scholarly journals The clinical effectiveness and cost-effectiveness of abatacept, adalimumab, etanercept and tocilizumab for treating juvenile idiopathic arthritis: a systematic review and economic evaluation

2016 ◽  
Vol 20 (34) ◽  
pp. 1-222 ◽  
Author(s):  
Jonathan Shepherd ◽  
Keith Cooper ◽  
Petra Harris ◽  
Joanna Picot ◽  
Micah Rose
Keyword(s):  
Quality Of Life ◽  
Juvenile Idiopathic Arthritis ◽  
Cost Effectiveness ◽  
Systematic Reviews ◽  
Clinical Effectiveness ◽  
Cost Utility ◽  
Open Label ◽  
Biologic Dmards

BackgroundJuvenile idiopathic arthritis (JIA) is characterised by joint pain, swelling and a limitation of movement caused by inflammation. Subsequent joint damage can lead to disability and growth restriction. Treatment commonly includes disease-modifying antirheumatic drugs (DMARDs), such as methotrexate. Clinical practice now favours newer drugs termed biologic DMARDs where indicated.ObjectiveTo assess the clinical effectiveness and cost-effectiveness of four biologic DMARDs [etanercept (Enbrel®, Pfizer), abatacept (Orencia®, Bristol-Myers Squibb), adalimumab (Humira®, AbbVie) and tocilizumab (RoActemra®, Roche) – with or without methotrexate where indicated] for the treatment of JIA (systemic or oligoarticular JIA are excluded).Data sourcesElectronic bibliographic databases including MEDLINE, EMBASE, The Cochrane Library and the Database of Abstracts of Reviews of Effects were searched for published studies from inception to May 2015 for English-language articles. Bibliographies of related papers, systematic reviews and company submissions were screened and experts were contacted to identify additional evidence.Review methodsSystematic reviews of clinical effectiveness, health-related quality of life and cost-effectiveness were undertaken in accordance with the Preferred Reporting Items for Systematic Reviews and Meta-Analyses statement. A cost–utility decision-analytic model was developed to compare the estimated cost-effectiveness of biologic DMARDs versus methotrexate. The base-case time horizon was 30 years and the model took a NHS perspective, with costs and benefits discounted at 3.5%.ResultsFour placebo-controlled randomised controlled trials (RCTs) met the inclusion criteria for the clinical effectiveness review (one RCT evaluating each biologic DMARD). Only one RCT included UK participants. Participants had to achieve an American College of Rheumatology Pediatric (ACR Pedi)-30 response to open-label lead-in treatment in order to be randomised. An exploratory adjusted indirect comparison suggests that the four biologic DMARDs are similar, with fewer disease flares and greater proportions of ACR Pedi-50 and -70 responses among participants randomised to continued biologic DMARDs. However, confidence intervals were wide, the number of trials was low and there was clinical heterogeneity between trials. Open-label extensions of the trials showed that, generally, ACR responses remained constant or even increased after the double-blind phase. The proportions of adverse events and serious adverse events were generally similar between the treatment and placebo groups. Four economic evaluations of biologic DMARDs for patients with JIA were identified but all had limitations. Two quality-of-life studies were included, one of which informed the cost–utility model. The incremental cost-effectiveness ratios (ICERs) for adalimumab, etanercept and tocilizumab versus methotrexate were £38,127, £32,526 and £38,656 per quality-adjusted life year (QALY), respectively. The ICER for abatacept versus methotrexate as a second-line biologic was £39,536 per QALY.LimitationsThe model does not incorporate the natural history of JIA in terms of long-term disease progression, as the current evidence is limited. There are no head-to-head trials of biologic DMARDs, and clinical evidence for specific JIA subtypes is limited.ConclusionsBiologic DMARDs are superior to placebo (with methotrexate where permitted) in children with (predominantly) polyarticular course JIA who have had an insufficient response to previous treatment. Randomised comparisons of biologic DMARDs with long-term efficacy and safety follow-up are needed to establish comparative effectiveness. RCTs for JIA subtypes for which evidence is lacking are also required.Study registrationThis study is registered as PROSPERO CRD42015016459.FundingThe National Institute for Health Research Health Technology Assessment programme.

