Assessing economics of treatments for gynecological cancer where clinical effectiveness meets value for money

2004 ◽  
Vol 14 (5) ◽  
pp. 762-771 ◽  
Author(s):  
B. Stoykova ◽  
R. Dowie ◽  
H. C. Kitchener
Keyword(s):  
Clinical Trials ◽  
Cost Effectiveness ◽  
Economic Analysis ◽  
Gynecological Cancer ◽  
Previous Treatment ◽  
Clinical Effectiveness ◽  
Decision Makers ◽  
Value For Money ◽  
Access To Treatment ◽  
Economic Methods

During the last 10 years, the management of gynecological cancer has been undergoing a great deal of change. This is due to a drive to reduce ineffective treatment and associated morbidity while at the same time maximizing the benefits of currently available treatment. The foundation for this approach has been high-quality clinical trials which have been performed in increasing numbers. These trials can provide strong evidence that treatments are equivalent or that a new drug adds superiority to previous treatment.The access that women have to the most effective forms of treatment often depends on the availability of healthcare resources and their affordability within the healthcare system. Healthcare decision makers increasingly require not just clinical effectiveness of treatments but also cost-effectiveness to be demonstrated. While health economic methods have been applied to many forms of cancer treatment and screening, there have been very few rigorous economic studies performed in gynecological cancer.This article discusses how economic analysis can be incorporated into clinical trials and how it can provide the sort of value for money determination that payers of healthcare are now requiring. Economic analysis may add a little to the cost of trials, but in the end, it may increase access to treatment by convincing decision makers of cost-effectiveness.

scholarly journals A systematic review and economic evaluation of adalimumab and dexamethasone for treating non-infectious intermediate uveitis, posterior uveitis or panuveitis in adults

2017 ◽  
Vol 21 (68) ◽  
pp. 1-170 ◽  
Author(s):  
Hazel Squires ◽  
Edith Poku ◽  
Inigo Bermejo ◽  
Katy Cooper ◽  
John Stevens ◽  
...  
Keyword(s):  
Clinical Trials ◽  
Cost Effectiveness ◽  
Current Practice ◽  
Clinical Effectiveness ◽  
Posterior Uveitis ◽  
Sensitivity Analyses ◽  
Dexamethasone Implant ◽  
Intermediate Uveitis ◽  
Sham Procedure ◽  
The Cost

