Charity financial support to motor neuron disease (MND) patients in Greater London: the impact of patients' socioeconomic status-a cross-sectional study.

OBJECTIVE: There is an immense socioeconomic burden for both the patients with motor neuron disease (MND) and their families. The aim of this study is to evaluate the extent to which the provision offered by the Motor Neurone Disease Association is distributed among patients with MND living in the ethnically and socially diverse area of Greater London, according to the patients' socioeconomic situation and needs. SETTING: Greater London, where age and sex-adjusted prevalence rates of MND in 2016 were calculated. PARTICIPANTS: Prevalent MND cases in Greater London, using anonymised data extracted from the Association's database. EXPOSURE: Demographic and socioeconomic characteristics PRIMARY AND SECONDARY OUTCOME MEASURES: Receiving a Motor Neurone Disease Association grant, and the amount of money received. RESULTS: 396 individuals with amyotrophic lateral sclerosis were detected, the age-specific and sex-specific prevalence of MND was 4.02 per 100 000 inhabitants, higher among men (5.13 per 100 000) than women (3.01 per 100 000). Men were statistically significantly 40% less likely to receive a grant compared with women; among grant recipients, the younger the age of the participant, the higher the size of the grant received. The Index of Multiple Deprivation was not associated with either receiving a grant nor the amount of money received, among recipients. CONCLUSION: Financial support by the Motor Neurone Disease Association is provided across individuals and across boroughs regardless of their socioeconomic circumstances. Differences that benefits women and younger patients were detected.

Supportive & palliative interventions in motor neurone disease: what we know from current literature?

Although there is no cure for motor neurone disease (MND), the advent of supportive interventions including multidisciplinary care (MDC) has improved treatment interventions and enhanced quality of life (QOL) for MND patients and their carers. Our integrative review showed evidence-based MDC, respiratory management and disease-modifying therapy that have improved the outcomes of patients diagnosed with MND. Supportive approaches to nutritional maintenance and optimization of symptomatic treatments, including management of communication and neuropsychiatric issues, improve the QOL for MND patients. Notwithstanding improvement to care and QOL, survival benefit has become evident with the advent of a MDC framework, early treatment with non-invasive ventilation (NIV). In addition, weight maintenance remains critical, as weight loss is associated with more rapid disease progression. The endof- life phase is poorly defined in MND patients and treatment remains challenging, yet effective symptom control through palliative care (PC) is achievable and essential.

The social and economic burden of frontotemporal degeneration.

OBJECTIVE: To quantify the socioeconomic burden of frontotemporal degeneration (FTD) compared to previously published data for Alzheimer disease (AD). METHODS: A 250-item internet survey was administered to primary caregivers of patients with behavioral-variant FTD (bvFTD), primary progressive aphasia, FTD with motor neuron disease, corticobasal syndrome, or progressive supranuclear palsy. The survey included validated scales for disease staging, behavior, activities of daily living, caregiver burden, and health economics, as well as investigator-designed questions to capture patient and caregiver experience with FTD. RESULTS: The entire survey was completed by 674 of 956 respondents (70.5%). Direct costs (2016 US dollars) equaled $47,916 and indirect costs $71,737, for a total annual per-patient cost of $119,654, nearly 2 times higher than reported costs for AD. Patients ≥65 years of age, with later stages of disease, and with bvFTD correlated with higher direct costs, while patients <65 years of age and men were associated with higher indirect costs. An FTD diagnosis produced a mean decrease in household income from $75,000 to $99,000 12 months before diagnosis to $50,000 to $59,999 12 months after diagnosis, resulting from lost days of work and early departure from the workforce. CONCLUSIONS: The economic burden of FTD is substantial. Counting productivity-related costs, per-patient costs for FTD appear to be greater than per-patient costs reported for AD. There is a need for biomarkers for accurate and timely diagnosis, effective treatments, and services to reduce this socioeconomic burden.