scholarly journals ImmunoCAP® ISAC and Microtest for multiplex allergen testing in people with difficult to manage allergic disease: a systematic review and cost analysis

2016 ◽  
Vol 20 (67) ◽  
pp. 1-178 ◽  
Author(s):  
Marie Westwood ◽  
Bram Ramaekers ◽  
Shona Lang ◽  
Nigel Armstrong ◽  
Caro Noake ◽  
...  
Keyword(s):  
Quality Of Life ◽  
Cost Effectiveness ◽  
Conference Proceedings ◽  
Citation Index ◽  
Clinical Effectiveness ◽  
Short Term ◽  
Cost Analyses ◽  
Immunocap Isac

BackgroundAllergy is a form of immune-mediated exaggerated sensitivity (hypersensitivity) to a substance that is either inhaled, swallowed, injected or comes into contact with the skin. Foreign substances that provoke allergies are called allergens. It has been claimed that multiplex allergen testing may help in diagnosing the cause of symptoms in patients with an unclear cause of allergy or who are allergic to more than one substance.ObjectivesTo evaluate multiplex allergen testing [devices that can measure the presence of multiple immunoglobulin E (IgE) antibodies in a patient’s blood at the same time], by assessing (1) clinical effectiveness (allergy symptoms, incidence of acute exacerbations, mortality, adverse events of testing and treatment, health-care presentations or admissions, health-related quality of life); (2) effects on treatment (diet, immunotherapy medications, other potential testing); (3) any additional diagnostic information provided by multiplex allergen testing; and (4) cost-effectiveness (cost of different assessment strategies).MethodsFifteen databases were searched from 2005 to April 2015, including MEDLINE (via OvidSp), MEDLINE In-Process Citations, MEDLINE Daily Update, PubMed (National Library of Medicine), EMBASE, Cochrane Database of Systematic Reviews (CDSR), Cochrane Central Register of Controlled Trials (CENTRAL), Database of Abstracts of Reviews of Effects (DARE), Health Technology Assessment (HTA) database, Science Citation Index (SCI), Conference Proceedings Citation Index-Science (CPCI-S), BIOSIS Previews, Latin American and Caribbean Health Sciences Literature (LILACS), National Institute for Health Research (NIHR) HTA programme, and the US Food and Drug Administration (FDA); supplementary searches of conference proceedings and trials registries were performed. Review methods followed published guidance from the Cochrane Collaboration and the Centre for Reviews and Dissemination, University of York, UK. The methodological quality of included studies was assessed using appropriate published tools or a review-specific tool designed by the project team. Studies were summarised in a narrative synthesis. Owing to a lack of data on the clinical effectiveness of multiplex allergen testing, no long-term cost-effectiveness model was developed. A conceptual model structure was developed and cost analyses were performed to examine the short-term costs of various possible diagnostic pathways.ResultsFifteen studies were included in the review. The very limited available data indicated that the addition of multiplex allergen testing [ImmunoCAP®Immuno Solid-phase Allergen Chip (ISAC), Thermo Fisher Scientific/Phadia AB, Uppsala, Sweden] to standard diagnostic work-up can change the clinicians’ views on the diagnosis, management and treatment of patients. There was some indication that the use of ImmunoCAP ISAC testing may be useful to guide decisions on the discontinuation of restrictive diets, the content of allergen-specific immunotherapy (SIT) prescriptions, and whether or not patients should receive SIT. However, none of the studies that we identified reported any information on clinical outcomes subsequent to changes in treatment or management. There was some evidence that ImmunoCAP ISAC may be useful for discriminating allergens that are structurally similar and are recognised by the same IgE antibody (cross-immunoreactive). No data were available for Microtest (Microtest Matrices Ltd, London, UK). Detailed cost analyses suggested that multiplex allergen testing would have to result in a substantial reduction of the proportions of patients receiving single IgE testing and oral food challenge tests in order to be cost-saving in the short term.ConclusionsNo recommendations for service provision can be made based on the analyses included in this report. It is suggested that a consensus-based protocol for the use of multiplex allergen testing be developed. The clinical effectiveness and cost-effectiveness of the proposed protocol should then be assessed by comparing long-term clinical and quality of life outcomes and resource use in patients managed using the protocol with those managed using a standard diagnostic pathway.Study registrationThis study is registered as PROSPERO CRD42015019739.FundingThis project was a Diagnostic Assessment Report commissioned by the NIHR HTA programme on behalf of the National Institute for Health and Care Excellence.

