{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,1,9]],"date-time":"2026-01-09T02:25:27Z","timestamp":1767925527593,"version":"3.49.0"},"reference-count":46,"publisher":"Public Library of Science (PLoS)","issue":"12","license":[{"start":{"date-parts":[[2021,12,17]],"date-time":"2021-12-17T00:00:00Z","timestamp":1639699200000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by\/4.0\/"}],"funder":[{"DOI":"10.13039\/100006108","name":"National Center for Advancing Translational Sciences","doi-asserted-by":"publisher","award":["UL1 TR002377"],"award-info":[{"award-number":["UL1 TR002377"]}],"id":[{"id":"10.13039\/100006108","id-type":"DOI","asserted-by":"publisher"}]},{"name":"Center of Excellence in Regulatory Science and Innovation Scholars Award to Yale and Mayo Clinic","award":["U01FD005938"],"award-info":[{"award-number":["U01FD005938"]}]}],"content-domain":{"domain":["www.plosone.org"],"crossmark-restriction":false},"short-container-title":["PLoS ONE"],"abstract":"<jats:p>The U.S. Food and Drug Administration (FDA) allows patients with serious illnesses to access investigational drugs for \u201ccompassionate use\u201d 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\u2014scientific rationale, risk-benefit ratio, functional status of the patient, and patient motivation\u2014influenced oncologists\u2019 decisions to request compassionate use drugs. Physicians struggled with a \u201cright-to-try\u201d framing of patient access to experimental drugs, noting instead their own responsibility to protect patients\u2019 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\u2019 sense of patients\u2019 expectations and their own internal sense of professional obligation to shepherd a safe process for patients at a vulnerable point in their care.<\/jats:p>","DOI":"10.1371\/journal.pone.0261478","type":"journal-article","created":{"date-parts":[[2021,12,17]],"date-time":"2021-12-17T18:47:56Z","timestamp":1639766876000},"page":"e0261478","update-policy":"https:\/\/doi.org\/10.1371\/journal.pone.corrections_policy","source":"Crossref","is-referenced-by-count":8,"title":["Oncologists\u2019 reflections on patient rights and access to compassionate use drugs: A qualitative interview study from an academic cancer center"],"prefix":"10.1371","volume":"16","author":[{"given":"Jeremiah","family":"Stout","sequence":"first","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-9723-891X","authenticated-orcid":true,"given":"Cambray","family":"Smith","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Jan","family":"Buckner","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Alex A.","family":"Adjei","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Mark","family":"Wentworth","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Jon C.","family":"Tilburt","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-3462-4546","authenticated-orcid":true,"given":"Zubin","family":"Master","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]}],"member":"340","published-online":{"date-parts":[[2021,12,17]]},"reference":[{"key":"pone.0261478.ref001","author":"FDA","year":"2018","journal-title":"Expanded Access Program Report"},{"issue":"6","key":"pone.0261478.ref002","doi-asserted-by":"crossref","first-page":"705","DOI":"10.1177\/2168479016656030","article-title":"Expanded Access of Investigational Drugs: The Experience of the Center of Drug Evaluation and Research Over a 10-Year Period.","volume":"50","author":"JP Jarow","year":"2016","journal-title":"Therapeutic Innovation and Regulatory Science"},{"key":"pone.0261478.ref003","unstructured":"FDA. Project Facilitate: Assisting Healthcare Providers with Expanded Access Requests for Investigational Oncology Products. https:\/\/www.fda.gov\/about-fda\/oncology-center-excellence\/project-facilitate. Published 2020. Updated 10\/16\/2020. Accessed 1\/2021."},{"key":"pone.0261478.ref004","unstructured":"Byrne J. Right to Try: A \u2019well-intentioned\u2019 but \u2019misguided\u2019 law. Healio. https:\/\/www.healio.com\/news\/hematology-oncology\/20200303\/right-to-try-a-wellintentioned-but-misguided-law. Published 2020. Updated March 10, 2020. Accessed 2021."},{"key":"pone.0261478.ref005","unstructured":"Goldwater Institute. Dead On Arrival: Federal \u201cCompassionate Use\u201d Leaves Little Hope for Dying Patients. http:\/\/righttotry.org\/dead-on-arrival\/. Published 2016. Accessed 2021."},{"issue":"8","key":"pone.0261478.ref006","doi-asserted-by":"crossref","first-page":"758","DOI":"10.1001\/jama.2015.7691","article-title":"Questions of Safety and Fairness Raised as Right-to-Try Movement Gains Steam","volume":"314","author":"JA Jacob","year":"2015","journal-title":"JAMA"},{"issue":"7618","key":"pone.0261478.ref007","doi-asserted-by":"crossref","first-page":"478","DOI":"10.1136\/bmj.39244.451192.AD","article-title":"Should terminally ill patients have the right to take drugs that pass phase I testing? 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Chahal","year":"2010","journal-title":"Journal of Medical Ethics"},{"issue":"9","key":"pone.0261478.ref011","doi-asserted-by":"crossref","first-page":"841","DOI":"10.1111\/imj.12530","article-title":"Dilemmas in the compassionate supply of investigational cancer drugs","volume":"44","author":"JR Lewis","year":"2014","journal-title":"Internal Medicine Journal"},{"issue":"1","key":"pone.0261478.ref012","doi-asserted-by":"crossref","first-page":"15","DOI":"10.1016\/S1470-2045(14)71161-1","article-title":"Expanded access programmes: patient interests versus clinical trial integrity","volume":"16","author":"P Lorigan","year":"2015","journal-title":"The Lancet Oncology"},{"issue":"9","key":"pone.0261478.ref013","doi-asserted-by":"crossref","first-page":"1275","DOI":"10.1517\/14656566.2015.1046837","article-title":"Should patients in need be given access to experimental drugs?","volume":"16","author":"AL Caplan","year":"2015","journal-title":"Expert Opinion on Pharmacotherapy"},{"issue":"10","key":"pone.0261478.ref014","doi-asserted-by":"crossref","first-page":"979","DOI":"10.1001\/jama.2016.0416","article-title":"The Ethical Challenges of Compassionate Use","volume":"315","author":"AL Caplan","year":"2016","journal-title":"JAMA"},{"issue":"3","key":"pone.0261478.ref015","doi-asserted-by":"crossref","first-page":"279","DOI":"10.1056\/NEJMhle1409465","article-title":"Practical, Legal, and Ethical Issues in Expanded Access to Investigational Drugs.","volume":"372","author":"JJ Darrow","year":"2015","journal-title":"New England Journal of Medicine"},{"key":"pone.0261478.ref016","article-title":"Make pre-approval Covid-19 vaccines available through expanded access, not an EUA.","author":"MW McCarthy","year":"2020"},{"key":"pone.0261478.ref017","article-title":"Pfizer and Moderna\u2019s \"95% effective\" vaccines\u2014let\u2019s be cautious and first see the full data.","author":"P Doshi","year":"2020","journal-title":"BMJ"},{"key":"pone.0261478.ref018","article-title":"FDA, under pressure from Trump, authorizes blood plasma as Covid-19 treatment","author":"N. 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