scholarly journals The delivery of chemotherapy at home: an evidence synthesis

2015 ◽  
Vol 3 (14) ◽  
pp. 1-182 ◽  
Author(s):  
Mark Corbett ◽  
Morag Heirs ◽  
Micah Rose ◽  
Alison Smith ◽  
Lisa Stirk ◽  
...  
Keyword(s):  
Quality Of Life ◽  
Cost Effectiveness ◽  
Clinical Effectiveness ◽  
Qualitative Studies ◽  
Economic Evaluations ◽  
Intravenous Chemotherapy ◽  
Outpatient Chemotherapy ◽  
Time And Energy ◽  
Effectiveness Studies

BackgroundRecent policy and guidance has focused on chemotherapy services being offered closer to home, but the clinical and economic implications of this are uncertain.ObjectivesTo compare the impact of delivering intravenous chemotherapy in different settings on a range of outcomes, including quality of life, safety and costs.DesignMultimethods approach: systematic review of clinical effectiveness, qualitative and cost-effectiveness studies; description of the patient pathway and brief survey of current provision; and development of a decision model to explore aspects of cost-effectiveness.SettingProvision of intravenous chemotherapy.ParticipantsChemotherapy patients.InterventionsSetting in which chemotherapy was administered (home, community or outpatient).Outcome measuresSafety, quality of life, preference, satisfaction, opinions/experiences, social functioning, clinical outcomes, costs and resource/organisational issues.Data sourcesSixteen electronic databases (including MEDLINE, EMBASE and The Cochrane Library) were searched from inception to October 2013 for published and unpublished studies.Review methodsTwo reviewers independently screened potentially relevant studies, extracted data and quality assessed the included studies. Study validity was evaluated using appropriate quality assessment tools. Clinical effectiveness and cost-effectiveness studies were summarised narratively, and qualitative studies were synthesised using meta-ethnography.ResultsOf the 67 eligible studies, 25 were comparative, with nine including a concurrent economic evaluation. Although some of the 10 randomised trials were designed to minimise avoidable biases, slow recruitment rates and non-participation of eligible patients for setting-related reasons meant that trial sample sizes were small and populations were inherently biased to favour the home or community settings. There was little evidence to suggest differences between settings in terms of quality of life, clinical outcomes, psychological outcomes or adverse events. All nine economic evaluations were judged as having low or uncertain quality, providing limited evidence to draw overall conclusions. Most were cost–consequence analyses, presenting cost outcomes alongside trial results but deriving no summary measure of benefit. Poor resource use reporting and use of different perspectives across settings made results difficult to compare. Seventeen qualitative studies (450 participants) were judged as moderate to good quality, although all compared new or proposed services with existing outpatient facilities and biased samples were used. The three main lines of argument were barriers to service provision, satisfaction with chemotherapy and making compromises to maintain normality. Most patients made explicit trade-offs between the time and energy required for outpatient chemotherapy, which reduced quality of life, and an increased sense of safety. A patient pathway was described, informed by expert advice and a brief survey of NHS and private providers, which identified wide variation in the ways in which home and community chemotherapy was delivered. Considering limitations of the available data and variation in provision, cost-effectiveness modelling results were not robust and were viewed as exploratory only; the results were highly unstable.ConclusionsPrimary studies comparing settings for administering intravenous chemotherapy appear difficult to conduct. Consequently, few robust conclusions can be made about the clinical effectiveness and cost-effectiveness. Qualitative studies indicate that the patient time and energy required for outpatient chemotherapy reduces quality of life. A nested randomised controlled trial within a larger observational cohort of patients is proposed to enhance recruitment and improve generalisability of results. Future economic evaluations require detailed patient characteristic, resource use, cost and quality-of-life data, although their results are likely to have limited generalisability.Study registrationThis study is registered as PROSPERO CRD42013004851.FundingThe National Institute for Health Research Health Services and Delivery Research programme.

scholarly journals Educational interventions to improve quality of life in people with chronic inflammatory skin diseases: systematic reviews of clinical effectiveness and cost-effectiveness

