No, Trump’s drug program for terminal patients did not save “thousands” of lives – my assessment

Former President Donald Trump has bragged in recent months about the “Right To Try,” a law he signed in 2018. It aims to improve terminally ill patients’ access to potentially life-saving medications that are not yet approved by the Food and Drug Administration (FDA).

“We have things to combat disease that won’t be approved for another five or six years and that people who are very sick or terminally ill should be able to use. But there was no mechanism for that,” Trump said on August 30 in Washington, DC in front of supporters of the conservative parental rights advocacy group Moms for Liberty.

He also said that through Right To Try, “we have saved thousands and thousands of lives.”

Trump similarly praised the program during an Aug. 17 rally in Pennsylvania, in a podcast interview with a conservative commentator and during his acceptance speech at the Republican National Convention: “Right To Try is a big deal,” Trump said at the time.

But medical experts who have studied the experimental treatment program say there is no evidence to support Trump’s claims. These experts say Right To Try has weakened regulations designed to protect patients.

What’s the right thing to try?

The Trickett Wendler, Frank Mongiello, Jordan McLinn and Matthew Bellina Right To Try Act, also known as the Right To Try Act, was passed by Congress on a bipartisan basis and signed into law in 2018. The goal was to streamline the process of obtaining potentially life-saving drugs that are not yet approved by the FDA for terminally ill patients. Speed ​​is important; Industry groups say it takes an average of 10 to 15 years for a new drug to reach pharmacy shelves.

However, a similar FDA program, the Expanded Access Pipeline, sometimes called “compassionate use,” has existed since the 1970s and became law in 1987.

And that is the root of many criticisms of Right To Try.

“Right To Try is essentially an ‘expanded access light,'” said Alison Bateman-House, a medical ethicist who studies access to investigational medical devices at New York University’s Grossman School of Medicine.

Right To Try targets fewer patients than expanded access and offers them fewer treatments, Bateman-House said.

Patients must meet specific but different criteria to qualify for one of the experimental drug programs.

To qualify for expanded use, patients must have a “serious or immediately life-threatening disease or condition” for which there is no “comparable or satisfactory alternative therapy to diagnose, monitor, or treat the disease or condition,” according to state regulations ” gives. Clinical trials must be unfeasible for patients and the use of these drugs must not interfere with ongoing trials. Additionally, the potential benefits must justify the risks, according to prescribers.

After identifying a treatment, the patient’s doctor must then seek approval from the manufacturer, the FDA, and the institutional review board that oversees the drug’s clinical trials.

The FDA said these steps exist so the agency can “fairly weigh the risks and benefits” of the drug and protect patient safety. The agency also collects data on the drugs’ clinical effects on patients and any side effects to include in the broader approval process for the drug.

Right To Try wanted to speed up this approval process. For example, under the new program, a doctor simply needs to identify an experimental drug and obtain permission from the manufacturer to use it. In most cases, the FDA does not have the authority to approve or reject the application and there is no review committee process to navigate.

However, because of the Right To Try program definitions, fewer patients and fewer medications are eligible.

Under the Right To Try, patients must have a “life-threatening” illness or condition, not just “serious” as in expanded access. Experimental drugs are only available after they complete Phase 1 clinical trials; Treatments accessed through the Expanded Access Program may be administered during a Phase 1 trial.

“Right To Try,” which includes liability protections for manufacturers and prescribers, also weakens requirements governing how doctors disclose the risks of experimental drugs to patients, leaving informed consent undefined. And it prevents the FDA from using information about patients’ tolerance of the drugs to “delay or negatively influence the review or approval of such drugs” unless top officials justify the public health benefit in writing.

Proponents say Right To Try is an example of successful deregulation and claim the more efficient approval process has saved lives. But critics see this as a major cause for concern because it “opens up the possibility of exploiting desperate patients,” said Holly Fernandez Lynch, a bioethicist who studies pharmaceutical policy at the University of Pennsylvania’s Perelman School of Medicine.

Government data shows that regulators were not the biggest hurdle for patients seeking experimental drugs. The FDA almost always approved expanded access requests, and did so quickly by the government’s standards.

According to a 2018 FDA report on the expanded access program, the FDA approved 99% of the approximately 9,000 applications received over the past five years and approved emergency applications for experimental drugs in less than a day on average. Recent data shows that the approval trend continues, even as the number of applications increases each year.

In rare cases where the FDA did not automatically approve applications, regulators often did not reject them but instead recommended adjustments to the requested dosage to address safety and effectiveness concerns.

Right To Try by the Numbers

The FDA does not provide detailed information about the number of doses provided or patients treated under the Right To Try. Instead, only an annual summary is published showing how many drugs have been approved under the program. The agency says it has approved 16 treatments since Right To Try began in 2018: 12 from 2018 to 2022 and four last year.

The FDA declined to provide additional information about the number of Right To Try applications or approvals.

Although the 16 drugs approved through Right To Try may have been administered to more than one patient each, experts believe it is extremely unlikely that thousands of patients were involved, as Trump said.

Trump’s claim represents an “egregious overestimation of the number of people using the right to try,” Fernandez Lynch said, noting that she believes the actual numbers are “very, very low.”

The Trump campaign did not respond to multiple requests for the source of the former president’s statistics. Karoline Leavitt, the campaign’s national press secretary, told KFF Health News that “in a second term, President Trump will of course remain open to other avenues to expand the ‘right to try’ to save more American lives.”

It remains unclear how Trump might expand the program, although the conservative Goldwater Institute is pushing for “Right To Try 2.0,” which would supposedly allow patients to receive customized therapeutics.

Experts noted that such drugs are already accessible through the expanded access program.

There is now evidence that the high price of experimental treatments, sometimes available through certain drug company programs but typically not covered by insurance, is a bigger hurdle for patients than regulatory guardrails.

“I don’t think people have a problem with the FDA blocking access to individualized therapeutics,” Bateman-House said. “I think the problem is that individualized therapeutics are incredibly expensive and there are only a very small number of researchers in the country who know how to make them.”

Our verdict

Trump has maintained throughout the campaign that his “Right To Try” program is new and has saved thousands of lives. But a similar program has been around for decades, and there is no evidence that Right To Try had anywhere near the impact Trump claimed.

Neither the Trump campaign nor Right To Try advocates provided evidence to support claims of widespread benefit. And government data shows that only 16 drugs were approved in the program’s first six years, with no information on how many patients took those drugs or what clinical outcomes were achieved.

Additionally, public health experts say Right To Try weakens patient protections and fails to address the real barriers to experimental drugs.

We rate Trump’s claim as false.

KFF Health News is a national newsroom that produces in-depth journalism on health issues and is one of the core operating programs of KFF – the independent source for health policy research, surveys and journalism. This story was produced in collaboration with PolitiFact. It may be republished for free.