Background The current standard of care for multiple myeloma requires several regimens of treatment, with patients experiencing high symptom burden and side effects, which negatively impact health-related quality of life (HRQoL). Thus, it is crucial to understand patient perceptions of multiple myeloma and how patients value different treatment options. Objective The purpose of this study was to conduct an exploratory investigation into concepts that could form attributes that influence treatment choices for patients with multiple myeloma and to identify trade-offs that patients are willing to make between treatment attributes. Methods In total, 30 patients with newly diagnosed or relapsed/refractory multiple myeloma from the UK, France, and Germany participated in semistructured interviews talking about their disease experience and symptoms, treatment benefits, treatment burden, perceived side effects, and benefit/risk trade-offs in treatment. The interview audio recordings were transcribed and analyzed using content analysis to identify treatment and disease aspects relevant to patients. Results Symptoms of fatigue and bone pain and treatment side effects of peripheral neuropathy, diarrhea, and constipation were cited by patients as the most disruptive to their HRQoL. Treatment duration was reported most frequently as a major treatment burden, and patients emphasized the importance of increased life expectancy as a treatment benefit. All patients showed good understanding of benefit/risk trade-offs in treatment, and some patients expressed a preference for more convenient modes of treatment administration. Conclusions Qualitative interviews identified key aspects of multiple myeloma treatment that are most important to patients. These findings will inform a wider patient-preferences study, which could improve treatment choice and HRQoL for patients with multiple myeloma.

Background As the number and type of cancer treatments available rises and patients live with the consequences of their disease and treatments for longer, understanding preferences for cancer care can help inform decisions about optimal treatment development, access, and care provision. Discrete choice experiments (DCEs) are commonly used as a tool to elicit stakeholder preferences; however, their implementation in oncology may be challenging if burdensome trade-offs (e.g. length of life versus quality of life) are involved and/or target populations are small. Objectives The aim of this review was to characterise DCEs relating to cancer treatments that were conducted between 1990 and March 2020. Data Sources EMBASE, MEDLINE, and the Cochrane Database of Systematic Reviews were searched for relevant studies. Study Eligibility Criteria Studies were included if they implemented a DCE and reported outcomes of interest (i.e. quantitative outputs on participants' preferences for cancer treatments), but were excluded if they were not focused on pharmacological, radiological or surgical treatments (e.g. cancer screening or counselling services), were non-English, or were a secondary analysis of an included study. Analysis Methods Analysis followed a narrative synthesis, and quantitative data were summarised using descriptive statistics, including rankings of attribute importance. Result Seventy-nine studies were included in the review. The number of published DCEs relating to oncology grew over the review period. Studies were conducted in a range of indications (n = 19), most commonly breast (n =10, 13%) and prostate (n = 9, 11%) cancer, and most studies elicited preferences of patients (n = 59, 75%). Across reviewed studies, survival attributes were commonly ranked as most important, with overall survival (OS) and progression-free survival (PFS) ranked most important in 58% and 28% of models, respectively. Preferences varied between stakeholder groups, with patients and clinicians placing greater importance on survival outcomes, and general population samples valuing health-related quality of life (HRQoL). Despite the emphasis of guidelines on the importance of using qualitative research to inform attribute selection and DCE designs, reporting on instrument development was mixed. Limitations No formal assessment of bias was conducted, with the scope of the paper instead providing a descriptive characterisation. The review only included DCEs relating to cancer treatments, and no insight is provided into other health technologies such as cancer screening. Only DCEs were included. Conclusions and Implications Although there was variation in attribute importance between responder types, survival attributes were consistently ranked as important by both patients and clinicians. Observed challenges included the risk of attribute dominance for survival outcomes, limited sample sizes in some indications, and a lack of reporting about instrument development processes. Protocol Registration PROSPERO 2020 CRD42020184232.

