Right to Try Law Fails Patients Despite FDA Rejection of RP1 Melanoma Therapy

The FDA’s recent rejection of RP1, an experimental immunotherapy for advanced melanoma, has reignited frustration among oncologists and patients who view it as another promising therapy denied to those in desperate need. Last month, the agency issued a “complete response letter”, declining to approve the treatment due to concerns about its effectiveness and the design of its clinical trial.

Oncologists and researchers have pushed back, arguing that the FDA overlooked encouraging response data and applied standards that may not align with the realities faced by patients with few remaining treatment options. For those with advanced melanoma who have exhausted standard therapies, the FDA’s decision is far from an abstract regulatory ruling—it can determine whether they have one last chance or no options at all.

This case raises a critical question: What happened to the Right to Try law, enacted during the first Trump administration? Designed for precisely this scenario, the policy was meant to empower terminally ill patients to access investigational treatments without enduring years of FDA approval delays. Lawmakers aimed to dismantle bureaucratic hurdles, granting patients, doctors, and drug developers greater flexibility.

Critics at the time warned that the law would deliver less than promised. They cautioned that the real obstacles were not solely FDA oversight but also manufacturer reluctance, institutional review requirements, and liability concerns. History has proven them correct.

The Right to Try Act: A Promise Unfulfilled

When President Donald Trump signed the Right to Try Act in 2018, surrounded by patients, he framed it as a “fundamental freedom” offering hope to the dying. Eight years later, the reality falls far short of the rhetoric. According to FDA reports, only a handful of drugs have been accessed under the law—12 drugs from 2018 to 2022, with just a few more each year since. Rather than creating a new pathway, the law has largely served as a permission slip that rarely translates into actual access.

The limitations of the law became evident almost immediately. In 2019, STAT News reported that an ALS patient—whose name was invoked during the signing of the law—was still unable to obtain treatment because companies refused to provide it. This was not an isolated incident. The law does not mandate that manufacturers, physicians, or hospitals participate, meaning the “right” to try ends where willingness begins.

Why the Law Fails in Practice

The Right to Try Act does not require drug manufacturers to supply investigational products—and it cannot, given the ethical and financial constraints they face. Companies often decline due to:

• High costs of providing unapproved treatments
• Liability concerns over potential adverse outcomes
• Risks that complications could jeopardize future FDA approval

Physicians and hospitals face similar pressures, including professional risks and institutional oversight, which may deter their participation. Meanwhile, because the FDA retains final approval authority, companies have strong incentives to avoid anything that could complicate their path to market.

The case of RP1 underscores this gap. Patients with advanced melanoma—many of whom have exhausted all standard therapies—may be willing to accept uncertainty in exchange for a potential benefit. Yet the current system does not prioritize patient risk tolerance or urgency. Instead, it remains mired in regulatory caution, leaving those in greatest need without recourse.

The Right to Try Act was intended to bridge this divide, but its implementation has fallen short. Without mandatory participation from manufacturers or institutions, the law’s promise remains largely unfulfilled for the patients it was designed to help.