2015 ◽  
Vol 19 (86) ◽  
pp. 1-176 ◽  
Author(s):  
Karen Pickett ◽  
Emma Loveman ◽  
Neelam Kalita ◽  
Geoff K Frampton ◽  
Jeremy Jones
Keyword(s):  
Quality Of Life ◽  
Cost Effectiveness ◽  
Skin Diseases ◽  
Clinical Effectiveness ◽  
Educational Interventions ◽  
Inflammatory Skin Diseases ◽  
Inflammatory Skin ◽  
Skin Conditions ◽  
Effectiveness Studies

BackgroundInflammatory skin diseases include a broad range of disorders. For some people, these conditions lead to psychological comorbidities and reduced quality of life (QoL). Patient education is recommended in the management of these conditions and may improve QoL.ObjectivesTo assess the clinical effectiveness and cost-effectiveness of educational interventions to improve health-related quality of life (HRQoL) in people with chronic inflammatory skin diseases.Data sourcesTwelve electronic bibliographic databases, including The Cochrane Library, MEDLINE and EMBASE, were searched to July 2014. Bibliographies of retrieved papers were searched and an Advisory Group contacted.Review methodsSystematic reviews were conducted following standard methodologies. Clinical effectiveness studies were included if they were undertaken in people with a chronic inflammatory skin condition. Educational interventions that aimed to, or could, improve HRQoL were eligible. Studies were required to measure HRQoL, and other outcomes such as disease severity were also included. Randomised controlled trials (RCTs) or controlled clinical trials were eligible. For the review of cost-effectiveness, studies were eligible if they were full economic evaluations, cost–consequence or cost analyses.ResultsSeven RCTs were included in the review of clinical effectiveness. Two RCTs focused on children with eczema and their carers. Five RCTs were in adults. Of these, two were of people with psoriasis, one was of people with acne and two were of people with a range of conditions. There were few similarities in the interventions (e.g. the delivery mode, the topics covered, the duration of the education), which precluded any quantitative synthesis. Follow-up ranged from 4 weeks to 12 months, samples sizes were generally small and, overall, the study quality was poor. There appeared to be positive effects on HRQoL in participants with psoriasis in one trial, but no difference between groups in another trial in which participants had less severe psoriasis. Carers of children in one RCT of eczema showed improvement in HRQoL; however, in a RCT evaluating a website intervention there were no demonstrable effects on HRQoL. Neither the RCT in those adults with acne nor the RCT in those adults with mixed skin conditions demonstrated an effect on HRQoL. One RCT reported subgroups with atopic dermatitis or psoriasis and education was effective for psoriasis only. Other outcomes also showed mixed results. It is unclear how clinically meaningful any of the observed improvements are. Three studies of cost-effectiveness were included. The interventions, comparators and populations varied across the studies and, overall, the studies provided limited information on cost-effectiveness. The studies did provide detailed information on resources and costs that could be useful to inform a future cost-effectiveness evaluation in this area.LimitationsThe application of the inclusion criterion around whether the interventions were aimed at improving HRQoL or the inference that they could improve HRQoL was difficult as information was rarely reported.ConclusionsThere is uncertainty regarding whether educational interventions addressing issues that could improve HRQoL in people with chronic skin conditions are effective. Tentative conclusions about the best approach to delivering these kinds of interventions are that face-to-face, group, sessions may be beneficial; however, text messages may also be effective. Delivery over a period of time and by a multidisciplinary team may also be associated with positive outcomes. There is uncertainty over whether or not educational interventions are cost-effective.Study registrationThis study is registered as PROSPERO CRD42014007426.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.

scholarly journals The clinical effectiveness and cost-effectiveness of second-eye cataract surgery: a systematic review and economic evaluation

2014 ◽  
Vol 18 (68) ◽  
pp. 1-206 ◽  
Author(s):  
Geoff Frampton ◽  
Petra Harris ◽  
Keith Cooper ◽  
Andrew Lotery ◽  
Jonathan Shepherd
Keyword(s):  
Quality Of Life ◽  
Systematic Review ◽  
Cost Effectiveness ◽  
Cataract Surgery ◽  
Patient Reported Outcomes ◽  
Clinical Effectiveness ◽  
Cost Effective ◽  
Eye Surgery ◽  
Patient Reported

