Christine Poulos

Introduction Benefit–risk profiles and mode/frequency of administration vary among the four US-approved treatments for anti-aquaporin-4 antibody-positive neuromyelitis optica spectrum disorder (AQP4-Ab+ NMOSD), but no published studies have reported treatment preferences among patients with NMOSD in the USA. Therefore, the objective of this study was to quantify preferences and predict treatment choices among patients with AQP4-Ab+ NMOSD in the USA. Methods A cross-sectional, web-based discrete choice experiment survey was administered to patients with self-reported AQP4-Ab+ NMOSD. Respondents evaluated hypothetical AQP4-Ab+ NMOSD treatment profile pairs defined by efficacy, safety, process-related attributes, and administration mode/frequency. Data were analyzed using a random-parameters logit model to estimate conditional relative importance of attributes, minimum acceptable benefit, and predicted treatment choice in pairwise treatment profile (ravulizumab-like, eculizumab-like, inebilizumab-like, and satralizumab-like) comparisons. Preference heterogeneity was investigated by exploratory subgroup analysis using interaction terms in the regression analysis. Results The 255 survey completers (mean ± standard deviation [SD] age, 41.4 ± 13.7 years) averaged 6.6 ± 5.4 years since diagnosis, and 63.5% identified as female and 46.7% as Black or African American. Rituximab was the most common treatment (34.1% of respondents), and 15.7%, 9.4%, or 7.1% of respondents were receiving eculizumab, inebilizumab, or satralizumab, respectively. Respondents placed greater importance on reducing chance of relapse within the first year of treatment compared with other attributes such as reducing administration frequency from every 2 weeks to every 8 weeks (Q8W); safety attributes (risks of serious opportunistic or recurrent infection, elevated liver enzymes, and meningococcal infection) were rated similar to each other in importance. Pairwise comparisons favored the ravulizumab-like profile (67.8–87.7%) over the other three treatment profiles (12.3–32.2%). Preferences did not vary by age, disease duration, disease impact, or relapses in the past 12 months, but subgroups defined by current treatment type demonstrated different (p = 0.066) preferences. Respondents receiving intravenous/subcutaneous injections alone or with oral immunosuppressive therapy/corticosteroid treatments placed greater importance on reducing chance of relapse versus those receiving oral treatments alone, but they were not more likely to select a Q24W treatment over a Q8W treatment. Conclusions Respondents with AQP4-Ab+ NMOSD placed the highest importance on reducing chance of relapse, placed a high importance on reduced administration frequency, and rated the safety attributes (avoiding treatments with a risk of meningococcal infection, risk of elevated liver enzymes, and risk of other infections) as comparable to each other but less influential than relapse prevention. Respondents were more likely to select a ravulizumab-like profile over comparators. These findings can inform shared decision-making in selecting treatments.

To quantify preferences for an adult combination vaccine for influenza and COVID-19 (flu + COVID) compared with standalone influenza and COVID-19 vaccines. This survey study used a series of direct-elicitation questions to assess preferences for a single-shot combination flu + COVID, standalone influenza, and standalone COVID-19 vaccines among US consumers (N = 601) and healthcare professionals (HCPs) (N = 299). Response frequencies described the proportion of each sample that would prefer a flu + COVID vaccine to standalone influenza and COVID-19 vaccines. A multivariate logit regression model explored how certain characteristics influenced the odds of selecting the flu + COVID vaccine over a standalone influenza vaccine. Most consumers (398/601; 66.2%) and HCPs (250/298; 83.9%) preferred a flu + COVID vaccine to a standalone influenza vaccine. When not forced to choose between flu + COVID and standalone influenza vaccines, most consumers again selected the flu + COVID vaccine (62.3%); 14.7% would prefer separate standalone influenza and COVID-19 vaccines, 8.3% a standalone influenza vaccine only, 7.3% a COVID-19 vaccine only, and 7.4% neither vaccine. Consumers aged ≥50 years with a body mass index ≥40, those aged ≥65 years who previously received a COVID-19 vaccine, and those who had previously experienced severe impacts from influenza were more likely to choose a flu + COVID vaccine over a standalone influenza vaccine than were consumers without these characteristics. HCPs whose practice stocks high-dose influenza vaccines were more likely to choose the flu + COVID vaccine for patients aged ≥65 with no risk factors and patients aged 18-64 with ≥1 risk factor over the standalone influenza vaccine. Results are subject to potential hypothetical, responder, selection, and information biases. Most US consumers and HCPs would likely prefer a single-shot combination flu + COVID vaccine compared with standalone influenza and COVID-19 vaccines. Given the low COVID-19 vaccination coverage rates in the US, the availability of a combination flu + COVID vaccine could help increase COVID-19 vaccine coverage.

