Master, Zubin

e13603 Background: Over half of Americans report favoring access to experimental treatments even if they have yet to be proven either safe or effective, yet only 3-5% are enrolled in clinical trials and a fraction of a percent in expanded access. The Expanded Access (EA) program (aka “compassionate use”) and Right-to-Try (RTT) are parallel federal mechanisms that offer qualifying patients access to off-trial experimental treatments. Our study aimed to capture the views and experiences of oncologists on obtaining eligible medicine using the FDA’s EA and is the first to report findings on physician attitudes toward RTT. Methods: We conducted 21 semi-structured interviews and qualitative analysis of oncologists at a major cancer center with 3 main sites across the U.S. Participants were purposely selected to have experience with EA. Results: Oncologists’ considerations for offering EA options to patients were based on a sound scientific rationale, reasonable risk-benefit ratio, functional status of the patient, and patient motivation. Although oncologists’ experience positively correlated with level of ease in navigating EA, most remained largely unfamiliar with RTT. Only 4 oncologists had experience discussing RTT with patients and none had obtained an experimental therapeutic under this pathway. When informed about RTT, oncologists articulated 3 sets of concerns including limited safety and oversight, unclear structure and lack of requirements to collect and report data, and the potential for inflating patient expectations. Published work by others have shown that patients desire experimental options to be offered and report that physicians do not always offer experimental medicine taking into account factors, such as, reimbursement. Given that oncologists may factor in variables such as patient motivation, and potentially affordability or health literacy, when considering whether experimental medicine is appropriate to offer their patient, there may be a need to develop a conversation aid so that clinically relevant options are presented to patients equitably and patient choice is enhanced. Conclusions: Further research to better understand patient perspectives and potential discordant views between patients and oncologists is needed in order to design shared decision-making tools to inform oncologists about off-trial preapproval pathways and help both oncologists and patients navigate the thorny issues of experimental oncology.

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.

While sexual and gender minorities are at increased risk for poor health outcomes, there is limited data regarding patient-provider interactions. In this study, we explored the perspectives of LGBTQ patients and their encounters with physicians in order to improve our understanding of patient-physician experiences. Using purposive selection of self-identified LGBTQ patients, we performed fourteen in-depth semi-structured interviews on topics of sexual orientation and gender identity, as well as their perceived role in the patient-provider relationship. Coding using a modified grounded theory approach was performed to generate themes. We identified three major themes that demonstrate the complexity of LGBTQ patient experiences. The first, Lacking trust, identifies mistrust and loss of the physician-patient relationship resulting from physicians’ poor or judgmental communication, or from physicians making assumptions about gender, using incorrect pronouns, and not recognizing heterogeneity within the transgender community. A second theme, Being vulnerable, describes the challenges and fears related to comfort of patients with disclosing their sexual orientation and/or gender identity. A final theme, Navigating discrimination, outlines racial or ethnic discrimination which creates an additional burden on top of illness and stigmatized identity. Our results reveal the complex needs of individuals with multiple stigmatized identities when developing relationships with providers. By using an intersectional perspective that appreciates the plurality of patients’ identities, providers can help to improve their relationships with LGBTQ patients. Incorporating intersectional training for medical students and residents could greatly benefit both LGBTQ patients and their physicians.