Experimental ‘Right to Try’ drug program has saved ‘thousands and thousands of lives’

Former President Donald Trump on August 30

Former President Donald Trump has in recent months touted his “Right to Try,” a law he signed into law in 2018. It aims to increase terminally ill patients’ access to potentially life-saving drugs not yet approved by the Food and Drug Administration.

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

He also said that because of the right to try, “I have saved thousands and thousands of lives.”

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

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

What is right to try?

The Trickett Wendler, Frank Mongiello, Jordan McLinn and Matthew Bellina act for the right to tryaka Right To Try, passed Congress on a bipartisan basis and was signed into law in 2018. It sought to streamline the process of obtaining potentially life-saving drugs that were not yet FDA-approved for terminally ill patients. Speed ​​matters; industry groups say it’s needed 10 to 15 years on average, for a new drug to reach pharmacy shelves.

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

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

“The right to try is basically ‘extended access light,'” said Alison Bateman-House, a medical ethicist who researches access to investigational medical products at New York University’s Grossman School of Medicine.

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

Facilitating access or clearing guarantees?

Patients must meet specific but different criteria to qualify for any 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 available to diagnose, monitor, or treat the disease or condition.” in accordance with government regulations. Clinical trials must be unfeasible for patients, and the use of these drugs must not interfere with any ongoing trials. Also, the potential benefits must justify the risks, according to prescribers.

Then, once a treatment is identified, the patient’s doctor must get approval from its manufacturer, the FDA, and the institutional review board that oversees clinical drug trials.

The FDA said these steps are in place so the agency can fairly weigh the drug’s risks and benefits 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 has attempted to expedite this approval process. Under the new program, for example, a physician only needs to identify an experimental drug and receive authorization for use from the manufacturer. In most cases, the FDA has no authority to approve or deny the application, and there is no review board process to navigate.

But because of Right To Try program definitions, fewer patients and fewer drugs qualify.

Under Right to Try, patients must have a “life-threatening” disease or condition, not just “serious,” as with expanded access. Experimental drugs are only available after they have completed phase 1 clinical trials; treatments accessed through the expanded access program may be administered during a phase 1 study.

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 how patients tolerate drugs to “delay or adversely affect the review or approval of such drug(s)” unless senior officials justify the public health benefit in writing.

Supporters say Right To Try is an example of successful deregulation and claim its more efficient approval process has saved lives. But critics see it as a key cause for concern because it “opens up the opportunity to exploit 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 regulatory agencies were not the main hurdle patients faced when seeking experimental drugs. The FDA has almost always approved expanded and expedited access applications according to government standards.

According to a 2018 FDA report on the expanded access program, the FDA has authorized 99 percent of the approximately 9,000 requests received over the past five years, approving emergency requests for experimental drugs in less than a day, on average. More recent data shows that the approval trend has continued, even as the number of applications has increased each year.

In the rare cases where the FDA did not automatically approve applications, regulators often did not reject them but recommended changes to the requested dose to address safety and efficacy concerns.

The right to try by numbers

The FDA does not share detailed information about the number of doses provided or the patients treated under Right to Try. Instead, just post one annual summary showing how many drugs were approved under the program. The agency says that since Right To Try began 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 through Right To Try were likely provided to more than one patient each, experts said it was highly unlikely that thousands of patients were involved, as Trump said.

Trump’s claim is a “gross overestimate of the number of people using Right To Try,” Fernandez Lynch said, noting that he believes the actual numbers are “very, very low.”

Trump’s campaign did not respond to multiple questions 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 ways to expand ‘Right to Try’ to save more American lives “.

It remains unclear how Trump might expand the program, though the conservative Goldwater Institute advocates “Right to Try 2.0,” which it claims will allow patients to receive individualized therapies.

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

Meanwhile, evidence shows that the high price of experimental treatments, which are sometimes available through certain drug company programs but are not typically covered by insurance, is a bigger hurdle for patients than regulatory buffers.

“I don’t think people have a problem with the FDA blocking access to individualized therapies,” 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 do 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 been around for decades, and there’s no evidence that Right To Try has ever had the impact Trump said it had.

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

In addition, public health experts said the Right to Try weakens patient protections and fails to address the real barriers to experimental drugs.

We rate Trump’s claim false.

our sources:

21 CFR §50.20

21 USC §360bbb-0a

Congressional Research Service, “Extended access and right to trial: access to investigational medicines”, March 16, 2021

Food and Drug Administration,”Extended access,” 28 February 2024

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Los Angeles Times, obituary of Marc HayutinJune 14, 2019

Phone interview with Alison Bateman-House, assistant professor at New York University Grossman School of Medicine, September 24, 2024

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