Effectiveness of mobile applications on healthy lifestyles aimed at diabetic patients: review of systematic reviews. (Preprint)

2019 ◽  
Author(s):  
Francisco Jesús Represas Carrera Sr ◽  
Ángel Alfredo Martínez Ques Sr ◽  
Ana Clavería Fontán Sr
Keyword(s):  
Quality Of Life ◽  
Diabetes Mellitus ◽  
Systematic Reviews ◽  
Mobile Applications ◽  
Control Group ◽  
Diabetic Patients ◽  
Healthy Lifestyles ◽  
Major Public Health Problem

BACKGROUND Diabetes mellitus is currently a major public health problem worldwide. It is traditionally approached in a clinical inpatient relationship between the patient and the healthcare professional. However, the rise of new technologies, particularly mobile applications, is revolutionizing the traditional healthcare model through the introduction of telehealthcare. OBJECTIVE (1) To assess the effects of mobile applications for improving healthy lifestyles on the quality of life and metabolic control of diabetes mellitus in adult patients. (2) To describe the characteristics of the mobile applications used, identify the healthy lifestyles they target, and describe any adverse effects their use may have. METHODS Review of systematic reviews and meta-analysis, following the guidelines of the Cochrane Collaboration and the Joanna Briggs Institute. We included studies that used any mobile application aimed at helping patients improve self-management of diabetes mellitus by focusing on healthy lifestyles. Studies needed to include a control group receiving regular care without the use of mobile devices. In May 2018, a search was conducted in Medline, Embase, Cochrane, LILACS, PsychINFO, Cinahl and Science Direct, updated in May 2019. The methodological quality of the studies was assessed using the Amstar-2 tool. RESULTS Seven systematic reviews of 798 articles were initially selected for analysis. The interventions had a duration of between 1 and 12 months. Mobile applications focused singly or simultaneously on different lifestyles aspects (diet, physical exercise, motivation, blood glucose levels, etc.). There are significant changes in HbA1c values, body weight and BMI, although in others, such as lipid profile, quality of life, or blood pressure, there is no clear improvement. CONCLUSIONS There is clear evidence that the use of mobile applications improves glycemic control in diabetic patients in the short term. There is a lack of evidence in its long-term benefits. It is thus necessary to carry out further studies to learn about the long-term effectiveness of mobile applications aimed at promoting the healthy lifestyles of diabetic patients. CLINICALTRIAL PROSPERO Register: CRD42019133685

scholarly journals Nine-year prospective efficacy and safety of brain-responsive neurostimulation for focal epilepsy

Neurology ◽  
2020 ◽  
Vol 95 (9) ◽  
pp. e1244-e1256 ◽  
Author(s):  
Dileep R. Nair ◽  
Kenneth D. Laxer ◽  
Peter B. Weber ◽  
Anthony M. Murro ◽  
Yong D. Park ◽  
...  
Keyword(s):  
Quality Of Life ◽  
Seizure Frequency ◽  
Focal Epilepsy ◽  
Responder Rate ◽  
Seizure Freedom ◽  
Open Label ◽  
Cognitive Domains ◽  
Signed Rank

