scholarly journals Immunosuppressive therapy for kidney transplantation in children and adolescents: systematic review and economic evaluation

2016 ◽  
Vol 20 (61) ◽  
pp. 1-324 ◽  
Author(s):  
Marcela Haasova ◽  
Tristan Snowsill ◽  
Tracey Jones-Hughes ◽  
Louise Crathorne ◽  
Chris Cooper ◽  
...  
Keyword(s):  
Kidney Transplantation ◽  
Economic Evaluation ◽  
Cost Effectiveness ◽  
Acute Rejection ◽  
Web Of Science ◽  
Graft Function ◽  
Clinical Effectiveness ◽  
Cost Effective ◽  
Controlled Trials ◽  
Online Library

BackgroundEnd-stage renal disease is a long-term irreversible decline in kidney function requiring kidney transplantation, haemodialysis or peritoneal dialysis. The preferred option is kidney transplantation followed by induction and maintenance immunosuppressive therapy to reduce the risk of kidney rejection and prolong graft survival.ObjectivesTo systematically review and update the evidence for the clinical effectiveness and cost-effectiveness of basiliximab (BAS) (Simulect,®Novartis Pharmaceuticals) and rabbit antihuman thymocyte immunoglobulin (Thymoglobuline,®Sanofi) as induction therapy and immediate-release tacrolimus [Adoport®(Sandoz); Capexion®(Mylan); Modigraf®(Astellas Pharma); Perixis®(Accord Healthcare); Prograf®(Astellas Pharma); Tacni®(Teva); Vivadex®(Dexcel Pharma)], prolonged-release tacrolimus (Advagraf,®Astellas Pharma); belatacept (BEL) (Nulojix,®Bristol-Myers Squibb), mycophenolate mofetil (MMF) [Arzip®(Zentiva), CellCept®(Roche Products), Myfenax®(Teva), generic MMF is manufactured by Accord Healthcare, Actavis, Arrow Pharmaceuticals, Dr Reddy’s Laboratories, Mylan, Sandoz and Wockhardt], mycophenolate sodium, sirolimus (Rapamune,®Pfizer) and everolimus (Certican,®Novartis Pharmaceuticals) as maintenance therapy in children and adolescents undergoing renal transplantation.Data sourcesClinical effectiveness searches were conducted to 7 January 2015 in MEDLINE (via Ovid), EMBASE (via Ovid), Cochrane Central Register of Controlled Trials (via Wiley Online Library) and Web of Science [via Institute for Scientific Information (ISI)], Cochrane Database of Systematic Reviews, Database of Abstracts of Reviews of Effects and Health Technology Assessment (HTA) (The Cochrane Library via Wiley Online Library) and Health Management Information Consortium (via Ovid). Cost-effectiveness searches were conducted to 15 January 2015 using a costs or economic literature search filter in MEDLINE (via Ovid), EMBASE (via Ovid), NHS Economic Evaluation Databases (via Wiley Online Library), Web of Science (via ISI), Health Economic Evaluations Database (via Wiley Online Library) and EconLit (via EBSCOhost).Review methodsTitles and abstracts were screened according to predefined inclusion criteria, as were full texts of identified studies. Included studies were extracted and quality appraised. Data were meta-analysed when appropriate. A new discrete time state transition economic model (semi-Markov) was developed; graft function, and incidences of acute rejection and new-onset diabetes mellitus were used to extrapolate graft survival. Recipients were assumed to be in one of three health states: functioning graft, graft loss or death.ResultsThree randomised controlled trials (RCTs) and four non-RCTs were included. The RCTs only evaluated BAS and tacrolimus (TAC). No statistically significant differences in key outcomes were found between BAS and placebo/no induction. Statistically significantly higher graft function (p < 0.01) and less biopsy-proven acute rejection (odds ratio 0.29, 95% confidence interval 0.15 to 0.57) was found between TAC and ciclosporin (CSA). Only one cost-effectiveness study was identified, which informed NICE guidance TA99. BAS [with TAC and azathioprine (AZA)] was predicted to be cost-effective at £20,000–30,000 per quality-adjusted life year (QALY) versus no induction (BAS was dominant). BAS (with CSA and MMF) was not predicted to be cost-effective at £20,000–30,000 per QALY versus no induction (BAS was dominated). TAC (with AZA) was predicted to be cost-effective at £20,000–30,000 per QALY versus CSA (TAC was dominant). A model based on adult evidence suggests that at a cost-effectiveness threshold of £20,000–30,000 per QALY, BAS and TAC are cost-effective in all considered combinations; MMF was also cost-effective with CSA but not TAC.LimitationsThe RCT evidence is very limited; analyses comparing all interventions need to rely on adult evidence.ConclusionsTAC is likely to be cost-effective (vs. CSA, in combination with AZA) at £20,000–30,000 per QALY. Analysis based on one RCT found BAS to be dominant, but analysis based on another RCT found BAS to be dominated. BAS plus TAC and AZA was predicted to be cost-effective at £20,000–30,000 per QALY when all regimens were compared using extrapolated adult evidence. High-quality primary effectiveness research is needed. The UK Renal Registry could form the basis for a prospective primary study.Study registrationThis study is registered as PROSPERO CRD42014013544.FundingThe National Institute for Health Research HTA programme.

