Treatment preferences of adults and adolescents with alopecia areata: A discrete choice experiment.

PRODUCTS with janus kinase (JAK) inhibition have been shown to promote hair regrowth in patients with alopecia areata (AA). To guide drug-approval and treatment decisions, it is important to understand patients' willingness to accept the potential risks of JAK inhibition in exchange for potential benefits. We quantified the treatment preferences of adult (≥18 years) and adolescent patients (12-17 years) with AA in the US and Europe to determine the trade-offs they are willing to make between benefits and risks. Preferences for oral AA treatment attributes were elicited using a discrete choice experiment consisting of 12 tasks in which patients chose between two hypothetical treatment alternatives and no treatment. Benefits included the probability of 80%-100% scalp hair regrowth (Severity of Alopecia Tool score ≤ 20) and achieving moderate-to-normal eyebrow and eyelash hair. Treatment-related risks included 3-year probabilities of serious infection, cancer, and blood clots. Preference estimates were used to calculate the maximum level of each risk that patients were willing to accept for increases in treatment benefits. The most important attribute to both adults (n = 201) and adolescents (n = 120) was a 50% probability of achieving hair regrowth on most or all the scalp; however, adolescents placed greater relative importance on this attribute than did adults. Adults were averse to the risks of serious infection, cancer, and blood clots, whereas adolescents were averse to the risk of cancer. For a 20% increase in the probability of 80%-100% scalp hair regrowth, adults were willing to accept a mean (95% confidence interval) 3-year risk of serious infection, cancer, and blood clots of 7.4% (5.5-9.3), 2.5% (1.9-3.1), and 9.3% (6.4-12.2). Adolescents were willing to accept a 3-year risk of cancer of 3.3% (2.4-4.2). Patients with AA in the US and Europe are willing to accept substantial risks to obtain an effective treatment.

Treatment preferences of patients with relapsed or refractory multiple myeloma in the United States, United Kingdom, Italy, Germany, France, and Spain: results from a discrete choice experiment.

Introduction: Newer treatment options for relapsed/refractory multiple myeloma (RRMM) with efficacy and safety profiles that differ from traditional therapies have facilitated personalized management strategies to optimize patient outcomes. In the context of such personalized management, understanding how treatment characteristics influence patients' preferences is essential. This study assessed patients' preferences for RRMM treatment attributes and determined trade-offs between potential benefits, administration procedures, and adverse effects. Methods: Patients' preferences were evaluated using a discrete choice experiment (DCE). Patients with RRMM who reported failing two lines of anti-myeloma treatment (immunomodulatory agent and a proteasome inhibitor [PI]) or ≥ 3 lines (including ≥1 PI, immunomodulatory agent, or anti-CD38 monoclonal antibody), were recruited across the US, UK, Italy, Germany, France, and Spain. DCE attributes and levels were identified using a targeted literature review, a review of clinical data for relevant RRMM treatments, qualitative patient interviews, and input from clinical and myeloma patient experts. The DCE was administered within an online survey from February-June 2022. Preference data were analyzed using an error-component logit model and willingness to make trade-offs for potential benefits, and relative attribute importance scores were calculated. Results: Overall, 296 patients from the US (n = 100), UK (n = 49), Italy (n = 45), Germany (n = 43), France (n = 39), and Spain (n = 20) participated in the DCE. Mean (standard deviation) age was 63.8 (8.0) years, 84% had a caregiver, and patients had a median of 3 (range: 2-8) prior lines of therapy. Efficacy attributes most influenced patients' preferences, with increasing overall response rate (25-85%) and overall survival (6 months to 2 years) contributing to ~50% of treatment decision-making. Administration procedures were also considered important to patients. Avoiding individual side effects was considered relatively less important, with patients willing to tolerate increases in side effects for gains in efficacy. Patient characteristics such as rate of disease progression, sociodemographics, or clinical characteristics also influenced treatment preferences. Conclusion: Patients with RRMM were willing to tolerate increased risk of side effects for higher efficacy. Preferences and risk tolerance varied between patients, with preference patterns differing by certain patient characteristics. This highlights the importance of shared decision-making for optimal treatment selection and patient outcomes.

Patient Preferences for Treatment of Bacillus Calmette-Guérin-unresponsive Non-muscle-invasive Bladder Cancer: A Cross-country Choice Experiment.

