Former President Donald Trump has boasted in recent months about the “right to try,” a law he signed in 2018. It aims to make it easier for terminally ill patients to access potentially life-saving medications that do not have not yet been approved by the Food and Drug Administration (FDA).

“We have means to fight diseases that will not be approved for five or six years and that very sick, terminally ill people should be able to use. But there was no mechanism to do it,” Trump said. said August 30speaking in Washington, D.C., to supporters of the conservative parental rights group Moms for Liberty.

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

Trump also praised the program during a Rally of August 17 in Pennsylvania, in a podcast interview with a conservative commentator, and at his Republican National Convention acceptance speech: “The right to try is a big deal,” Trump said then.

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

What is right to try?

THE Trickett Wendler, Frank Mongiello, Jordan McLinn and Matthew Bellina have the right to try actingaka Right To Try, was passed by Congress on a bipartisan basis and was signed into law in 2018. It aimed to streamline the process of obtaining potentially life-saving drugs that were not yet approved by the FDA for patients in terminal. Speed ​​matters; industry groups say we need to 10 to 15 years on average for a new drug to reach pharmacy shelves.

However, a similar FDA program, the expanded access a pipeline, sometimes called “compassionate use,” existed since the 1970s and became law in 1987.

And this is the origin of many criticisms of Right To Try.

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

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

Patients must meet specific, but different, criteria to be eligible for either investigational drug program.

To qualify for expanded use, patients must have a “serious or life-threatening disease or condition” for which there is no “comparable or satisfactory alternative therapy available to diagnose, monitor, or treat the disease or condition.” ‘affection’, according to government regulations. Clinical trials must be infeasible for patients and the use of these drugs must not interfere with ongoing studies. In addition, the potential benefits must justify the risks, according to prescribing physicians.

Then, after identifying a treatment, the patient’s doctor must receive approval from its manufacturer, the FDA, and the institutional review board overseeing the drug’s clinical trials.

The FDA said these measures exist so the agency can “fairly evaluate the risks and benefits” of the drug and protect patient safety. The agency also collects data on the clinical impact of drugs on the patient and any adverse effects to inform the broader drug approval process.

Right To Try sought to expedite this approval process. Under the new program, for example, a doctor must simply identify an investigational drug and receive permission from the manufacturer to use it. In most cases, the FDA has no authority to approve or deny the application and there is no review board process to follow.

But, due to Right To Try program definitions, fewer patients and fewer medications are eligible.

Under the right to try, patients must have a “life-threatening” disease or condition, not just a “serious” one, as under expanded access. Investigational drugs are only available after completing phase 1 clinical trials; treatments accessible through the expanded access program can be administered during a phase 1 study.

Right To Try, which includes liability protections for manufacturers and prescribing doctors, also weakens requirements that govern how doctors disclose the risks of investigational drugs to patients, leaving informed consent undefined. And it prevents the FDA from using information about how patients tolerate drugs to “delay or harm the review or approval of such drug(s)” unless senior officials justify in writing the benefit for public health.

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

Government data shows that regulatory agencies were not the main barrier patients faced when seeking experimental drugs. The FDA has almost always approved requests for expanded access, and quickly by government standards.

According to a 2018 FDA report on the Expanded Access Program, the FDA authorized 99% of the approximately 9,000 applications received over the previous five years, approving emergency applications for investigational drugs in less than a day on average. More recent data shows that the approval trend has continued, even though the number of applications has increased each year.

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

The right to try in numbers

The FDA does not share detailed information about the number of doses provided or which patients are treated under the right to try. Instead, he only publishes a annual summary indicating how many drugs have been approved under the program. The agency says that since Right To Try launched in 2018, it has approved 16 treatments: 12 from 2018 to 2022 and four last year.

The FDA declined to provide additional information on the number of right-to-try applications or approvals.

Although the 16 drugs approved under Right To Try were likely provided to more than one patient each, experts said it was extremely unlikely that thousands of patients were involved, as Trump said.

Trump’s claim represents a “gross overestimate of the number of people who use 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 inquiries about 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 advocates “Right to Try 2.0”, which he believes will allow patients to receive individualized therapies.

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

At the same time, data show that the high price of experimental treatments, which are sometimes available through some drug company programs but are generally not covered by insurance, poses a greater barrier to patients than are guarded. -regulatory fools.

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

Our decision

Trump claimed throughout the campaign that his Right To Try program was new and had saved thousands of lives. But a similar program has existed for decades and there is no evidence that Right To Try has had an impact comparable to what Trump claimed.

Neither the Trump campaign nor Right To Try advocates have provided evidence to support claims of widespread benefit. And government data shows that only 16 drugs were approved under the program in its first six years, without taking into account the number of patients who used those drugs or their clinical outcomes.

Additionally, public health experts have said the right to try weakens patient protections and fails to remove real barriers to experimental drugs.

We believe Trump’s assertion is false.

KFF Health News is a national newsroom that produces in-depth journalism on health issues and is one of the major operating programs of KFF — the independent source for health policy research, polling and journalism. This story was produced in partnership with Political fact. This can be republished for free.