Assessing the burden of illness from cervical dystonia using the Toronto Western Spasmodic Torticollis Rating Scale scores and health utility: a meta-analysis of baseline patient-level clinical trial data.

PURPOSE: This study aimed to explore the burden of illness associated with cervical dystonia (CD), including possible demographic and humanistic correlates of baseline disease severity. METHODS: The analysis involved the five multinational randomized, placebo-controlled clinical trials that had evaluated the efficacy and safety of Dysport® in patients with CD, including assessment using the Toronto Western Spasmodic Torticollis Rating Scale (TWSTRS). Patient-level TWSTRS scores from the individual studies were meta-analysed to estimate disease severity at baseline. One of the studies had reported Short Form-36 (SF-36) Health Survey quality-of-life measures, and these data were used to investigate whether the severity of CD was associated with humanistic outcomes, as measured by health utility. A generalized regression model was then applied to explore potential correlation between TWSTRS scores and utilities. RESULTS: The estimated pooled mean baseline severity of CD in clinical trial entrants, as measured by TWSTRS score, was 43.23 (95% CI = 39.31-47.15). In general, disease severity was significantly greater in patients aged over 40 years (compared to the reference group aged 18-30 years). However, there was no correlation between disease severity and other demographic characteristics (e.g., weight, height, gender). Higher TWSTRS scores correlated with worse health-related quality of life as perceived by patients and was reflected in health utility (R(2 )= 0.133). CONCLUSIONS: This study was able to define TWSTRS scores in patients with CD in terms of associated utility. This approach could help in capturing the disease's burden through measures that are more tangible than TWSTRS scores to patients, carers, clinicians, and healthcare payers.

Epidemiological, humanistic, and economic burden of illness of lower limb spasticity in adults: a systematic review.

BACKGROUND: The purpose of this study was to investigate the epidemiological, humanistic, and economic burden of illness associated with adult lower limb spasticity (LLS) and its complications. METHODS: A systematic search of MEDLINE and EMBASE identified 23 studies published between January 2002 and October 2012 that assessed the epidemiology, impact, and resource use associated with LLS. A hand-search of four neurology conferences identified abstracts published between 2010 and 2012. RESULTS: LLS was found to occur in one third of adults after stroke, half to two thirds with multiple sclerosis, and three quarters with cerebral palsy. LLS limits mobility and reduces quality of life. No clear association was found between LLS and occurrence of pain, development of contractures, or risk of falls. CONCLUSION: The evidence on the burden of LLS and its complications is surprisingly limited given the condition's high prevalence among adults with common disorders, such as stroke. Further research is needed to clarify the impact of LLS, including the likelihood of thrombosis in spastic lower limbs. The dearth of high-quality evidence for LLS suggests a lack of awareness of, and interest in, the problem, and therefore, the unmet need among patients and their carers.

Evaluation of the cost per patient per injection of botulinum toxin A in upper limb spasticity: comparison of two preparations in 19 countries.

BACKGROUND: Botulinum toxin A injections are indicated for the management of movement disorders, including upper limb spasticity. The aim of this study was to compare the cost per patient per injection for two botulinum toxin A preparations in 19 countries. METHODS: Doses of botulinum toxin A are expressed in noninterchangeable units (U), ie, Botox(®) in 100 Allergan units (100 U) and Dysport(®) in 500 Speywood units (500 U). Recommended dosages were derived from country-specific summaries of product characteristics or prescribing information. Cost analysis was based on official list prices and expressed in 2011 Euros. The cost per patient per injection was calculated using the recommended dosage in upper limb spasticity combined with price per vial in each country. RESULTS: For upper limb spasticity, the recommended dosage for Dysport in the summary of product characteristics is 1000 U per patient, whereas for Botox the recommended dosage when recommendations were made is 300 U. Allowing for different prices per vial in each country, the cost per patient per injection for upper limb spasticity was less for Dysport than for Botox in 18 (95%) of the 19 countries (mean 17% less across countries). The difference was 20% or higher in nearly half (47%) of the countries. Sensitivity analyses considering available "real-world" dosing showed consistent results, with Dysport being less costly than Botox in all 19 countries. CONCLUSION: Considering costs per patient per injection based on analysis of recommended dosages in the summary of product characteristics, Dysport remains cheaper than Botox in most countries. Thus, when extrapolated to a national level, substantial savings could be realized by using Dysport in the treatment of upper limb spasticity.

