Four Issues in Cost-Effectiveness Analysis and Health Technology Assessment: a View from the Touch-line

2015 ◽  
pp. 77-94
Author(s):  
Anthony J. Culyer
Keyword(s):  
Cost Effectiveness ◽  
Health Technology Assessment ◽  
Technology Assessment ◽  
Health Technology ◽  
Cost Effectiveness Analysis ◽  
Effectiveness Analysis

A Bayesian Net Benefit Approach to Cost‐effectiveness Analysis in Health Technology Assessment

2009 ◽  
Vol 16 (3) ◽  
pp. 323-345 ◽  
Author(s):  
Elías Moreno ◽  
Francisco Javier Girón ◽  
Francisco José Vázquez‐Polo ◽  
Miguel A. Negrín
Keyword(s):  
Cost Effectiveness ◽  
Health Technology Assessment ◽  
Technology Assessment ◽  
Health Technology ◽  
Cost Effectiveness Analysis ◽  
Net Benefit ◽  
Effectiveness Analysis

Gesundheitspolitik – Eine neue Methode zur Bestimmung der optimalen „Willingness to Pay“ in Kosteneffektivitätsanalysen

2020 ◽  
Vol 25 (03) ◽  
pp. 129-130
Keyword(s):  
Cost Effectiveness ◽  
Willingness To Pay ◽  
Health Technology Assessment ◽  
Technology Assessment ◽  
Health Technology ◽  
Cost Effectiveness Analysis ◽  
New Method ◽  
Effectiveness Analysis

Phelps CE. A New Method to Determine the Optimal Willingness to Pay in Cost-Effectiveness Analysis. Value Health. 2019; 22 (7): 785–791 Die Studie liefert einen neuen Ansatz zur Bestimmung der optimalen „Willingness to Pay“(WTP) für HTA‘s (health technology assessment). Die Analyse definiert den Nutzen als eine Funktion des Einkommens. Die Kalibrierung wurde mithilfe der abgeschätzten relativen Risikoaversion (r*) durchgeführt, von der die optimale WTP anhand der Ergebnisse von Garber und Phelps‘ aus dem Jahre 1997 bestimmt werden kann.

scholarly journals Sixteen-week versus standard eight-week prednisolone therapy for childhood nephrotic syndrome: the PREDNOS RCT

2019 ◽  
Vol 23 (26) ◽  
pp. 1-108 ◽  
Author(s):  
Nicholas JA Webb ◽  
Rebecca L Woolley ◽  
Tosin Lambe ◽  
Emma Frew ◽  
Elizabeth A Brettell ◽  
...  
Keyword(s):  
Nephrotic Syndrome ◽  
Cost Effectiveness ◽  
Adverse Events ◽  
Health Technology Assessment ◽  
Technology Assessment ◽  
Health Technology ◽  
Cost Effectiveness Analysis ◽  
Prednisolone Therapy ◽  
Effectiveness Analysis ◽  
First Relapse

Background The optimal corticosteroid regimen for treating the presenting episode of steroid-sensitive nephrotic syndrome (SSNS) remains uncertain. Most UK centres use an 8-week regimen, despite previous systematic reviews indicating that longer regimens reduce the risk of relapse and frequently relapsing nephrotic syndrome (FRNS). Objectives The primary objective was to determine whether or not an extended 16-week course of prednisolone increases the time to first relapse. The secondary objectives were to compare the relapse rate, FRNS and steroid-dependent nephrotic syndrome (SDNS) rates, requirement for alternative immunosuppressive agents and corticosteroid-related adverse events (AEs), including adverse behaviour and costs. Design Randomised double-blind parallel-group placebo-controlled trial, including a cost-effectiveness analysis. Setting One hundred and twenty-five UK paediatric departments. Participants Two hundred and thirty-seven children presenting with a first episode of SSNS. Participants aged between 1 and 15 years were randomised (1 : 1) according to a minimisation algorithm to ensure balance of ethnicity (South Asian, white or other) and age (≤ 5 or ≥ 6 years). Interventions The control group (n = 118) received standard course (SC) prednisolone therapy: 60 mg/m2/day of prednisolone in weeks 1–4, 40 mg/m2 of prednisolone on alternate days in weeks 5–8 and matching placebo on alternate days in weeks 9–18 (total 2240 mg/m2). The intervention group (n = 119) received extended course (EC) prednisolone therapy: 60 mg/m2/day of prednisolone in weeks 1–4; started at 60 mg/m2 of prednisolone on alternate days in weeks 5–16, tapering by 10 mg/m2 every 2 weeks (total 3150 mg/m2). Main outcome measures The primary outcome measure was time to first relapse [Albustix® (Siemens Healthcare Limited, Frimley, UK)-positive proteinuria +++ or greater for 3 consecutive days or the presence of generalised oedema plus +++ proteinuria]. The secondary outcome measures were relapse rate, incidence of FRNS and SDNS, other immunosuppressive therapy use, rates of serious adverse events (SAEs) and AEs and the incidence of behavioural change [using Achenbach Child Behaviour Checklist (ACBC)]. A comprehensive cost-effectiveness analysis was performed. The analysis was by intention to treat. Participants were followed for a minimum of 24 months. Results There was no significant difference in time to first relapse between the SC and EC groups (hazard ratio 0.87, 95% confidence interval 0.65 to 1.17; log-rank p = 0.3). There were also no differences in the incidence of FRNS (SC 50% vs. EC 53%; p = 0.7), SDNS (44% vs. 42%; p = 0.8) or requirement for other immunosuppressive therapy (56% vs. 54%; p = 0.8). The total prednisolone dose received following completion of study medication was 5475 mg vs. 6674 mg (p = 0.07). SAE rates were not significantly different (25% vs. 17%; p = 0.1) and neither were AEs, except poor behaviour (yes/no), which was less frequent with EC treatment. There were no differences in ACBC scores. EC therapy was associated with a mean increase in generic health benefit [0.0162 additional quality-adjusted life-years (QALYs)] and cost savings (£4369 vs. £2696). Limitations Study drug formulation may have prevented some younger children who were unable to swallow whole or crushed tablets from participating. Conclusions This trial has not shown any clinical benefit for EC prednisolone therapy in UK children. The cost-effectiveness analysis suggested that EC therapy may be cheaper, with the possibility of a small QALY benefit. Future work Studies investigating EC versus SC therapy in younger children and further cost-effectiveness analyses are warranted. Trial registration Current Controlled Trials ISRCTN16645249 and EudraCT 2010-022489-29. Funding This project was funded by the National Institute for Health Research (NIHR) Health Technology Assessment programme and will be published in full in Health Technology Assessment; Vol. 23, No. 26. See the NIHR Journals Library website for further project information.

