Search

Introduction: Loss of cognitive function is a common feature in schizophrenia. However, generic measures of health-related quality of life favored by decision-makers, such as the EQ-5D, are not designed to detect changes in cognitive function. We report the valuation of the Schizophrenia Cognition Rating Scale (SCoRS), a schizophrenia-specific measure of cognitive impairment. Methods: Expert opinion and psychometric analysis of the SCoRS from clinical trial data was undertaken to select 5 key items from the measure. These items were combined orthogonally to develop health-state vignettes. Vignettes were valued using composite time trade-off (cTTO) in one-on-one video calls. Several econometric models were fitted to the data to estimate disutilities. Performance of EQ-5D- and SCoRS-based utilities were compared in the trial data. Results: The SCoRS items selected for the valuation study represented attention, learning, processing speed, social cognition and memory. Four hundred respondents participated in the valuation study. The best observed health state was valued at 0.855 [standard deviation (SD) = 0.179] and the worst at 0.152 (SD = 0.575). At the most severe levels, 'social cognition' received the largest disutility followed by 'learning' and 'memory'. The final model to estimate utilities had 15 parameters. SCoRS-based utilities were sensitive to change in cognition, but the EQ-5D was not. Conclusion: It is feasible to value different dimensions of cognition separately using a validated instrument for proxy assessment. The resulting utilities indicate loss of quality of life due to reduced cognitive functioning. [ABSTRACT FROM AUTHOR]

Background: Previous research has extensively documented the impact of migraine episodes ('ictal') on patients' health-related quality of life. Few studies have looked at the impact of migraine on migraine-free days ('interictal'). This study was designed to describe interictal burden of migraine in a mixed group of people affected by migraine and to explore patient characteristics associated with interictal burden. Methods: People with migraine in the United States (US) and Germany were recruited for a cross-sectional online survey, including a subgroup treated with calcitonin gene-related peptide (CGRP) monoclonal antibody (mAb). The survey included the Migraine Interictal Burden Scale (MIBS-4), Headache Impact Test (HIT-6), and items measuring patient demographics, clinical and treatment background. Data were analyzed using descriptive statistics and linear regression. Results: Five hundred six people with migraine completed the survey (US: n = 257; Germany: n = 249), of whom 195 had taken a CGRP mAb for three or more months. Participants had a mean of 8.5 (SD = 6.4) Monthly Migraine Days (MMD) and 10.4 (SD = 7.1) Monthly Headache Days (MHD). The mean MIBS-4 score was 6.3 (SD = 3.4), with 67% reporting severe interictal burden (MIBS-4: ≥5). The mean HIT-6 score was 65.3 (SD = 6.0), with 86% reporting severe migraine impact (HIT-6: ≥60). MIBS-4 was correlated with the HIT-6 (r = 0.37), MMD and MHD (both r = 0.27). The HIT-6, MMD, MHD, CGRP mAb treatment, and depression all had an independent positive association with the MIBS-4. Conclusion: Two-thirds of the study sample reported substantial interictal burden. Whilst interictal burden was associated with migraine frequency and impact of migraine attacks, study results also show it represented a distinct aspect of the overall disease burden. Study findings further indicate unique associations between interictal burden and depression. A unique positive association between interictal burden and CGRP mAb treatment suggests a remaining unmet need among people affected by migraine treated with CGRP mAb. [ABSTRACT FROM AUTHOR]

