An Overview of Specialist Services for Huntington's Disease in the United Kingdom.

BACKGROUND: Huntington's disease (HD) is a rare inherited neurodegenerative disorder characterized by complex evolving needs that change as the condition progresses. There is limited understanding about the organization of HD clinical services and their resourcing in the United Kingdom (UK). OBJECTIVE: To understand the organization and resourcing of specialist HD services for people with HD (PwHD) in the UKMethods:This cross-sectional study collected quantitative data via on online survey, and qualitative data via telephone semi-structured interviews. Descriptive statistics were used to describe quantitative outcomes, and qualitative results were analyzed using content analysis. RESULTS: A total of 31 specialist services for HD were identified. Of the 27 services that completed the online survey, 23 had an active multidisciplinary team of healthcare professionals (HCPs) and were led primarily by a mental health trust (26%) or tertiary referral hospital (26%). Specialist services offered outpatient clinics (96%), outreach in the community (74%), telemedicine (70%), inpatient beds (26%) and satellite clinics (26%). Many services indicated that their capacity (ability to see patients as often as needed with current resources) was difficult, with some services reporting more difficulty at the early or later stages of HD. Key resourcing gaps were identified with access to facilities, HCPs and referral networks. CONCLUSIONS: This research highlights the variation in organization and capacity within individual HD services as well as current resourcing and gaps in access that influence this capacity. Further research should be done to understand the impact of service organization and current resourcing gaps in access on the quality of care provided for PwHD in the UK.

Need, demand, supply in health care: working definitions, and their implications for defining access.

Effective policymaking in health care systems begins with a clear typology of the terminology - need, demand, supply and access to care - and their interrelationships. However, the terms are contested and their meaning is rarely stated explicitly. This paper offers working definitions of need, demand and supply. We draw on the international literature and use a Venn diagram to explain the terms. We then define access to care, reviewing alternative and competing definitions from the literature. We conclude by discussing potential applications of our conceptual framework to help to understand the interrelationships and trade-offs between need, demand, supply and access in health care.

Humanistic burden of problem joints for children and adults with haemophilia.

INTRODUCTION: The "problem joint" (PJ) concept was developed to address patient-centric needs for a more holistic assessment of joint morbidity for people with haemophilia (PwH). AIM: To quantify the humanistic burden of PJs in PwH to further support validation of the PJ outcome measure. METHODS: Multivariable regression models evaluated the relationship between PJs and health-related quality of life (HRQoL, EQ-5D-5L) and overall work productivity loss (WPL) using data from the 'Cost of HaEmophilia: a Socioeconomic Survey' population studies (adults: CHESS II, CHESS US+; children/adolescents: CHESS-Paeds). Covariates included were haemophilia severity, age, comorbidities and education. RESULTS: The CHESS II sample included 292 and 134 PwH for HRQoL and WPL analyses, mean age 38.6 years (39% ≥1 PJ, 61% none). CHESS US+ included 345 and 239 PwH for HRQoL and WPL, mean age 35 years (43% ≥1 PJ, 57% none). CHESS-Paeds included 198 PwH aged 4-17 (HRQoL only), mean age 11.5 years (19% ≥1 PJ, 81% none). In CHESS II and CHESS US+, presence of PJs was associated with worse HRQoL (Both p < .001). Few CHESS-Paeds participants had PJs, with no significant correlation with HRQoL. In CHESS II, upper body PJs were significantly correlated to WPL (p < .05). In CHESS US+, having ≥1 PJ or upper and lower body PJs were significantly correlated to WPL (vs. none; both p < .05). CONCLUSION: This study has shown a meaningful burden of PJs on PwH, which should be considered in clinical and health policy assessments of joint health.

Economic burden of Huntington disease in Europe and the USA: Results from the Huntington's Disease Burden of Illness study.