BackgroundNon-infectious intermediate uveitis, posterior uveitis and panuveitis are a heterogeneous group of inflammatory eye disorders. Management includes local and systemic corticosteroids, immunosuppressants and biological drugs.ObjectivesTo evaluate the clinical effectiveness and cost-effectiveness of subcutaneous adalimumab (Humira®; AbbVie Ltd, Maidenhead, UK) and a dexamethasone intravitreal implant (Ozurdex®; Allergan Ltd, Marlow, UK) in adults with non-infectious intermediate uveitis, posterior uveitis or panuveitis.Data sourcesElectronic databases and clinical trials registries including MEDLINE, EMBASE, Cochrane Database of Systematic Reviews, Database of Abstracts of Reviews of Effects and the World Health Organization’s International Clinical Trials Registry Platform were searched to June 2016, with an update search carried out in October 2016.Review methodsReview methods followed published guidelines. A Markov model was developed to assess the cost-effectiveness of dexamethasone and adalimumab, each compared with current practice, from a NHS and Personal Social Services (PSS) perspective over a lifetime horizon, parameterised with published evidence. Costs and benefits were discounted at 3.5%. Substantial sensitivity analyses were undertaken.ResultsOf the 134 full-text articles screened, three studies (four articles) were included in the clinical effectiveness review. Two randomised controlled trials (RCTs) [VISUAL I (active uveitis) and VISUAL II (inactive uveitis)] compared adalimumab with placebo, with limited standard care also provided in both arms. Time to treatment failure (reduced visual acuity, intraocular inflammation, new vascular lesions) was longer in the adalimumab group than in the placebo group, with a hazard ratio of 0.50 [95% confidence interval (CI) 0.36 to 0.70;p < 0.001] in the VISUAL I trial and 0.57 (95% CI 0.39 to 0.84;p = 0.004) in the VISUAL II trial. The adalimumab group showed a significantly greater improvement than the placebo group in the 25-item Visual Function Questionnaire (VFQ-25) composite score in the VISUAL I trial (mean difference 4.20;p = 0.010) but not the VISUAL II trial (mean difference 2.12;p = 0.16). Some systemic adverse effects occurred more frequently with adalimumab than with placebo. One RCT [HURON (active uveitis)] compared a single 0.7-mg dexamethasone implant against a sham procedure, with limited standard care also provided in both arms. Dexamethasone provided significant benefits over the sham procedure at 8 and 26 weeks in the percentage of patients with a vitreous haze score of zero (p < 0.014), the mean best corrected visual acuity improvement (p ≤ 0.002) and the percentage of patients with a ≥ 5-point improvement in VFQ-25 score (p < 0.05). Raised intraocular pressure and cataracts occurred more frequently with dexamethasone than with the sham procedure. The incremental cost-effectiveness ratio (ICER) for one dexamethasone implant in one eye for a combination of patients with unilateral and bilateral uveitis compared with limited current practice, as per the HURON trial, was estimated to be £19,509 per quality-adjusted life-year (QALY) gained. The ICER of adalimumab for patients with mainly bilateral uveitis compared with limited current practice, as per the VISUAL trials, was estimated to be £94,523 and £317,547 per QALY gained in active and inactive uveitis respectively. Sensitivity analyses suggested that the rate of blindness has the biggest impact on the model results. The interventions may be more cost-effective in populations in which there is a greater risk of blindness.LimitationsThe clinical trials did not fully reflect clinical practice. Thirteen additional studies of clinically relevant comparator treatments were identified; however, network meta-analysis was not feasible. The model results are highly uncertain because of the limited evidence base.ConclusionsTwo RCTs of systemic adalimumab and one RCT of a unilateral, single dexamethasone implant showed significant benefits over placebo or a sham procedure. The ICERs for adalimumab were estimated to be above generally accepted thresholds for cost-effectiveness. The cost-effectiveness of dexamethasone was estimated to fall below standard thresholds. However, there is substantial uncertainty around the model assumptions. In future work, primary research should compare dexamethasone and adalimumab with current treatments over the long term and in important subgroups and consider how short-term improvements relate to long-term effects on vision.Study registrationThis study is registered as PROSPERO CRD42016041799.FundingThe National Institute for Health Research Health Technology Assessment programme.

scholarly journals A case of misalignment: the perspectives of local and national decision-makers on the implementation of psychological treatment by telephone in the Improving Access to Psychological Therapies Service

2019 ◽  
Vol 19 (1) ◽  
Author(s):  
Kelly Rushton ◽  
Claire Fraser ◽  
Judith Gellatly ◽  
Helen Brooks ◽  
Peter Bower ◽  
...  
Keyword(s):  
Focus Group ◽  
Best Practice ◽  
Psychological Treatment ◽  
Cost Savings ◽  
Clinical Effectiveness ◽  
Service Level ◽  
Patient Choice ◽  
Decision Makers ◽  
Normalisation Process Theory ◽  
Access To Treatment

Abstract Background Psychological treatment delivered by telephone is recommended by the National Institute for Health and Care Excellence (NICE) for mild to moderate depression and anxiety, and forms a key part of the Improving Access to Psychological Therapy (IAPT) programme in the UK. Despite evidence of clinical effectiveness, patient engagement is often not maintained and psychological wellbeing practitioners (PWPs) report lacking confidence and training to deliver treatment by telephone. This study aimed to explore the perspectives of professional decision makers (both local and national) on the barriers and facilitators to the implementation of telephone treatment in IAPT. Methods Sixteen semi-structured qualitative telephone interviews and one focus group were carried out with decision makers (n = 21) who were involved locally and nationally in policy, practice and research. The interviews and focus group were coded thematically, and then mapped onto the four core constructs of Normalisation Process Theory (NPT). Results The use of telephone for psychological treatment was universally recognised amongst participants as beneficial for improving patient choice and access to treatment. However, at service level, motives for the implementation of telephone treatments are often misaligned with national objectives. Pressure to meet performance targets has become a key driver for the use of telephone treatment, with promises of increased efficiency and cost savings. These service-focussed objectives challenge the integration of telephone treatments, and PWP acceptance of telephone treatments as non-inferior to face-to-face. Ambivalence among a workforce often lacking the confidence to deliver telephone treatments leads to reluctance among PWPs to ‘sell’ treatments to a patient population who are not generally expecting treatment in this form. Conclusions Perceptions of a need to ‘sell’ telephone treatment in IAPT persist from top-level decision makers down to frontline practitioners, despite their conflicting motives for the use of telephone. The need for advocacy to highlight the clinical benefit of telephone treatment, along with adequate workforce support and guidance on best practice for implementation is critical to the ongoing success and sustainability of telephone treatment in primary care mental health programmes.

