Patients Have a Right To Try. Why Can’t They Use It?

The recent Food and Drug Administration (FDA) rejection of RP1, an experimental immunotherapy for advanced melanoma, has sparked frustration among oncologists and patients who see it as another promising therapy kept out of reach.

Last month, the agency issued a “complete response letter” declining to approve the therapy, citing insufficient evidence of effectiveness and concerns about the trial’s design.

Oncologists and researchers have pushed back, arguing that the FDA dismissed encouraging response data and applied standards that may not fit patients with few remaining options. For patients with advanced melanoma who have exhausted standard treatments, that decision is not an abstract regulatory judgment—it can mean the difference between having one more option and having none.

The case raises an obvious question: Whatever happened to “Right to Try,” the policy enacted during the first Trump administration? It was intended for precisely this situation. Yet even when patients are told they have a right to try, that right often exists more on paper than in practice.

Congress passed the federal Right to Try Act in 2018 with a straightforward idea: Terminally ill patients with limited options should be able to access investigational therapies without waiting years for full FDA approval. Lawmakers sought to remove bureaucratic barriers and give patients, their doctors, and drug developers greater flexibility.

Critics warned at the time that the law would do less than its supporters claimed. They argued that the real barriers were not just FDA oversight, but manufacturer reluctance, institutional review requirements, and liability concerns.

In that respect, they were right.

The sales pitch was sweeping. When President Donald Trump signed Right to Try in 2018, surrounded by patients, he promised a “fundamental freedom” that would give dying patients hope.

Eight years later, the record is far thinner than the rhetoric. The FDA reports only a handful of uses each year—12 drugs from 2018 to 2022, and just a few more annually since. It wasn’t a new pathway so much as a permission slip that rarely translates into access.

The gap was evident almost immediately. In 2019, STAT News reported that an ALS patient—whose name appears in the law—still couldn’t obtain treatment, as companies declined to provide it.

That wasn’t a fluke. The law doesn’t require manufacturers, physicians, or hospitals to participate. In practice, the “right” to try ends where others’ willingness begins.

The law does not require drug manufacturers to provide investigational products—and it shouldn’t. Companies often decline because of cost, liability concerns, and the risk that adverse outcomes could complicate approval. Physicians and hospitals face similar pressures, including professional risk and institutional oversight, and they, too, must be free to exercise their judgment or decline involvement. Meanwhile, because the FDA still controls final approval, companies have strong incentives to avoid anything that might jeopardize it.

The RP1 case illustrates this gap. Patients with advanced melanoma—many of whom have exhausted standard therapies—may be willing to accept uncertainty in exchange for a chance at benefit. But the current system does not primarily ask what risks patients are willing to accept. It asks what risks regulators are willing to tolerate on their behalf.

That approach may be understandable when dealing with routine conditions or widely used medications. But it is far harder to justify when applied to patients with terminal illnesses, for whom delay is not a neutral outcome but often the worst possible one.

Supporters of the status quo argue that lowering evidentiary standards would open the door to ineffective or unsafe treatments. That concern is legitimate. But it overlooks an equally important point: The current system already rations access, just in a different way. Instead of allowing patients to weigh risks and benefits with input from their physicians, it assigns that judgment to regulators, who make it on everyone’s behalf through a process that is necessarily slow, cautious, and one-size-fits-all.

More fundamentally, we should reconsider the assumption that a single federal agency must decide when patients may access experimental care.

One alternative would be to shift from centralized control to independent certification. Rather than giving the FDA the power to block access, independent organizations—public or private—could evaluate investigational therapies and provide clear, competing assessments of their safety and effectiveness.

That approach would preserve rigorous evaluation while restoring patient choice. Patients and physicians would still have access to expert guidance, but the final decision would not rest with regulators acting on behalf of everyone. It would rest with the individuals who bear the risk. We rely on independent certification in many other areas of life without giving those organizations the power to ban access altogether. Medicine should be no different.

Right to Try fails because it leaves the existing gatekeeping structure intact. Independent certification would take a different approach: inform patients rather than decide for them.

Independent certification would not guarantee access to every investigational therapy, nor should it. Patients, physicians, and manufacturers each have their own rights and responsibilities, and no system can—or should—override them. But we can do far better than a framework that defaults to delay and denial.

For patients facing life-threatening illness, uncertainty is not the enemy. Delay is. The current system asks them to wait for proof that they may never live long enough to see. A right to try that cannot be exercised is no right at all.