Several years ago, Anita Turk, MD, assistant professor of clinical medicine at the Indiana University School of Medicine in Indianapolis, was treating a patient with advanced gastrointestinal cancer. The woman was in a dire situation: She faced a life-threatening diagnosis and had no FDA-approved treatment options or clinical trial opportunities.

But there was one possibility, an investigational drug that had completed phase 1 testing that Turk could potentially access through a 2018 federal law called the Right to Try Act.

Right to Try provides patients with life-threatening conditions a pathway to receive investigational treatments after they’ve exhausted FDA-approved and clinical trial avenues. Under the pathway, the FDA and institutional review board (IRB) review are not required before treatment.

When Right to Try became law, proponents promised speed and autonomy to help patients access investigational therapies when other, more mainstream options had run out. But after nearly 8 years, available data suggest limited uptake of the pathway, raising questions about whether it has meaningfully helped patients with life-threatening diagnoses or largely created a symbolic alternative to the more widely used expanded access pathway, which includes more robust safety guardrails.

Right to Try “has been mostly a nothing burger,” said Christopher Robertson, JD, PhD, a professor of health law, policy, and management, Boston University School of Law in Boston.

“It’s been a rhetorical and political win. But on the ground, it’s made very little difference,” explained Robertson, a member of the Working Group on Compassionate Use and Preapproval Access at NYU Langone Health.

One of the biggest worries is patient safety. Critics have expressed concerns about the ethics of forgoing FDA and IRB oversight and potentially giving patients false hope that drugs at such an early stage of development will have any meaningful benefit.

But Turk’s case was unusual.

On the safety front, “I just knew the data well. And I felt comfortable giving the medicine,” said Turk. The expanded access pathway for the investigational drug, which Turk declined to name for privacy reasons, was not open to new patients.

Importantly, the patient understood the realities and risks of going this route. She knew the treatment was not a cure — at most, it would buy her some time, Turk said.

The patient had also cleared another big hurdle of Right to Try: cost. The manufacturer provided the drug for free, and the patient’s insurance agreed to cover its administration, which Turk described as highly unusual.

It “is not my go-to practice to use Right to Try,” stressed Turk. But in this case, it was “probably our only option.”

A Murky Uptake Picture

Although the Right to Try and expanded access pathways overlap in many ways, expanded access is much more popular.

A 2021 study in the Journal of the National Cancer Institute revealed that most oncologists have limited familiarity with Right to Try, but after learning more, they preferred the expanded access route, given its requisite review by the FDA and an IRB.

Outside of greater safety oversight, the expanded access program includes broader options because treatments can still be in a phase 1 trial, while drugs must have completed phase 1 testing to use Right to Try.

In recent years, FDA officials have allowed more than 99% of expanded access requests for individual patients to proceed. In fiscal year 2023, 1318 of the 1326 nonemergency requests were given the go-ahead, as were all 634 emergency requests, according to the latest online data from FDA’s Center for Drug Evaluation and Research.

Emergency requests are usually granted immediately by phone, and nonemergency requests are processed in a median of 4 days, according to congressional testimony by FDA official Peter Lurie, MD, MPH, in 2016.

During that testimony, Lurie explained that even though nearly all expanded access applications are approved, FDA officials made safety-related changes, such as monitoring guidance or modifying dosing, in 11% of applications.

Gauging how often physicians have used Right to Try is more challenging.

Although Right to Try guidance stipulates that manufacturers report annually to the FDA about the drugs provided along with details, such as the number of doses, the number of patients treated, what the drug was used for, and any serious adverse events, publicly available information on its use remains limited.

A brief online summary reveals that a total of 21 drugs or biological products have been provided between 2018 and 2024. The summary does not specify drug names, number of patients involved, type of condition, or adverse events.

But the number of drugs listed may not reflect the total pool of patients who have benefitted from Right to Try, said Naomi Lopez, a senior fellow at the Goldwater Institute, which advocated for the legislation.

A small, nonrandomized cohort study, for instance, suggested a potential benefit of this pathway for patients with an aggressive form of brain cancer. The analysis reported that survival outcomes for 21 patients with recurrent glioblastoma, most of whom had received an investigational drug under Right to Try, exceeded historic average survival outcomes for this patient population.