scholarly journals Immunosuppressive therapy for kidney transplantation in adults: a systematic review and economic model

2016 ◽  
Vol 20 (62) ◽  
pp. 1-594 ◽  
Author(s):  
Tracey Jones-Hughes ◽  
Tristan Snowsill ◽  
Marcela Haasova ◽  
Helen Coelho ◽  
Louise Crathorne ◽  
...  
Keyword(s):  
Kidney Transplantation ◽  
Cost Effectiveness ◽  
Maintenance Therapy ◽  
Web Of Science ◽  
Graft Loss ◽  
Clinical Effectiveness ◽  
Cost Effective ◽  
Health Technology ◽  
Controlled Trials ◽  
Online Library

BackgroundEnd-stage renal disease is a long-term irreversible decline in kidney function requiring renal replacement therapy: kidney transplantation, haemodialysis or peritoneal dialysis. The preferred option is kidney transplantation, followed by immunosuppressive therapy (induction and maintenance therapy) to reduce the risk of kidney rejection and prolong graft survival.ObjectivesTo review and update the evidence for the clinical effectiveness and cost-effectiveness of basiliximab (BAS) (Simulect®, Novartis Pharmaceuticals UK Ltd) and rabbit anti-human thymocyte immunoglobulin (rATG) (Thymoglobulin®, Sanofi) as induction therapy, and immediate-release tacrolimus (TAC) (Adoport®, Sandoz; Capexion®, Mylan; Modigraf®, Astellas Pharma; Perixis®, Accord Healthcare; Prograf®, Astellas Pharma; Tacni®, Teva; Vivadex®, Dexcel Pharma), prolonged-release tacrolimus (Advagraf®Astellas Pharma), belatacept (BEL) (Nulojix®, Bristol-Myers Squibb), mycophenolate mofetil (MMF) (Arzip®, Zentiva; CellCept®, Roche Products; Myfenax®, Teva), mycophenolate sodium (MPS) (Myfortic®, Novartis Pharmaceuticals UK Ltd), sirolimus (SRL) (Rapamune®, Pfizer) and everolimus (EVL) (Certican®, Novartis) as maintenance therapy in adult renal transplantation.MethodsClinical effectiveness searches were conducted until 18 November 2014 in MEDLINE (via Ovid), EMBASE (via Ovid), Cochrane Central Register of Controlled Trials (via Wiley Online Library) and Web of Science (via ISI), Cochrane Database of Systematic Reviews, Database of Abstracts of Reviews of Effects and Health Technology Assessment (The Cochrane Library via Wiley Online Library) and Health Management Information Consortium (via Ovid). Cost-effectiveness searches were conducted until 18 November 2014 using a costs or economic literature search filter in MEDLINE (via Ovid), EMBASE (via Ovid), NHS Economic Evaluation Database (via Wiley Online Library), Web of Science (via ISI), Health Economic Evaluations Database (via Wiley Online Library) and the American Economic Association’s electronic bibliography (via EconLit, EBSCOhost). Included studies were selected according to predefined methods and criteria. A random-effects model was used to analyse clinical effectiveness data (odds ratios for binary data and mean differences for continuous data). Network meta-analyses were undertaken within a Bayesian framework. A new discrete time–state transition economic model (semi-Markov) was developed, with acute rejection, graft function (GRF) and new-onset diabetes mellitus used to extrapolate graft survival. Recipients were assumed to be in one of three health states: functioning graft, graft loss or death.ResultsEighty-nine randomised controlled trials (RCTs), of variable quality, were included. For induction therapy, no treatment appeared more effective than another in reducing graft loss or mortality. Compared with placebo/no induction, rATG and BAS appeared more effective in reducing biopsy-proven acute rejection (BPAR) and BAS appeared more effective at improving GRF. For maintenance therapy, no treatment was better for all outcomes and no treatment appeared most effective at reducing graft loss. BEL + MMF appeared more effective than TAC + MMF and SRL + MMF at reducing mortality. MMF + CSA (ciclosporin), TAC + MMF, SRL + TAC, TAC + AZA (azathioprine) and EVL + CSA appeared more effective than CSA + AZA and EVL + MPS at reducing BPAR. SRL + AZA, TAC + AZA, TAC + MMF and BEL + MMF appeared to improve GRF compared with CSA + AZA and MMF + CSA. In the base-case deterministic and probabilistic analyses, BAS, MMF and TAC were predicted to be cost-effective at £20,000 and £30,000 per quality-adjusted life-year (QALY). When comparing all regimens, only BAS + TAC + MMF was cost-effective at £20,000 and £30,000 per QALY.LimitationsFor included trials, there was substantial methodological heterogeneity, few trials reported follow-up beyond 1 year, and there were insufficient data to perform subgroup analysis. Treatment discontinuation and switching were not modelled.Future workHigh-quality, better-reported, longer-term RCTs are needed. Ideally, these would be sufficiently powered for subgroup analysis and include health-related quality of life as an outcome.ConclusionOnly a regimen of BAS induction followed by maintenance with TAC and MMF is likely to be cost-effective at £20,000–30,000 per QALY.Study registrationThis study is registered as PROSPERO CRD42014013189.FundingThe National Institute for Health Research Health Technology Assessment programme.