Background: The debilitating nature of migraine attacks is widely established; however, less is known about how the interictal burden (i.e., how patients are affected in-between migraine episodes) of migraine impacts on patients' health-related quality of life (HRQL). Acute and preventive treatments may lift the burden of the disease, but they often have unwanted side effects and limited effectiveness. The objective of this study was to understand the interictal burden of migraines, from the patient perspective, and to explore patient experience with migraine treatments. Methods: Participants (n=35) with a self-reported diagnosis of migraine were recruited in the US, UK and Canada, including a subgroup of patients who had taken calcitonin gene-related peptide monoclonal antibody (CGRP mAb) treatment for at least three months. Participants completed a background questionnaire, followed by a semi-structured interview via telephone or video call. The interviews explored patients' migraine symptoms, perception of interictal burden and treatment experience. The interview transcripts were analysed using thematic analysis. Results: The most reported migraine symptom was migraine pain, followed by aura, sensory sensitivity and nausea. Most participants reported interictal impact on HRQL, lifestyle changes they made to avoid triggers or in anticipation of an attack, impacts on work, career, daily activities and relationships. Emotional impacts were reported by all participants, including anger, depression, anxiety and hopelessness. Many participants who took preventive treatments reported improvements in HRQL and functioning but still experienced breakthrough attacks. Among patients who took CGRP mAbs, participants noted varying consistency of treatment effectiveness between treatment administrations. Conclusion: This study detailed the additional HRQL impact of migraine in-between migraine attacks and described the unmet need for effective treatment options to prevent and mitigate migraine attacks. [ABSTRACT FROM AUTHOR]

Background: Spinal muscular atrophy (SMA) is a rare neuromuscular disease that affects motor neurons, resulting in progressive skeletal muscle weakness and atrophy. Objective: The aim of this study was to examine treatment preferences of patients and caregivers of patients with Type 2 and non-ambulatory Type 3 SMA in the Netherlands, Belgium, Finland, Ireland and Portugal. Methods: A discrete choice experiment (DCE) survey was developed to elicit the preferences of adult patients and caregivers regarding different treatment aspects of SMA. This survey built on the design of a similar study undertaken in the UK. The DCE described choice questions in terms of attributes and levels combined using a D-efficient design. The attributes described improvements or worsening in motor and breathing function. The mode of treatment administration (intrathecal injection, single intravenous infusion or regular oral therapy) was described. Treatment risks and side effects related to currently available treatments including risk of liver injury, fatigue, headache, nausea, diarrhoea and rash were described. Lastly, an attribute described whether a treatment had evidence of treatment effectiveness in different SMA types. Participants were recruited via patient advocacy associations to complete an online survey. A clustered conditional logit model was used to estimate treatment preferences. Results: Participants (n = 65) were 4.8 times and 8.1 times more likely to choose a treatment with stable or improved (vs worse) motor function, respectively. Similarly, participants were 4.3 times and 5.8 times more likely to choose stable or improved (vs worse) breathing function, respectively. Treatments with a risk of liver injury, fatigue, headache and nausea were 1.6 times less likely to be chosen than treatments with a risk of diarrhoea and rash. Treatments with demonstrated effectiveness in Type 1 SMA only were 2.3 times less likely to be chosen than those with demonstrated effectiveness in Types 1–3 SMA. Treatments administered via intrathecal injections were also 1.8 times less likely to be chosen than daily oral treatments. Discussion: Study results show the importance of improvement as well as stabilisation of motor and breathing function to patients and caregivers, and a preference for oral treatments, treatments with demonstrated effectiveness in Types 2–3 SMA, and avoidance of liver injury risk. [ABSTRACT FROM AUTHOR]

