The “Right to Try” experimental drug program saved “thousands and thousands of lives”
Former President Donald Trump on August 30
Former President Donald Trump has boasted in recent months about the “Right to Try,” a law he signed in 2018. Its goal is to boost access for terminally ill patients to potentially life-saving medications that have not yet been approved. by the Food and Drug Administration.
“We have things to combat diseases that won’t be approved for five or six years and that people who are very sick, with terminal illnesses, should be able to use. But there was no mechanism to do it,” Trump said on August 30, speaking in Washington, DC, 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 an Aug. 17 rally 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 in that moment.
However, 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 weakens regulations meant to protect patients.
What is right to try?
The Right to Try Act by Trickett Wendler, Frank Mongiello, Jordan McLinn and Matthew Bellina, also known as the Right to Try, was passed by Congress on a bipartisan basis and became law in 2018. It sought to streamline the process for obtaining medications that potentially life-saving and that were not available. It is not yet approved by the FDA for terminally ill patients. Speed matters; Industry groups say it takes 10 to 15 years on average for a new drug to reach pharmacies.
However, a similar FDA program, expanded access, sometimes called “compassionate use,” has existed since the 1970s and became law in 1987.
And that is the root of many criticisms of the Right to Try.
“The right to try is basically ‘expanded access light,’” said Alison Bateman-House, a medical ethicist who researches access to investigational medical products at New York University Grossman School of Medicine.
Right To Try serves fewer patients than expanded access and offers them fewer treatments, Bateman-House said.
Facilitate access or remove safeguards?
Patients must meet specific, but different, criteria to qualify for any of the experimental medication 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.” , according to government regulations. . Clinical trials should be unfeasible for patients and the use of these medications should not interfere with any ongoing studies. Additionally, the potential benefits must justify the risks, according to prescribing doctors.
Then, after identifying a treatment, the patient’s doctor must receive approval from its manufacturer, the FDA, and the institutional review board that oversees clinical trials of the drug.
The FDA said these steps 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 drugs’ clinical impact on the patient and any adverse effects to inform the drug’s broader approval process.
Right To Try sought to expedite this approval process. Under the new program, for example, a doctor must simply identify an experimental drug and receive authorization 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 navigate.
But, because of the definitions of the Right To Try program, fewer patients and fewer medications qualify.
Under right to try, patients must have a “life-threatening” illness or condition, not just “serious,” as is the case under expanded access. Experimental drugs are available only after Phase 1 clinical trials have been completed; Treatments accessed 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 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 drugs,” unless top 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 saved lives. But critics see this as a key cause for concern, because “it opens up the opportunity to exploit desperate patients,” said Holly Fernández 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 obstacle patients faced when seeking experimental drugs. The FDA almost always approved expanded access requests quickly according to government standards.
According to a 2018 FDA report on the expanded access program, the FDA authorized 99% of the approximately 9,000 applications it received in the previous five years, approving emergency applications 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 rare cases where the FDA did not automatically approve requests, regulators often did not deny them but recommended adjustments to the requested dosage to address safety and efficacy concerns.
Right to try by the numbers
The FDA does not share detailed information about the number of doses administered or patients treated under Right to Try. Instead, it publishes only an annual summary showing how many drugs have been approved under the program. The agency says since Right To Try started 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 is extremely unlikely that thousands of patients were involved, as Trump claimed.
Trump’s claim represents an “egregious overestimation of the number of people using the right to try,” Fernández Lynch said, noting that he 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’s still unclear how Trump might expand the program, although the conservative Goldwater Institute advocates for “Right to Try 2.0,” which it says will allow patients to receive individualized therapies.
Experts noted that such medications 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 typically not covered by insurance, is a bigger obstacle for patients than regulatory barriers. .
“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 make them.”
Our failure
Trump has claimed throughout the campaign that his Right to Try program is innovative and has saved thousands of lives. But a similar program has been around for decades, and there is no evidence that Right To Try has had anywhere near the impact Trump said it had.
Neither the Trump campaign nor right-to-try advocates provided evidence to support claims of widespread benefit. And government data show that only 16 drugs have been approved under the program in its first six years, without accounting for how many patients used those drugs or their clinical outcomes.
Additionally, public health experts have said Right To Try weakens patient protections and fails to address real barriers to experimental drugs.
We rate Trump’s claim as false.
our sources:
21 CFR §50.20
21 USC §360bbb-0a
Congressional Research Service, “Expanded Access and the Right to Try: Access to Investigational Drugs,” March 16, 2021
Food and Drug Administration, “Expanded Access,” February 28, 2024
Food and Drug Administration, “Expanded Access Submission Data (Compassionate Use),” May 2, 2024
Food and Drug Administration, “Expanded Access Program Report,” May 2018
Food and Drug Administration, “Expanded Access to Investigational Drugs for Therapeutic Use: Questions and Answers, Guidance for Industry,” November 2022
Food and Drug Administration, “FDA Fact Sheet: Right to Try,” accessed September 29, 2024
Food and Drug Administration, “Right to Try,” January 23, 2023
Food and Drug Administration, “Right to Try Annual Report Summary,” June 6, 2024
Goldwater Institute, “Right to Try Individualized Treatments (Right to Try 2.0),” accessed September 29, 2024
Goldwater Institute, “The Right to Try Works,” accessed September 29, 2024
Goldwater Institute, “The Right to Try,” October 5, 2014
Goldwater Institute, “What is the right thing to try?” Accessed September 29, 2024.
Los Angeles Times, Marc Hayutin obituary, June 14, 2019
Telephone interview with Alison Bateman-House, assistant professor at New York University Grossman School of Medicine, September 24, 2024
Telephone interview with Holly Fernandez Lynch, associate professor of medical ethics and law at the Perelman School of Medicine at the University of Pennsylvania, September 17, 2024
PhRMA, “Research and Development Policy Framework,” accessed September 29, 2024
Roll Pass, “Speech: Donald Trump Holds Political Rally in Wilkes-Barre, Pennsylvania,” August 17, 2024
The Singju Post, “Full transcript: Trump addresses Moms for Freedom 2024 Summit,” August 31, 2024
