“We will be saving — I don’t even want to say thousands, because I think it’s going to be much more, thousands and thousands, hundreds of thousands. We’re going to be saving tremendous numbers of lives.”
The legislation that Trump signed into law Wednesday allows terminally ill patients to seek access to experimental medicine that is not yet fully approved by the Food and Drug Administration.
Whether it will save “hundreds of thousands of lives” is a prediction that is, at best, unclear. But the effect of similar laws in some states has been muted.
A program known as compassionate use, or expanded access, has been in place since the 1970s. It allows patients with a serious disease or condition to obtain experimental medicines; the FDA says it authorizes 99 percent of the requests for expanded access that it receives.
The new national law — like similar laws in more than three dozen states — allows patients and doctors to ask drug companies directly for access to the experimental drugs, rather than wait for approval by the agency.
Yet these laws “do not ensure that manufacturers will provide the drug or that insurance companies will cover the cost,” according to a policy report from Rice University. Obtaining the medicines from manufacturers can be more cumbersome than going through the FDA’s existing program, the report found.
Colorado enacted the first right-to-try law in 2014. Since then, “there have been no documented cases of anyone receiving access, because of a right-to-try law, to an experimental product that would not have been available via the FDA’s expanded access program,” a 2017 study by researchers from New York University concluded.
Alison Bateman-House, a professor of medical ethics and an author of the study, called it “extremely unlikely” that the national right-to-try law would directly save hundreds of thousands of lives, given that drug companies are using — and accustomed to — the existing process.
“It’s impossible to tell how many people will be helped by the federal right-to-try law,” said Christina Sandefur, the executive vice president of the Goldwater Institute, the conservative group that spearheaded the effort.
But “if it saves one person, it’s worth it,” she said, adding, “For the patients who are turned away from clinical trials and who are unable to navigate the complex expanded access process, right to try will give them a last chance — and the right to hope.”
While the law itself may have a minimal effect, Bateman-House said, the right-to-try movement and the attention that Trump has drawn to it are positive steps toward raising patients’ awareness for experimental treatment. She predicted that the number of patients asking for developing medicines will increase — just under the current expanded access program.
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