scholarly journals Adjunctive colposcopy technologies for examination of the uterine cervix – DySIS, LuViva Advanced Cervical Scan and Niris Imaging System: a systematic review and economic evaluation

2013 ◽  
Vol 17 (8) ◽  
pp. i-239 ◽  
Author(s):  
R Wade ◽  
E Spackman ◽  
M Corbett ◽  
S Walker ◽  
K Light ◽  
...  
Keyword(s):  
Economic Evaluation ◽  
Cost Effectiveness ◽  
Citation Index ◽  
Imaging System ◽  
Cervical Screening ◽  
Clinical Effectiveness ◽  
Health Technology ◽  
Risk Of Bias ◽  
Controlled Trials ◽  
Higher Sensitivity

BackgroundWomen in England (aged 25–64 years) are invited for cervical screening every 3–5 years to assess for cervical intraepithelial neoplasia (CIN) or cancer. CIN is a term describing abnormal changes in the cells of the cervix, ranging from CIN1 to CIN3, which is precancerous. Colposcopy is used to visualise the cervix. Three adjunctive colposcopy technologies for examination of the cervix have been included in this assessment: Dynamic Spectral Imaging System (DySIS), the LuViva Advanced Cervical Scan and the Niris Imaging System.ObjectiveTo determine the clinical effectiveness and cost-effectiveness of adjunctive colposcopy technologies for examination of the uterine cervix for patients referred for colposcopy through the NHS Cervical Screening Programme.Data sourcesSixteen electronic databases [Allied and Complementary Medicine Database (AMED), BIOSIS Previews, Cochrane Database of Systematic Reviews (CDSR), Cochrane Central Register of Controlled Trials (CENTRAL), Cumulative Index to Nursing and Allied Health Literature (CINAHL), Database of Abstracts of Reviews of Effects (DARE), EMBASE, Health Management Information Consortium (HMIC), Health Technology Assessment (HTA) database; Inspec, Inside Conferences, MEDLINE, NHS Economic Evaluation Database (NHS EED), PASCAL, Science Citation Index Expanded (SCIE) and Science Citation Index (SCI) – Conference Proceedings], and two clinical trial registries [ClinicalTrials.gov and Current Controlled Trials (CCT)] were searched to September–October 2011.Review methodsStudies comparing DySIS, LuViva or Niris with conventional colposcopy were sought; a narrative synthesis was undertaken. A decision-analytic model was developed, which measured outcomes in terms of quality-adjusted life-years (QALYs) and costs were evaluated from the perspective of the NHS and Personal Social Services with a time horizon of 50 years.ResultsSix studies were included: two studies of DySIS, one study of LuViva and three studies of Niris. The DySIS studies were well reported and had a low risk of bias; they found higher sensitivity with DySIS (both the DySISmap alone and in combination with colposcopy) than colposcopy alone for identifying CIN2+ disease, although specificity was lower with DySIS. The studies of LuViva and Niris were poorly reported and had limitations, which indicated that their results were subject to a high risk of bias; the results of these studies cannot be considered reliable. The base-case cost-effectiveness analysis suggests that both DySIS treatment options are less costly and more effective than colposcopy alone in the overall weighted population; these results were robust to the ranges tested in the sensitivity analysis. DySISmap alone was more costly and more effective in several of the referral