scholarly journals Imaginary Worlds and Efficiency Frontiers: Should We Abandon the IQWiG Health Technology Assessment Model?

2017 ◽  
Vol 8 (1) ◽  
Author(s):  
Paul C Langley
Keyword(s):  
Cost Effectiveness ◽  
Health Technology Assessment ◽  
Technology Assessment ◽  
Health Technology ◽  
Time Frame ◽  
Cost Effectiveness Analysis ◽  
Assessment Model ◽  
Efficiency Frontier ◽  
Effectiveness Analysis ◽  
Life Years

The contribution of cost-effectiveness analysis to the pricing of pharmaceuticals in Germany is at best marginal and in many, if not most cases, absent. While this may reflect a reasonable belief that cost-effectiveness analysis adds little if anything to pricing and formulary placement decisions, its marginalization reflects considerable dissatisfaction, if not frustration, with modeling efforts by the Institut für Qualität und Wirtschaftlichkeit im Gesundheitseesen (IQWiG). In part, this reflects the rejection of quality adjusted life years (QALYs) as the common outcome standard, together with the adoption of the efficiency frontier as the default framework for modeled claims. The purpose of this commentary is to consider the merits, in the German context, of an efficiency frontier framework for cost-effectiveness and pricing decisions. The commentary concludes that the efficiency frontier framework for health technology assessment, in supporting the creation of non-evaluable claims from models or simulations, fails of to meet the standards of normal science: it fails to support claims that are credible, evaluable and replicable. It should be abandoned. If cost-effectiveness modeling is to play a constructive role in pricing negotiations in Germany then manufacturers should be required to submit evaluable claims. The most effective way of ensuring this is to require manufacturers to accompany any submission for a new product with a protocol detailing how their claims, to include those for clinical outcomes, cost-effectiveness and budget impact, are to be evaluated and reported to decision makers in a meaningful time frame.   Type: Commentary

scholarly journals Expanding HTA – Correcting a Misattribution, Clarifying the Scope of HTA and CEA Comment on "Ethics in HTA: Examining the ‘Need for Expansion’"

2019 ◽  
Vol 8 (12) ◽  
pp. 732-733
Author(s):  
Anthony J. Culyer
Keyword(s):  
Cost Effectiveness ◽  
Health Technology Assessment ◽  
Technology Assessment ◽  
Health Technology ◽  
Cost Effectiveness Analysis ◽  
Effectiveness Analysis

Abrishami, Oortwijn, and Hofman (AOH) attribute to me a position I do not hold and an argument I did not make. The purpose of this note is make clear what my position actually is and to clarify the main differences between health technology assessment (HTA) and cost-effectiveness analysis (CEA).

scholarly journals Health technology assessment in the United Kingdom

2009 ◽  
Vol 25 (S1) ◽  
pp. 178-181 ◽  
Author(s):  
Michael Drummond ◽  
David Banta
Keyword(s):  
Health Care ◽  
United Kingdom ◽  
Cost Effectiveness ◽  
Health Technology Assessment ◽  
Technology Assessment ◽  
Clinical Excellence ◽  
Present Situation ◽  
Health Technology ◽  
The United Kingdom ◽  
Short Run

Objectives: The aim of this study was to describe generally the development and present situation with health technology assessment (HTA) in the United Kingdom.Methods: The methods used are a review of important materials that have described the development process and present situation, supplemented by some personal experiences.Results: The United Kingdom has been characterized historically as a country with a strong interest in evidence in health care, both clinical trials for efficacy and cost-effectiveness analyses. However, this evidence was not well-linked to the needs of the National Health Services (NHS) before formation of the NHS R&D Programme in 1991, The R&D Programme brought substantial resources into HTA and related activities, with the central aim of improving health care in Britain and increasing value for money. However, policy makers as well as staff of the R&D Programme were dissatisfied with the use of the HTA results in clinical and administrative practice. Therefore, the National Institute of Clinical Excellence (NICE) was formed in 1999. NICE issues guidance intended to influence practical decision making in health care at the national and local levels, based on efficacy information and, in some cases, economic analyses. NICE is now also seeking ways to maximize impacts on practice.Conclusions: The UK experience shows that information on clinical and cost-effectiveness may not be enough to change practice, at least in the short-run. Still, one may conclude that the United Kingdom now has one of the few most important and influential HTA programs in the world.

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