To quantify physicians' preferences for adjuvant renal cell carcinoma (RCC) treatments. A discrete-choice experiment was administered online to board-certified/eligible physicians. Physicians chose between pairs of hypothetical adjuvant therapies for a high-risk patient who had recently undergone a radical nephrectomy. Data were analyzed using random-parameters logit and latent-class models. Physicians (n = 250; 64% oncologists; 36% urologists) placed most importance on improvements in the chance of 5-year overall survival, followed by increased median disease-free survival and reduced risk of side effects. The analyses also highlighted their willingness to make tradeoffs between these benefits and risks. Physicians were generally tolerant of increases in the risks of treatment-related severe diarrhea, dizziness, and fatigue and were willing to accept increases in these risks in exchange for improvements in overall or disease-free survival. Subgroup analysis revealed heterogeneity between oncologists and urologists, and latent-class analysis revealed significant heterogeneity among the whole physician sample. Most physicians in this study would recommend adjuvant therapy to a typical high-risk postnephrectomy RCC patient.

To quantify patients' preferences for adjuvant renal cell carcinoma (RCC) treatments. Preferences were elicited using a discrete-choice experiment requiring RCC patients to choose between 2 hypothetical treatments. Data were analyzed using random-parameters logit and latent-class models. Patients (n = 250) preferred treatments that increase disease-free and overall survival (OS), are taken less frequently, require no concomitant medication, have a shorter duration, and have lower side-effect risks. The analyses also highlighted their willingness to make tradeoffs between these benefits and risks. Patients were generally tolerant of increases in the risks of treatment-related severe diarrhea, dizziness, and fatigue and were willing to accept increases in these risks in exchange for improvements in overall or disease-free survival. Latent-class analysis identified 3 classes: class 1 (37.5%) and class 2 (26.9%) preferred not to opt out of treatment and prioritized increased OS and disease-free survival, respectively; class 3 (35.5%) preferred to opt out and prioritized mode, duration, and risks. Heterogeneity suggests patient-physician discussions are important when considering RCC treatments.

To understand the preferences of healthcare providers (HCPs) in Switzerland for pediatric hexavalent vaccine attributes. A discrete-choice experiment included a series of choices between 2 hypothetical pediatric hexavalent vaccines with varying attributes: device type (including preparation time and risk of dosage errors), proportion of infants seroprotected against Haemophilus influenzae type b (Hib) at 11-12 months (pre-booster), packaging size, years on the market, and the thermostability at room temperature. Odds ratios (ORs) and conditional relative attribute importance (CRAI) were calculated using random-parameters logit. HCPs (150 pediatricians and 40 nursing staff) in Switzerland were unlikely to choose a vaccine conferring 50% (OR 0.00; 95% CI 0.00-0.00) or 70% (OR 0.01; 95% CI 0.00-0.01) of infants with Hib seroprotection at 11-12 months (pre-booster) compared with a vaccine conferring 90% seroprotection. The odds of choosing a vaccine available on the market for more than 3 years were nearly 5 times the odds of choosing a vaccine available on the market for less than 1 year (OR 4.76; 95% CI 1.87-7.65). The odds of choosing a vaccine in a prefilled syringe were nearly 3 times the odds of choosing a reconstituted vaccine (OR 2.77; 95% CI 1.39-4.15), and the odds of choosing a vaccine with a smaller package size were nearly 2 times the odds of choosing a vaccine with larger package size (OR 1.89; 95% CI 1.23-2.55). HCPs were equally likely to choose vaccines that can stay at room temperature for 6 versus 3 days (OR 1.07; 95% CI 0.73-1.42). According to CRAI, the most important attribute was Hib seroprotection, followed by years on the market, device type, and packaging size. Hib seroprotection at 11-12 months was the most important hexavalent vaccine attribute to HCPs in this study.