groups but the incremental cost-effectiveness ratio (ICER) was never higher than £1687 per QALY. DySIS plus colposcopy was less costly and more effective in all reasons for referral. Only indicative analyses were carried out on Niris and LuViva and no conclusions could be made on their cost-effectiveness.LimitationsThe assessment is limited by the available evidence on the new technologies, natural history of the disease area and current treatment patterns.ConclusionsDySIS, particularly in combination with colposcopy, has higher sensitivity than colposcopy alone. There is no reliable evidence on the clinical effectiveness of LuViva and Niris. DySIS plus colposcopy appears to be less costly and more effective than both the DySISmap alone and colposcopy alone; these results were robust to the sensitivity analyses undertaken. Given the lack of reliable evidence on LuViva and Niris, no conclusions on their potential cost-effectiveness can be drawn. There is some uncertainty about how generalisable these findings will be to the population of women referred for colposcopy in the future, owing to the introduction of the human papillomavirus (HPV) triage test and uptake of the HPV vaccine.Study registrationPROSPERO Record CRD42011001614.FundingThe National Institute for Health Research Health Technology Assessment programme.

Clinical effectiveness and cost-effectiveness of implantable cardioverter defibrillators for arrhythmias: A systematic review and economic evaluation

2007 ◽  
Vol 23 (1) ◽  
pp. 63-70 ◽  
Author(s):  
Jackie Bryant ◽  
Hakan Brodin ◽  
Emma Loveman ◽  
Andrew Clegg
Keyword(s):  
Systematic Review ◽  
Cost Effectiveness ◽  
Cardiac Death ◽  
Clinical Effectiveness ◽  
Cost Effective ◽  
Controlled Trials ◽  
Implantable Cardioverter Defibrillators ◽  
Inclusion Criteria ◽  
Randomized Controlled ◽  
Cardioverter Defibrillators

Objectives:The clinical effectiveness and cost-effectiveness of implantable cardioverter defibrillators (ICD) for arrhythmias was assessed.Methods:A systematic review of the literature of systematic reviews and randomized controlled trials that reported mortality outcomes associated with implantable cardioverter defibrillators compared with antiarrhythmic drug therapy in people at risk of sudden cardiac death due to arrhythmias was undertaken. Economic evaluations were also sought. Inclusion criteria, data extraction, and quality assessment were undertaken by standard methodology. A decision analytic model was constructed using best available evidence to determine cost-effectiveness in a UK setting.Results:Eight randomized controlled trials, two systematic reviews, and a meta-analysis met the inclusion criteria and were of variable quality. Evidence suggests that ICDs reduce mortality in both secondary and primary prevention, although the magnitude of benefit depends on baseline risk for sudden cardiac death. Incremental cost per quality-adjusted life year ranged from £52,000 ($98,000) to over £200,000 ($379,000), depending on mortality risk and assumptions made.Conclusions:Evidence suggests that ICDs reduce total mortality but may be cost-effective only in some subgroups of patients at high risk of ventricular arrhythmias. Further research is needed on risk stratification of patients in whom ICDs are most likely to be clinically and cost-effective.

