Typically, Goldwater gets involved in state legislative efforts, but in this case the federal law hampered the state laws Goldwater advocated. “There was no grand master plan,” Coleman says when asked if federal legislation was the ultimate goal.

Right-to-try laws at the state level are constitutionally questionable, says Alison Bateman-House, an NYU medical ethicist.

Alison Bateman-House, a medical ethicist at New York University who co-chairs an academic working group that advises the FDA on compassionate use, calls Texas “sort of a weird creature.” State right-to-try laws are “constitutionally questionable,” she says, “because states cannot claim for themselves federal powers.”

State right-to-try laws are collecting cobwebs for other reasons, says Bateman-House, including lack of physician awareness or interest; institutional reluctance to allow their physicians to participate; and pharmaceutical company reluctance to put their investigational drugs out there for fear of an adverse event that could derail FDA review and approval. The dearth of patients means, in Bateman-House’s view, that right to try is “a marketing slogan.”

Coleman acknowledges that state right-to-try laws are largely based on model legislation the Goldwater Institute drafted. The organization also helped draft the legislation that Senate sponsor Ron Johnson, a Wisconsin Republican, pushed through the Senate last summer and served as a template for the House version.

The Senate took up legislation when Johnson threatened to use his senatorial privilege to block reauthorization of FDA funding. The Senate was “backed into a corner,” says Bateman-House. “The Senate did not want to pass the right-to-try bill.” But senators wanted to reauthorize FDA funding, so the bill passed by a nearly unanimous vote of 94 to 1.

In the House, the legislation couldn’t even make it out of committee till Johnson and Vice President Mike Pence started applying some pressure. The bill couldn’t muster the two-thirds majority needed when it advanced on a fast-track procedure. A week later when only a simple majority was needed, 35 Democrats joined with 232 Republicans to push it across the finish line. Now the legislation goes back to the Senate.

Critics see far-reaching consequences if the bill becomes law. Andrew McFadyen, executive director of the Isaac Foundation, an advocacy group for patients with rare diseases, told Politico the aim is to dismantle the regulatory process. “Tear down as many regulations as possible, take away all oversight, and let it be the Wild West of medicine,” he said.

Fixing compassionate use

Gottlieb has defended what the FDA is doing to grant terminally ill patients access to investigational drugs. In testimony before a House subcommittee last fall, Gottlieb said his agency is simplifying the compassionate-use application process, providing clearer instructions on how to submit requests, disseminating information about ongoing clinical trials, and setting up an online navigator to guide applicants.

He also argued for keeping the FDA involved in access to investigational agents that are outside the confines of a clinical trial. “While the agency permits almost all expanded access applications to proceed, it makes meaningful changes in approximately 10% of these cases to enhance patient safety,” Gottlieb said in his testimony. PhRMA agrees with Gottlieb and says that FDA oversight needs to continue.

Dying people shouldn’t have to apply to the federal government to save their own lives, says Starlee Coleman of the Goldwater Institute.

But Goldwater’s Coleman says the FDA’s current process can be onerous for people with only a short time to live. For example, she says that the requirement that an institutional review board oversee the protocol is a major barrier for people in rural areas who can’t travel to one of the academic medical centers that typically runs the protocols. But the institute’s objections are more fundamental: “Our biggest issue with the existing process is that we do not believe that dying people should have to file an application to the federal government to save their own life,” Coleman says. “They don’t have time.”

Getting right-to-try laws right

Despite having some momentum, the right-to-try legislation has run into some opposition. Seventy-five patient safety groups sent a letter to House leaders criticizing the House bill before the first vote in March. In a statement to the Washington Post, former FDA Commissioners Robert Califf, Margaret Hamburg, Andrew von Eschenbach, and Mark McClellan said there was no evidence that the House or Senate bills “would meaningfully improve access for patients.”

In taking up the House right-to-try bill, the Senate would have to accept a few changes from the bill it passed last year. The original Senate bill says “prescriber, dispenser, or other individual entity” would not be liable for anything that goes wrong in a treatment protocol. The House version would hold a “physician, clinical investigator, or hospital” liable for misconduct and negligence. Both bills say manufacturers would have no liability, although the language differs.

The Senate bill defines eligible people as having a “life-threatening disease or condition”; the House bill defines them as people with a “terminal illness.” As Bateman-House points out, diabetes could be construed as a life-threatening disease. Gottlieb has suggested this language: “a stage of disease in which there is a reasonable likelihood that death will occur within a matter of months.”

What happens next could determine if Delpassand is a pioneer or a just a lone wolf.


Legislation and regulation
By Richard Mark Kirkner
Legislation and regulation
By Richard Mark Kirkner

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