With patients demanding experimental drugs, ‘right to try’ is becoming the law of the land

Written by Bob Tedeschi for Stat News.

Harrisburg, Pa. — To hear Bob Godshall tell it, he has no business being alive.

Eleven years ago, at age 72, he was diagnosed with multiple myeloma, a type of bone cancer. His doctors told him to get his affairs in order. He’d be gone within a year.

But when Godshall learned some myeloma patients bought time with bone marrow transplants, he asked his doctors if he could get one, too. “No way,” they said, citing his advanced age.

Godshall, who had at that point been a member of the Pennsylvania state legislature for more than 20 years, made a few calls. He got the treatment — after signing “stacks of paper” assuring doctors they would face no liability if he died — and lived long enough to see the approval of a new drug that has since kept his disease under control.

The experience convinced him that no terminally ill patient should ever hear “no way” again.

He’s not alone. Over the past three years, “right-to-try” advocates in 33 states have helped enact legislation to eliminate legal obstacles blocking terminally ill patients from treatments that aren’t yet approved by the Food and Drug Administration. Those advocates are showing considerable momentum in the remaining 17 states, potentially upending the established order for experimental drugs.

The movement has been fueled in no small part by the anti-regulatory sentiment that propelled Donald Trump’s rise to the presidency and by the explicit support of Vice President Mike Pence.

Some physicians, ethicists, and regulatory officials say the laws could harm more patients than they help — but many are reluctant to publicly oppose the laws for fear of being seen as opposing any one patient’s quest to save his or her life. Lawmakers, critics say, can stand on high moral ground as champions of the dying, while opponents struggle to demonstrate potential harms to faceless patients.

“It’s extremely hard to articulate a position against this because it sounds like you’re not sympathetic to a patient’s plight,” said Beth E. Roxland, senior adviser on law, ethics, and policy at NYU Langone Medical School.

Roxland, who previously led Johnson & Johnson’s bioethics and business strategy, said if the laws are enacted in all 50 states and are extensively used, “our entire drug development system would fail, frankly.”

Those arguments have not moved Godshall, a Republican who authored the right-to-try bill that is now under consideration in Pennsylvania.

“I just don’t feel that other people that aren’t in the Legislature shouldn’t have the same right I had,” he said in a recent interview.

Asked to handicap the bill’s chances, Godshall said he had some favors to call in.

“I’ll get it through,” he said.

The movement to provide experimental drugs to the terminally ill has its roots in the HIV epidemic, when people with the virus sought access to experimental drugs through sometimes illicit means, as dramatized in the movie “Dallas Buyers Club.”

But the current spate of legislation began in 2014, largely at the prompting of the Goldwater Institute, a Phoenix-based libertarian think tank that objected to what it saw as unnecessary governmental interference between patients, their doctors, and pharmaceutical companies.

Before right-to-try laws were passed, patients who exhausted conventional medical treatments but wanted to try experimental drugs had only two options. They could seek a clinical trial, which often involves travel and health-related requirements that are hard for many dying patients to meet. Or they could petition the FDA for access to drugs under the agency’s “expanded access,” or “compassionate use” program.

Under that program, doctors, patients, and drug manufacturers ask the FDA for the right to use a drug or device that has entered, but not yet completed, the agency’s drug approval process. A doctor’s office becomes a satellite clinical trial site, complete with an oversight panel.

The FDA approves nearly all such requests.

But critics say the process for applying for expanded access is still complex and time-consuming, especially for doctors with no experience offering experimental treatments, and right-to-try proponents say that discourages doctors and drug makers from entering the process.

Their new state laws, built on model legislation from the Goldwater Institute, seek to remove the FDA from the equation, allowing doctors, patients, and drug makers to strike their own deals for drugs that have cleared the safety phase of FDA testing.

Last year, in a move that was seen by some as a response to right-to-try laws, the FDA overhauled its compassionate use program to ease the application burden, but by that time the right-to-try movement had gained significant momentum.

California, Connecticut, Georgia, Idaho, Maine, New Hampshire, South Carolina, and West Virginia passed versions of the law in 2016. Of the 17 states that have not passed right-to-try laws, the biggest are New York, New Jersey, Maryland, Massachusetts, Washington, and Pennsylvania.