Background: Patients with non-muscle-invasive bladder cancer (NMIBC) that is unresponsive to bacillus Calmette-Guérin (BCG) immunotherapy face a difficult choice. Immediate radical cystectomy (RC) is effective but might represent overtreatment. Continuing bladder preservation with medical therapy is an alternative, but it risks progression to muscle-invasive bladder cancer (MIBC) and a reduction in survival. Objective: To understand the trade-offs patients are willing to make in selecting treatments for BCG-unresponsive NMIBC. Design setting and participants: Adults with NMIBC from the UK, France, Germany, and Canada who reported current receipt of BCG, disease unresponsive to BCG, or receipt of RC in the previous 12 mo after failure of BCG were recruited to participate in an online choice experiment. Patients were asked to make repeated choices between two hypothetical medical treatments and the option to undergo immediate RC. The medical treatments required trade-offs between the time to RC, the mode and frequency of administration, the risk of experiencing serious side effects, and the risk of disease progression. Outcome measurements and statistical analysis: Error component logit models were used to calculate relative attribute importance (RAI) scores as the maximum percentage contribution to a preference and acceptable benefit-risk trade-offs. Results and limitations: Most of the 107 participants (average age 63 yr) never selected RC (89%) as their preferred option in the choice experiment. Preferences were most affected by time to RC (RAI 55%), followed by risk of progressing to MIBC (RAI 25%), medication administration (RAI 12%), and the risk of serious side effects (RAI 8%). To increase the time to RC from 1 yr to 6 yr, patients accepted a 43.8% increase in the risk of progression and a 66.1% increase in the risk of serious side effects. Conclusions: Patients with BCG-treated NMIBC valued bladder-sparing treatments and were willing to make substantial benefit-risk trade-offs to delay RC. Patient summary: Adults with bladder cancer not invading the bladder muscle completed an online experiment in which they chose between hypothetical medications and bladder removal. The results show that patients would be willing to accept different risks associated with medications to delay bladder removal. Patients considered disease progression the most important risk of medicinal treatment.

Current Health State Affected Patient Preferences More Than Disease Status: A Discrete Choice Experiment in Multiple Myeloma.

OBJECTIVES: To examine how disease status and current health state influence treatment preferences of patients with multiple myeloma (MM). METHODS: Participants with MM from France, Germany, and the United Kingdom completed a web-based survey that included a discrete choice experiment (DCE) and EQ-5D assessment. The DCE elicited preferences for 8 attributes: increased life expectancy, increased time to relapse, pain, fatigue, risk of infection, administration (route and duration), frequency of administration, and monitoring. Multinomial logit models were used to analyze DCE preference data and to calculate life expectancy trade-offs. RESULTS: Three hundred participants with MM (newly diagnosed, transplant eligible, n = 108; newly diagnosed, transplant ineligible, n = 105; relapsed-refractory, n = 87) completed the survey. The most valued attributes were pain, fatigue, and increased life expectancy. Participants would want an additional 2.7 years of life expectancy (95% confidence interval [CI] 2.4-3.1 years) to tolerate extreme pain and an additional 2.0 years of life expectancy (95% CI 1.6-2.3 years) to tolerate constant fatigue. Participants in a better health state (third EQ-5D score quartile [0.897]) required less additional life expectancy than participants with a worse health state (first EQ-5D score quartile [0.662]) to tolerate extreme pain (2.3 years [95% CI 1.9-2.6 years] vs 3.0 years [95% CI 2.6-3.4 years]; P = .007). There was little difference in treatment preferences between newly diagnosed and relapsed-refractory patients for pain, fatigue, and increased life expectancy. CONCLUSIONS: Current health state influenced treatment preferences of patients with MM more than disease status and should be considered when making treatment decisions.

Patient Preferences for Multi-Cancer Early Detection (MCED) Screening Tests.

BACKGROUND: Emerging blood-based multi-cancer early detection (MCED) tests can detect a variety of cancer types across stages with a range of sensitivity, specificity, and ability to predict the origin of the cancer signal. However, little is known about the general US population's preferences for MCED tests. OBJECTIVE: To quantify preferences for MCED tests among US adults aged 50-80 years using a discrete choice experiment (DCE). METHODS: To quantify preferences for attributes of blood-based MCED tests, an online DCE was conducted with five attributes (true positives, false negatives, false positives, likelihood of the cancer type unknown, number of cancer types detected), among the US population aged 50-80 years recruited via online panels and social media. Data were analyzed using latent class multinomial logit models and relative attribute importance was obtained. RESULTS: Participants (N = 1700) were 54% female, mean age 63.3 years. Latent class modeling identified three classes with distinct preferences for MCED tests. The rank order of attribute importance based on relative attribute importance varied by latent class, but across all latent classes, participants preferred higher accuracy (fewer false negatives and false positives, more true positives) and screenings that detected more cancer types and had a lower likelihood of cancer type unknown. Overall, 72% of participants preferred to receive an MCED test in addition to currently recommended cancer screenings. CONCLUSIONS: While there is significant heterogeneity in cancer screening preferences, the majority of participants preferred MCED screening and the accuracy of these tests is important. While the majority of participants preferred adding an MCED test to complement current cancer screenings, the latent class analyses identified a small (16%) and specific subset of individuals who value attributes differently, with particular concern regarding false-negative and false-positive test results, who are significantly less likely to opt-in.

