Stout, Jeremiah

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.

Background: Electronic health records (EHRs) may help enable reliable, rapid data management for many uses, such as facilitating communication of advance care planning (ACP). However, issues with validity and accuracy of EHRs hinder the use of ACP information for practical applications. Design: We present a cross-sectional pilot study of 433 older adults with cancer from three large health care systems, participating in an ongoing multisite pragmatic trial (4UH3AG060626-02). We compared data extracted from dedicated structured EHR fields for ACP to a chart review of corresponding ACP documentation contained in the medical chart. Results: Structured ACP data existed for 43.2% of patients and varied by site (25.7% −48.9%). Of the identified structured ACP data elements, 59.2% of recorded elements were correct, 23.7% were incorrect, and 17.1% were duplicates with heterogeneity across sites. Conclusion: Structured ACP data in EHRs were frequently incorrect. This represents a problem for patients and their families, as well as quality improvement and research efforts. Clinical Trials Registration: NCT03609177.

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.

Purpose: As many as 20% of oncology patients receive chemotherapy in the last 14 days of their lives. This study characterized conversations between patients and cancer clinicians on chemotherapy cessation in the setting of advanced cancer. Methods: This 3-site study captured real-time, audio-recorded interviews between oncology clinicians and patients with cancer during actual clinic visits. Audio-recordings were reviewed for discussion of chemotherapy cessation and were analyzed qualitatively. Results: Among 525 recordings, 14 focused on stopping chemotherapy; 14 patients participated with 11 different clinicians. Two types of nonmutually exclusive conversation elements emerged: direct and specific elements that described an absence of effective therapeutic options and indirect elements. An example of a direct element is as follows: “…You know this is…always really tough…But I—I think that you may need more help…I think we’re close to stopping chemotherapy…And hospice is really helpful to have in place…” In contrast, the second conversation element was more convoluted: “…transplant is not an option and surgery is not an option…The options…are taking a pill…It doesn’t shrink the tumor…It may help you live a little longer. But I’m worried if [you] had the pill, it’s still a therapy and it still has side effects. I [am] worried if I give it to you now, that you’re so weak, it will make you worse.” No relationship seemed apparent between conversation elements and chemotherapy cessation. Conclusions: Conversations on chemotherapy cessation are complex; multiple factors appear to drive the decision to stop.