“We feel like we’re at the tipping point,” said Starlee Coleman, a spokeswoman for the Goldwater Institute.

In Maryland, state senators were showing bipartisan support for a right-to-try bill earlier this month, according to the state Senator Bryan Simonaire, who authored the legislation.

Simonaire, who is a Republican, said he and the bill’s sponsor in the House of Delegates, Karen Lewis Young, who is a Democrat, withdrew the bill last year to gather more information on behalf of various interest groups that had questions. He said representatives from Johns Hopkins Medicine, for example, were worried that some patients might avoid clinical trials by seeking experimental treatments directly from drug makers — thereby undermining the clinical trial system.

As a result, Simonaire changed the bill to require patients to seek their preferred treatment first through a clinical trial.

Simonaire said he expects “smooth sailing” once the bill reaches the Senate floor, which, he said, could happen as soon as next week.

“One of the things that’s fascinating about this issue and why it worked so quickly, is that there wasn’t a lot of organized opposition,” said Coleman. “Rarely will someone testify against a bill. There are people who say they are concerned about particular aspect of the language, but not a lot of people who say I’m opposed to this bill and here’s why.”

Roxland, the NYU ethicist, is watching, dismayed, as the right-to-try bills sweep the country.

“What I see is, unfortunately, very little pushback in terms of what the downsides could be,” she said. “If anything, it’s just picking up steam.”

Roxland said she has spoken to medical industry leaders and policymakers who support the rights of patients to seek potentially lifesaving treatments, but who feel the right-to-try laws, as written, will ultimately harm patients. She said these leaders are frequently reluctant to to express nuanced objections in the face of more emotionally charged arguments. “It’s hard to argue against when it’s framed as the terminally ill having the right to save their lives,” she said. “How do you argue against that?”

This could be one reason why the FDA and major trade groups, including the American Medical Association and the Pharmaceutical Research and Manufacturers of America, have taken no public stance on the legislation.

More recently, though, high-profile figures have voiced concerns. In US Senate hearings on federal right-to-try legislation in the fall, Dr. Peter Lurie, associate FDA commissioner, defended the agency’s expanded access program, and cautioned against “changes that would reduce FDA’s role in expanded access, or that might undermine the crucial clinical trial development process.”

Meanwhile, Dr. John Jenkins, who, until this year, oversaw the FDA’s drug development program, told STAT that right-to-try initiatives “open a Pandora’s box for potential harm to the patient.”

By removing the FDA from the discussion between patients and doctors, he said, the laws prevent patients from receiving important information about investigational drugs and place patients “at unnecessary risk of harm and exposure to potential medical fraud.”

Influential patient advocates, too, have begun speaking against the legislation — while stressing their support for the right of the terminally ill to seek treatments.

In an interview with STAT last week, the founder of Friends of Cancer Research, a Washington-based advocacy group, related the experience of her sister, Gale, who 20 years ago died as a result of an experimental bone-marrow transplant procedure she sought in the late stages of a cancer battle.

Clinical trials, said Ellen V. Sigal, have since shown that the therapy “made things much worse for patients.”

Sigal said proposed federal right-to-try legislation, as well as state versions, “won’t help patients. It will hurt them.”

Sigal and other critics say that while the state legislation requires drug makers to offer information about possible “worst outcomes,” these drug makers may not know all of those outcomes based on limited data from an early trial. They may also lack information that the FDA would have in hand, such as how patients with similar physical circumstances fared with similar investigational drugs from other manufacturers.

For many of the drugs patients might receive through right-to-try channels, Sigal said, “there’s no efficacy data at all. At least for dying patients, give them something that has some promise, some data.”

Godshall, who is the most senior member of Pennsylvania’s House of Representatives, was preparing to travel again recently to Philadelphia for his quarterly checkup, where he would give seven vials of blood and await word on his condition.

He said his cancer has begun catching up with him, to the point where he will not run for re-election when his current term expires next year. “I can’t play golf, I can’t go hunting,” he said. “The cancer has taken all my physical strength away from me.”

The years following his transplant were often difficult: He lost both his wife and a son to cancer. Yet he believes his legislation could define his legacy.

“This bill gives people a chance, like I had, to live,” he said. “I don’t think any bill I’ve done — which is a lot — would be more important.”