scholarly journals The clinical effectiveness and cost-effectiveness of open mesh repairs in adults presenting with a clinically diagnosed primary unilateral inguinal hernia who are operated in an elective setting: systematic review and economic evaluation

2015 ◽  
Vol 19 (92) ◽  
pp. 1-142 ◽  
Author(s):  
Pawana Sharma ◽  
Dwayne Boyers ◽  
Neil Scott ◽  
Rodolfo Hernández ◽  
Cynthia Fraser ◽  
...  
Keyword(s):  
Inguinal Hernia ◽  
Economic Evaluation ◽  
Cost Effectiveness ◽  
Mesh Repair ◽  
Clinical Effectiveness ◽  
Risk Of Bias ◽  
Controlled Trials ◽  
Unilateral Inguinal Hernia ◽  
Meta Analyses ◽  
Open Mesh

BackgroundsCurrent open mesh techniques for inguinal hernia repair have shown similar recurrence rates. However, chronic pain has been associated with Lichtenstein mesh repair, the most common surgical procedure for inguinal hernia in the UK. The position of the mesh is probably an important factor. The Lichtenstein method requires dissection of the inguinal wall and fixation of the mesh. In contrast, in the open preperitoneal approach the mesh is placed in the preperitoneal space and held in place with intra-abdominal pressure. Currently, there is no consensus regarding the best open approach for repair of inguinal hernia.ObjectivesTo determine the clinical effectiveness and cost-effectiveness of open preperitoneal mesh repair compared with Lichtenstein mesh repair in adults presenting with a clinically diagnosed primary unilateral inguinal hernia.Data sourcesWe searched major electronic databases (e.g. MEDLINE, MEDLINE In-Process & Other Non-Indexed, EMBASE, Cochrane Controlled Trials Register) from inception to November 2014 and contacted experts in the field.Review methodsEvidence was considered from randomised controlled trials (RCTs) that compared open preperitoneal mesh repair with Lichtenstein mesh repair for the treatment of inguinal hernia. Two reviewers independently selected studies for inclusion. One reviewer completed data extraction and assessed risk of bias for included studies, and two reviewers independently cross-checked the details extracted. Meta-analyses techniques were used to combine results from included studies. A Markov model was developed to assess the cost-effectiveness of open mesh procedures from a NHS health services perspective over a 25-year time horizon.ResultsTwelve RCTs involving 1568 participants were included. Participants who underwent open preperitoneal mesh repair returned to work and normal activities significantly earlier than those who underwent Lichtenstein mesh repair [mean difference –1.49 days, 95% confidence interval (CI) –2.78 to –0.20 days]. Although no significant differences were observed between the two open approaches for incidence of pain [risk ratio (RR) 0.50, 95% CI 0.20 to 1.27], numbness (RR 0.48, 95% CI 0.15 to 1.56), recurrences (Peto odds ratio 0.76, 95% CI 0.38 to 1.52) or postoperative complications, fewer events were generally reported after open preperitoneal mesh repair. The results of the economic evaluation indicate that the open preperitoneal mesh repair was £256 less costly and improved health outcomes by 0.041 quality-adjusted life-years (QALYs) compared with Lichtenstein mesh repair. The open preperitoneal procedure was the most efficient and dominant treatment strategy with a high (> 98%) probability of being cost-effectiveness for the NHS at a willingness to pay of £20,000 for a QALY. Results were robust to a range of sensitivity analyses. However, the magnitude of cost saving or QALY gain was sensitive to some model assumptions.LimitationsOverall, the included trials were of small sample size (mean 130.7 participants) and at high or unclear risk of bias. Meta-analyses results demonstrated significant statistical heterogeneity for most of the assessed outcomes.ConclusionsOpen preperitoneal mesh repair appears to be a safe and efficacious alternative to Lichtenstein mesh repair. Further research is required to determine the long-term effects of these surgical procedures as well as the most effective open preperitoneal repair technique in terms of both clinical efficacy and costs.Study registrationThis study is registered as PROSPERO CRD42014013510.FundingThe National Institute for Health Research Health Technology Assessment programme.