BackgroundElective cataract surgery is the most commonly performed surgical procedure in the NHS. In bilateral cataracts, the eye with greatest vision impairment from cataract is operated on first. First-eye surgery can improve vision and quality of life. However, it is unclear whether or not cataract surgery on the second eye provides enough incremental benefit to be considered clinically effective and cost-effective.ObjectiveTo conduct a systematic review of clinical effectiveness and analysis of cost-effectiveness of second-eye cataract surgery in England and Wales, based on an economic model informed by systematic reviews of cost-effectiveness and quality of life.Data sourcesTwelve electronic bibliographic databases, including MEDLINE, EMBASE, Web of Science, The Cochrane Library and the Centre for Reviews and Dissemination databases were searched from database inception to April 2013, with searches updated in July 2013. Reference lists of relevant publications were also checked and experts consulted.Review methodsTwo reviewers independently screened references, extracted and checked data from the included studies and appraised their risk of bias. Based on the review of cost-effectiveness, a de novo economic model was developed to estimate the cost-effectiveness of second-eye surgery in bilateral cataract patients. The model is based on changes in quality of life following second-eye surgery and includes post-surgical complications.ResultsThree randomised controlled trials (RCTs) of clinical effectiveness, three studies of cost-effectiveness and 10 studies of health-related quality of life (HRQoL) met the inclusion criteria for the systematic reviews and, where possible, were used to inform the economic analysis. Heterogeneity of studies precluded meta-analyses, and instead data were synthesised narratively. The RCTs assessed visual acuity, contrast sensitivity, stereopsis and several measures of HRQoL. Improvements in binocular visual acuity and contrast sensitivity were small and unlikely to be of clinical significance, but stereopsis was improved to a clinically meaningful extent following second-eye surgery. Studies did not provide evidence that second-eye surgery significantly affected HRQoL, apart from an improvement in the mental health component of HRQoL in one RCT. In the model, second-eye surgery generated 0.68 incremental quality-adjusted life-years with an incremental cost-effectiveness ratio of £1964. Model results were most sensitive to changes in the utility gain associated with second-eye surgery, but otherwise robust to changes in parameter values. The probability that second-eye surgery is cost-effective at willingness-to-pay thresholds of £10,000 and £20,000 is 100%.LimitationsClinical effectiveness studies were all conducted more than 9 years ago. Patients had good vision pre surgery which may not represent all patients eligible for second-eye surgery. For some vision-related patient-reported outcomes and HRQoL measures, thresholds for determining important clinical effects are either unclear or have not been determined.ConclusionsSecond-eye cataract surgery is generally cost-effective based on the best available data and under most assumptions. However, more up-to-date data are needed. A well-conducted RCT that reflects current populations and enables the estimation of health state utility values would be appropriate. Guidance is required on which vision-related, patient-reported outcomes are suitable for assessing effects of cataract surgery in the NHS and how these measures should be interpreted clinically.Study registrationThis project is registered as PROSPERO CRD42013004211.FundingThis project was funded by the National Institute for Health Research Health Technology Assessment programme.

scholarly journals Infliximab for the treatment of ulcerative colitis

2009 ◽  
Vol 13 (Suppl 3) ◽  
pp. 7-12
Author(s):  
C Hyde ◽  
S Bryan ◽  
A Juarez-Garcia ◽  
L Andronis ◽  
A Fry-Smith
Keyword(s):  
Quality Of Life ◽  
Ulcerative Colitis ◽  
Cost Effectiveness ◽  
Evidence Review Group ◽  
Clinical Effectiveness ◽  
Quality Adjusted Life Year ◽  
Single Technology Appraisal ◽  
Nice Guidance ◽  
Related Quality

This paper presents a summary of the evidence review group (ERG) report into the clinical effectiveness and cost-effectiveness of infliximab for moderately to severely active ulcerative colitis (UC) based upon a review of the manufacturer’s submission to the National Institute for Health and Clinical Excellent (NICE) as part of the single technology appraisal (STA) process. The submission indicated that the efficacy of infliximab (5 mg/kg) had been demonstrated in terms of higher response rates and a sustained response in health-related quality of life. For the cost-effectiveness analysis, the manufacturer built a Markov model to compare infliximab with standard care. It estimated the incremental cost per quality-adjusted life-year (QALY) gained was between £25,044 and £33,866 depending on the strategy used. The ERG report generally agreed with the evidence on effectiveness of infliximab for subacute exacerbations of UC. However, there were several areas of uncertainty, of which the interpretation of the importance of the quality of life changes in the subacute situation and the assessment of the adequacy of the evidence of effectiveness of infliximab in the acute hospital-based situation were considered pre-eminent by the ERG. This challenged the estimates of cost-effectiveness offered and suggested that there should be a separate assessment of infliximab for acute exacerbations of moderately to severely active UC. The summary of the NICE guidance issued in April 2008 as a result of the STA states that: infliximab is not recommended for the treatment of subacute manifestations of moderately to severely active UC.

