In a recent Viewpoint in JAMA Oncology, a group of oncology ethicists and clinicians examine the ethical challenges surrounding “Right to Try” (RTT) access to investigational cancer therapies. The article wrestles with how physicians, patients, and health systems should weigh individual hope against collective evidence-based practice — especially as new targeted therapies emerge rapidly but lack definitive proof of safety and effectiveness.
The authors explore the ethical implications of Right to Try (RTT) access to investigational oncology agents, using a recent case of off-protocol use of an unapproved drug to illustrate the risks and dilemmas involved.
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Real-world ethical tension
The article uses a 2024 case of a woman with stage IV pancreatic cancer who accessed an experimental KRAS inhibitor through RTT after standard therapy failed. Despite her oncologist’s caution about severe toxicity and unproven efficacy, she pursued the drug and later died of liver failure without tumour regression. The authors use this case to highlight the inherent clinical and ethical tension in bypassing controlled trial oversight.
Patient autonomy versus safety
RTT is often justified on the basis of respecting patient choice in the face of terminal illness. However, the authors argue that patient autonomy should not override the responsibility to protect patients from harm, particularly when evidence of benefit is lacking and risks are significant.
Collective evidence and research integrity
The article warns that unrestricted access to unproven agents may undermine cancer research and the accrual of high-quality evidence needed to benefit future patients. In oncology, where clinical trials are the foundation for progress, circumventing regulatory safeguards can jeopardise both individual and public health goals.
This Viewpoint sits at the intersection of ethics, policy, and clinical care, and it raises questions that many clinicians face at the bedside:
The discussed case underscores that unproven treatments can carry serious, even fatal, risks — a caution especially salient in oncology, where therapies often have narrow therapeutic indices.
While respecting patient hope matters, unregulated access to experimental agents risks harming individual well-being and cancer care’s collective progress.
RTT pathways could divert patients away from structured clinical trials, slowing evidence generation for new therapies and weakening the scientific basis for future approvals and standards of care.
While affirming the importance of compassion and autonomy in clinical decision-making, the authors argue for a framework that:
- Upholds rigorous evidence standards before wide use of investigational agents
- Supports transparent physician–patient discussions about realistic benefits and risks
- Protects the integrity of clinical research platforms that ultimately generate lifesaving data
Clinicians must balance empathy for patient suffering with a commitment to evidence-based practice. As the authors note, “while respecting patient hope matters, unregulated access to experimental agents risks harming individual well-being and cancer care’s collective progress.”
Paper: Zhu X, Yan L, Zhu X, Wang Y. The Ethics of Right to Try Investigational Agents in Oncology—Balancing Hope With Evidence. JAMA Oncol. Published online January 08, 2026. doi:10.1001/jamaoncol.2025.5771 Access online here.
