scholarly journals The cost-effectiveness of using performance-based financing to deliver the basic package of health services in Afghanistan

2020 ◽  
Vol 5 (9) ◽  
pp. e002381
Author(s):  
Ahmad S Salehi ◽  
Josephine Borghi ◽  
Karl Blanchet ◽  
Anna Vassall
Keyword(s):  
Cost Effectiveness ◽  
Health Services ◽  
Unit Cost ◽  
Health Benefit ◽  
Standard Of Care ◽  
Child Health Services ◽  
Randomised Control Trial ◽  
Economic Evaluations ◽  
Performance Based Financing ◽  
The Cost

Performance-based financing (PBF) is a mechanism to improve the quality and the utilisation of health benefit packages. There is a dearth of economic evaluations of PBF in the ‘real world’. Afghanistan implemented PBF between 2010 and 2015 and evaluated the programme using a pragmatic cluster-randomised control trial. We conducted a cost-effectiveness analysis of the PBF programme in Afghanistan, compared with the standard of care, from the provider payer’s perspective. The incremental cost-effectiveness ratio of PBF compared with the standard of care was US$1242 per disability-adjusted life year averted; not cost-effective when compared with an opportunity cost threshold of US$349. Incentive payments were the main contributor to PBF financial cost (70%) followed by data verification (23%), staff time (5%) and administration (2%). The unit cost per case of antenatal care (ANC), skilled birth attendance (SBA) and postnatal care (PNC) services in the standard of care was US$0.96 (95% CI 0.92–1.0), US$4.8 (95% CI 4.1–6.3) and US$1.3 (95% CI 1.2–1.4), respectively, whereas the cost of ANC, SBA and PNC services per case in PBF areas were US$4.72 (95% CI 4.68–5.7), US$48.5 (95% CI 48.0–52.5) and US$5.4 (95% CI 5.1–5.9), respectively. To conclude, our study found that PBF, as implemented in the Afghan context, was not the best use of funds to strengthen the delivery of maternal and child health services. The cost-effectiveness of alternative PBF designs needs to be appraised before using PBF at scale to support health benefit packages. PBF needs to be considered in the context of funding the range of constraints that inhibit health service performance improvement.

scholarly journals Systematic literature review of the economic burden of spinal muscular atrophy and economic evaluations of treatments

2021 ◽  
Vol 16 (1) ◽  
Author(s):  
Tamara Dangouloff ◽  
Camille Botty ◽  
Charlotte Beaudart ◽  
Laurent Servais ◽  
Mickaël Hiligsmann
Keyword(s):  
Cost Effectiveness ◽  
Spinal Muscular Atrophy ◽  
Annual Cost ◽  
Muscular Atrophy ◽  
Standard Of Care ◽  
Severe Form ◽  
Cost Of Care ◽  
Economic Evaluations ◽  
Life Years ◽  
The Cost

Abstract Background Spinal muscular atrophy (SMA) is a rare and devastating condition for which new disease-modifying treatments have recently been approved. Given the increasing importance of economic considerations in healthcare decision-making, this review summarizes the studies assessing the cost of SMA and economic evaluations of treatments. A systematic review of the literature in PubMed and Scopus up to 15 September 2020 was conducted according to PRISMA guidelines. Results Nine studies reporting the annual cost of care of patients with SMA and six evaluations of the cost-effectiveness of SMA treatments were identified. The average annual cost of SMA1, the most frequent and severe form in which symptoms appear before the age of 6 months were similar according to the different studies, ranged from $75,047 to $196,429 per year. The yearly costs for the forms of the later-onset form, called SMA2, SMA3, and SMA4, which were usually pooled in estimates of healthcare costs, were more variable, ranging from $27,157 to $82,474. The evaluations of cost-effectiveness of treatment compared nusinersen treatment against standard of care (n = 3), two treatments (nusinersen and onasemnogene abeparvovec) against each other and no drug treatment (n = 1), nusinersen versus onasemnogene abeparvovec (n = 1), and standard of care versus nusinersen with and without newborn screening (n = 1). The incremental cost-effectiveness ratio (ICER) of nusinersen compared to standard of care in SMA1 ranged from $210,095 to $1,150,455 per quality-adjusted life years (QALY) gained and that for onasemnogene abeparvovec ranged from $32,464 to $251,403. For pre-symptomatic patients, the ICER value ranged from $206,409 to $735,519. The ICERs for later-onset forms of SMA (2, 3 and 4) were more diverse ranging from $275,943 to $8,438,049. Conclusion This review confirms the substantial cost burden of standard of care for SMA patients and the high cost-effectiveness ratios of the approved drugs at the current price when delivered in post-symptomatic patients. Since few studies have been conducted so far, there is a need for further prospective and independent economic studies in pre- and post-symptomatic patients.