scholarly journals Certolizumab pegol (CIMZIA®) for the treatment of rheumatoid arthritis

2010 ◽  
Vol 14 (Suppl 2) ◽  
pp. 1-10
Author(s):  
M Connock ◽  
S Tubeuf ◽  
K Malottki ◽  
A Uthman ◽  
J Round ◽  
...  
Keyword(s):  
Quality Of Life ◽  
Rheumatoid Arthritis ◽  
Cost Effectiveness ◽  
Treatment Option ◽  
English Language ◽  
Certolizumab Pegol ◽  
Clinical Effectiveness ◽  
Quality Of Life Measures ◽  
Language Studies

This paper presents a summary of the evidence review group (ERG) report into the clinical effectiveness and cost-effectiveness of certolizumab pegol (CZP) for adults with active rheumatoid arthritis (RA) that have not responded adequately to treatment with conventional disease modifying anti-rheumatic drugs (DMARDs) including methotrexate (MTX), in accordance with the licensed indication, based upon the evidence submission from the manufacturer to the National Institute for Health and Clinical Excellence (NICE) as part of the single technology appraisal (STA) process. The outcome measures included American College of Rheumatology (ACR) 20, 50 and 70 response rates and quality of life measures after 3 months and 6 months of treatment. The ERG examined the submission’s search strategies and considered they appeared comprehensive and that it was unlikely that relevant studies would have been missed. Only English language studies were considered in the submission and non-English language studies relevant to the decision problem may possibly have been ignored. The ERG analysed the first submitted economic model so as to itemise in detail clarification points that were brought to the attention of the manufacturer. In response the manufacturer submitted a modified cost-effectiveness analysis. The ERG undertook further analysis of this second model and other additional submitted evidence. The clinical evidence was derived from two multicentre blinded randomised controlled trials (RCTs) comparing CZP + MTX to placebo + MTX (the RAPID 1 and RAPID 2 trials). RAPID 1 lasted 52 weeks with 982 patients and RAPID 2 24 weeks with 619 patients. Evidence for clinical effectiveness of CZP in mono-therapy came from the 24-week FAST4WARD trial with 220 patients that compared CZP (400 mg every 4 weeks) versus placebo. The three key RCTs demonstrated statistically significant superiority of CZP + MTX versus placebo + MTX and of CZP versus placebo with respect to a variety of outcomes including ACR 20, ACR 50 and ACR 70 measures and quality of life measures at 3 and 6 months. On the basis of results from the indirect comparison meta-analyses, the manufacturer suggested that CZP may be at least as effective as other ‘biological’ DMARD (bDMARD) comparators and, in a few ACR measures at 3 and 6 months, more effective. CZP is an effective therapy for adult RA patients whose disease has failed to respond adequately to cDMARDs including MTX or who are intolerant of MTX. The cost-effectiveness of CZP relative to other bDMARDs is unclear because the economic modelling undertaken may have ignored relevant effectiveness data and potential differences between trial populations, and so may have included effectiveness results that were biased in favour of CZP; underestimated uncertainty in the relative effectiveness of compared DMARDs; and ignored the potential influence of differences between bDMARDs with regard to adverse events and their related costs and health impacts. The NICE guidance issued in October 2009 states that: the Committee is minded not to recommend certolizumab pegol as a treatment option for people with RA; and the Committee recommends that NICE asks the manufacturer of CZP for more information on the clinical effectiveness and cost-effectiveness of CZP for the treatment of people with RA. On receipt of this information and details of a patient access scheme NICE issued final guidance recommending CZP, under certain criteria, as a treatment option for people with RA.