BACKGROUND AND PURPOSE: The prevalence of Huntington disease (HD) has increased over time; however, there is a lack of up-to-date evidence documenting the economic burden of HD by disease stage. This study provides an estimate of the annual direct medical, nonmedical, and indirect costs associated with HD from participants in the Huntington's Disease Burden of Illness (HDBOI) study in five European countries and the USA. METHODS: The HDBOI is a retrospective, cross-sectional study. Data collection was conducted between September 2020 and May 2021. Participants were recruited by their HD-treating physicians and categorized as early stage (ES), mid stage (MS), or advanced stage (AS) HD. Data were collected via three questionnaires: a case report form, completed by physicians who collected health care resource use associated with HD to compute direct medical cost, and optional patient and caregiver questionnaires, which included information used to compute nondirect medical and indirect costs. Country-specific unit cost sources were used. RESULTS: HDBOI cost estimates were €12,663 (n = 2094) for direct medical costs, €2984 (n = 359) for nondirect medical costs, and €47,576 (n = 436) for indirect costs. Costs are higher in patients who are at later stages of disease; for example, direct medical costs estimates were €9220 (n = 846), €11,885 (n = 701), and €18,985 (n = 547) for ES, MS, and AS, respectively. Similar trends were observed for nondirect and indirect costs. Costs show large variations between patients and countries. CONCLUSIONS: Cost estimates from the HDBOI study show that people with HD and their caregivers bear a large economic burden that increases as disease progresses.

Humanistic Burden of Huntington Disease: Evidence From the Huntington Disease Burden of Illness Study.

Background and Objectives: Huntington disease (HD) is a rare, inherited, and highly complex neurodegenerative disorder with no currently approved disease-modifying treatments. We investigated the effect of HD on health-related quality of life and other patient-reported outcomes in the Huntington's Disease Burden of Illness (HDBOI) study. Methods: The HDBOI study is a retrospective, cross-sectional study conducted between September 2020 and May 2021 in France, Germany, Italy, Spain, the United Kingdom, and the United States. People with symptomatic onset HD (PwHD) were recruited by their HD-treating physicians and categorized as early (ES), mid (MS), or advanced stage (AS) HD. Physicians provided sociodemographic and clinical information from the participant's medical records in electronic case report forms (eCRF); participants or their proxies completed online Patient Public Involvement Engagement questionnaires (PPIE-P). Patient-reported outcomes included the 5-level EQ-5D version (EQ-5D-5L), Short-Form-(SF)-36 v2 (and SF-6-Dimension [SF-6D] utility), Huntington Quality of Life Instrument (H-QoL-I), and the Work Productivity and Activity Impairment Specific Health Problem. All outcomes were summarized using descriptive statistics, and differences between disease stages were assessed by Kruskal-Wallis tests. Results: A total of 2,094 PwHD were enrolled with completed eCRFs (100%) and PPIE-P forms (n = 482, 23%). Participants' mean age was 47.3 years; they were generally evenly distributed across countries, with the majority being ES (40%) followed by MS (33%) and LS (26%). The mean EQ-5D-5L (n = 336) utility score was 0.59 (SD, 0.27), with the highest mean utility scores [SD] in ES (0.72 [0.22]) followed by MS (0.62 [0.18]) and AS (0.37 [0.30]), p < 0.001. The mean SF-6D score (n = 482) was 0.57 (SD, 0.10), with mean values decreasing with advanced disease (ES, 0.61; MS, 0.56; AS, 0.50, p < 0.001). H-QoL-I mean scores (n = 482) also worsened with more advanced disease, from 0.58 for ES to 0.49 for MS and 0.37 for AS, p < 0.001. Impairment in daily activities and in work productivity also increased with more advanced disease. Overall proxy respondents reported on average worse outcomes than PwHD (self-reported) across all outcomes and disease stages suggesting a possible unawareness of deficits by PwHD. Discussion: The HDBOI study provides new insights into the characteristics and humanistic burden of PwHD and offers a meaningful contribution to this underserved research area.

Differential humanistic and economic burden of mild, moderate and severe haemophilia in european adults: a regression analysis of the CHESS II study.