Patients with Inflammatory Bowel Disease Have Heterogeneous Treatment Preferences That Are Largely Determined by the Avoidance of Abdominal Pain and Side Effects [P-POWER IBD Study].

BACKGROUND AND AIMS: Patient-centric management of inflammatory bowel disease [IBD] is important, with consensus considering patient-reported outcomes alongside clinical and endoscopic assessment by healthcare providers. However, evidence regarding patients' treatment priorities is still limited. This study aimed to elicit benefit-risk trade-offs that patients with IBD are willing to make, to help inform discussions about patient-centric treatment targets. METHODS: This was a cross-sectional online survey of adults with self-confirmed Crohn's disease [CD] or ulcerative colitis [UC] receiving IBD treatment. The impact of efficacy, administration and safety on treatment preferences was elicited using a discrete choice experiment. Relative attribute importance [RAI] and maximum acceptable risk of mild-to-moderate side effects [SEs] were estimated from a mixed logit model. RESULTS: In total, 400 patients [CD: 54%; UC: 46%; female: 38.0%; age range: 18-78 years] were recruited. Efficacy, administration and safety affected treatment preferences to varying degrees, with abdominal pain being most important [RAI 33%] followed by risks of mild-to-moderate SEs [RAI 27%] and serious infections [RAI 16%]. To reduce abdominal pain from severe to moderate/mild, patients accepted an additional 18.8% or 30.6% risk of mild-to-moderate SEs, respectively. While average preferences between patients with CD and UC were similar, patients with CD placed greater importance on abdominal pain [p < 0.05], and patients with UC on bowel urgency [p < 0.05]. However, preferences varied notably. CONCLUSIONS: While avoiding abdominal pain, SEs and serious infections had on average the highest treatment priority, preferences varied between patients. Treatment strategies should consider the trade-offs individuals are willing to make.

How do study design features and participant characteristics influence willingness to participate in clinical trials? Results from a choice experiment.

BACKGROUND: Research about the decision to participate in a clinical study has tended to be limited to single indications and has focused on narrow sets of study and participant characteristics. This study applied stated preference methods to understand the clinical trial design attributes that most influence willingness to participate and how this varied with participant characteristics. METHODS: Adults residing in the US, China, or Poland with a self-reported diagnosis of cancer, heart disease, migraine, rheumatoid arthritis, or multiple sclerosis completed an online survey. Participants were asked whether they would participate in clinical studies defined by seventeen attributes within five categories (payment/support, administration/procedures, treatment-related, study location/time commitment, and data collection/feedback). Participants saw six different hypothetical clinical study profiles. Depending on their participation decision to an initial clinical study profile, the subsequent five questions had one design attribute (randomly selected per question) consecutively improved or deteriorated to elicit preferences. A logistic regression was used to determine which participant characteristics influenced participation decisions. A latent class logit model was used to identify how the influence of study design features varied between participants and whether groups of participants with similar preferences could be identified. RESULTS: The survey was completed by 487 participants (32% China, 35% Poland, 33% US; 8%-19% per indication). Willingness to participate was found to be a function of participant age, certain elements of quality of life, and previous treatment experience, in particular number of lines of treatment received and experience of adverse events. Willingness to participate was influenced by study design features such as payment, study duration, and time commitment - both the overall time and whether the time was at home or away from home, with the latter being particularly relevant to participants experiencing fatigue due to their disease. CONCLUSIONS: This study quantifies how study designs influence willingness to participate and how this varies with participant types. These findings suggest that it is how an indication influences quality of life and treatment experience, rather than the indication alone, that impacts participation rates, opening the way for insights that are transferrable across indications, which may be particularly useful when considering rare diseases.

Patient preferences for frontline therapies for Philadelphia chromosome-positive acute lymphoblastic leukemia: a discrete choice experiment.