scholarly journals The cost-effectiveness of domiciliary non-invasive ventilation in patients with end-stage chronic obstructive pulmonary disease: a systematic review and economic evaluation

2015 ◽  
Vol 19 (81) ◽  
pp. 1-246 ◽  
Author(s):  
Janine Dretzke ◽  
Deirdre Blissett ◽  
Chirag Dave ◽  
Rahul Mukherjee ◽  
Malcolm Price ◽  
...  
Keyword(s):  
Quality Of Life ◽  
Cost Effectiveness ◽  
Hospital Admissions ◽  
Cost Effective ◽  
Invasive Ventilation ◽  
Non Invasive ◽  
Non Invasive Ventilation ◽  
The Cost ◽  
Effectiveness Studies

BackgroundChronic obstructive pulmonary disease (COPD) is a chronic progressive lung disease characterised by non-reversible airflow obstruction. Exacerbations are a key cause of morbidity and mortality and place a considerable burden on health-care systems. While there is evidence that patients benefit from non-invasive ventilation (NIV) in hospital during an acute exacerbation, evidence supporting home use for more stable COPD patients is limited. In the UK, domiciliary NIV is considered on health economic grounds in patients after three hospital admissions for acute hypercapnic respiratory failure.ObjectiveTo assess the clinical effectiveness and cost-effectiveness of domiciliary NIV by systematic review and economic evaluation.Data sourcesBibliographic databases, conference proceedings and ongoing trial registries up to September 2014.MethodsStandard systematic review methods were used for identifying relevant clinical effectiveness and cost-effectiveness studies assessing NIV compared with usual care or comparing different types of NIV. Risk of bias was assessed using Cochrane guidelines and relevant economic checklists. Results for primary effectiveness outcomes (mortality, hospitalisations, exacerbations and quality of life) were presented, where possible, in forest plots. A speculative Markov decision model was developed to compare the cost-effectiveness of domiciliary NIV with usual care from a UK perspective for post-hospital and more stable populations separately.ResultsThirty-one controlled effectiveness studies were identified, which report a variety of outcomes. For stable patients, a modest volume of evidence found no benefit from domiciliary NIV for survival and some non-significant beneficial trends for hospitalisations and quality of life. For post-hospital patients, no benefit from NIV could be shown in terms of survival (from randomised controlled trials) and findings for hospital admissions were inconsistent and based on limited evidence. No conclusions could be drawn regarding potential benefit from different types of NIV. No cost-effectiveness studies of domiciliary NIV were identified. Economic modelling suggested that NIV may be cost-effective in a stable population at a threshold of £30,000 per quality-adjusted life-year (QALY) gained (incremental cost-effectiveness ratio £28,162), but this is associated with uncertainty. In the case of the post-hospital population, results for three separate base cases ranged from usual care dominating to NIV being cost-effective, with an incremental cost-effectiveness ratio of less than £10,000 per QALY gained. All estimates were sensitive to effectiveness estimates, length of benefit from NIV (currently unknown) and some costs. Modelling suggested that reductions in the rate of hospital admissions per patient per year of 24% and 15% in the stable and post-hospital populations, respectively, are required for NIV to be cost-effective.LimitationsEvidence on key clinical outcomes remains limited, particularly quality-of-life and long-term (> 2 years) effects. Economic modelling should be viewed as speculative because of uncertainty around effect estimates, baseline risks, length of benefit of NIV and limited quality-of-life/utility data.ConclusionsThe cost-effectiveness of domiciliary NIV remains uncertain and the findings in this report are sensitive to emergent data. Further evidence is required to identify patients most likely to benefit from domiciliary NIV and to establish optimum time points for starting NIV and equipment settings.Future work recommendationsThe results from this report will need to be re-examined in the light of any new trial results, particularly in terms of reducing the uncertainty in the economic model. Any new randomised controlled trials should consider including a sham non-invasive ventilation arm and/or a higher- and lower-pressure arm. Individual participant data analyses may help to determine whether or not there are any patient characteristics or equipment settings that are predictive of a benefit of NIV and to establish optimum time points for starting (and potentially discounting) NIV.Study registrationThis study is registered as PROSPERO CRD42012003286.FundingThe National Institute for Health Research Health Technology Assessment programme.

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.

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