Decision-tree model for health economic comparison of two long-acting somatostatin receptor ligand devices in France, Germany, and the UK.

BACKGROUND: Long-acting somatostatin receptor ligands (SRL) with product-specific formulation and means of administration are injected periodically in patients with acromegaly and neuroendocrine tumors. A simple decision-tree model aimed at comparing cost savings with ready-to-use Somatuline Autogel(®) (lanreotide) and Sandostatin LAR(®) (octreotide) for the UK, France, and Germany. The drivers of cost savings studied were the reduction of time to administer as well as a reduced baseline risk of clogging during product administration reported for Somatuline Autogel(®). METHODS: The decision-tree model assumed two settings for SRL administration, ie, by either hospital-based or community-based nurses. In the case of clogging, the first dose was assumed to be lost and a second injection performed. Successful injection depended on the probability of clogging. Direct medical costs were included. A set of scenarios were run, varying the cost drivers, such as the baseline risk of clogging, SRL administration time, and percentage of patients injected during a hospital stay. RESULTS: Costs per successful injection were less for Somatuline Autogel(®)/Depot, ranging from Euros (EUR) 13-45, EUR 52-108, and EUR 127-151, respectively, for France, Germany, and the UK. The prices for both long-acting SRL were the same in France, and cost savings came to 100% from differences other than drug prices. For Germany and the UK, the proportion of savings due to less clogging and shorter administration time was estimated to be around 32% and 20%, respectively. Based on low and high country-specific patient cohort size estimations of individuals eligible for SRL treatment among the patient population with acromegaly and neuroendocrine tumors, annual savings were estimated to be up to EUR 2,000,000 for France, EUR 6,000,000 for Germany, and EUR 7,000,000 for the UK. CONCLUSION: This model suggests that increasing usage of the Somatuline device for injection of SRL might lead to substantial savings for health care providers across Europe.

Budget impact of tegaserod on a managed care organization formulary.

We sought to develop a budget impact model that assesses the economic effect of adding tegaserod for the management of irritable bowel syndrome (IBS) with constipation to the formulary of a managed care organization (MCO). The model estimates the per patient economic impact and the per member, per month (PMPM) economic impact of patients 6 months before and 6 months after the initiation of tegaserod. Resource utilization data, taken from medical and pharmacy administrative claims data, were based on a retrospective, longitudinal study of 3365 patients administered tegaserod through a large, geographically diverse MCO. Costs were estimated for 2 patient subgroups, women with IBS and other gastrointestinal (GI) diagnoses. Sensitivity analyses were performed by varying several model input parameters. The base-case model resulted in an incremental PMPM budget impact associated with the use of tegaserod of 0.01 dollars. Total per patient budget impact (for all resources, including tegaserod) for a 6-month period was 274.34 dollars for women with IBS and 301.84 dollars for women with other GI diagnoses. Overall, 25.9% (29.0% for women with IBS group and 21.9% for women with other GI diagnoses group) of the cost of tegaserod was offset by decreases in resource utilization. Key drivers of post-tegaserod reductions in resource costs were hospital stays, outpatient office visits, emergency department visits, endoscopic procedures, and nonendoscopic procedures. Tegaserod therapy can decrease GI-related resource utilization, resulting in a significant cost-offset percentage. When the associated budget impact of adding tegaserod to its formulary is absorbed across an entire MCO population, the PMPM impact of tegaserod is small.