scholarly journals Topotecan, pegylated liposomal doxorubicin hydrochloride, paclitaxel, trabectedin and gemcitabine for advanced recurrent or refractory ovarian cancer: a systematic review and economic evaluation

2015 ◽  
Vol 19 (7) ◽  
pp. 1-480 ◽  
Author(s):  
Steven J Edwards ◽  
Samantha Barton ◽  
Elizabeth Thurgar ◽  
Nicola Trevor
Keyword(s):  
Ovarian Cancer ◽  
Cost Effectiveness ◽  
Liposomal Doxorubicin ◽  
Pegylated Liposomal Doxorubicin ◽  
Clinical Effectiveness ◽  
Cost Effective ◽  
Economic Evaluations ◽  
Controlled Trials ◽  
Platinum Sensitive ◽  
Additional Qaly

BackgroundOvarian cancer is the fifth most common cancer in the UK, and the fourth most common cause of cancer death. Of those people successfully treated with first-line chemotherapy, 55–75% will relapse within 2 years. At this time, it is uncertain which chemotherapy regimen is more clinically effective and cost-effective for the treatment of recurrent, advanced ovarian cancer.ObjectivesTo determine the comparative clinical effectiveness and cost-effectiveness of topotecan (Hycamtin®, GlaxoSmithKline), pegylated liposomal doxorubicin hydrochloride (PLDH; Caelyx®, Schering-Plough), paclitaxel (Taxol®, Bristol-Myers Squibb), trabectedin (Yondelis®, PharmaMar) and gemcitabine (Gemzar®, Eli Lilly and Company) for the treatment of advanced, recurrent ovarian cancer.Data sourcesElectronic databases (MEDLINE®, EMBASE, Cochrane Central Register of Controlled Trials, Health Technology Assessment database, NHS Economic Evaluations Database) and trial registries were searched, and company submissions were reviewed. Databases were searched from inception to May 2013.MethodsA systematic review of the clinical and economic literature was carried out following standard methodological principles. Double-blind, randomised, placebo-controlled trials, evaluating topotecan, PLDH, paclitaxel, trabectedin and gemcitabine, and economic evaluations were included. A network meta-analysis (NMA) was carried out. A de novo economic model was developed.ResultsFor most outcomes measuring clinical response, two networks were constructed: one evaluating platinum-based regimens and one evaluating non-platinum-based regimens. In people with platinum-sensitive disease, NMA found statistically significant benefits for PLDH plus platinum, and paclitaxel plus platinum for overall survival (OS) compared with platinum monotherapy. PLDH plus platinum significantly prolonged progression-free survival (PFS) compared with paclitaxel plus platinum. Of the non-platinum-based treatments, PLDH monotherapy and trabectedin plus PLDH were found to significantly increase OS, but not PFS, compared with topotecan monotherapy. In people with platinum-resistant/-refractory (PRR) disease, NMA found no statistically significant differences for any treatment compared with alternative regimens in OS and PFS. Economic modelling indicated that, for people with platinum-sensitive disease and receiving platinum-based therapy, the estimated probabilistic incremental cost-effectiveness ratio [ICER; incremental cost per additional quality-adjusted life-year (QALY)] for paclitaxel plus platinum compared with platinum was £24,539. Gemcitabine plus carboplatin was extendedly dominated, and PLDH plus platinum was strictly dominated. For people with platinum-sensitive disease and receiving non-platinum-based therapy, the probabilistic ICERs associated with PLDH compared with paclitaxel, and trabectedin plus PLDH compared with PLDH, were estimated to be £25,931 and £81,353, respectively. Topotecan was strictly dominated. For people with PRR disease, the probabilistic ICER associated with topotecan compared with PLDH was estimated to be £324,188. Paclitaxel was strictly dominated.LimitationsAs platinum- and non-platinum-based treatments were evaluated separately, the comparative clinical effectiveness and cost-effectiveness of these regimens is uncertain in patients with platinum-sensitive disease.ConclusionsFor platinum-sensitive disease, it was not possible to compare the clinical effectiveness and cost-effectiveness of platinum-based therapies with non-platinum-based therapies. For people with platinum-sensitive disease and treated with platinum-based therapies, paclitaxel plus platinum could be considered cost-effective compared with platinum at a threshold of £30,000 per additional QALY. For people with platinum-sensitive disease and treated with non-platinum-based therapies, it is unclear whether PLDH would be considered cost-effective compared with paclitaxel at a threshold of £30,000 per additional QALY; trabectedin plus PLDH is unlikely to be considered cost-effective compared with PLDH. For patients with PRR disease, it is unlikely that topotecan would be considered cost-effective compared with PLDH. Randomised controlled trials comparing platinum with non-platinum-based treatments might help to verify the comparative effectiveness of these regimens.Study registrationThis study is registered as PROSPERO CRD42013003555.FundingThe National Institute for Health Research Health Technology Assessment programme.