Extrapolation of Cost-Effectiveness Information to Local Settings

1998 ◽  
Vol 3 (2) ◽  
pp. 108-112 ◽  
Author(s):  
Stirling Bryan ◽  
Jackie Brown
Keyword(s):  
Cost Effectiveness ◽  
Unit Cost ◽  
Economic Evaluations ◽  
Local Policy ◽  
Local Setting ◽  
History Of ◽  
Sources Of Variation ◽  
Reducing Costs ◽  
The Cost ◽  
Aid Decision

Providers and purchasers of health care are increasingly looking to the results of economic evaluations for guidance when making their decisions. In this paper the authors argue that there are dangers involved in the naive and unthinking use of published cost-effectiveness information outside the setting in which the information was generated. In considering whether the results of a published study are likely to be relevant locally, decision-makers are encouraged to assess whether the values of the key parameters reported in the published study apply locally. The possible sources of variation are described: unit cost differences; differences in the prevalence, incidence or natural history of disease; and differences in the comparators. In situations where the only source of variation is that local unit costs are different, local values can be substituted in the published analysis and local cost-effectiveness results estimated. Where the other sources of variation exist, the decision-maker is required to make an assumption about the nature of the interaction between the sources of variation and the values of the cost-effectiveness parameters. Using an example, the authors argue that local threshold analysis can aid decision-making where the policy change being considered has a high probability either of increasing effectiveness or reducing costs. Without local reanalysis, there is a danger that local policy changes in line with the recommendations of published studies will promote inefficiency. Re-analysis in the local setting is, however, reliant on authors of economic evaluations being explicit about their methods and the comparators used in their analyses.

scholarly journals Cost-effectiveness of oral anticancer drugs and associated individualised dosing approaches in patients with cancer: protocol for a systematic review

BMJ Open ◽  
2021 ◽  
Vol 11 (8) ◽  
pp. e047173
Author(s):  
Madelé van Dyk ◽  
Norma Bulamu ◽  
Chelsea Boylan ◽  
Anna M Mc Laughlin ◽  
Ganessan Kichenadasse ◽  
...  
Keyword(s):  
Systematic Review ◽  
Economic Evaluation ◽  
Cost Effectiveness ◽  
Anticancer Drugs ◽  
Health Economic Evaluation ◽  
Health Benefit ◽  
Bibliographic Databases ◽  
Economic Evaluations ◽  
The Cost ◽  
Individualised Dosing