BACKGROUND: The lifelong nature of haemophilia makes patient-centred and societal assessments of its impact important to clinical and policy decisions. Quantifying the humanistic and economic burden by severity is key to assessing the impact on healthcare systems. We analysed the annual direct medical (excluding factor replacement therapy costs) and non-medical costs as well as societal costs and health-related quality of life (HRQoL) of mild, moderate and severe disease among adults with haemophilia A or B without inhibitors in Europe. Participants in the CHESS II study reported their HRQoL, non-medical costs, and work impairment; physicians provided costs and consultation history from the medical chart. Descriptive statistics summarized patient characteristics, costs, and HRQoL scores. Regression models estimated differences in outcomes for moderate and severe versus mild disease, adjusting for age, body mass index, country, comorbidities, weight-adjusted factor consumption and education. RESULTS: The analytic sample included 707 patients with a mean age of 38 years; the majority of patients had haemophilia A (81%), and 47% had severe disease, followed by moderate (37%) and mild disease (16%). Patients with severe or moderate disease had on average higher direct costs, €3105 and €2469 respectively, versus mild disease. Societal costs were higher for patients with severe and moderate disease by €11,115 and €2825, respectively (all P < 0.01). HRQoL scores were also significantly worse for severe and moderate patients versus those with mild disease. CONCLUSION: Severity of haemophilia is predictive of increasing economic and humanistic burden. The burden of moderate disease, as measured by direct costs and HRQoL, did not appear to be substantially different than that observed among patients with severe haemophilia.

Health-related quality of life, direct medical and societal costs among children with moderate or severe haemophilia in Europe: multivariable models of the CHESS-PAEDs study.

BACKGROUND: Haemophilia bears substantial humanistic and economic burden on children and their caregivers. Characterising the differential impact of severe versus moderate paediatric haemophilia is important for clinical and health policy decisions. We analysed health-related quality of life (HRQoL), annual direct medical (excluding factor treatment costs), non-medical and societal costs among children and adolescents with moderate and severe haemophilia A or B without inhibitors from the European CHESS-PAEDs study. Information was reported by physicians and caregivers; patients aged ≥ 8 years self-reported their HRQoL. Descriptive statistics summarised demographic and clinical characteristics, costs, and HRQoL scores (EQ-5D-Y). Regression models estimated differences in HRQoL and costs for moderate versus severe haemophilia adjusting for age, body mass index z-score, country, number of comorbidities, and weight-adjusted annual clotting factor consumption. RESULTS: The analytic sample comprised 794 patients with a mean age of 10.5 years; most had haemophilia A (79%) and 58% had severe haemophilia. Mean predicted direct medical costs in moderate patients were two-thirds of the predicted costs for severe disease (€3065 vs. €2047; p < 0.001; N = 794), while societal costs were more than half of the predicted costs for children with severe haemophilia (€6950 vs. €3666; p < 0.001; N = 220). Mean predicted HRQoL scores were 0.74 and 0.69 for moderate and severe disease, respectively (p < 0.05; N = 185). CONCLUSION: Children with haemophilia and their caregivers displayed a significant economic and humanistic burden. While severe patients showed the highest direct medical and societal costs, and worse HRQoL, the burden of moderate haemophilia on its own was substantial and far from negligible.

Becoming a resident in a high demanded medical specialty: an unequal race? Evidence from the Spanish resident market.

BACKGROUND: Gender occupational segregation in medicine is associated with several undesired consequences such as earnings disparity, shortages of specialists or lower quality of care among others. This paper focuses on the persistent gender gap observed in the most popular specialties of the Spanish resident market. In particular, it explores the role of the specialty allocation system in perpetuating the occupational segregation. For that purpose, this paper studies the effect of a policy change in the ranking system that determines doctors' specialty choice order. The change increased the competitiveness of the process by increasing the weight of an entry examination from 75% to 90%, in detriment of doctors' grade point average that decreased from 25% to 10%. Findings from previous literature suggest that that male and female doctors might have reacted differently to the increased competitiveness of the process. METHODS: Data come from administrative records of doctors' specialty choices for the years 2013 and 2015 and they are used to compute the difference between doctors' pre and post-change ranking positions. Then, differences in the distribution of rank differences between male and female doctors are tested by means of parametric (T-test) and non-parametric (Wilcoxon rank) approaches. RESULTS: Results show that the policy change has overall favoured male doctors. On average, female doctors lose ranking positions, with respect to the position they would have achieved with the old weights, whilst male doctors gain positions. The differences are more pronounced in the top half of the ranking distribution, meaning that female doctors on average have reduced their probability of accessing the most demanded specialties. CONCLUSIONS: The objective of the policy was the enhancement of the prospects of Spanish-graduate doctors with respect to international graduates by giving more weight to the less prone to bias examination scores. Nonetheless, the change have had the unintended consequence of reducing the probability of female doctors accessing highly demanded specialties and thus exacerbating the gender gap. The allocation system needs revision to make it accountable for the actual role of doctors in society.

