Background Chronic inflammatory demyelinating polyneuropathy (CIDP) is a rare neurological disorder characterised by muscle weakness and impaired sensory function. The present study provides a comprehensive literature review of the burden of illness of CIDP. Methods Systematic literature search of PubMed, Embase, and key conferences in May 2019. Search terms identified studies on the epidemiology, humanistic burden, current treatment, and economic burden of CIDP published since 2009 in English. Results Forty-five full texts and nineteen conference proceedings were identified on the epidemiology (n = 9), humanistic burden (n = 7), current treatment (n = 40), and economic burden (n = 8) of CIDP. Epidemiological studies showed incidence and prevalence of 0.2-1.6 and 0.8-8.9 per 100,000, respectively, depending on geography and diagnostic criteria. Humanistic burden studies revealed that patients experienced physical and psychosocial burden, including impaired physical function, pain and depression. Publications on current treatments reported on six main types of therapy: intravenous immunoglobulins, subcutaneous immunoglobulins, corticosteroids, plasma exchange, immunosuppressants, and immunomodulators. Treatments may be burdensome, due to adverse events and reduced independence caused by treatment administration setting. In Germany, UK, France, and the US, CIDP economic burden was driven by direct costs of treatment and hospitalisation. CIDP was associated with indirect costs driven by impaired productivity. Conclusions This first systematic review of CIDP burden of illness demonstrates the high physical and psychosocial burden of this rare disease. Future research is required to fully characterise the burden of CIDP, and to understand how appropriate treatment can mitigate burden for patients and healthcare systems.
Objective To assess the impact of components of the national programme for information technology (NPfIT) on measures of clinical and operational efficiency. Design Quasi-experimental controlled before and after study using routinely collected patient level data. Setting Four NHS acute hospital trusts in England. Data sources Inpatient admissions and outpatient appointments, 2000-5. Interventions A system for ordering pathology tests and browsing results (computerised physician order entry, CPOE) and a system for requesting radiological examinations and displaying images (picture archiving and communications system, PACS). Main outcome measures Requests per inpatient, outpatient, or day case patient for full blood count, urine culture and urea and electrolytes tests, and plain x ray film, computed tomography, and ultrasonography examinations.Results CPOE was associated with a reduction in the proportion of outpatient appointments at which full blood count (odds ratio 0.25, 95% confidence interval 0.16 to 0.40), urea and electrolytes (0.55, 0.39 to 0.77), and urine culture (0.30, 0.17 to 0.51) tests were ordered, and at which full blood count tests were repeated (0.73, 0.53 to 0.99). Conversely, the same system was associated with an almost fourfold increase in the use of urea and electrolytes tests among day case patients (3.63, 1.66 to 7.94). PACS was associated with a reduction in repeat plain x ray films at outpatient appointments (0.62, 0.44 to 0.88) and a reduction in inpatient computed tomography (0.83, 0.70 to 0.98). Conversely, it was associated with increases in computed tomography requested at outpatient appointments (1.89, 1.26 to 2.84) and computed tomography repeated within 48 hours during an inpatient stay (2.18, 1.52 to 3.14). Conclusions CPOE and PACS were associated with both increases and reductions in tests and examinations. The magnitude of the changes is potentially important with respect to the efficiency of provision of health care. Better information about the impact of modern IT is required to enable healthcare organisations to manage implementation optimally.
Objectives: Inherited RPE65-related retinal dystrophy is a rare, progressive disease, which until now has had no treatment options. Patients experience decreasing functional vision often from childhood and half of all patients are legally blind by age 18. This study evaluates the cost-effectiveness of voretigene neparvovec, a gene therapy which is the first treatment option ever developed, in a Danish setting. Methods: A Markov model with annual cycles was developed to describe the disease progression with and without treatment respectively. In the model, patients could go from moderate visual impairment through four additional states of visual impairment, with death as an option from all other states. Costs were based on list prices of drugs and either DRG costs or prices derived from a Danish eye care hospital clinic for cost of treatments. In line with Danish convention, the model focused on the perspective of a healthcare payer and thus, social costs were not included. Discount rates in Denmark follow national guidance in which the rate is 4% in the beginning with a lowering over time. The average rate, weighted by survivors in the model, ended up at 3,5% in the base case. In line with earlier NICE guidance, sensitivity analyses were performed also on lower discount rates given the upfront nature of payments. Results: In the base case, the ICER lands at DKK 834,241 with a QALY gain of 6.27. The results are mostly affected by choice of discount rate and assumptions regarding the duration of treatment effect. It is particularly worth noting that a lower discount rate of 1.5% almost halves the ICER, to DKK 447,034 with a QALY gain of 11.7. The undiscounted QALY gain is 20.1. Conclusions: With an assumed ICER threshold of DKK 1,000,000 for orphan indications, voretigene neparvovec is cost-effective in a Danish setting.