Aim: To describe development of a shared decision making (SDM) aid in treating primary immunodeficiency diseases (PID) with immunoglobulin replacement therapy (IGRT). Materials & methods: Expert en... Plain language summary Shared decision making and developing a decision aid Shared decision making happens when patients and doctors work together to choose treatment options based on a patient's c... Tweetable abstract For patients with #primaryimmunodeficiency considering how to access immunoglobulin replacement therapy, a shared decision-making aid using best–worst scaling may improve treatme...

Objectives To adapt a patient-reported outcome (PRO) measure, the Western Ontario McMaster Universities Osteoarthritis Index (WOMAC), into efficacy attributes for a discrete choice experiment (DCE) survey designed to quantify the relative importance of endpoints commonly used in knee osteoarthritis (KOA) trials. Methods The adaptation comprised four steps: (1) selecting domains of interest; (2) determining presentation and framing of selected attributes; (3) determining attribute levels; and (4) developing choice tasks. This process involved input from multiple stakeholders, including regulators, health preference researchers, and patients. Pretesting was conducted to evaluate if patients comprehended the adapted survey attributes and could make trade-offs among them. Results The WOMAC pain and function domains were selected for adaption to two efficacy attributes. Two versions of the discrete choice experiment (DCE) instrument were created to compare efficacy using (1) total domain scores and (2) item scores for “walking on a flat surface.” Both attributes were presented as improvement from baseline scores by levels of 0%, 30%, 50%, and 100%. Twenty-six participants were interviewed in a pretest of the instrument (average age 60 years; 58% female; 62% had KOA for ≥ 5 years). The participants found both versions of attributes meaningful and relevant for treatment decision-making. They demonstrated willingness and ability to tradeoff improvements in pain and function separately, though many perceived them as inter-related. Conclusions This study adds to the growing literature regarding adapting PRO measures for patient preference studies. Such adaptation is important for designing a preference study that can incorporate a clinical trial’s outcomes with PRO endpoints.

Physicians’ preferences for attributes of medical treatments for endometriosis-associated pain have not previously been quantified. US obstetrician-gynecologists completed an online discrete-choice experiment survey. In a series of questions, physicians chose a medical treatment for a hypothetical patient with endometriosis experiencing severe, persistent dysmenorrhea, nonmenstrual pelvic pain, and/or dyspareunia. Each question presented two hypothetical medical treatments for endometriosis-associated pain, defined by seven attributes with varying levels. Preferences weights and conditional relative importance (CRI) were calculated using a random-parameters logit model. Respondents (N = 250) had an average age of 53 years; 36% were female. The most important attribute, conditional on the attributes and levels evaluated, was risk of moderate-to-severe hot flashes (CRI, 3.34). In descending order of importance, the CRIs of the other attributes were 2.13 for improvement in nonmenstrual pelvic pain, 2.04 for improvement in dyspareunia, 1.88 for improvement in dysmenorrhea, 1.16 for risk of pregnancy-related complications if pregnancy occurs during treatment, 0.62 for increased risk of bone fracture later in life, and 0.48 for mode of administration. In addition to valuing pain reduction, respondents prioritized avoiding moderate-to-severe hot flashes, followed by less common and less immediate risks of pregnancy-related complications and bone fracture.