Dynamic Cost-Effectiveness: A More Efficient Reimbursement Criterion

2008 ◽  
Vol 11 (2) ◽  
Author(s):  
Douglas Lundin ◽  
Joakim Ramsberg
Keyword(s):  
Life Cycle ◽  
Cost Effectiveness ◽  
Theoretical Model ◽  
Patent Protection ◽  
Decision Makers ◽  
Value For Money ◽  
Entire Life ◽  
Entire Life Cycle ◽  
The Cost ◽  
Over Time

Basing drug reimbursement on cost-effectiveness provides too little incentives for R&D. The reason for this is that cost-effectiveness is concerned with immediate value for money. But since the price of a drug usually declines over time, the drug might well provide value for money as seen over its entire life cycle, even though its price during patent protection is too high to warrant reimbursement according to the cost-effectiveness decision rule. We show in a theoretical model that welfare could be improved if decision-makers took a longer perspective and initially allowed higher prices than immediate value for money can motivate. We also discuss the real world relevance of applying dynamic cost-effectiveness.

HEALTH TECHNOLOGY ASSESSMENT

2002 ◽  
Vol 18 (2) ◽  
pp. 184-191 ◽  
Author(s):  
Francois Schubert
Keyword(s):  
Clinical Trials ◽  
Economic Evaluation ◽  
Cost Effectiveness ◽  
Health Technology Assessment ◽  
Technology Assessment ◽  
Health Technology ◽  
Decision Makers ◽  
Clinical Endpoints ◽  
Real World Evidence ◽  
The Many

Increasingly, health technology assessment (HTA) is used to aid decisions on the reimbursement of pharmaceuticals or recommendations for their use. The pharmaceutical industry seeks to work in partnership with HTA agencies; however, this presents a number of challenges. Clinical trials will need to include appropriate measures that capture economic and patient benefits as well as relevant clinical endpoints, and the industry will want to seek international harmonization of the many guidelines for economic evaluation. The problem of demonstrating cost-effectiveness of a product before it is available for use must be addressed, possibly by conditional reimbursement to allow collection of real world evidence. It is also important that reimbursement decision makers minimize bias, play fair, and adhere to the written rules they issue. If the industry fairly demonstrates the value of a product using the best available evidence, HTA agencies should be transparent in the rationale for their recommendations.

Economic Analysis of NCIC CTG JBR.10: A Randomized Trial of Adjuvant Vinorelbine Plus Cisplatin Compared With Observation in Early Stage Non–Small-Cell Lung Cancer—A Report of the Working Group on Economic Analysis, and the Lung Disease Site Group, National Cancer Institute of Canada Clinical Trials Group

2007 ◽  
Vol 25 (16) ◽  
pp. 2256-2261 ◽  
Author(s):  
Raymond Ng ◽  
Baktiar Hasan ◽  
Nicole Mittmann ◽  
Marie Florescu ◽  
Frances A. Shepherd ◽  
...  
Keyword(s):  
Health Care ◽  
Clinical Trials ◽  
Cost Effectiveness ◽  
Adjuvant Chemotherapy ◽  
Economic Analysis ◽  
National Cancer Institute ◽  
Early Stage ◽  
Public Health Care ◽  
Cost Effective Treatment ◽  
Utilization Data