IntroductionOral anticancer drugs (OADs) have rapidly expanded with more than 70 OADs targeting several molecular targets. Many of the OADs exert an exposure–response relationship but still, a ‘one-size fits-all’ dose is used, ignoring interindividual variability. Several of these OADs share similar mechanisms of actions and thus target the same cancer and has resulted in a substantial research focus on comparing the health benefit of each. However, significantly less is known about the cost–benefit associated with OADs. This paper will provide a protocol to systematically review studies that have evaluated the cost-effectiveness of OADs and their associated individualised dosing interventions.Methods and analysisSystematic review methodology will be applied to identify, select and extract data from published economic evaluation (costs and outcomes/benefits) studies of OADs and their associated individualised dosing interventions. Bibliographic databases (eg, Ovid EMBASE, Ovid MEDLINE) will be used to perform the systematic literature search (between 1 January 2000 and October 2020). Only full economic evaluations will be included, but no restrictions on study outcomes will be applied. The quality of included primary studies will be assessed using the Consolidated Health Economic Evaluation Reporting Standards checklist for reporting economic evaluations. Studies with low-quality evidence will be excluded. A narrative synthesis of the results from the included studies will be undertaken, with a subgroup analysis where appropriate.Ethics and disseminationThis systematic review will not require ethics approval as there will not be any collection of primary data. Findings of this review will be disseminated through publications in peer-reviewed journals, presentations at workshops or conferences and sharing through a media release. Findings from this review will provide evidence to direct and inform policy-makers where cost-neutral strategies may be effective or where dose individualising strategies may be economically beneficial. Additionally, gaps will be identified in the current literature to inform future-related research.PROSPERO registration numberCRD42020218170.Electronic supplemental materialThe online version of this article contains supplemental material, which is available to authorised users.

scholarly journals Implementation of a performance-based financing scheme in Malawi and resulting externalities on the quality of care of non-incentivized services

2021 ◽  
Vol 21 (1) ◽  
Author(s):  
Stephan Brenner ◽  
Caterina Favaretti ◽  
Julia Lohmann ◽  
Jobiba Chinkhumba ◽  
Adamson S. Muula ◽  
...  
Keyword(s):  
Quality Of Care ◽  
Health Services ◽  
Antenatal Care ◽  
Service Provision ◽  
Child Health Services ◽  
Negative Externalities ◽  
Positive Externalities ◽  
Performance Based Financing ◽  
A Performance

Abstract Background Countries in Africa progressively implement performance-based financing schemes to improve the quality of care provided by maternal, newborn and child health services. Beyond its direct effects on service provision, evidence suggests that performance-based financing can also generate positive externalities on service utilization, such as increased use of those services that reached higher quality standards after effective scheme implementation. Little, however, is known about externalities generated within non-incentivized health services, such as positive or negative effects on the quality of services within the continuum of maternal care. Methods We explored whether a performance-based financing scheme in Malawi designed to improve the quality of childbirth service provision resulted positive or negative externalities on the quality of non-targeted antenatal care provision. This non-randomized controlled pre-post-test study followed the phased enrolment of facilities into a performance-based financing scheme across four districts over a two-year period. Effects of the scheme were assessed by various composite scores measuring facilities’ readiness to provide quality antenatal care, as well as the quality of screening, prevention, and education processes offered during observed antenatal care consultations. Results Our study did not identify any statistically significant effects on the quality of ANC provision attributable to the implemented performance-based financing scheme. Our findings therefore suggest not only the absence of positive externalities, but also the absence of any negative externalities generated within antenatal care service provision as a result of the scheme implementation in Malawi. Conclusions Prior research has shown that the Malawian performance-based financing scheme was sufficiently effective to improve the quality of incentivized childbirth service provision. Our findings further indicate that scheme implementation did not affect the quality of non-incentivized but clinically related antenatal care services. While no positive externalities could be identified, we also did not observe any negative externalities attributable to the scheme’s implementation. While performance-based incentives might be successful in improving targeted health care processes, they have limited potential in producing externalities – neither positive nor negative – on the provision quality of related non-incentivized services.