ObjectiveTo prospectively evaluate safety and efficacy of brain-responsive neurostimulation in adults with medically intractable focal onset seizures (FOS) over 9 years.MethodsAdults treated with brain-responsive neurostimulation in 2-year feasibility or randomized controlled trials were enrolled in a long-term prospective open label trial (LTT) to assess safety, efficacy, and quality of life (QOL) over an additional 7 years. Safety was assessed as adverse events (AEs), efficacy as median percent change in seizure frequency and responder rate, and QOL with the Quality of Life in Epilepsy (QOLIE-89) inventory.ResultsOf 256 patients treated in the initial trials, 230 participated in the LTT. At 9 years, the median percent reduction in seizure frequency was 75% (p < 0.0001, Wilcoxon signed rank), responder rate was 73%, and 35% had a ≥90% reduction in seizure frequency. We found that 18.4% (47 of 256) experienced ≥1 year of seizure freedom, with 62% (29 of 47) seizure-free at the last follow-up and an average seizure-free period of 3.2 years (range 1.04–9.6 years). Overall QOL and epilepsy-targeted and cognitive domains of QOLIE-89 remained significantly improved (p < 0.05). There were no serious AEs related to stimulation, and the sudden unexplained death in epilepsy (SUDEP) rate was significantly lower than predefined comparators (p < 0.05, 1-tailed χ2).ConclusionsAdjunctive brain-responsive neurostimulation provides significant and sustained reductions in the frequency of FOS with improved QOL. Stimulation was well tolerated; implantation-related AEs were typical of other neurostimulation devices; and SUDEP rates were low.ClinicalTrials.gov identifierNCT00572195.Classification of evidenceThis study provides Class IV evidence that brain-responsive neurostimulation significantly reduces focal seizures with acceptable safety over 9 years.

scholarly journals Economic evaluation of online computerised cognitive–behavioural therapy without support for depression in primary care: randomised trial

2010 ◽  
Vol 196 (4) ◽  
pp. 310-318 ◽  
Author(s):  
S. A. H. Gerhards ◽  
L. E. de Graaf ◽  
L. E. Jacobs ◽  
J. L. Severens ◽  
M. J. H. Huibers ◽  
...  
Keyword(s):  
Quality Of Life ◽  
Cost Effectiveness ◽  
Cognitive Behavioural Therapy ◽  
Behavioural Therapy ◽  
Cost Utility ◽  
Significant Group ◽  
Efficient Treatment ◽  
Cognitive Behavioural ◽  
The Cost

BackgroundEvidence about the cost-effectiveness and cost utility of computerised cognitive–behavioural therapy (CCBT) is still limited. Recently, we compared the clinical effectiveness of unsupported, online CCBT with treatment as usual (TAU) and a combination of CCBT and TAU (CCBT plus TAU) for depression. The study is registered at the Netherlands Trial Register, part of the Dutch Cochrane Centre (ISRCTN47481236).AimsTo assess the cost-effectiveness of CCBT compared with TAU and CCBT plus TAU.MethodCosts, depression severity and quality of life were measured for 12 months. Cost-effectiveness and cost-utility analyses were performed from a societal perspective. Uncertainty was dealt with by bootstrap replications and sensitivity analyses.ResultsCosts were lowest for the CCBT group. There are no significant group differences in effectiveness or quality of life. Cost-utility and cost-effectiveness analyses tend to be in favour of CCBT.ConclusionsOn balance, CCBT constitutes the most efficient treatment strategy, although all treatments showed low adherence rates and modest improvements in depression and quality of life.

SHOULD CHRONIC HEPATITIS B BE TREATED AS EARLY AS POSSIBLE?

2013 ◽  
Vol 29 (1) ◽  
pp. 35-41 ◽  
Author(s):  
Frank Hulstaert ◽  
Christoph Schwierz ◽  
Frederik Nevens ◽  
Nancy Thiry ◽  
Mohamed Gamil ◽  
...  
Keyword(s):  
Quality Of Life ◽  
Chronic Hepatitis ◽  
Cost Effectiveness ◽  
Hepatitis B ◽  
Chronic Hepatitis B ◽  
Disease Stage ◽  
Multicenter Survey ◽  
Cirrhotic Patients