Health-related quality of life (HRQoL) outcome can be used to monitor health and well-being and is one of the important outcomes that may predict overall survival (OS) in cancer patients. This study examined the association of OS and HRQoL measured before (T0), after cancer diagnosis (T1), and HRQoL changes from T0 to T1 (T1-T0) in patients diagnosed with bladder cancer (BC). Methods: This longitudinal retrospective cohort study used the 1998-2013 Surveillance, Epidemiology and End Results (SEER) database linked with Medicare Health Outcomes Survey (MHOS). HRQoL was measured using the SF-36 (1998-2005)/ Veterans Rand-12 (2006 onwards) and reported as physical (PCS), mental (MCS) component scores, and 8 domain scores. Adjusted hazard ratios (aHRs [95%CI]) were estimated using multivariable logistic regression models, adjusting for demographics, comorbidities, and cancer specific characteristics. Results: 438 BC patients aged $65 years who had HRQoL data at both T0 and T1 were identified. Among those, 220 (50.2%) died and 218 (49.8%) remained alive (censored). Compared to deceased patients, the censored patients were younger (75.366.0 vs. 77.266.8 years; p,0.01), had longer OS (90.4639.9 vs. 67.9642.0 months; p,0.001), had a lower-stage tumor at diagnosis (p,0.001), and had non-invasive tumors (p,0.01). Risk of death was negatively associated with increased PCS at T0 and T1 (aHR T0 =0.

Health technology assessment (HTA) agencies vary in their use of quantitative patient preference data (PP) and the extent to which they have formalized this use in their guidelines. Based on the authors' knowledge of the literature, we identified six different PP “use cases” that integrate PP into HTA in five different ways: through endpoint selection, clinical benefit rating, predicting uptake, input into economic evaluation, and a means to weight all HTA criteria. Five types of insight are distinguished across the use cases: understanding what matters to patients, predicting patient choices, estimating the utility generated by treatment benefits, estimating the willingness to pay for treatment benefits, and informing distributional considerations. Summarizing the literature on these use cases, we recommend circumstances in which PP can add value to HTA and the further research and guidance that is required to support the integration of PP in HTA. Where HTA places more emphasis on clinical outcomes, novel endpoints are available; or where there are already many treatment options, PP can add value by helping decision makers to understand what matters to patients. Where uptake is uncertain, PP can be used to estimate uptake probability. Where indication-specific utility functions are required or where existing utility measures fail to capture the value of treatments, PP can be used to generate or supplement existing utility estimates. Where patients are paying out of pocket, PP can be used to estimate willingness to pay.

BackgroundAntiretroviral treatment (ART) for patients living with HIV (PLHIV) has improved greatly, however, challenges with daily oral dosing remain. New ART options with reduced dosing frequency and innovative delivery methods may help address these challenges. This study assesses patient and physician satisfaction with current treatments and preferences for switching to a monthly or every other month long-acting injectable (LAI) ART.MethodsThis is a cross-sectional online survey of PLHIV and physicians treating PLHIV in United States and Canada. A literature review, clinical expert input, and qualitative and quantitative pilots informed survey design. Eligible PLHIV were on ART for ≥ 6 months and virally suppressed (self-reported). Survey questions for patients evaluate satisfaction and adherence to current ART. Treatment preferences are assessed using a discrete choice experiment (DCE), where respondents choose between staying on current ART, switching to another oral ART or switching to a LAI ART. DCE treatment attributes include dosing frequency, side effects, forgivability, food/mealtime restrictions, and mode of administration. Pilot data for US patients is included here; the main survey will include approximately 550 patients and 450 physicians.ResultsOf 51 PLHIV completing the pilot survey, 80% were male, mean age was 54 years, and 63% were on ART for ≥10 years. Switching ART was common, with 55% reporting changing their ART ≥ 3 times. Just under half of patients (47%) were not totally satisfied with their current ART. Most common reasons for dissatisfaction included daily reminder of having HIV (31%) and having to take medicine every day (28%). Just over a quarter of patients (28%) reported forgetting to take their ART in the prior month. Across all DCE choices, patients preferred to remain on their current treatment 47% of the time, while 45% of the time patients preferred switching to the LAI, and for the remaining 8%, patients chose switching to another oral ART regimen.ConclusionDespite advances in ART, treatment challenges remain. Among the treatment-experienced PLHIV in this pilot survey, over half of their choices resulted in switching to an alternative regimen, and when opting to switch, most patients preferred the long-acting injectable treatment regimen.Disclosures All authors: No reported disclosures.