Still, given safety and ethical uncertainties, Right to Try can be a hard sell for clinicians and drug manufacturers.

“Right to Try doesn’t equal right to receive,” said Julie R. Gralow, MD, chief medical officer and executive vice president of the American Society of Clinical Oncology.

Companies may choose not to provide the drug for various reasons, including preferring to retain a limited supply for early-phase clinical trials, she said.

And if a death occurs in those early phases, it “could look really bad to investors,” Gralow noted.

Pharmaceutical companies prefer the backstop of an IRB to cover ethical and safety concerns and “to make sure that patients are giving robust and informed consent,” Robertson added.

The False Hope Problem

Desperate patients may lobby for desperate measures, not aware of the potential risks that drugs in early-phase trials may pose, Turk said.

“Patients will sign up for ‘anything,’” she said. “But they don’t really know what that means. It could be life-threatening. It could destroy their quality of life, even if they survive the horrible event.”

Meanwhile, oncologists must commit substantial time and resources to navigate these pathways — often uncompensated hours that can eat into a physician’s already busy schedule, Robertson said. An oncologist interviewed in the 2021 Journal of the National Cancer Institute study reported that an informed consent conversation consumed nearly 2 hours. The patient wanted to access an investigational drug through Right to Try rather than risk random assignment to a clinical trial’s control arm, but the oncologist had to explain that the patient didn’t qualify for Right to Try because they had a clinical trial option.

Along with the time investment of Right to Try, physicians may worry about potentially misleading their patients, Robertson said. “Am I giving my patient false hope? Why am I doing that? And maybe I should have an IRB and the FDA doublecheck everything before we go too far down this road.”

The Right to Try law does include protections from liability for the physician, manufacturer, and others involved in administrating an investigational drug, unless they engage in gross negligence or other serious malfeasance. But in practical terms, physicians may have more inherent liability protection under the expanded access pathway, Robertson said.

“Expanded access helps reduce the chance of an actual bad outcome,” he said, “and lets you point to FDA and IRB as backing you up.” Even so, Robertson noted that he and other colleagues have not found any instances of physician lawsuits under either pathway.

As for safety concerns, there are also safeguards with Right to Try, explained Lopez, from the Goldwater Institute. For instance, physicians and manufacturers don’t have to provide the requested drug, she noted.

And even though it’s not required with Right to Try, “my understanding is that a lot of these cases are still using IRBs,” Lopez said. “I don’t think this law has done anything to undermine patient safety.”

There’s also a broader argument about the right to patient autonomy, particularly when facing a devastating and potentially terminal diagnosis, Lopez said. “It’s the question of, ‘Should the federal government have a veto stamp over a treatment that is safe enough to use in clinical trials, when your physician thinks that it might help you, and when the manufacturer is able and willing to provide it, and when the patient wants that treatment.’”

Balancing Expectations

Despite the anemic uptake of Right to Try, related legislation continues to pass. As of last summer, more than 40 states had Right to Try laws on the books. Leaders at the Goldwater Institute are lobbying for another federal law, dubbed Right to Try 2.0, which they say would improve access to highly individualized treatments, such as a cancer vaccine based on a patient’s genetic mutations.

But such an update would not address oncologists’ biggest concern: Safety.

For Right to Try to become more relevant, there would need to be more safety oversight, including FDA involvement, said Zubin Master, PhD, associate professor at Wake Forest University School of Medicine in Winston-Salem, North Carolina, and a bioethicist who has researched oncologists’ understanding of the law. “But then you are just making the Right to Try law look like expanded access,” said Master.

Gralow couldn’t recall a time prior to leaving clinical practice in 2021 that a patient with a life-threatening diagnosis suggested Right to Try to obtain a drug therapy. Turk’s experience has been quite different. Patients regularly ask her about the law after their standard treatment options have failed and they’ve become too ill to qualify for a clinical trial.

“It’s not a fun conversation because they feel like I am withholding some magic wand that I know is not there,” Turk said.

While Turk has not used Right to Try again, in her sole experience with the law, “it was worth the work it took to have it happen,” she said. Her patient did respond to the experimental drug and is still alive today.

Still, Turk remains ambivalent about whether she’d use it again. “I’m personally not a fan of the Right to Try pathway,” she said. “It doesn’t advance the science.”

Experts interviewed for this piece had no relevant disclosures.