The impact of moving to a 12h shift pattern on employee wellbeing: A qualitative study in an acute mental health setting.

BACKGROUND: Against a backdrop of increasing demand for mental health services, and difficulties in recruitment and retention of mental health staff, employers may consider implementation of 12 h shifts to reduce wage costs. Mixed evidence regarding the impact of 12 h shifts may arise because research is conducted in divergent contexts. Much existing research is cross sectional in design and evaluates impact during the honeymoon phase of implementation. Previous research has not examined the impact of 12 h shifts in mental health service settings. OBJECTIVE: To evaluate how employees in acute mental health settings adapt and respond to a new 12 h shift system from a wellbeing perspective. DESIGN: A qualitative approach was adopted to enable analysis of subjective employee experiences of changes to organisation contextual features arising from the shift pattern change, and to explore how this shapes wellbeing. SETTING(S): Six acute mental health wards in the same geographical area of a large mental health care provider within the National Health Service in England. PARTICIPANTS: 70 participants including modern matrons, ward managers, clinical leads, staff nurses and healthcare assistants. METHODS: Semi-structured interviews with 35 participants at 6 months post-implementation of a new 12 h shift pattern, with a further 35 interviewed at 12 months post-implementation. RESULTS: Thematic analysis identified unintended consequences of 12 h shifts as these patterns changed roles and the delivery of care, diminishing perceptions of quality of patient care, opportunities for social support, with reports of pacing work to preserve emotional and physical stamina. These features were moderated by older age, commitment to the public healthcare sector, and fit to individual circumstances in the non-work domain leading to divergent work-life balance outcomes. CONCLUSIONS: Findings indicate potential exists for differential wellbeing outcomes of a 12 h shift pattern and negative effects are exacerbated in a stressful and dynamic acute mental health ward context. In a tight labour market with an ageing workforce, employee flexibility and choice are key to retention and wellbeing. Compulsory 12 h shift patterns should be avoided in this setting.

Trends in and drivers of healthcare expenditure in the English NHS: a retrospective analysis.

BACKGROUND: In England, rises in healthcare expenditure consistently outpace growth in both GDP and total public expenditure. To ensure the National Health Service (NHS) remains financially sustainable, relevant data on healthcare expenditure are needed to inform decisions about which services should be delivered, by whom and in which settings. METHODS: We analyse routine data on NHS expenditure in England over 9 years (2008/09 to 2016/17). To quantify the relative contribution of the different care settings to overall healthcare expenditure, we analyse trends in 14 healthcare settings under three broad categories: Hospital Based Care (HBC), Diagnostics and Therapeutics (D&T) and Community Care (CC). We exclude primary care and community mental health services settings due to a lack of consistent data. We employ a set of indices to aggregate diverse outputs and to disentangle growth in healthcare expenditure that is driven by activity from that due to cost pressures. We identify potential drivers of the observed trends from published studies. RESULTS: Over the 9-year study period, combined NHS expenditure on HBC, D&T and CC rose by 50.2%. Expenditure on HBC rose by 54.1%, corresponding to increases in both activity (29.2%) and cost (15.7%). Rises in expenditure in inpatient (38.5%), outpatient (57.2%), and A&E (59.5%) settings were driven predominately by higher activity. Emergency admissions rose for both short-stay (45.6%) and long-stay cases (26.2%). There was a switch away from inpatient elective care (which fell by 5.1%) and towards day case care (34.8% rise), likely reflecting financial incentives for same-day discharges. Growth in expenditure on D&T (155.2%) was driven by rises in the volume of high cost drugs (270.5%) and chemotherapy (110.2%). Community prescribing grew by 45.2%, with costs falling by 24.4%. Evidence on the relationship between new technologies and healthcare expenditure is mixed, but the fall in drug costs could reflect low generic prices, and the use of health technology assessment or commercial arrangements to inform pricing of new medicines. CONCLUSIONS: Aggregate trends in HCE mask enormous variation across healthcare settings. Understanding variation in activity and cost across settings is an important initial step towards ensuring the long-term sustainability of the NHS.

