Oncologists’ reflections on patient rights and access to compassionate use drugs: A qualitative interview study from an academic cancer center

Jeremiah Stout, Cambray Smith, Jan Buckner, Alex A. Adjei, Mark Wentworth, Jon C. Tilburt, Zubin Master

Research output: Contribution to journalArticlepeer-review


The U.S. Food and Drug Administration (FDA) allows patients with serious illnesses to access investigational drugs for “compassionate use” outside of clinical trials through expanded access (EA) Programs. The federal Right-to-Try Act created an additional pathway for non-trial access to experimental drugs without institutional review board or FDA approval. This removal of oversight amplifies the responsibility of physicians, but little is known about the role of practicing physicians in non-trial access to investigational drugs. We undertook semi-structured interviews to capture the experiences and opinions of 21 oncologists all with previous EA experience at a major cancer center. We found five main themes. Participants with greater EA experience reported less difficulty accessing drugs through the myriad of administrative processes and drug company reluctance to provide investigational products while newcomers reported administrative hurdles. Oncologists outlined several rationales patients offered when seeking investigational drugs, including those with stronger health literacy and a good scientific rationale versus others who remained skeptical of conventional medicine. Participants reported that most patients had realistic expectations while some had unrealistic optimism. Given the diverse reasons patients sought investigational drugs, four factors—scientific rationale, risk-benefit ratio, functional status of the patient, and patient motivation—influenced oncologists’ decisions to request compassionate use drugs. Physicians struggled with a “right-to-try” framing of patient access to experimental drugs, noting instead their own responsibility to protect patients’ best interest in the uncertain and risky process of off-protocol access. This study highlights the willingness of oncologists at a major cancer center to pursue non-trial access to experimental treatments for patients while also shedding light on the factors they use when considering such treatment. Our data reveal discrepancies between physicians’ sense of patients’ expectations and their own internal sense of professional obligation to shepherd a safe process for patients at a vulnerable point in their care.

Original languageEnglish (US)
Article numbere0261478
JournalPloS one
Issue number12 December
StatePublished - Dec 2021

ASJC Scopus subject areas

  • General


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