Subcutaneous immunoglobulin treatment in CIDP and MMN. Efficacy, treatment satisfaction and costs.

Subcutaneous administration of immunoglobulin (SCIG) in chronic inflammatory demyelinating polyneuropathy (CIDP) and multifocal motor neuropathy (MMN) has been reported in several case reports and in a few randomized trials during the last decade. In this review we present the studies on SCIG in CIDP and MMN with special focus on the clinical effects. Moreover, the effect on quality of life, side effects to SCIG and the health economic perspectives are reviewed. Nine case studies, three randomized trials and six long-term, follow-up studies were identified. Most of the studies are conducted in patients switched from regular IVIG to SCIG treatment; one study involves treatment-naïve patients. The review shows that none of the studies have been powered to demonstrate an effect on disability. SCIG can maintain muscle strength for a period of 1 to 2years and ability seems preserved for a similar period. Quality of life is generally unchanged or improved after switch to SCIG and generalized side-effects seem fewer, whereas local reactions at the injection site occur. Health economic analyses favour SCIG at the doses used in the reviewed studies.

Economic Studies in Motor Neurone Disease: A Systematic Methodological Review.

BACKGROUND: Motor neurone disease (MND) is a devastating condition which greatly diminishes patients' quality of life and limits life expectancy. Health technology appraisals of future interventions in MND need robust data on costs and utilities. Existing economic evaluations have been noted to be limited and fraught with challenges. OBJECTIVE: The aim of this study was to identify and critique methodological aspects of all published economic evaluations, cost studies, and utility studies in MND. METHODS: We systematically reviewed all relevant published studies in English from 1946 until January 2016, searching the databases of Medline, EMBASE, Econlit, NHS Economic Evaluation Database (NHS EED) and the Health Economics Evaluation Database (HEED). Key data were extracted and synthesised narratively. RESULTS: A total of 1830 articles were identified, of which 15 economic evaluations, 23 cost and 3 utility studies were included. Most economic studies focused on riluzole (n = 9). Six studies modelled the progressive decline in motor function using a Markov design but did not include mutually exclusive health states. Cost estimates for a number of evaluations were based on expert opinion and were hampered by high variability and location-specific characteristics. Few cost studies reported disease-stage-specific costs (n = 3) or fully captured indirect costs. Utilities in three studies of MND patients used the EuroQol EQ-5D questionnaire or standard gamble, but included potentially unrepresentative cohorts and did not consider any health impacts on caregivers. CONCLUSION: Economic evaluations in MND suffer from significant methodological issues such as a lack of data, uncertainty with the disease course and use of inappropriate modelling framework. Limitations may be addressed through the collection of detailed and representative data from large cohorts of patients.

£2.5m for specialist nurses in Scotland.

An online campaign has helped secure an additional £2.5 million for specialist nurses in Scotland and double the number of nurses caring for patients with motor neurone disease.

Diagnostic yield and cost-effectiveness of investigations in patients presenting with isolated lower motor neuron signs.

Our objective was to investigate the yield and cost-effectiveness of investigations and therapeutic trials of intravenous immunoglobulin (IVIg) in patients presenting with isolated lower motor neuron (LMN) signs. We performed a retrospective chart review of cases diagnosed between January 2007 and September 2013. Investigation results and their impact on outcome, and outcome of IVIg treatment trials were abstracted. Cost was calculated in Canadian dollars (C$). Fifty-nine of 333 patients presented with isolated LMN signs. The majority of patients (61%) evolved to amyotrophic lateral sclerosis (ALS) within 36 months of presentation, while 37.3% remained with progressive muscular atrophy (PMA) with mean follow-up 29.6 months. Of the 1210 tests performed, 4.9% were abnormal. The diagnosis was changed in only one patient where a muscle biopsy revealed a distal myopathy. Fourteen patients received therapeutic trials of IVIg to rule out an IVIg-responsive inflammatory motor neuropathy with no objective clinical benefit. Total group cost was C$630,484.72 (C$10,686.18/patient). IVIg represented 58.7% of total costs. In conclusion, extensive investigations and treatment trials of IVIg have low yield in the work-up of patients with isolated LMN signs and are not cost-effective when clinical features do not suggest an alternative diagnosis to PMA.