Marginal assessment of the cost and benefits of aphasia treatment: Evidence from community-based telerehabilitation treatment for aphasia

2021 ◽  
pp. 1357633X2098277
Author(s):  
Molly Jacobs ◽  
Patrick M Briley ◽  
Heather Harris Wright ◽  
Charles Ellis
Keyword(s):  
Cost Effectiveness ◽  
Economies Of Scale ◽  
Reference Group ◽  
Unit Cost ◽  
Community Based ◽  
Face To Face ◽  
Conduction Aphasia ◽  
Aphasia Treatment ◽  
The Cost ◽  
The Relationship

Introduction Few studies have reported information related to the cost-effectiveness of traditional face-to-face treatments for aphasia. The emergence and demand for telepractice approaches to aphasia treatment has resulted in an urgent need to understand the costs and cost-benefits of this approach. Methods Eighteen stroke survivors with aphasia completed community-based aphasia telerehabilitation treatment, utilizing the Language-Oriented Treatment (LOT) delivered via Webex videoconferencing program. Marginal benefits to treatment were calculated as the change in Western Aphasia Battery-Revised (WAB-R) score pre- and post-treatment and marginal cost of treatment was calculated as the relationship between change in WAB-R aphasia quotient (AQ) and the average cost per treatment. Controlling for demographic variables, Bayesian estimation evaluated the primary contributors to WAB-R change and assessed cost-effectiveness of treatment by aphasia type. Results Thirteen out of 18 participants experienced significant improvement in WAB-R AQ following telerehabilitation delivered therapy. Compared to anomic aphasia (reference group), those with conduction aphasia had relatively similar levels of improvement whereas those with Broca’s aphasia had smaller improvement. Those with global aphasia had the largest improvement. Each one-point of improvement cost between US$89 and US$864 for those who improved (mean = US$200) depending on aphasia type/severity. Discussion Individuals with severe aphasia may have the greatest gains per unit cost from treatment. Both improvement magnitude and the cost per unit of improvement were driven by aphasia type, severity and race. Economies of scale to aphasia treatment–cost may be minimized by treating a variety of types of aphasia at various levels of severity.

Modeling the cost-effectiveness of a new treatment for MS (natalizumab) compared with current standard practice in Sweden

2008 ◽  
Vol 14 (5) ◽  
pp. 679-690 ◽  
Author(s):  
G Kobelt ◽  
J Berg ◽  
P Lindgren ◽  
B Jonsson ◽  
L Stawiarz ◽  
...  
Keyword(s):  
Clinical Trial ◽  
Cost Effectiveness ◽  
Societal Perspective ◽  
Functional Disability ◽  
Health Benefit ◽  
Base Case ◽  
Current Standard ◽  
Disease Modifying Drugs ◽  
New Treatment ◽  
The Cost

Objective To estimate the cost-effectiveness of a new treatment (natalizumab) for multiple sclerosis (MS) compared with current standard therapy with disease-modifying drugs (DMDs) in Sweden. Methods A Markov model was constructed to illustrate disease progression based on functional disability (the Expanded Disability Status Scale (EDSS)). The effectiveness of natalizumab was based on a 2-year clinical trial in 942 patients (AFFIRM). The effectiveness of current DMDs was estimated from a matched sample of 512 patients in the Stockholm MS registry. Patients withdrawing from treatment were assumed to follow the disease course of 824 patients with relapsing–remitting disease at onset in the Ontario natural history cohort. Costs and utilities are based on a recent observational study in 1339 patients. All data sets were available at the patient level. Main results are presented from the societal perspective, over a 20-year time frame, in 2005 Euros (€1 = 9.25 SEK). Results In the base case, treatment with natalizumab was less expensive and more effective than treatment with current DMDs. When only healthcare costs were considered, the cost per quality-adjusted life year gained with natalizumab was €38 145. Results are sensitive only to the time horizon of the analysis and assumptions about effectiveness of natalizumab beyond the trial. Conclusions This cost-effectiveness analysis used registry data, cohort and observational studies to extrapolate the efficacy findings of natalizumab from the AFFIRM clinical trial to measure effectiveness in clinical practice. The analysis results suggest that for the population considered, natalizumab provides an additional health benefit at a similar cost to current DMDs from a societal perspective.