Purpose National Cancer Institute of Canada Clinical Trials Group JBR.10 study is among the landmark trials that have established third generation platinum-based adjuvant chemotherapy as the standard of care after resection of stages IB-II NSCLC, improving absolute 5-year survival by 15% and median survival by 21 months. This cost-effectiveness analysis of adjuvant chemotherapy from the perspective of Canada's public health care system was undertaken based on the JBR.10 study population. Patients and Methods The primary outcome of the study was the incremental cost effectiveness ratio (ICER) expressed in dollars per life-year gained (LYG). Direct medical resource utilization data were collected retrospectively from trial data and medical records of patients enrolled in the JBR.10 study at the five largest accruing Canadian centers, from the time of random assignment until death or study closure (April 2004). Survival and available costs (2005 Canadian dollars [$CAD]) are presented both with and without discounting at 5% per year. Results Utilization data were collected from 172 Canadian patients (36% of the trial population), 85 randomly assigned to observation and 87 randomly assigned to chemotherapy. The mean costs of treatment per patient in the observation and adjuvant chemotherapy arms were $23,878 and $31,319, respectively, with an ICER of CAD$7,175/LYG discounted (95% CI, −$3,463 to $41,565), and $10,096/LYG undiscounted (95% CI, −$819 to $55,651). Conclusion Adjuvant vinorelbine plus cisplatin is a highly cost effective treatment that compares very favorably with other standard health care interventions.

scholarly journals Infliximab, adalimumab and golimumab for treating moderately to severely active ulcerative colitis after the failure of conventional therapy (including a review of TA140 and TA262): clinical effectiveness systematic review and economic model

2016 ◽  
Vol 20 (39) ◽  
pp. 1-326 ◽  
Author(s):  
Rachel Archer ◽  
Paul Tappenden ◽  
Shijie Ren ◽  
Marrissa Martyn-St James ◽  
Rebecca Harvey ◽  
...  
Keyword(s):  
Systematic Review ◽  
Ulcerative Colitis ◽  
Cost Effectiveness ◽  
Economic Analysis ◽  
Citation Index ◽  
Clinical Effectiveness ◽  
Health Technology ◽  
Controlled Trials ◽  
Beneficial Effects ◽  
Biological Treatments

BackgroundUlcerative colitis (UC) is the most common form of inflammatory bowel disease in the UK. UC can have a considerable impact on patients’ quality of life. The burden for the NHS is substantial.ObjectivesTo evaluate the clinical effectiveness and safety of interventions, to evaluate the incremental cost-effectiveness of all interventions and comparators (including medical and surgical options), to estimate the expected net budget impact of each intervention, and to identify key research priorities.Data sourcesPeer-reviewed publications, European Public Assessment Reports and manufacturers’ submissions. The following databases were searched from inception to December 2013 for clinical effectiveness searches and from inception to January 2014 for cost-effectiveness searches for published and unpublished research evidence: MEDLINE, EMBASE, Cumulative Index to Nursing and Allied Health Literature, The Cochrane Library including the Cochrane Systematic Reviews Database, Cochrane Controlled Trials Register, Database of Abstracts of Reviews of Effects, the Health Technology Assessment database and NHS Economic Evaluation Database; ISI Web of Science, including Science Citation Index, and the Conference Proceedings Citation Index-Science and Bioscience Information Service Previews. The US Food and Drug Administration website and the European Medicines Agency website were also searched, as were research registers, conference proceedings and key journals.Review methodsA systematic review [including network meta-analysis (NMA)] was conducted to evaluate the clinical effectiveness and safety of named interventions. The health economic analysis included a review of published economic evaluations and the development of a de novo model.ResultsTen randomised controlled trials were included in the systematic review. The trials suggest that adult patients receiving infliximab (IFX) [Remicade®, Merck Sharp & Dohme Ltd (MSD)], adalimumab (ADA) (Humira®, AbbVie) or golimumab (GOL) (Simponi®, MSD) were more likely to achieve clinical response and remission than those receiving placebo (PBO). Hospitalisation data were limited, but suggested more favourable outcomes for ADA- and IFX-treated patients. Data on the use of surgical intervention were sparse, with a potential benefit for intervention-treated patients. Data were available from one trial to support the use of IFX in paediatric patients. Safety issues identified included serious infections, malignancies and administration site reactions. Based on the NMA, in the induction phase, all biological treatments were associated with statistically significant beneficial effects relative to PBO, with the greatest effect associated with IFX. For patients in response following induction, all treatments except ADA and GOL 100 mg at 32–52 weeks were associated with beneficial effects when compared with PBO, although these were not significant. The greatest effects at 8–32 and 32–52 weeks were associated with 100 mg of GOL and 5 mg/kg of IFX, respectively. For patients in remission following induction, all treatments except ADA at 8–32 weeks and GOL 50 mg at 32–52 weeks were associated with beneficial effects when compared with PBO, although only the effect of ADA at 32–52 weeks was significant. The greatest effects were associated with GOL (at 8–32 weeks) and ADA (at 32–52 weeks). The economic analysis suggests that colectomy is expected to dominate drug therapies, but for some patients, colectomy may not be considered acceptable. In circumstances in which only drug options are considered, IFX and GOL are expected to be ruled out because of dominance, while the incremental cost-effectiveness ratio for ADA versus conventional treatment is approximately £50,300 per QALY gained.LimitationsThe health economic model is subject to several limitations: uncertainty associated with extrapolating trial data over a lifetime horizon, the model does not consider explicit sequential pathways of non-biological treatments, and evidence relating to complications of colectomy was identified through consideration of approaches used within previous models rather than a full systematic review.ConclusionsAdult patients receiving IFX, ADA or GOL were more likely to achieve clinical response and remission than those receiving PBO. Further data are required to conclusively demonstrate the effect of interventions on hospitalisation and surgical outcomes. The economic analysis indicates that colectomy is expected to dominate medical treatments for moderate to severe UC.Study registrationThis study is registered as PROSPERO CRD42013006883.FundingThe National Institute for Health Research Health Technology Assessment programme.

