Cambray Smith1, Jeremiah Stout2, Alex A Adjei3, Jan Buckner3, Mark Wentworth4, Jon Tilburt5, Zubin Master6. 1. Biomedical Ethics Research Program, Mayo Clinic, Rochester, MN, USA and School of Medicine, University of North Carolina - Chapel Hill, Chapel Hill, NC, USA. 2. Biomedical Ethics Research Program and Alix School of Medicine, Mayo Clinic, Rochester, MN, USA. 3. Department of Oncology, Mayo Clinic, Rochester, MN, USA. 4. Office of Research Regulatory Support, Mayo Clinic, Rochester, MN, USA. 5. Biomedical Ethics Research Program and General Internal Medicine, Mayo Clinic, Rochester, MN, USA. 6. Biomedical Ethics Research Program and Center for Regenerative Medicine, Mayo Clinic, Rochester, MN, USA.
Abstract
BACKGROUND: The federal Right-to-Try (RTT) Act created an alternate regulatory pathway for preapproval access to investigational drugs. A few studies have examined the experiences of physicians with the Food and Drug Administration's Expanded Access Programs, but to our knowledge, no study has yet to examine their attitudes and experiences toward RTT. METHODS: This study explored the views of 21 oncologists at a major cancer center with 3 main sites across the United States using semi-structured interviews and qualitative analysis. Participants were selected to have experience with Expanded Access Programs. RESULTS: Most oncologists had limited familiarity with RTT, and several reported confusion about the legislation, including whether patients have a right to investigational drugs and an obligation for companies to provide them. Although oncologists were interested in decreased regulatory burdens, 3 areas of concern were articulated: lack of safety and oversight, unclear structure and no provision for data collection, and potential heightening of patient expectations. Only 4 oncologists had experience discussing RTT, and none formally attempted to obtain the drug through this mechanism. Participants questioned the practicality of RTT legislation and suggested alternative ways to improve access. CONCLUSIONS: The study provides foundational empirical data underlying challenging ambiguities by experienced oncologists familiar with off-trial use of investigational therapeutics and reaffirms the role of physicians and regulatory bodies in mitigating the risks of investigational drugs. Our findings highlight the need for medical centers to inform oncologists about RTT and other preapproval pathways so that they are able to address questions from patients interested in nontrial investigational drugs.
BACKGROUND: The federal Right-to-Try (RTT) Act created an alternate regulatory pathway for preapproval access to investigational drugs. A few studies have examined the experiences of physicians with the Food and Drug Administration's Expanded Access Programs, but to our knowledge, no study has yet to examine their attitudes and experiences toward RTT. METHODS: This study explored the views of 21 oncologists at a major cancer center with 3 main sites across the United States using semi-structured interviews and qualitative analysis. Participants were selected to have experience with Expanded Access Programs. RESULTS: Most oncologists had limited familiarity with RTT, and several reported confusion about the legislation, including whether patients have a right to investigational drugs and an obligation for companies to provide them. Although oncologists were interested in decreased regulatory burdens, 3 areas of concern were articulated: lack of safety and oversight, unclear structure and no provision for data collection, and potential heightening of patient expectations. Only 4 oncologists had experience discussing RTT, and none formally attempted to obtain the drug through this mechanism. Participants questioned the practicality of RTT legislation and suggested alternative ways to improve access. CONCLUSIONS: The study provides foundational empirical data underlying challenging ambiguities by experienced oncologists familiar with off-trial use of investigational therapeutics and reaffirms the role of physicians and regulatory bodies in mitigating the risks of investigational drugs. Our findings highlight the need for medical centers to inform oncologists about RTT and other preapproval pathways so that they are able to address questions from patients interested in nontrial investigational drugs.
Authors: Scott Moerdler; Lindy Zhang; Elena Gerasimov; Chong Zhu; Tamar Wolinsky; Michael Roth; Nancy Goodman; Daniel A Weiser Journal: Pediatr Blood Cancer Date: 2018-11-08 Impact factor: 3.167
Authors: M E Blair Holbein; Jelena P Berglund; Kevin Weatherwax; David E Gerber; Joan E Adamo Journal: Clin Transl Sci Date: 2015-01-15 Impact factor: 4.689
Authors: Jeremiah Stout; Cambray Smith; Jan Buckner; Alex A Adjei; Mark Wentworth; Jon C Tilburt; Zubin Master Journal: PLoS One Date: 2021-12-17 Impact factor: 3.240