A Systematic Review of Cost-Effectiveness Analyses Alongside Randomised Controlled Trials of Acupuncture

2012 ◽  
Vol 30 (4) ◽  
pp. 273-285 ◽  
Author(s):  
Song-Yi Kim ◽  
Hyangsook Lee ◽  
Younbyoung Chae ◽  
Hi-Joon Park ◽  
Hyejung Lee
Keyword(s):  
Cost Effectiveness ◽  
Usual Care ◽  
Randomised Controlled Trials ◽  
Risk Of Bias ◽  
Economic Evaluations ◽  
Controlled Trials ◽  
German Studies ◽  
Life Years ◽  
Randomised Controlled ◽  
The Cost

Objective To summarise the evidence on the cost-effectiveness of acupuncture. Methods We identified full economic evaluations such as cost-effectiveness analysis (CEA), cost-utility analysis (CUA) and cost-benefit analysis (CBA) alongside randomised controlled trials (RCTs) that assessed the consequences and costs of acupuncture for any medical condition. Eleven electronic databases were searched up to March 2011 without language restrictions. Eligible RCTs were assessed using the Cochrane criteria for risk of bias and a modified version of the checklist for economic evaluation. The general characteristics and the results of each economic analysis such as incremental cost-effectiveness ratios (ICERs) were extracted. Results Of 17 included studies, nine were CUAs that measured quality-adjusted life years (QALYs) and eight were CEAs that assessed effectiveness of acupuncture based on improvements in clinical symptoms. All CUAs showed that acupuncture with or without usual care was cost-effective compared with waiting list control or usual care alone, with ICERs ranging from ¢3011/QALY (dysmenorrhoea) to ¢22 298/QALY (allergic rhinitis) in German studies, and from £3855/QALY (osteoarthritis) to £9951/QALY (headache) in UK studies. In the CEAs, acupuncture was beneficial at a relatively low cost in six European and Asian studies. All CUAs were well-designed with a low risk of bias, but this was not the case for CEAs. Conclusions Overall, this review demonstrates the cost-effectiveness of acupuncture. Despite such promising results, any generalisation of these results needs to be made with caution given the diversity of diseases and the different status of acupuncture in the various countries.

scholarly journals Cost-Effectiveness of Peer-Delivered HIV Self-Tests for MSM in Uganda

2021 ◽  
Vol 9 ◽  
Author(s):  
Stephen Okoboi ◽  
Barbara Castelnuovo ◽  
Jean-Pierre Van Geertruyden ◽  
Oucul Lazarus ◽  
Lung Vu ◽  
...  
Keyword(s):  
Cost Effectiveness ◽  
Incremental Cost ◽  
Hiv Testing ◽  
Cost Effective ◽  
Hiv Infections ◽  
Standard Of Care ◽  
Sub Saharan Africa ◽  
High Risk Population ◽  
Self Testing ◽  
The Cost