Aim: We examined the preferences of adults with Philadelphia chromosome-positive acute lymphoblastic leukemia (Ph+ ALL) for benefits and risks of tyrosine kinase inhibitors combined with chemotherapy for first-line treatment. Methods: In a discrete choice experiment, 201 patients chose between hypothetical treatment alternatives with varied levels of remission duration and overall survival (OS), and risks of major cardiovascular (CV) events and myelosuppression. Results: Although OS was the most important attribute to patients with Ph+ ALL, they were willing to tolerate a 2.9% increase in CV risk for 1 additional month of OS. Older patients (>59 years) and patients not in remission were less likely to tolerate increased CV risk. Conclusion: Preferences and risk tolerance varied between patients, highlighting the importance of shared decision making when selecting treatments for Ph+ ALL.

Treatments differ in their potential benefits and side effects they may come with. Patients should be involved in deciding which treatments they receive. This is because patients may have different views than physicians on how the benefits and side effects of treatment would affect their quality of life. Additionally, patients may have different risk tolerances. This study shows how patients with a form of leukemia valued survival benefits and side effects of treatments, and the trade-offs that they were willing to make between these. On average, longer survival had most value to patients. They were willing to accept a higher risk of a major cardiovascular side effect (e.g., having a stroke) if the treatment would allow them to live longer. However, not all patients had the same opinion, and some groups of patients were less willing to accept risks to receive longer survival. By involving patients in treatment decisions, we can help ensure they receive treatments that match their personal preferences.

Evaluating the Consistency of Patient Preference Estimates: Systematic Variation in Survival-Adverse Event Trade-Offs in Patients with Cancer or Cardiovascular Disease.

BACKGROUND: The increased use of patient preference data in healthcare decision making has raised concerns about the reliability and consistency of the estimates generated by patient preference studies. However, literature reviews to assess the consistency of preferences are confounded by heterogeneity in study designs. METHODS: This paper adopted a novel approach to evaluating preference consistency: comparing estimates of a single trade-off-the marginal rate of substitution (MRS) between survival improvements and risks of adverse events-across multiple patient groups and using meta-regression to assess whether MRS varied systematically between patients. A log-linear, random effects regression was run, weighted for the sample sizes of studies from which estimates were extracted. RESULTS: Using studies identified in published reviews of patient preference data, 42 estimates of MRS were generated from the 12 studies. On average, patients obtained the same utility from a 2.3% reduction in the risk of an adverse event as from a 1-month increase in survival, with a range of 0.002-13.5%. The regression model had an R2 of over 90% and suggests that MRS depended on patients' expected survival and the type of adverse event being traded. CONCLUSION: These results suggest that although preferences vary between patients, they may do so in systematic and predictable ways. Further, they do so in ways consistent with societal preferences and decision maker priorities toward end-of-life settings. Further work is required to replicate this result in other patient groups and to explore the consistency of preferences for other treatment attributes.

Patient Preferences of Low-Dose Aspirin for Cardiovascular Disease and Colorectal Cancer Prevention in Italy: A Latent Class Analysis.

BACKGROUND: Patients taking low-dose aspirin to prevent cardiovascular disease (CVD) may also benefit from a reduced risk of colorectal cancer (CRC). OBJECTIVE: The aim was to examine the preferences of people eligible for preventive treatment with low-dose aspirin and the trade-offs they are willing to make between CVD prevention, CRC prevention, and treatment risks. METHODS: A cross-sectional study using a discrete choice experiment (DCE) survey was conducted in Italy in 2019 to elicit preferences for three benefit attributes (prevention of ischemic stroke, myocardial infarction, and CRC) and four risk attributes (intracranial and gastrointestinal bleeding, peptic ulcer, and severe allergic reaction) associated with use of low-dose aspirin. Latent class logit models were used to evaluate variation in treatment preferences. RESULTS: The DCE survey was completed by 1005 participants eligible for use of low-dose aspirin. A four-class model had the best fit for the primary CVD prevention group (n = 491), and a three-class model had the best fit for the secondary CVD prevention group (n = 514). For the primary CVD prevention group, where classes differed on age, education level, type 2 diabetes, exercise, and low-dose aspirin use, the most important attributes were intracranial bleeding (two classes), myocardial infarction (one class), and CRC (one class). For the secondary CVD prevention group, where classes differed on various comorbidities, self-reported health, exercise, and CVD medication use, the most important attributes were intracranial bleeding (two classes), myocardial infarction (one class), and gastrointestinal bleeding (one class). CONCLUSION: Patient preferences for the benefits and risks of low-dose aspirin differ significantly among people eligible for treatment as primary or secondary CVD prevention.

Paid work, household work, or leisure? Time allocation pathways among women following a cancer diagnosis.