Dose-escalation strategy in refractory metastatic colorectal cancer: A change in terms of cost-effectiveness

2021 ◽  
pp. 107815522199254
Author(s):  
Jacopo Giuliani ◽  
Francesco Fiorica ◽  
Giovanni Ponturo ◽  
Maurizio Azzurro ◽  
Andrea Ruzzenente ◽  
...  
Keyword(s):  
Colorectal Cancer ◽  
Cost Effectiveness ◽  
Metastatic Colorectal Cancer ◽  
Dose Escalation ◽  
Lower Cost ◽  
Cost Effective ◽  
Phase Iii ◽  
Controlled Trials ◽  
Pivotal Phase ◽  
Randomized Controlled

The analysis was conducted to assess the pharmacological costs of regorafenib and trifluridine/tipiracil in the treatment of refractory metastatic colorectal cancer (mCRC). Pivotal phase III randomized controlled trials (RCTs) of regorafenib and trifluridine/tipiracil in the treatment of refractory mCRC were considered. We have also considered the ReDOS trial, in order to verify if the dose-escalation strategy (practice changing for regorafenib) could influences the results. Differences in OS (expressed in months) between the different arms were calculated and compared with the pharmacological costs (at the Pharmacy of our Hospital and expressed in euros (€)) needed to get one month of OS. Trifluridine/tipiracil resulted the less expensive, with 1167.50 €per month OS-gained. The ReDOS trial further reduce costs with 510.41 €per month OS-gained in favour of regorafenib with the escalation-dose strategy. Both regorafenib and trifluridine/tipiracil can be considered economically sustainable treatments for refractory mCRC, apparently with a lower cost of trifluridine/tipiracil. The adoption of a dose-escalation strategy (ReDOS trial) could reverse the situation making regorafenib more cost-effective than trifluridine/tipiracil.

scholarly journals Early invasive strategy in senior patients with non-ST-segment elevation myocardial infarction: is it cost-effective? - a decision-analytic model and value of information analysis

BMJ Open ◽  
2019 ◽  
Vol 9 (9) ◽  
pp. e030678 ◽  
Author(s):  
Julija Simpson ◽  
Mehdi Javanbakht ◽  
Luke Vale
Keyword(s):  
Myocardial Infarction ◽  
Cost Effectiveness ◽  
Elderly Patients ◽  
Value Of Information ◽  
Clinical Effectiveness ◽  
Cost Effective ◽  
Information Analysis ◽  
Invasive Strategy ◽  
Early Invasive Strategy

BackgroundNon-ST-elevation myocardial infarction (NSTEMI) is the most common type of heart attack in the UK and it is becoming increasingly prevalent among older people. An early invasive treatment strategy may be effective and cost-effective for treating NSTEMI but evidence is currently unclear.ObjectivesTo assess the cost-effectiveness of the early invasive strategy versus medical management in elderly patients with NSTEMI and to provide guidance for future research in this area.MethodsA long-term Markov state transition model was developed. Model inputs were systematically derived from a number of sources most appropriate to a UK relevant analysis, such as published studies and national routine data. Costs were estimated from the perspective of National Health Service and Personal Social Services. The model was developed using TreeAge Pro software. Based on a probabilistic sensitivity analysis, a value of information analysis was carried out to establish the value of decision uncertainty both overall and for specific input parameters.ResultsIn 2017 UK £, the incremental cost-effectiveness ratio of the early invasive strategy was £46 916 for each additional quality-adjusted life-year (QALY) gained, with a probability of being cost-effective of 23% at a cost-effectiveness threshold of £20 000/QALY. There was a considerable decision uncertainty with these results. The value of removing all this uncertainty was up to £1 920 000 annually. Most uncertainty related to clinical effectiveness parameters and the optimal study design to remove this uncertainty would be a randomised controlled trial.ConclusionBased on current evidence, the early invasive strategy is not likely to be cost-effective for elderly patients with NSTEMI. This conclusion should be interpreted with caution mainly due to the absence of NSTEMI-specific data and long-term clinical effectiveness estimates.