Intervention, Comparators, Outcomes, Settings, Time, Effects, Sensitivity) framework for an incremental cost effectiveness/utility, cost benefit, cost consequence, cost minimization, budget impact, willingness to pay, value of life or value of information analysis. FAE included sequential assessment of publications based on Pharmaceuticals Pricing Board, Consolidated Health Economic Evaluation Reporting Standards (CHEERS) and two earlier criteria. LoEE was built based on existing CCC classification (strong, moderate, minor/weak, no evidence) and extended significantly to cover HEEs. RESULTS: 41 publications fulfilled the search criteria and 15 publications were fully reviewed. In FAE, the most effort was used to assess the representativeness of patients, costs, quality of life and other inputs as well as modelling rationale for the Finnish setting. Generally, the applicability of foreign HEE results to the established Finnish CCC setting as such was poor and only one treatment had strong negative HEE evidence (multiple non-conflicting incremental HEE results). Most drugs had minor-to-moderate (one or conflicting) HEE evidence and three drugs had no valid HEE evidence. SLR, FAE and LoEE of HEEs was beneficial for the CCC. CONCLUSIONS: PICOSTEPS formed a working basis for the structural inclusion criteria to carry out the SLR. FAE was done based on the guidelines and demonstrated that there is significant need for local HEEs, especially if the published HEEs are rare or poorly generalizable to Finland. LoEE is an important part of review and should conclude the SLR statements based on HEEs.OBJECTIVES: Transfusion-dependent b-thalassemia (TDT) is a rare genetic disease that affects the production of healthy red blood cells. Conventional treatment involves regular blood transfusions and iron chelation, which is associated with potential adverse events and a substantial impact on quality of life. The only approved curative therapy is allogenic hematopoietic stem cell transplant (allo-HSCT), which carries the risk of complications, including graft-versus-host disease (GvHD). One novel treatment approach is gene therapy involving autologous stem cell transplant of the patient's own genetically modified hematopoietic stem cells. As new treatments for TDT are introduced, cost-utility analyses are needed to examine their value. The purpose of this study was to estimate utilities associated with treatment for TDT. METHODS: General population respondents in England valued eight health state vignettes (developed based on literature review and clinician, patient, and parent interviews) in time tradeoff interviews. There were two pre-transplant health states, three health states describing the year in which a transplant occurs, and three post-transplant health states. RESULTS: A total of 207 participants completed interviews (49.8% female; mean age ¼ 43.2 years; Newcastle, n¼87; London, n¼72; Bristol, n¼48). Mean (SD) utilities for the pre-transplant health states were 0.73 (0.25) for transfusion with oral chelation and 0.63 (0...
e14061 Background: Colorectal cancer (CRC) is the third and second most common cancer in males and females, respectively, worldwide. Based on cases diagnosed in 2008, the age-adjusted incidence rate of CRC was 47.2/100,000/year in the US. Approximately 15-25% of all new cases are diagnosed with metastases and half of all cases result in metastases, representing an increased health and economic burden. The objective of this study was to gain a more in-depth understanding of the epidemiology, clinical and economic burden of second-line CRC in the US, Europe big 5, BRIC-M countries, and Japan. Methods: A Cochrane standard systematic literature review was performed using EMBASE, PubMed, NHSEED databases, and data from key congresses. Searches were conducted using specifically tailored search algorithms for years 2006-2011. Results: Of the 1,377 citations reviewed, 10 studies were identified (6 were epidemiology studies). No information on the number of patients treated for second-line mCRC was found. Estimates of median overall survival were short and varied from 7-18.7 months. New chemotherapy regimens, younger patients, and patients without KRAS mutations are associated with longer overall survival. Information on clinical burden of disease was also limited. The majority of patients presented with liver metastases (73%) and cardiovascular disease (63%). Three clinical trials evaluated quality of life (QoL) with significant heterogeneities in study design, population, treatment regimens and assessment instruments used. No statistically significant results favored targeted therapy. Only 1 study conducted in the US assessed economic burden in patients treated with either bevacizumab or cetuximab. At 6 months the total health care cost was $73,712 and $81,781 respectively, with main cost drivers being outpatient and targeted therapy costs. Conclusions: A substantial evidence gap regarding knowledge of burden of illness in second-line mCRC limits informed clinical and economic decision making. Further research is required to evaluate country-specific epidemiologic and survival data, QoL, resource utilization and cost variation associated with active treatment and end of life care. Research funded by Sanofi/Regeneron.
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