Two recent Wall Street Journal editorials argue that the Food and Drug Administration’s (FDA) “inexplicable” rejection of RP1, an immunotherapy for melanoma, may cost lives by blocking access to a promising therapy for patients with few remaining options. That critique captures a real frustration. But the deeper problem goes beyond any single FDA decision.
In Your Body, Your Health Care, I wrote:
“Ideally, Congress should end the FDA’s power to deny consumers their right to self-test and self-medicate through its power over the drug and medical device approval process… Private-sector organizations can perform the same function as the FDA in certifying that drugs and devices are safe and effective. The crucial difference is that private certification organizations can provide consumers with the information they need to make educated treatment decisions but cannot deny them access to medical care.”
That principle is most urgent for patients with terminal illness.
These patients are often told that the law protects their right to try investigational treatments. But “Right to Try” laws are largely symbolic. They permit access to experimental therapies, but the system is structured in ways that often prevent patients from obtaining them.
Existing pathways, such as expanded access and the federal “Right to Try” law, technically allow patients to seek experimental therapies. But access depends on whether manufacturers are willing to provide the drug and whether physicians can navigate a cumbersome process to administer it. The result is a system in which patients may be legally guaranteed the right to try but lack the practical ability to do so.
Competent adults should be free to decide whether to accept the risks of investigational therapies, even when efficacy is uncertain.
This is not prohibition in the classic sense. But for patients facing terminal illness, it can function much the same way.
The Real Barrier: A System Built for Populations, Not Patients
Consider a patient with metastatic melanoma who has exhausted all approved therapies and has limited time remaining. Obtaining an investigational drug outside a clinical trial, however, typically requires manufacturer agreement, FDA authorization, and institutional review board (IRB) approval (see FDA guidance on expanded access for physicians and how to request single-patient expanded access).
Each step has a rationale. Together, the steps create delays and uncertainty that are incompatible with the realities of terminal illness.
The deeper issue is structural. The system is designed to answer a population-level question—whether a drug is safe and effective for the market—not whether an individual patient, facing imminent death, should take the risk. It answers that question by restricting individual choice.
In that system, time is a feature, not a bug. For patients, it is the opposite.
Why Existing Solutions Fall Short
I have proposed international drug reciprocity as a way to speed access when regulators in different countries reach different conclusions. That approach can be valuable when a therapy is approved abroad but delayed at home. But it offers no help when no regulator has yet approved a therapy. In those cases, the barrier to treatment is not delay—it is uncertainty.
Right to Try has a different limitation. It removes the FDA from the process but leaves the underlying constraints intact: manufacturer willingness, clinical infrastructure, and liability concerns.
It creates the appearance of access without changing the conditions that prevent access. Right to Try did not fail because it went too far. It failed because it left the existing system intact.
Respecting Autonomy—on Both Sides
Any reform must begin with a basic principle: Autonomy applies not only to patients but also to manufacturers—and to the physicians asked to administer these therapies.
Patients should be free to accept risk in pursuit of potential benefit. But companies cannot be compelled to provide investigational drugs if doing so risks undermining clinical trials, depleting a limited supply, or jeopardizing eventual approval. Physicians likewise face malpractice and regulatory risks when using therapies outside the standard of care. A policy that ignores these constraints would simply replace one form of coercion with another. Meanwhile, the patient—who bears the ultimate risk—remains the least empowered decisionmaker in the process.
The challenge, then, is not to mandate access but to stop systematically discouraging it.
Making Access Possible, Not Theoretical
Expanded access already exists as a pathway. The problem is that it is too slow, opaque, and burdensome to function when patients do not have time.
Policymakers could take several steps to make expanded access viable without compelling participation:
- Preauthorized expanded-access protocols
Allow manufacturers to establish flexible, standing protocols that physicians can use without case-by-case negotiation, reducing delay and administrative uncertainty. - Simplified physician attestation
Replace lengthy applications with a brief certification that the patient has a serious condition, lacks alternatives, and has provided informed consent. - Centralized IRB review
Federal rules already allow a single IRB, but institutions often duplicate review. Encouraging reliance on central IRBs—or allowing limited pre-clearance with post hoc oversight—would reduce redundant review and delay. - Faster FDA response times
Formalize rapid turnaround (e.g., 24–48 hours) to reduce uncertainty when authorization is required. - Clearer liability protections
Current protections are incomplete and ambiguous. Clarifying them would reduce a major deterrent for both physicians and manufacturers while preserving accountability for fraud or gross negligence. - Targeted reporting requirements
Limit reporting to serious and unexpected adverse events and basic outcomes rather than layering on requirements that begin to resemble those of a clinical trial.
These changes would not guarantee access. They would simply ensure that when patients, physicians, and manufacturers are willing to proceed, the system does not stand in their way.
From Legal Rights to Real Choices
For patients with terminal illness, time is the scarcest resource. A system that recognizes their autonomy in principle but withholds it in practice is not neutral—it is making the decision for them.
We do not need to guarantee access to every investigational therapy. But we should stop pretending that a theoretical right is meaningful when the system is built to prevent its use. For patients who are out of time, that distinction is not theoretical.
Ideally, policy should respect the autonomy of patients, manufacturers, and physicians alike. In practice, that means there will be cases where not all parties are satisfied. The goal is not to eliminate those tensions but to ensure that the system does not structurally prevent willing participants from acting.
The right to try is not the same as the ability to try. Until policy reflects that distinction, many patients will continue to have one without the other.