scholarly journals Smaller clinical trials for decision making; using p-values could be costly

F1000Research ◽  
2018 ◽  
Vol 7 ◽  
pp. 1176 ◽  
Author(s):  
Nicholas Graves ◽  
Adrian G. Barnett ◽  
Edward Burn ◽  
David Cook
Keyword(s):  
Decision Making ◽  
Clinical Trials ◽  
Economic Analysis ◽  
Hypothesis Testing ◽  
Sample Size ◽  
Statistical Significance ◽  
Decision Makers ◽  
Statistical Hypothesis ◽  
Statistical Hypothesis Testing ◽  
Good Investment

Background: Clinical trials might be larger than needed because arbitrary high levels of statistical confidence are sought in the results. Traditional sample size calculations ignore the marginal value of the information collected for decision making. The statistical hypothesis testing objective is misaligned with the goal of generating information necessary for decision-making. The aim of the present study was to show that a clinical trial designed to test a prior hypothesis against an arbitrary threshold of confidence may recruit too many participants, wasting scarce research dollars and exposing participants to research unnecessarily. Methods: We used data from a recent RCT powered for traditional rules of statistical significance. The data were also used for an economic analysis to show the intervention led to cost savings and improved health outcomes. Adoption represented a good investment for decision-makers. We examined the effect of reducing the trial’s sample size on the results of the statistical hypothesis-testing analysis and the conclusions that would be drawn by decision-makers reading the economic analysis. Results: As the sample size reduced it became more likely that the null hypothesis of no difference in the primary outcome between groups would fail to be rejected. For decision-makers reading the economic analysis, reducing the sample size had little effect on the conclusion about whether to adopt the intervention. There was always high probability the intervention reduced costs and improved health. Conclusions: Decision makers managing health services are largely invariant to the sample size of the primary trial and the arbitrary p-value of 0.05. If the goal is to make a good decision about whether the intervention should be adopted widely, then that could have been achieved with a much smaller trial. It is plausible that hundreds of millions of research dollars are wasted each year recruiting more participants than required for RCTs.

scholarly journals The basics of economic evaluation in mental healthcare

2015 ◽  
Vol 21 (5) ◽  
pp. 345-353 ◽  
Author(s):  
James Shearer ◽  
Sarah Byford
Keyword(s):  
Economic Evaluation ◽  
Cost Effectiveness ◽  
Treatment Options ◽  
Mental Healthcare ◽  
Decision Makers ◽  
Economic Evaluations ◽  
Relative Cost ◽  
Value For Money ◽  
Relative Value ◽  
Randomised Controlled

SummaryEconomic evaluation involves the comparative analysis of the costs and consequences of alternative (different) treatment options. Economic evaluations provide decision makers with information about the relative value for money, or cost-effectiveness, of various treatment programmes. The relative cost-effectiveness of new interventions is a key consideration in health technology assessments by the UK's National Institute for Health and Care Excellence, and economic evaluations alongside randomised controlled trials are routinely requested by funders such as the National Institute for Health Research. This article outlines some of the key concepts and issues in the economic evaluation of mental healthcare.

Sign in / Sign up

Export Citation Format

Share Document