IntroductionPulmonary arterial hypertension (PAH) is a rare, incurable disease associated with decreased life expectancy and a marked impact on quality of life (QoL). There are three classes of drugs available for treatment: endothelin receptor antagonists (ERA), drugs acting on nitric oxide pathway (riociguat and phosphodiesterase type 5 inhibitors [PDE5i]), and drugs acting on prostacyclin pathway. The latter have widely different modes of administration – continuous intravenous infusion, continuous subcutaneous infusion, inhaled, and oral – each associated with variable treatment burden, and implications for health economic assessment. This study aimed to establish utility values associated with different modes of administration of drugs acting on the prostacyclin pathway for use in economic evaluations of PAH treatments.MethodsA UK general public sample completed the EQ-5D-5L and valued four health states in time trade-off interviews. The health states drafted from literature and interviews with PAH experts (n=3) contained identical descriptions of PAH and ERA/PDE5i treatment, but differed in description of administration including oral (tablets), inhaled (nebulizer), continuous subcutaneous infusion, and continuous intravenous infusion.ResultsA total of 150 participants (63% female; mean age 37 years) completed interviews. Utilities are presented as values between 0 and 1, with 0 representing the state of being dead and 1 representing being in full health. The mean (SD) utility for oral health state was 0.85 (0.16), while all other health states were significantly lower at 0.74 (0.27) for inhaled (p=0.001), 0.59 (0.31) for subcutaneous (p<0.001) and 0.54 (0.32) for intravenous (p<0.001), indicating that there are disutilities (negative differences) associated with non-oral health states. Disutilities were −0.11 for inhaled, −0.26 for subcutaneous, and −0.31 for intravenous administration.ConclusionThe results demonstrate quantifiable QoL differences between modes of administration of drugs acting on the prostacyclin pathway. QoL burden should be considered for economic evaluation of drugs for PAH treatment.
Introduction: With increasing availability of different treatments for chronic obstructive pulmonary disease (COPD), we sought to understand patient preferences for COPD treatment in the UK, USA, and Germany using a discrete choice experiment (DCE). Methods: Qualitative research identified six attributes associated with COPD maintenance treatments: ease of inhaler use, exacerbation frequency, frequency of inhaler use, number of different inhalers used, side effect frequency, and out-of-pocket costs. A DCE using these attributes, with three levels each, was designed and tested through cognitive interviews and piloting. It comprised 18 choice sets, selected using a D-efficient experimental design. Demographics and disease history were collected and the final DCE survey was completed online by participants recruited from panels in the UK, USA and Germany. Responses were analyzed using mixed logit models, with results expressed as odds ratios (ORs). Results: Overall, 450 participants (150 per country) completed the DCE; most (UK and Germany, 97.3%; USA, 98.0%) were included in the final analysis. Based on relative attribute importance, avoidance of side effects was found to be most important (UK: OR 11.65; USA: OR 7.17; Germany: OR 11.45; all p<0.0001), followed by the likelihood of fewer exacerbations (UK: OR 2.22; USA: OR 1.63; Germany: OR 2.54; all p<0.0001) and increased ease of use (UK: OR 1.84; USA: OR 1.84; Germany: OR 1.60; all p<0.0001). Number of inhalers, outof-pocket costs, and frequency of inhaler use were found to be less important. Preferences were relatively consistent across the three countries. All participants required a reduction in exacerbations to accept more frequent inhaler use or use of more inhalers. Conclusion: When selecting COPD treatment, individuals assigned the highest value to the avoidance of side effects, experiencing fewer exacerbations, and ease of inhaler use. Ensuring that patients' preferences are considered may encourage treatment compliance.
Objectives:This study aims to explore the impact of Charcot-Marie-Tooth disease type 1A (CMT1A) and its treatment on patients in European (France, Germany, Italy, Spain, and the United Kingdom) and US real-world practice. Methods:Adults with CMT1A (n ¼ 937) were recruited to an ongoing observational study exploring the impact of CMT. Data were collected via CMT&Me, an app through which participants completed patientreported outcome measures. Results:Symptoms ranked with highest importance were weakness in the extremities, difficulty in walking, and fatigue. Almost half of participants experienced a worsening of symptom severity since diagnosis. Anxiety and depression were each reported by over one-third of participants. Use of rehabilitative interventions, medications, and orthotics/walking aids was high. Conclusions:Patient-reported burden of CMT1A is high, influenced by difficulties in using limbs, fatigue, pain, and impaired quality of life. Burden severity appears to differ across the population, possibly driven by differences in rehabilitative and prescription-based interventions, and country-specific health care variability.
Relational memory is the ability to flexibly organize and integrate multiple sources of information to produce emergent outcomes. In tests for one type of relational memorystimulus equivalence-arbitrary stimuli become related in ways not explicitly trained. Little is known however about whether stimulus equivalence-based relational memory ability differentially emerges during offline periods of either sleep or wake. Here, fifty-one, healthy young adults learned a series of interconnected conditional relations involving arbitrary visual images (A-B, A-C, and AD), and were immediately tested for maintenance of these relations. Following a 12-hour offline period consisting of either sleep or wake, both groups were tested for novel inferences-symmetry (B-A, C-A, and D-A) and equivalence relations (B-C, C-B, C-D, and D-C)-as well as retention of the trained relations. Results from delayed testing, supported by Bayesian statistics, showed that accuracy did not differ between the sleep and wake groups. Potential limitations of this preliminary investigation and directions for future research are discussed.
NICE have published nine HST guidance, all with positive recommendations, a median of 20 months (range 7-38) after European MA. An additional 11 HST guidance are in development with MAs for a median of 14 months (range: 0-53) with six having draft guidance issued, all being "not recommended". Of the 20 HSTs with NICE guidance published/in-development, 16, 14, 8, 6, 2, and 6 were assessed by HAS, G-BA, NCPE, SMC, TLV, and ZIN, respectively. Of these, 22/30 (73%) and 7/22 (32%) of assessments made by clinical-effectiveness and cost-effectiveness HTA bodies received positive outcomes, respectively, with median delays between European MA and positive appraisal outcomes of 7 and 37 months, respectively. Conclusions: Although NICE HST appraisals have more positive recommendations and have faster time to recommendations following European MA than other costeffectiveness HTA bodies, time to positive recommendation is still substantially delayed compared to clinical-effectiveness HTA bodies. In 2018, a new SMC appraisal framework was introduced, whereby ultra-orphan therapies would be made available for $3-years while additional evidence is collected pending a final SMC appraisal. This could potentially prove a more suitable best-practice model.
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