Has the 'Right to Try' Act Saved Thousands of Lives? What the Data Shows

The Legislative Background: Bypassing the FDA

The "Trickett Wendler, Frank Mongiello, Jordan McLinn, and Matthew Bellina Right to Try Act" was signed into law on May 30, 2018 [1]. The legislation was designed to establish a new pathway for patients diagnosed with life-threatening diseases or conditions who have exhausted all approved treatment options, are unable to participate in clinical trials, and seek access to experimental drugs [6].

To qualify under the law, an investigational drug must have completed a Phase I clinical trial—which establishes basic safety in humans—and be under active development or review by the FDA [6]. Crucially, the Right to Try Act allows patients and physicians to request these drugs directly from the manufacturer, bypassing the FDA's traditional role of reviewing and authorizing such individual requests, as well as the requirement for approval by an Institutional Review Board (IRB) [2][6].

What the Data Shows: A Tale of Two Pathways

Proponents of the legislation, including President Trump, have frequently praised its impact, stating that the law has saved "thousands and thousands" or even "hundreds of thousands" of lives [1][2]. However, the official data compiled by the FDA under mandatory reporting rules tells a different story.

Under a final rule implemented in late 2022, drug manufacturers and sponsors who supply investigational drugs through the Right to Try pathway must submit an annual summary to the FDA detailing the number of doses supplied, the number of patients treated, and any serious adverse events [3]. The FDA's consolidated Right to Try Annual Reporting Summaries indicate that the pathway is rarely used:

• 2018–2022: 12 total drugs were reported as accessed under the pathway [3].
• 2023: 4 total drugs were reported [3].
• 2024: 5 total drugs were reported [3].

While the FDA's public summaries disclose the number of drugs used rather than the total count of individual patients, policy researchers and fact-checkers note that the number of patients per drug is typically very low, often estimated to be in the single or low double digits per drug [8]. Even under generous assumptions, the total number of patients treated under the federal Right to Try Act since 2018 is likely in the dozens or low hundreds, far below the public claims of thousands of lives saved.

Instead of the Right to Try pathway, the vast majority of patients seeking compassionate use of unapproved drugs continue to utilize the FDA’s long-standing Expanded Access program [8]. Established in the 1980s, the Expanded Access program has a well-documented history of high volume and efficiency. In Fiscal Year 2023 alone, the FDA's Center for Drug Evaluation and Research (CDER) and Center for Biologics Evaluation and Research (CBER) received 1,518 individual patient requests, allowing 1,491 to proceed—an authorization rate of 98.2% [5]. Additionally, the Center for Devices and Radiological Health (CDRH) approved 722 requests for experimental medical devices [5]. Historically, the FDA approves more than 99% of all evaluable expanded access requests, often within days, or hours in emergency cases [4].

Why Right to Try Usage Remains Low

Health policy experts and bioethicists point to several structural reasons why the Right to Try pathway is rarely utilized. The law removed the requirement for FDA permission, but it did not address the primary practical and economic hurdles that limit access to experimental medicines:

1. No Mandate for Manufacturers: The law does not require pharmaceutical companies to provide experimental drugs [6]. A company is free to deny a patient's request, and they frequently do [8].
2. Supply Constraints: Investigational drugs are typically manufactured in very small batches for clinical trials. Companies often lack the physical supply to distribute drugs outside of active studies.
3. Risk to Clinical Trials: Drug developers invest hundreds of millions of dollars in clinical trials. They are often highly risk-averse, fearing that a severe adverse event or death in a terminally ill patient using the drug under Right to Try could negatively influence FDA regulators during the final approval process, even though the law provides some liability protections [9].
4. Financial Barriers: Insurance companies are not mandated to cover the costs of experimental treatments or the medical care required to administer them. Under the law, manufacturers are permitted to charge patients for the direct cost of the drug, which can run into tens or hundreds of thousands of dollars, making the pathway financially inaccessible for most patients.
5. Physician Reluctance: Without FDA guidance on dosing, safety protocols, and drug interactions, many oncologists and specialists are hesitant to prescribe unapproved, experimental compounds to vulnerable patients [10].

The Nuance: A Legal Safety Valve and Individual Lifelines

Despite its low overall utilization, the Right to Try Act has had a significant impact on specific families and served as an important legal statement. The law's advocates argue that measuring its success solely by patient numbers misses a broader philosophical point: the restoration of medical autonomy. The Goldwater Institute, a conservative think tank that championed the legislation, argues that terminally ill patients have a moral right to seek experimental treatments without needing permission from a federal regulatory agency [2][7].

Furthermore, the law has provided real, documented lifelines for individual patients. A prominent example is Matt Bellina, a former U.S. Navy pilot diagnosed with ALS (amyotrophic lateral sclerosis) in 2014 [7]. Bellina was a leading advocate for the Right to Try movement, and the federal law is named in part after him [1]. In late 2018, Bellina successfully utilized the newly enacted Right to Try law to gain access to Nurown, an investigational stem cell therapy developed by BrainStorm Cell Therapeutics [7]. Bellina and his family have credited the treatment with slowing the progression of his disease and extending his life [7].

For patients like Bellina, the Right to Try Act provided a crucial alternative when they were ineligible for clinical trials and could not navigate the administrative requirements of the traditional FDA Expanded Access program. In this sense, the law serves as a vital "safety valve" in the healthcare system, offering a parallel path of last resort for patients who have run out of options.

Conclusion

The debate over the Right to Try Act highlights the ongoing tension in health policy between regulatory safety and patient autonomy. The claim that the law has "saved thousands of lives" is not supported by the data; the FDA's annual reports show that only 21 drugs have been accessed through this pathway over six years. The vast majority of compassionate drug access continues to flow through the FDA's Expanded Access program, which remains highly efficient and widely approved.

Nonetheless, the Right to Try Act has established a permanent legal alternative that bypasses federal regulation, empowering patients and physicians in specific, critical circumstances. For the select individuals who have successfully used it, the law has been a success, demonstrating that even a rarely used pathway can hold profound value for families facing terminal illness.