Background: Atypical hemolytic uremic syndrome is a rare disease caused by complement dysregulation that can lead to progressive kidney damage or death if untreated. Owing to its rarity, the impact of atypical hemolytic uremic syndrome and available therapies (eculizumab and ravulizumab) on patients' health-related quality of life is difficult to describe, but such data are required for an economic evaluation.Objective: The objective of this study was to estimate utility values for atypical hemolytic uremic syndrome-related attributes in five countries for an economic evaluation.Methods: Using discrete choice experiment surveys, key atypical hemolytic uremic syndrome-related attributes (life expectancy, administration frequency, risk of meningitis, need for hospitalization, and risk of kidney impairment) were evaluated in adult general population samples from Australia, Canada, the Netherlands, Sweden, and the UK. Survey choice sets were constructed using a published orthogonal array. A mixed-effects logit model estimated preference strength for each attribute. Utilities were estimated using marginal substitution rates between overall survival and other attributes, weighted against average life expectancy.Results: Across all countries (N = 2382), utility weights revealed a consistent pattern: participants were averse to the risk of kidney impairment (disutility/utility weight range: -0.185 to -0.158), risk of meningitis (-0.041 to -0.032), and the need for hospitalization (-0.063 to -0.048), but preferred 8-weekly vs 2-weekly infusions over 1 h (0.013-0.039).Conclusions: Although all attributes played a role in determining treatment preferences, the largest drivers were life expectancy and risk of kidney impairment. Participants favored 8-weekly dosing (corresponding to ravulizumab administration frequency) vs 2-weekly dosing. The discrete choice experiment was designed such that estimated (dis)utility weights can be used in future cost-effectiveness models in atypical hemolytic uremic syndrome. [ABSTRACT FROM AUTHOR]

Introduction: Various treatment options are available for metastatic hormone-sensitive prostate cancer. This study aimed to quantify how men with prostate cancer in the United Kingdom (UK), Germany, and Spain perceive the risks and benefits of hypothetical abiraterone acetate plus prednisone treatment and docetaxel-based chemotherapy treatment options.Methods: A targeted literature review, exploratory interviews with prostate cancer patients and oncologists, and pre-test interviews were used to develop a discrete choice experiments (DCE). The final DCE included 32 choice sets, selected using a main-effects orthogonal design, divided into two survey blocks. Paired profiles presented hypothetical treatments for prostate cancer through six attributes that could be presented at two or four levels each. Preference estimates were estimated using a conditional logit regression model. Preference results were stratified by cancer stage.Results: A total of 152 participants (mean age 69 years) completed the DCE in the UK, Germany, and Spain. Treatment effectiveness was the main concern for the patients (difference in preference estimates between 8 and 32 months 1.443). Participants wanted to avoid pain that was not well controlled (preference dummy coding estimate - 1.157). Participants valued a change from an oral medication to an intravenous treatment (change in preference estimate - 0.416) more negatively than a change from a 1% to a 5% risk of infection (change in preference estimate - 0.313).Conclusions: This study shows that treatment effectiveness and pain control were the most important attributes for patients with prostate cancer. These two attributes influenced more than 50% of their decision-making in this study. The risk of fatigue and mode of administration were least prioritised by patients. This study highlights the relative importance that Spanish, German, and British patients place on various aspects of treatment options for prostate cancer. Understanding patient preference and taking them into consideration shall help physicians when developing their treatment strategies for their patients.Funding: Janssen. [ABSTRACT FROM AUTHOR]

Purpose: Long-term levodopa therapy and related fluctuating plasma concentrations are associated with between-dose periods of 'off time' resulting in substantial variation in symptoms and functioning throughout the day in people with Parkinson's (PwP).Methods: PwP across UK, France, Spain and Italy completed an online survey to explore: the impact of 'off time' on (1) health-related quality of life (HRQL) and (2) on functioning and ability to undertake usual activities; (3) the value of 'off time' relative to other factors associated with Parkinson's through a stated preference discrete choice experiment (SPDCE).Results: In total, 305 PwP completed the online survey. Overall mean HRQL (utility) score was significantly lower for 'off time' (0.37) than for 'on time' (0.60). All attributes within the SPDCE were significant predictors of treatment choice, although increased duration of 'on time' (per hour per day: odds ratio (OR) = 1.40) and predictability of 'off time' to within 30 min (OR = 1.42) were valued most highly.Conclusions: 'On time' and predictability of 'off time' are highly valued by PwP. Due to substantial diurnal variation of Parkinson's symptoms, standard patient-reported outcome (PRO) assessments may not adequately capture the impact of 'off time' on HRQL and participation in daily activities. [ABSTRACT FROM AUTHOR]