Epidemiology and medical expenses of motor neuron diseases in Taiwan.

BACKGROUND: The purpose of the study is to investigate the epidemiology and associated medical expenses of motor neuron diseases (MND) in Taiwan. METHODS: Data from the National Health Insurance Research Database, involving diagnoses of MND and Certificates of Serious Accidents and Diseases, were retrieved from January 2000 to December 2005. Age- and sex-specific incidences of MND were estimated by dividing the incidence number by population data obtained from the Department of Statistics, Ministry of the Interior. RESULTS: In total, 1,554 patients with MND were identified. The average yearly incidence was 1.05 per 100,000 people. The male to female ratio was 1.51. The incidence rate, which was 1.25 in men and 0.84 in women, tended to increase with age, and peaked in the ranges of 65-69 years in men and 70-74 years in women. The expenses of the medical services associated with MND increased year by year, except for a sudden drop in inpatient expenses in 2005. CONCLUSIONS: The incidence of MND in Taiwan was 1.05 per 100,000/year, which was lower than that of Caucasians. The incidence had moderate male predominance and increased with age. With an aging population, case numbers and medical expenses associated with these diseases are expected to rise in future decades.

Effects of the early diagnosis of amyotrophic lateral sclerosis on the patient: disadvantages.

While the early diagnosis of disease is generally desirable, this is not always so. There is reason to be concerned about the reliability of clinical diagnosis of amyotrophic lateral sclerosis (ALS), based on currently available methods, early in the disease process. Applying the standard of cost-effectiveness it is possible to estimate the effort required to extend the lifespan of persons who are detected through a screening programme. Until it is possible to alter the treatment outcome, patients have little to gain by the early detection of ALS.

The role of the amyotrophic lateral sclerosis and motor neurone disease community in health-care resourcing.

The Amyotrophic Lateral Sclerosis and Motor Neurone Disease (ALS/MND) Associations operate in the same financially constrained environment as everyone else. Increasing competition for limited health-care resources means that we are having to become better at competing for them on behalf of people affected by ALS/MND. This paper highlights the problems associated with making a case for ALS/MND, explores a range of method available to us, and illustrates using examples the recent activities undertaken by the UK MND Association.

Support that prolongs life. Ventilation in motor neurone disease.

In several countries, including the US, active management of MND is practised using ventilatory support to manage respiratory insufficiency. Respiratory failure should be anticipated, so that rational and informed choices for survival can be made by patient, family and healthcare team. The choice to withdraw from life support should be available. Some productivity and quality of life can be maintained in the most disabled patients.

Ethics and the clinician: the daily experience with motor neurone disease.

Ethical issues in health care are typically perceived as arising from extreme situations which do not usually confront the average clinician. However, knowingly or otherwise, clinicians working with motor neurone disease deal daily with ethical issues in the form of value judgements, the application of choice limiting principles and the language of clinician-patient interaction.

Research and the associations: an era for partnership.

A growing volume of research is being conducted into amyotrophic lateral sclerosis/motor neurone disease, driven in part by a perceived rise in incidence of the disease. This increase in research coincides with the emergence of the voluntary associations. Originally critical of the lack of research, the associations have started to raise funds for a variety of studies. Many of these require the active involvement of patients, and, given the relatively small numbers, there are dangers of the associations, research and good clinical care becoming too closely interdependent. There are also dangers of pharmaceutical companies and the voluntary associations being put under pressure to produce results extremely quickly. As well as having a valuable part to play in research, the voluntary associations are also involved in patient education, and in trying to spread the word on which available options are the subject of 'informed choice'.