Objectives: We studied the cost-effectiveness of tenofovir and entecavir in e antigen positive (CHBe+) and negative (CHBe-) chronic hepatitis B.Methods: Using a multicenter survey including 544 patients we measured patient quality of life and attributable costs by clinical disease stage. Natural disease progression was studied in 278 patients in a single center. A Markov model was constructed to follow hypothetical cohorts of treated and untreated 40-year-old CHBe+ and CHBe- patients and 50-year-old patients with compensated cirrhosis.Results: We did not find an improvement in quality of life when viral load was reduced under treatment. Transition rates to liver cirrhosis were found to be age-dependent. Assuming equal effectiveness, tenofovir dominates the entecavir strategy because of its lower price in Belgium. The incremental cost-effectiveness ratio (ICER) of tenofovir after 20 years is more favorable for treating Caucasian cirrhotic patients (mean ICER €29,000/quality-adjusted life-year [QALY]) compared with treating non-cirrhotic patients (mean ICER €110,000 and 131,000/QALY for CHB e+ and e-, respectively). Within the non-cirrhotic patients the ICER decreases with increasing cohort starting age from 30 to 50 years.Conclusions: Results of long-term models for tenofovir or entecavir treatment of CHB need to be interpreted with caution as long-term trials with hard end points are lacking. Especially the effect on HCC remains highly uncertain. Based on cost-effectiveness considerations such antiviral treatment should be targeted at patients with cirrhosis or at risk of rapid progression to this disease stage.

scholarly journals Sustained Improvement in Health-Related Quality of Life in Patients with Hemophilia A with or without Inhibitors Treated with Fitusiran Prophylaxis

Blood ◽  
2021 ◽  
Vol 138 (Supplement 1) ◽  
pp. 3197-3197
Author(s):  
Viridiana Cano ◽  
José Bartelt-Hofer ◽  
Wenruo Hu ◽  
Shauna R. Andersson ◽  
Pronabesh Dasmahapatra ◽  
...  
Keyword(s):  
Quality Of Life ◽  
Board Of Directors ◽  
Phase 1 ◽  
Publicly Traded ◽  
Open Label ◽  
Advisory Committees ◽  
Open Label Extension ◽  
Related Quality

Abstract BACKGROUND Fitusiran is an investigational, subcutaneous (SC), prophylactically administered small interfering RNA (siRNA) therapeutic. It targets antithrombin and restores thrombin generation sufficient to rebalance hemostasis in people with hemophilia A or B, with or without inhibitors. Long-term exposure to fitusiran is being studied in a Phase 1/2, 6-year open-label extension study (NCT02554773) in patients with hemophilia (PwH) A or B with or without inhibitors. Improvement in the health-related quality of life (HRQoL) as measured by Haemophilia Quality of Life Questionnaire for Adults (Haem-A-QoL) total score, and specifically in the physical health domain was previously reported by von Mackensen et al. for PwHA with inhibitors (NCT02554773, N=17, von Mackensen et al. Blood 2020;136(1):23-24). This analysis aimed to present HRQoL changes from baseline to last available measure (LAM), as measured by the Haem-A-QoL total score and domains (filtered by actual treatment exposure following 2 voluntary dosing holds) in PwHA, with or without inhibitors, who continued into the Phase 2 open-label extension study. METHODS Participants who completed the Phase 1 study (NCT02035605) were eligible to participate in Phase 2 long-term exposure study (NCT02554773). All participants received fixed monthly doses of 50 mg (N=9) or 80 mg (N=18) of fitusiran. HRQoL data were collected in 3-month lapse periods. Mean changes from baseline to LAM were calculated for the Haem-A-QoL (total score and domains). RESULTS As of February 2021, 26 severe and 1 moderate PwHA (13 with, 14 without inhibitors) with mean baseline age (SD) 37.3 (9.7) were treated for up to mean (SD) 33.32 (17.06) months. For the total Haem-A-QoL score and each of 10 domains, mean improvements from baseline to LAM (lower scores denoting better HRQoL) were consistently observed in PwHA (except for the sport & leisure domain that was higher in the non-inhibitor group). Mean estimated reductions (SD) in the Haem-A-QoL total score were -8.33 (13.84) and -9.16 (14.55) while that in the physical health domain score were -13.21 (25.07) and -7.14 (22.80), for PwHA with and without inhibitors respectively (Table 1). When comparing Haem-A-QoL domains in inhibitor to non-inhibitor participants, results suggest a modest favorable trend for non-inhibitor patients (greater reductions in 4 vs 6 domains, respectively). CONCLUSIONS Mean improvements in the Haem-A-QoL total scores and 9 out of 10 domains suggest sustained HRQoL improvements in PwHA. Small sample size and outlying results might limit the interpretation. Further research in HRQoL for fitusiran will include a larger population on the new 50 mg every other month dose regimen, and notably Phase III results from NCT03549871 (open-label study in patients switching to fitusiran from previous prophylaxis or bypassing agents) and NCT03754790 (open-label, long-term safety and efficacy study of fitusiran) in various subpopulations. Figure 1 Figure 1. Disclosures Cano: Sanofi Genzyme: Current Employment, Current equity holder in publicly-traded company; Takeda: Ended employment in the past 24 months. Bartelt-Hofer: Sanofi: Current Employment, Current equity holder in publicly-traded company, Current holder of individual stocks in a privately-held company. Hu: Sanofi: Current Employment. Andersson: Sanofi: Current Employment, Current equity holder in publicly-traded company; WEST advisory committee member: Membership on an entity's Board of Directors or advisory committees. Dasmahapatra: Sanofi: Current Employment, Current equity holder in publicly-traded company. Von Mackensen: Biomarin: Speakers Bureau; Novo Nordisk: Consultancy; Sanofi: Consultancy; Sobi: Consultancy, Membership on an entity's Board of Directors or advisory committees, Research Funding, Speakers Bureau; University Medical Centre Hamburg-Eppendorf: Current Employment; CSL Behring: Speakers Bureau; Chugai/Roche: Membership on an entity's Board of Directors or advisory committees.