The impact of extending nurse working hours on staff sickness absence: Evidence from a large mental health hospital in England.

BACKGROUND: A pressing international concern is the issue of mental health workforce capacity, which is also of concern in England where staff attrition rates are significantly higher than in physical health services. Increasing demand for mental health services has led to severe financial pressures resulting in staff shortages, increased workloads, and work-related stress, with health care providers testing new models of care to reduce cost. Previous evidence suggests shift work can negatively affect health and wellbeing (increased accidents, fatigue, absenteeism) but can be perceived as beneficial by both employers and employees (fewer handovers, less overtime, cost savings). OBJECTIVE: This study reports an evaluation of the impact of extending the shifts of nurses and health care assistants from 8 to 12 hours. Using data before and after the policy change, the effect of extended working hours on short term sickness (< 7 days) on staff is examined. SETTING: The setting is six inpatient wards within a large mental health hospital in England where the shift extension took place between June and October 2017. The Data come from wards administrative records and the analysis is performed using weekly data (N=463). METHODS: Causal inference methods (Interrupted Time Series and Difference-in-Difference) are used to compare staff sickness rates before and after the implementation, where the outcome variable is defined as the ratio of total sickness hours over the total scheduled working hours (full time equivalents) in a given week. Patient casemix, staff demographics, ward and time variables are included as controls. RESULTS: Estimation results establish that the extended shifts are associated with an increased percentage of sickness hours per week of between 0.73% and 0.98%, the equivalent of a complete shift per week per ward. CONCLUSION: This is the first study to use causal inference to measure the impact of longer shifts on sickness absences for mental health workforce. The analysis is relevant to other providers which may increasingly look towards these shift patterns as a means of cost saving.

Getting the right balance? A mixed logit analysis of the relationship between UK training doctors' characteristics and their specialties using the 2013 National Training Survey.

OBJECTIVE: To analyse how training doctors' demographic and socioeconomic characteristics vary according to the specialty that they are training for. DESIGN: Descriptive statistics and mixed logistic regression analysis of cross-sectional survey data to quantify evidence of systematic relationships between doctors' characteristics and their specialty. SETTING: Doctors in training in the United Kingdom in 2013. PARTICIPANTS: 27 530 doctors in training but not in their foundation year who responded to the National Training Survey 2013. MAIN OUTCOME MEASURES: Mixed logit regression estimates and the corresponding odds ratios (calculated separately for all doctors in training and a subsample comprising those educated in the UK), relating gender, age, ethnicity, place of studies, socioeconomic background and parental education to the probability of training for a particular specialty. RESULTS: Being female and being white British increase the chances of being in general practice with respect to any other specialty, while coming from a better-off socioeconomic background and having parents with tertiary education have the opposite effect. Mixed results are found for age and place of studies. For example, the difference between men and women is greatest for surgical specialties for which a man is 12.121 times more likely to be training to a surgical specialty (relative to general practice) than a woman (p-value<0.01). Doctors who attended an independent school which is proxy for doctor's socioeconomic background are 1.789 and 1.413 times more likely to be training for surgical or medical specialties (relative to general practice) than those who attended a state school (p-value<0.01). CONCLUSIONS: There are systematic and substantial differences between specialties in respect of training doctors' gender, ethnicity, age and socioeconomic background. The persistent underrepresentation in some specialties of women, minority ethnic groups and of those coming from disadvantaged backgrounds will impact on the representativeness of the profession into the future. Further research is needed to understand how the processes of selection and the self-selection of applicants into specialties gives rise to these observed differences.