scholarly journals Diagnostic and therapeutic medical devices for safer blood management in cardiac surgery: systematic reviews, observational studies and randomised controlled trials

2017 ◽  
Vol 5 (17) ◽  
pp. 1-406 ◽  
Author(s):  
Gavin J Murphy ◽  
Andrew D Mumford ◽  
Chris A Rogers ◽  
Sarah Wordsworth ◽  
Elizabeth A Stokes ◽  
...  
Keyword(s):  
Systematic Review ◽  
Cardiac Surgery ◽  
Applied Research ◽  
Near Infrared ◽  
Predictive Accuracy ◽  
Clinical Effectiveness ◽  
Cost Effective ◽  
Controlled Trials ◽  
Red Cell ◽  
Red Cell Transfusion

BackgroundAnaemia, coagulopathic bleeding and transfusion are strongly associated with organ failure, sepsis and death following cardiac surgery.ObjectiveTo evaluate the clinical effectiveness and cost-effectiveness of medical devices used as diagnostic and therapeutic tools for the management of anaemia and bleeding in cardiac surgery.Methods and resultsWorkstream 1 – in the COagulation and Platelet laboratory Testing in Cardiac surgery (COPTIC) study we demonstrated that risk assessment using baseline clinical factors predicted bleeding with a high degree of accuracy. The results from point-of-care (POC) platelet aggregometry or viscoelastometry tests or an expanded range of laboratory reference tests for coagulopathy did not improve predictive accuracy beyond that achieved with the clinical risk score alone. The routine use of POC tests was not cost-effective. A systematic review concluded that POC-based algorithms are not clinically effective. We developed two new clinical risk prediction scores for transfusion and bleeding that are available as e-calculators. Workstream 2 – in the PAtient-SPecific Oxygen monitoring to Reduce blood Transfusion during heart surgery (PASPORT) trial and a systematic review we demonstrated that personalised near-infrared spectroscopy-based algorithms for the optimisation of tissue oxygenation, or as indicators for red cell transfusion, were neither clinically effective nor cost-effective. Workstream 3 – in the REDWASH trial we failed to demonstrate a reduction in inflammation or organ injury in recipients of mechanically washed red cells compared with standard (unwashed) red cells.LimitationsExisting studies evaluating the predictive accuracy or effectiveness of POC tests of coagulopathy or near-infrared spectroscopy were at high risk of bias. Interventions that alter red cell transfusion exposure, a common surrogate outcome in most trials, were not found to be clinically effective.ConclusionsA systematic assessment of devices in clinical use as blood management adjuncts in cardiac surgery did not demonstrate clinical effectiveness or cost-effectiveness. The contribution of anaemia and coagulopathy to adverse clinical outcomes following cardiac surgery remains poorly understood. Further research to define the pathogenesis of these conditions may lead to more accurate diagnoses, more effective treatments and potentially improved clinical outcomes.Study registrationCurrent Controlled Trials ISRCTN20778544 (COPTIC study) and PROSPERO CRD42016033831 (systematic review) (workstream 1); Current Controlled Trials ISRCTN23557269 (PASPORT trial) and PROSPERO CRD4201502769 (systematic review) (workstream 2); and Current Controlled Trials ISRCTN27076315 (REDWASH trial) (workstream 3).FundingThis project was funded by the National Institute for Health Research (NIHR) Programme Grants for Applied Research programme and will be published in full inProgramme Grants for Applied Research; Vol. 5, No. 17. See the NIHR Journals Library website for further project information.

Sign in / Sign up

Export Citation Format

Share Document