PP126 Radiofrequency Ablation For Metastatic Spinal Lesions

2021 ◽  
Vol 37 (S1) ◽  
pp. 20-21
Author(s):  
Alezandra Torres-castaño ◽  
Amado Rivero-Santana ◽  
Lilisbeth Perestelo-Pérez ◽  
Ana Toledo-chávarri ◽  
Andrea Duarte-Diaz ◽  
...  
Keyword(s):  
Quality Of Life ◽  
Cost Effectiveness ◽  
Radiofrequency Ablation ◽  
Systematic Reviews ◽  
Palliative Treatment ◽  
Case Series ◽  
Controlled Trials ◽  
Vertebral Metastases ◽  
Randomized Controlled

IntroductionAbout 70 percent of metastatic breast, lung, and prostate cancers affect the bones. When this phase of the disease affects the spine, the mobility and quality of life of patients are severely impaired. Radiofrequency ablation (RFA) has become a feasible option in the palliative treatment of vertebral metastases due to its minimal invasiveness and short procedure time. This health technology assessment report aimed to identify, evaluate, and synthesize evidence on the safety, effectiveness, and cost effectiveness of RFA for vertebral metastases.MethodsA systematic search was conducted to identify literature published from December 2016 to July 2019 in the following databases: Medline, Embase, the Cochrane Library, and the Centre for Reviews and Dissemination. Systematic reviews, randomized and non-randomized controlled trials, and case series studies evaluating the efficacy and safety of RFA in patients with vertebral metastases were included.ResultsSixteen studies were included: two systematic reviews, 13 case series studies, and one comparative study. None of the systematic reviews identified any randomized controlled trials. Of the 14 included primary studies, 10 evaluated RFA in combination with vertebroplasty, three evaluated RFA in combination with kyphoplasty, and one study evaluated a combination of RFA and radiation therapy. In all cases, the evaluated patients had different types of cancer (e.g., breast, lung, or liver). The follow-up periods varied between the studies from one day to 12 months. The most commonly used RFA devices were the STAR™ Tumour Ablation System (Merit Medical Systems) and the OsteoCool™ Radiofrequency Ablation System (Medtronic).ConclusionsRFA reduces pain, improves functional capacity, and provides greater local control of disease, potentially giving patients a higher quality of life, even in the context of metastatic disease. Although there is evidence on the safety and efficacy of this technology for the palliative treatment of vertebral metastases, more studies with higher methodological quality are needed. There were no studies available on the cost effectiveness of RFA for this indication.

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