BACKGROUND: There is extensive evidence on the relationship between receiving a cancer diagnosis and labour market outcomes. However, there is limited evidence on the relationship between a cancer diagnosis and non-labour market outcomes such as household work, passive leisure and physical leisure. Furthermore, most current research focuses on time allocation at a given time point, ignoring both the life course perspective and long term effects. METHODS: Using data from the Australian Longitudinal Study on Women's Health (ALSWH), we observe time allocation for 91 women diagnosed with breast cancer and 486 women diagnosed with skin cancer, and 2711 women living without cancer. Our analysis is unique in combining sequence analysis and multivariate multinomial logit modelling. Using sequence analysis, we first analyse life-courses post a cancer diagnosis for paid work, household work, passive leisure and physical leisure from 2004 to 2016. Using multivariate multinomial logit modelling, we test whether a cancer diagnosis influences such life-courses. We consider the robustness of results to cancer severity and change in time allocation before and after a cancer diagnosis. RESULTS: We identify three clusters for paid work and two clusters for household work, passive leisure and physical leisure. Neither a breast cancer nor skin cancer diagnosis has a significant effect on the pathways of time allocation. Such results are robust to severity of diagnosis with 'cured' and 'non-cured' breast cancer, and skin cancer treated with and without 'chemotherapy or radiotherapy'. Women post a breast cancer diagnosis are less likely to maintain their pre-cancer's time use on paid work, household work and passive leisure; however, such effects are not significant. CONCLUSION: Being diagnosed with breast cancer or skin cancer has no significant long-term effect on the pathways of time allocation. Future work should explore the relationship between time allocation and wellbeing and the preference for time allocation.

Evaluating the Trade-Offs Men with Localized Prostate Cancer Make between the Risks and Benefits of Treatments: The COMPARE Study.

PURPOSE: The COMPARE (COMparing treatment options for ProstAte cancer) study aimed to evaluate and quantify the trade-offs patients make between different aspects of active surveillance and definitive therapy. MATERIALS AND METHODS: A discrete choice experiment tool was used to elicit patient preferences for different treatment characteristics in 34 urology departments. Patients with localized prostate cancer completed the discrete choice experiment within 1 week of being diagnosed and before they made treatment decisions. The discrete choice experiment was pretested (5) and piloted (106) with patients. Patients chose their preferred treatment profile based on the 6 characteristics of treatment type (active surveillance, focal therapy, radical therapy), return to normal activities, erectile function, urinary function, not needing more cancer treatment and 10 to 15-year cancer specific survival. Different tools were designed for patients with low-intermediate (468) and high risk (166) disease. An error components conditional logit model was used to estimate preferences and trade-offs between treatment characteristics. RESULTS: Patients with low-intermediate risk disease were willing to trade 6.99% absolute decrease in survival to have active surveillance over definitive therapy. They were willing to trade 0.75%, 0.46% and 0.19% absolute decrease in survival for a 1-month reduction in time to return to normal activities and 1% absolute improvements in urinary and sexual function, respectively. Patients with high risk disease were willing to trade 3.10%, 1.04% and 0.41% absolute decrease in survival for a 1-month reduction in time to return to normal activities and 1% absolute improvements in urinary and sexual function, respectively. CONCLUSIONS: Patients with low-intermediate risk prostate cancer preferred active surveillance to definitive therapy. Patients of all risk levels were willing to trade cancer specific survival for improved quality of life.

What are GPs' preferences for financial and non-financial incentives in cancer screening? Evidence for breast, cervical, and colorectal cancers.

General practitioners (GPs) play a key role in the delivery of preventive and screening services for breast, cervical, and colorectal cancers. In practice, GPs' involvement varies considerably across types of cancer and among GPs, raising important questions about the determinants of GPs' implication in screening activities: what is the relative impact of financial and non-financial incentives? Are GPs' preferences for financial and non-financial incentives cancer-specific? Is there preference heterogeneity and how much does it differ according to the screening context? This study investigates the determinants of GPs' involvement in cancer screening activities using the discrete choice experiment (DCE) methodology. A representative sample of 402 GPs' was recruited in France between March and April 2014. Marginal rates of substitution were used to compare GPs' preferences for being involved in screening activities across three types of cancers: breast, cervical, and colorectal. Variability of preferences was investigated using Hierarchical Bayes mixed logit models. The results indicate that GPs are sensitive to both financial and non-financial incentives, such as a compensated training and systematic transmission of information about screened patients, aimed to facilitate communication between doctors and patients. There is also evidence that the level and variability of preferences differ across screening contexts, although the variations are not statistically significant on average. GPs appear to be relatively more sensitive to financial incentives for being involved in colorectal cancer screening, whereas they have higher and more heterogeneous preferences for non-financial incentives in breast and cervical cancers. Our study provides new findings for policymakers interested in prioritizing levers to increase the supply of cancer screening services in general practice.