Adult ADHD impairs HRQoL, work productivity, and regular daily activities.

'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.

Chronic lymphocytic leukemia (CLL) is a largely incurable disease which affects patients' health related quality of life (HRQL). Treatment is often initiated when symptoms affect HRQL, and patients can experience many rounds of treatment throughout their life. Therefore, the economic burden of CLL can be high. Utility or preference weights for health states reflect the value of HRQL of a given health state and range from 1 (full health) to 0 (dead) and below (negative values possible). Nine health states were developed representing different CLL treatment lines or disease stages. One hundred members of the UK general public valued each health state using the time trade-off methodology. Progression-free survival (PFS) without therapy (mean utility = 0.82) was the least burdensome, with relapsed lines of treatment (mean utility = 0.42) representing the greatest burden. The results underline the value in maintaining a state of PFS for as long as possible.

Purpose Neovascular age-related macular degeneration (nAMD) and diabetic macular edema (DME) patients treated with intravitreally injected anti-vascular endothelial growth factor (anti-VEGF) monotherapies achieve lower vision improvements compared with patients in clinical trials. This qualitative research study aimed to better understand the real-world anti-VEGF treatment experience from nAMD and DME patients’, caregivers’, and retina specialists’ perspectives. Methods One-time, semi-structured, individual interviews were conducted with adult patients with nAMD or DME treated with anti-VEGF injections for ≥12 months, their caregivers, and experienced retina specialists. Interview transcripts were analyzed qualitatively using a thematic analysis approach. Results A total of 49 nAMD and 46 DME patients, 47 nAMD and 33 DME caregivers, and 62 retina specialists were interviewed in the USA, Canada, France, Germany, Italy and Spain. Most (79%) patients and caregivers reported disruptions to their routine on the day before, the day of, or the day after anti-VEGF injection. Seven nAMD patients (14%) and 14 DME patients (30%) reported having missed an injection visit. The most frequently reported driver for adherence for patients was the doctor–patient relationship (n=66, 70%), whereas for caregivers, it was the ease of booking an appointment (n=25, 32%). Retina specialists reported patient education on the treatment (n=28, 45%) as the most important driver. Treatment barriers could be grouped into four categories: tolerability, clinical factors, logistical parameters and human factors. The most frequently reported barrier to adherence for patients and caregivers was related to side effects (pain/discomfort/irritation: n=63, 67% of patients; n=52, 66% of caregivers), whereas for retina specialists it was logistical parameters (travel logistics: n=44, 71%). Conclusion This study highlights the importance of the doctor–patient relationship and patient education as key drivers, and treatment tolerability and logistics as key barriers to treatment adherence. Improved doctor–patient relationship/communication and patient education together with new therapies offering convenience, long-acting effectiveness, and better tolerability may improve treatment adherence.

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.

Background There is considerable burden of illness in hereditary angioedema (HAE). However, instruments to assess health-related quality of life (HRQoL) in HAE are limited. The Angioedema Quality of Life Questionnaire (AE-QoL) was developed to measure HRQoL in patients with recurrent angioedema; the validity of the AE-QoL in patients with HAE is described. Methods To identify disease-related experiences with a focus on the impact of HAE on HRQoL, interviews were conducted with a group of clinician experts and patients with HAE from Canada, France, Germany, Spain, the United Kingdom, and the United States, along with a targeted literature review. Concepts were mapped to the AE-QoL to assess item relevance, interpretation, and conceptual coverage. Cognitive interviews assessed item clarity and relevance. A psychometric validation was performed using data from a phase 3 trial. Results Interviews were conducted with seven clinicians and 40 adult patients. Patients reported 35 unique impacts of HAE on their lives, the most frequent being on work/school, social relationships, physical activities, and emotions, particularly fear/worrying and anxiety. Saturation for these impacts was reached, and all concepts covered in the AE-QoL were reported during the interviews. Patients agreed that the questionnaire items and response options were clear and relevant, and the 4-week recall period was appropriate. The psychometric validation included data from 64 patients. For AE-QoL total scores, excellent internal consistency (Cronbach’s alpha > 0.90), test-retest reliability (intraclass coefficient > 0.80), convergent validity with the Sheehan Disability Scale (r = 0.663), divergent validity with the EQ-5D-5L index (r = 0.292) and EQ-VAS (r = 0.337), and known-groups validity (p < 0.0001; ɳ2 = 0.56) were demonstrated. Conclusions Qualitative and psychometric analyses showed that the AE-QoL is a reliable and valid instrument for measuring HRQoL in adult patients with HAE from six countries.

Increased severity and frequency of aPPHG episodes in patients with insulin-treated diabetes is associated with greater burden and experience of symptoms, and can negatively impact daily functioning.

IntroductionEuropean data on health-related quality of life (HRQoL) in adults with attention deficit/hyperactivity disorder (ADHD) in the general population is sparse.Aims and objectivesTo report HRQoL in UK adults with ADHD.MethodsUK residents aged 18–55 years with a diagnosis of adult ADHD completed an online, cross-sectional survey including questions on disease history, the EuroQol Five Dimensions questionnaire with five-levels (EQ-5D-5L) and the Work productivity and activity impairment questionnaire: general health (WPAI:GH). ADHD symptom severity was assessed by telephone using ADHD rating scale version IV with adult prompts (ADHD-RS-IV).ResultsThe survey was completed by 233 participants (65.2% women; 77.3% white British), mean age 32.6 years (standard deviation [SD] 9.5), mean ADHD-RS-IV total score 43.46 (SD 7.88). Their mean EQ-5D-5L utility score of 0.74 (SD 0.21) was lower than the UK population norm of 0.86 (SD 0.23).[1] WPAI:GH scores indicated that health problems resulted in impairments of 32.04% in work productivity and 45.79% in regular daily activities. Regression analyses adjusting for gender, age and comorbidities demonstrated associations between EQ-5D-5L utility scores and gender (men had lower scores, P < 0.001), work impairment due to health problems (increasing impairment was associated with lower scores, P = 0.005) and age (for each additional year of age, scores decreased by 0.007, P = 0.010).ConclusionsThese results highlight the impact on health utility, work productivity and regular daily activities, and add to the description of the disease burden of adult ADHD in the UK.This study was funded by Shire Development LLC.Disclosure of interestKings College London research support account for Asherson received honoraria for consultancy to Shire, Eli-Lilly and Novartis educational/research awards from Shire, Lilly, Novartis, Vifor Pharma, GW Pharma and QbTech speaker at sponsored events for Shire, Lilly and Novartis.