Introduction: Distribution of HIV self-testing (HIVST) kits through MSM peer networks is a novel and effective strategy to increase HIV testing coverage in this high-risk population. No study has evaluated the cost or cost effectiveness of peer distribution of HIVST strategies among MSM in sub-Saharan Africa.Methods: From June to August 2018, we conducted a pilot study of secondary MSM peer HIVST kit distribution at The AIDS Support Organization at Entebbe and Masaka. We used an ingredients approach to estimate the cost of MSM peer HIVST kit distribution relative to standard-of-care (SOC) hotspot testing using programme expenditure data reported in US dollars. The provider perspective was used to estimate incremental cost-effective ratios per HIV infection averted using the difference in HIV annual transmission rates between MSM with HIV who knew their status and were not virologically suppressed and MSM with HIV who did not know their status.Results: We enrolled 297 participants of whom 150 received MSM peer HIVST kit distribution (intervention group) and 147 received TASO standard of care HIV testing (control group). Provider cost for the intervention was $2,276 compared with $1,827 for SOC during the 3-month study period. Overall, the intervention resulted in higher HIV positivity yield (4.9 vs. 1.4%) and averted more HIV infections per quarter (0.364 vs. 0.104) compared with SOC. The cost per person tested was higher for the intervention compared to SOC ($15.90 vs. $12.40). Importantly, the cost per new HIV diagnosis ($325 vs. $914) and cost per transmission averted ($6,253 vs. $ 17,567) were lower for the intervention approach relative to SOC. The incremental cost per HIV transmission averted by the self-testing program was $1,727. The incremental cost to providers per additional HIV-positive person identified by the intervention was $147.30.Conclusion: The intervention strategy was cost-effective, and identified more undiagnosed HIV infections than SOC hotspot testing at a cost-effectiveness threshold of US $2,129. Secondary distribution of HIVST kits through peers should further be evaluated with longer duration aimed at diagnosing 95% of all persons with HIV by 2030; the first UNAIDS 95-95-95 target.

scholarly journals Cost-effectiveness of treatment optimisation with biomarkers for immunotherapy in solid tumours: a systematic review protocol

BMJ Open ◽  
2021 ◽  
Vol 11 (9) ◽  
pp. e048141
Author(s):  
Sara Mucherino ◽  
Valentina Lorenzoni ◽  
Valentina Orlando ◽  
Isotta Triulzi ◽  
Marzia Del Re ◽  
...  
Keyword(s):  
Systematic Review ◽  
Cost Effectiveness ◽  
Immune Checkpoint ◽  
Cost Utility ◽  
Solid Tumours ◽  
Economic Evaluations ◽  
Monetary Benefit ◽  
The Cost ◽  
Net Monetary Benefit

IntroductionThe combination of biomarkers and drugs is the subject of growing interest both from regulators, physicians and companies. This study protocol of a systematic review is aimed to describe available literature evidences about the cost-effectiveness, cost-utility or net-monetary benefit of the use of biomarkers in solid tumour as tools for customising immunotherapy to identify what further research needs.Methods and analysisA systematic review of the literature will be carried out according to the Preferred Reporting Items for Systematic Reviews and Meta-Analyses statement guidelines. PubMed and Embase will be queried from June 2010 to June 2021. The PICOS model will be applied: target population (P) will be patients with solid tumours treated with immune checkpoint inhibitors (ICIs); the interventions (I) will be test of the immune checkpoint predictive biomarkers; the comparator (C) will be any other targeted or non-targeted therapy; outcomes (O) evaluated will be health economic and clinical implications assessed in terms of incremental cost-effectiveness ratio, net health benefit, net monetary benefit, life years gained, quality of life, etc; study (S) considered will be economic evaluations reporting cost-effectiveness analysis, cost-utility analysis, net-monetary benefit. The quality of the evidence will be graded according to Grading of Recommendations Assessment, Development and Evaluation.Ethics and disseminationThis systematic review will assess the cost-effectiveness implications of using biomarkers in the immunotherapy with ICIs, which may help to understand whether this approach is widespread in real clinical practice. This research is exempt from ethics approval because the work is carried out on published documents. We will disseminate this protocol in a related peer-reviewed journal.PROSPERO registration numberCRD42020201549.

scholarly journals Lenvatinib and sorafenib for differentiated thyroid cancer after radioactive iodine: a systematic review and economic evaluation

2020 ◽  
Vol 24 (2) ◽  
pp. 1-180 ◽  
Author(s):  
Nigel Fleeman ◽  
Rachel Houten ◽  
Adrian Bagust ◽  
Marty Richardson ◽  
Sophie Beale ◽  
...  
Keyword(s):  
Thyroid Cancer ◽  
Cost Effectiveness ◽  
Systematic Reviews ◽  
Observational Studies ◽  
Differentiated Thyroid Cancer ◽  
Radioactive Iodine ◽  
Clinical Effectiveness ◽  
Economic Evaluations ◽  
Base Case ◽  
The Cost

