Research Participants' High Expectations of Benefit in Early-Phase Oncology Trials: Are We Asking the Right Question?

Abstract

A B S T R A C T PurposeTo determine whether patients' expectations of benefit in early-phase oncology trials depend on how patients are queried and to explore whether expectations are associated with patient characteristics. Patients and MethodsParticipants were 171 patients in phase I or II oncology trials in the United States. After providing informed consent for a trial but before receiving the investigational therapy, participants answered questions about expectations of benefit. We randomly assigned participants to one of three groups corresponding to three queries about expectations: frequency type, belief type, or both. Main outcomes were differences in expectations by question type and the extent to which expectations were associated with demographic characteristics, numeracy, dispositional optimism, religiousness/spirituality, understanding of research, and other measures. ResultsThe belief-type group had a higher mean expectation of benefit (64.4 of 100) than the combination group (51.6; P ϭ .01) and the frequency-type group (43.1; P Ͻ .001). Mean expectations in the combination and frequency groups were not significantly different (P ϭ .06). Belief-type expectations were associated with a preference for nonquantitative information (r ϭ Ϫ0.19; 95% CI, Ϫ0.19 to Ϫ0.36), knowledge about research (r ϭ Ϫ0.21; 95% CI, Ϫ0.38 to Ϫ0.03), dispositional optimism (r ϭ 0.20; 95% CI, 0.01 to 0.37), and spirituality (r ϭ 0.22; 95% CI, 0.03 to 0.38). Frequency-type expectations were associated with knowledge about clinical research (r ϭ Ϫ0.27; 95% CI, Ϫ0.27 to Ϫ0.51). ConclusionIn early-phase oncology trials, patients' reported expectations of benefit differed according to how patients were queried and were associated with patient characteristics. These findings have implications for how informed consent is obtained and assessed.

A study of motivations and expectations of patients seen in phase 1 oncology clinics

“…Patients have demonstrated unrealistically high expectations of personal benefit or even a cure from these investigational agents 15, 16, 17. Much of the research has attributed these idealistic expectations to suboptimal patient‐physician communication and a patient‐based culture of needing to remain optimistic as treatment options diminish 11, 12, 14, 15, 18…”

Patients' understanding of how genotype variation affects benefits of tamoxifen therapy for breast cancer.

“…Recent research has questioned whether high expectations of direct benefit, which patients consistently report in oncology trials, are due to a "therapeutic misestimation" among participants (suggesting that patients misunderstand the purpose of the trial, undermining the validity of the informed consent process) or are expressions of optimism for the best possible outcome [17][18][19]. Our study was not designed to test this complex question, but highlights the potential for patients to misunderstand what new genomic tests can offer [20,21].…”

Attitudes toward participating in Phase I clinical trials: an investigation with patient–family–physician triads

“…Based on the literature regarding the efficacy and side effects of previous Phase I chemotherapeutic agents (2,17), we detailed the expected tumor responses along with probabilities, expected clinical benefits and potential serious adverse effects. We used frequency-type probability statements because they are the most appropriate for conveying objective knowledge of the trial in a way that participants could comprehend (6,18). To prevent negative connotations of the term 'no therapy' and to protect from suggestions of coercion, enrollment in a palliative care program for pain and symptom management was offered provided as an alternative to participation in the trial (19)(20)(21).…”

“…There is concern that such 'unrealistic' optimism could compromise the validity of informed consent (4), suggesting that patients with fair understanding of benefit-risk ratios would not participate in Phase I trials (5). Such high expectation of benefits in Phase I trials would be an expression of hope rather than a misunderstanding of benefit-risk ratio (1,6,7). Yet, it is not clear how much a misunderstanding or expression of hope contributes to patients' choices.…”