Background Recently approved second‐generation androgen receptor inhibitors (SGARIs) for non‐metastatic castration‐resistant prostate cancer (nmCRPC) have similar efficacy but differ in safety profiles. We used a discrete choice experiment (DCE) to examine how nmCRPC patients and caregivers perceive the benefits versus risks of these new treatments. Methods An online DCE survey with 14 treatment choice questions was administered to nmCRPC patients and caregivers. Each choice question compared two hypothetical medication profiles varying in terms of 5 safety attributes (risk or severity of adverse events [AEs]: fatigue, skin rash, cognitive problems, serious fall, and serious fracture) and two efficacy attributes (duration of overall survival [OS] and time to pain progression). Random parameters logit models were used to estimate each attribute's relative importance. We also estimated the amounts of OS that respondents were willing to forego for a reduction in AEs. Results In total, 143 nmCRPC patients and 149 caregivers viewed the AEs in following order of importance (most to least): serious fracture, serious fall, cognitive problems, fatigue, and skin rash. On average, patients were willing to trade 5.8 and 4.0 months of OS to reduce the risk of serious fracture and fall, respectively, from 3% to 0%; caregivers were willing to trade 6.6 and 5.4 months of OS. Conclusions nmCRPC patients and caregivers preferred treatments with lower AE burdens and were willing to forego OS to reduce the risk and severity of AEs. Our results highlight the importance of carefully balancing risks and benefits when selecting treatments in this relatively asymptomatic population.

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Background. Prader-Willi syndrome (PWS) is a rare neurodevelopmental disorder causing quality of life impairments such as insatiable hunger (hyperphagia) and obesity. We explored caregivers’ willingness to assume treatment risk in exchange for reduced hyperphagia according to a PWS-validated observer-reported outcome measure. Methods. We partnered with PWS patient organizations to develop a discrete-choice experiment exploring caregivers’ benefit-risk tradeoffs for emerging PWS treatments. The treatment benefit was a reduction in hyperphagia (as measured by a 0-, 5-, or 10-point change on the Hyperphagia Questionnaire for Clinical Trials [HQ-CT]). Treatment risks included weight gain (none, 5%, 10%), added risk of skin rash (none, 10%, 20%), and risk of liver damage (none, 1 in 1000, 10 in 1000). Preference models were estimated using mixed logistic regression and maximum acceptable risk. We explored differences in preferences across familial caregivers of patients with and without hyperphagia. Results. Four hundred sixty-eight caregivers completed the online survey. The majority of caregivers reported that patients experienced hyperphagia (68%) and half of patients experienced obesity (52%). Caregivers of patients without hyperphagia were willing to accept greater weight gain (16.4% v. 8.1%, P = 0.004) and a higher risk of skin rash (11.7% v. 6.2% P = 0.008) as compared to caregivers of patients with hyperphagia. Caregivers of patients with hyperphagia would accept a higher risk of liver damage as compared to caregivers of patients without hyperphagia (11.9 out of 1000 v. 6.4 out of 1000, P = 0.04). Conclusions. This research demonstrates that caregivers are willing to accept risk in exchange for a five-point improvement on the HQ-CT, a smaller marginal improvement than had been previously classified as meaningful. Patient experience with hyperphagia is a modifier in how much risk caregivers will accept.

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196 Background: Second-generation androgen receptor inhibitors (SGARIs) have been recently approved for the management of nmCRPC based on documented improvements in metastasis-free survival. We used a discrete choice experiment (DCE) to examine how nmCRPC patients (pts) and caregivers perceive the benefits versus risks of SGARIs. Methods: nmCRPC pts and caregivers were invited to complete an online DCE survey of 14 treatment choice questions, each comparing 2 hypothetical medication profiles varying in terms of 5 safety attributes (frequency or severity of adverse events [AEs]: fatigue, skin rash, cognitive problems, serious falls, and serious fractures) and 2 efficacy attributes (duration of overall survival [OS] and time to pain progression). These attributes were selected based on in-depth qualitative interviews with pts, caregivers, and physicians. We used random parameters logit models to estimate preference weights and relative importance scores for each attribute. Results: 143 nmCRPC pts and 149 caregivers were included in the analysis. Both pts and caregivers viewed safety attributes in the following decreasing order of importance: fractures, falls, cognitive problems, fatigue, and rash. Compared to a reduction in rash severity from moderate to none, a similar reduction in cognitive problems severity was considered nearly as important by patients but twice as important by caregivers. On average, pts were willing to trade 5.8 and 4.0 months of OS to reduce the risks of serious fractures and falls, respectively, from 3% to 0%; the corresponding figures caregivers were willing to trade were 6.6 and 5.4 months of OS. Of note, 8.4% of pts and 14.8% of caregivers consistently chose the treatment profile with the lowest fall or fracture risk, regardless of the other attributes' values. Conclusions: nmCRPC pts and caregivers preferred treatments with lower AE burdens and were willing to trade substantial amounts of OS to avoid AEs. They viewed the reduction of fractures, falls, and cognitive problems as most important. In this relatively asymptomatic population, carefully balancing risks and benefits when selecting SGARI treatments is important to optimizing quality of survival.