Background Thyroid cancer is a rare cancer, accounting for only 1% of all malignancies in England and Wales. Differentiated thyroid cancer (DTC) accounts for ≈94% of all thyroid cancers. Patients with DTC often require treatment with radioactive iodine. Treatment for DTC that is refractory to radioactive iodine [radioactive iodine-refractory DTC (RR-DTC)] is often limited to best supportive care (BSC). Objectives We aimed to assess the clinical effectiveness and cost-effectiveness of lenvatinib (Lenvima®; Eisai Ltd, Hertfordshire, UK) and sorafenib (Nexar®; Bayer HealthCare, Leverkusen, Germany) for the treatment of patients with RR-DTC. Data sources EMBASE, MEDLINE, PubMed, The Cochrane Library and EconLit were searched (date range 1999 to 10 January 2017; searched on 10 January 2017). The bibliographies of retrieved citations were also examined. Review methods We searched for randomised controlled trials (RCTs), systematic reviews, prospective observational studies and economic evaluations of lenvatinib or sorafenib. In the absence of relevant economic evaluations, we constructed a de novo economic model to compare the cost-effectiveness of lenvatinib and sorafenib with that of BSC. Results Two RCTs were identified: SELECT (Study of [E7080] LEnvatinib in 131I-refractory differentiated Cancer of the Thyroid) and DECISION (StuDy of sorafEnib in loCally advanced or metastatIc patientS with radioactive Iodine-refractory thyrOid caNcer). Lenvatinib and sorafenib were both reported to improve median progression-free survival (PFS) compared with placebo: 18.3 months (lenvatinib) vs. 3.6 months (placebo) and 10.8 months (sorafenib) vs. 5.8 months (placebo). Patient crossover was high (≥ 75%) in both trials, confounding estimates of overall survival (OS). Using OS data adjusted for crossover, trial authors reported a statistically significant improvement in OS for patients treated with lenvatinib compared with those given placebo (SELECT) but not for patients treated with sorafenib compared with those given placebo (DECISION). Both lenvatinib and sorafenib increased the incidence of adverse events (AEs), and dose reductions were required (for > 60% of patients). The results from nine prospective observational studies and 13 systematic reviews of lenvatinib or sorafenib were broadly comparable to those from the RCTs. Health-related quality-of-life (HRQoL) data were collected only in DECISION. We considered the feasibility of comparing lenvatinib with sorafenib via an indirect comparison but concluded that this would not be appropriate because of differences in trial and participant characteristics, risk profiles of the participants in the placebo arms and because the proportional hazard assumption was violated for five of the six survival outcomes available from the trials. In the base-case economic analysis, using list prices only, the cost-effectiveness comparison of lenvatinib versus BSC yields an incremental cost-effectiveness ratio (ICER) per quality-adjusted life-year (QALY) gained of £65,872, and the comparison of sorafenib versus BSC yields an ICER of £85,644 per QALY gained. The deterministic sensitivity analyses show that none of the variations lowered the base-case ICERs to < £50,000 per QALY gained. Limitations We consider that it is not possible to compare the clinical effectiveness or cost-effectiveness of lenvatinib and sorafenib. Conclusions Compared with placebo/BSC, treatment with lenvatinib or sorafenib results in an improvement in PFS, objective tumour response rate and possibly OS, but dose modifications were required to treat AEs. Both treatments exhibit estimated ICERs of > £50,000 per QALY gained. Further research should include examination of the effects of lenvatinib, sorafenib and BSC (including HRQoL) for both symptomatic and asymptomatic patients, and the positioning of treatments in the treatment pathway. Study registration This study is registered as PROSPERO CRD42017055516. Funding The National Institute for Health Research Health Technology Assessment programme.

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