What Trump got wrong about 'right to try'

Kadia Tubman
Reporter
Donald Trump gestures during a rally in El Paso, Texas, Feb.11, 2019. (Photo: Leah Millis/Reuters)

At a rally in El Paso, Texas, Monday night, President Trump bragged about his “right-to-try” law, as he’d done earlier in his State of the Union address. Only this time he added how much the law, which allows terminally ill patients access to unapproved experimental drugs, is saving lives.

“Nobody will understand the lifesaving effects,” said Trump. “People are getting better that were thinking that they were going to die.”

The crowd cheered in response.

“We have drugs in the pipeline that are showing tremendous progress,” he said. “If somebody is terminally ill let’s let them have access to our drugs.”

In his first address to Congress, Trump called for a federal “right-to-try” law, modeled on existing laws in some states. The law, which passed Congress and was signed by Trump last May, gives patients with fatal diseases the ability to request experimental treatment directly from drug companies without approvals from the Food and Drug Administration (FDA).

But it appears only two patients have been treated under the act, and it is much too soon to tell whether it has saved any lives.

“I always wondered why we didn’t do it before,” Trump said before moving onto his next rally topic.

Before “right to try,” there was a program in place that also allowed patients with fatal conditions to try experimental treatments: The FDA’s expanded access program, also called “compassionate use,” which began in the 1970s. But “expanded access” required case-by-case approval by the FDA — which was granted in virtually every instance in recent years, although not necessarily immediately. “Right to try” involves a direct transaction with the drug manufacturer.

Applications to try experimental drugs under compassionate use increased during the HIV epidemic, and have picked up again in the last five years, particularly in response to the Trump administration’s deregulation push.

“‘Right to try’ is about an individual and the individual’s right to try to save their own life,” said Naomi Lopez Bauman, director of health care policy at Goldwater Institute, the libertarian think tank behind the first state-level “right-to-try” law in 2014. “No one’s compelled to participate, but it creates another pathway so that patients can pursue potentially lifesaving treatments.”

She added: “This is very early on [in the process]. The law will be a year old in May. It’s going to be a long time before the FDA has the data about how many people have been treated. And ultimately, more patients over time sharing their stories.”

Trump displays the signed “Right to Try” Act, which gives terminally ill patients the right to use experimental medications not yet approved by the FDA, May 30, 2018. (Photo: Leah Millis/Reuters)

“[Right to try] is an alternate pathway to the same end as what we previously had,” Alison Bateman-House, co-chair of the NYU School of Medicine Working Group on Compassionate Use and Pre-Approval Access (CUPA), told Yahoo News. “And at this point, we’re only aware of two patients who have taken advantage of it.”

The two cases are a brain cancer patient who has received an experimental anticancer vaccine and an ALS patient who has been receiving experimental treatment. A year is not enough to determine the new law’s success rate, but decades with the past program speak for itself.

Compassionate use “involves the FDA and involves something called the institutional review board, both of which were there to make sure that patient got the best possible chance at succeeding at their attempt and not being exploited by someone looking to take advantage of a desperate patient,” said Bateman-House. “[Trump’s] path has gotten rid of both of those safeguards.”

The White House did not immediately respond to a request for comment.

Opponents to “right to try” argue that the law gives terminally people false hope and opens the door to exploitation by drug companies. “All they’ve done is taken away some of the protections that [patients] should have,” said Bateman-House.

But supporters say that by sidestepping the FDA’s approval, which was granted 99 percent of the time from 2010-2014, they can more readily access drugs that could save their lives.

But the law doesn’t ensure companies will provide the treatments or drugs. Less than a year after “right to try” became law, patients are still waiting for time-sensitive treatment.

Before “right to try,” dying patients could also obtain experimental drugs through clinical trials, which were selective and required traveling. Under “compassionate use,” access could be denied by the FDA if the risks were considered too great or there was a potential for an alternative treatment.

Drugs approved by the FDA typically go through a three-step process of clinical trials to determine a safe and effective dose. Pharmaceutical companies spend hundreds of millions of dollars testing new drugs under rigorous conditions, comparing them to placebos and to existing treatments, before they can be approved. The system was designed both to shield consumers from dangerous and ineffective drugs, and companies from demands from patients for untested and possibly unsafe therapies.

Now, under “right to try,” one trial for safety will suffice. The FDA offers guidance on “right to try.” But patients, doctors and drug makers can arrange their own terms for treatment. Companies will have to determine if they want to risk treating certain patients or if they have enough supply to meet demands, and patients may be asked to pay for the drugs out of pocket.

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