For several decades, the incremental cost-effectiveness ratio has been routinely used by health technology assessment agencies around the world to summarise the results of economic evaluations of ...health interventions. Yet reporting and considering incremental cost-effectiveness ratios is unnecessary. Alternative summary measures exist, based on the concept of ‘net benefit’. The incremental cost-effectiveness ratio and measures of net benefit share several commonalities but some important distinctions. As a result, different methods are required to calculate and interpret incremental cost-effectiveness ratios compared to measures of net benefit. The aim of this practical application is to introduce readers to these methods, using a hypothetical example to illustrate key issues. First, the methods used to calculate each measure are described. Next, for each measure, consideration is made of whether and how each measure may be interpreted to perform the following tasks, each of which may be of interest to health technology assessment agencies: (1) identifying the single most cost-effective strategy; (2) ranking strategies from ‘most’ to ‘least’ cost-effective (on an ordinal scale); (3) determining the magnitude to which a strategy is more or less cost-effective than another strategy (on a cardinal scale); and (4) determining whether a strategy is more or less cost-effective following a sensitivity or scenario analysis. This practical application also introduces a novel approach for visually interpreting measures of net benefit using the cost-effectiveness plane, which addresses a number of limitations of the conventional cost-effectiveness ‘efficiency frontier’. By the end of this practical application, readers should have an understanding of how to calculate and interpret each measure, as well as the relative strengths and limitations of each.
Although probabilistic analysis has become the accepted standard for decision analytic cost-effectiveness models, deterministic one-way sensitivity analysis continues to be used to meet the need of ...decision makers to understand the impact that changing the value taken by one specific parameter has on the results of the analysis. The value of a probabilistic form of one-way sensitivity analysis has been recognised, but the proposed methods are computationally intensive. Deterministic one-way sensitivity analysis provides decision makers with biased and incomplete information whereas, in contrast, probabilistic one-way sensitivity analysis (POSA) can overcome these limitations, an observation supported in this study by results obtained when these methods were applied to a previously published cost-effectiveness analysis to produce a conditional incremental expected net benefit curve. The application of POSA will provide decision makers with unbiased information on how the expected net benefit is affected by a parameter taking on a specific value and the probability that the specific value will be observed.
Probabilistic sensitivity analysis (PSA) demonstrates the parameter uncertainty in a decision problem. The technique involves sampling parameters from their respective distributions (rather than ...simply using mean/median parameter values). Guidance in the literature, and from health technology assessment bodies, on the number of simulations that should be performed suggests a ‘sufficient number’, or until ‘convergence’, which is seldom defined. The objective of this tutorial is to describe possible outcomes from PSA, discuss appropriate levels of accuracy, and present guidance by which an analyst can determine if a sufficient number of simulations have been conducted, such that results are considered to have converged. The proposed approach considers the variance of the outcomes of interest in cost-effectiveness analysis as a function of the number of simulations. A worked example of the technique is presented using results from a published model, with recommendations made on best practice. While the technique presented remains essentially arbitrary, it does give a mechanism for assessing the level of simulation error, and thus represents an advance over current practice of a round number of simulations with no assessment of model convergence.
As high-cost medicines put increasing pressure on public health care budgets, the need to identify ‘fair’ prices for medicines has never been greater. This paper proposes a framework, built upon ...fundamental economic principles, that allows for the consideration of ‘fair’ prices for medicines. The framework incorporates key considerations from conventional supply-side and demand-side approaches for specifying a cost-effectiveness ‘threshold’, including the health opportunity cost borne by other patients (
k
) and society’s willingness to pay for marginal improvements in population health (
v
). The costs incurred by manufacturers in developing and supplying new medicines are also considered, as are the incentives for manufacturers to strategically price up to any common price per unit of benefit (cost-effectiveness ‘threshold’) specified by the payer. The framework finds that, at any ‘fair’ price, a medicine’s dynamically calculated incremental cost-effectiveness ratio (ICER) lies below
k
. When pricing medicines collectively, the framework finds that a common price below
k
is required to maximize population health (consumer surplus) or to maximize total welfare (consumer and producer surplus). This framework has important policy implications for payers who wish to improve population health outcomes from constrained health care budgets. In particular, existing approaches to ‘value-based pricing’ should be reconsidered to ensure that patients receive a ‘fair’ share of the resulting economic surplus.
In considering these methods, some fundamental weaknesses of the ICER become apparent. 2 ICERs are More Laborious to Calculate The ICER is simple to calculate between two strategies; however, ...calculating ICERs in evaluations of three or more strategies can be laborious. Since the ICER is a pairwise measure, multiple ICERs need to be calculated. By contrast, calculating net benefit is simple, regardless of the number of strategies. Since it is not a pairwise measure, the net benefit of each strategy is not dependent on other strategies. Since ICERs are not calculated between dominated or extendedly dominated strategies, ranking such strategies using ICERs is impossible. ...there is no advantage to using ICERs if the threshold is 'unknown'.
Purpose
To develop an evidence-based guideline for the management of grades I–III neck pain and associated disorders (NAD).
Methods
This guideline is based on recent systematic reviews of ...high-quality studies. A multidisciplinary expert panel considered the evidence of effectiveness, safety, cost-effectiveness, societal and ethical values, and patient experiences (obtained from qualitative research) when formulating recommendations. Target audience includes clinicians; target population is adults with grades I–III NAD <6 months duration.
Recommendation 1
Clinicians should rule out major structural or other pathologies as the cause of NAD. Once major pathology has been ruled out, clinicians should classify NAD as grade I, II, or III.
Recommendation 2
Clinicians should assess prognostic factors for delayed recovery from NAD.
Recommendation 3
Clinicians should educate and reassure patients about the benign and self-limited nature of the typical course of NAD grades I–III and the importance of maintaining activity and movement. Patients with worsening symptoms and those who develop new physical or psychological symptoms should be referred to a physician for further evaluation at any time during their care.
Recommendation 4
For NAD grades I–II ≤3 months duration, clinicians may consider structured patient education in combination with: range of motion exercise, multimodal care (range of motion exercise with manipulation or mobilization), or muscle relaxants. In view of evidence of no effectiveness, clinicians should not offer structured patient education alone, strain-counterstrain therapy, relaxation massage, cervical collar, electroacupuncture, electrotherapy, or clinic-based heat.
Recommendation 5
For NAD grades I–II >3 months duration, clinicians may consider structured patient education in combination with: range of motion and strengthening exercises, qigong, yoga, multimodal care (exercise with manipulation or mobilization), clinical massage, low-level laser therapy, or non-steroidal anti-inflammatory drugs. In view of evidence of no effectiveness, clinicians should not offer strengthening exercises alone, strain-counterstrain therapy, relaxation massage, relaxation therapy for pain or disability, electrotherapy, shortwave diathermy, clinic-based heat, electroacupuncture, or botulinum toxin injections.
Recommendation 6
For NAD grade III ≤3 months duration, clinicians may consider supervised strengthening exercises in addition to structured patient education. In view of evidence of no effectiveness, clinicians should not offer structured patient education alone, cervical collar, low-level laser therapy, or traction.
Recommendation 7
For NAD grade III >3 months duration, clinicians should not offer a cervical collar. Patients who continue to experience neurological signs and disability more than 3 months after injury should be referred to a physician for investigation and management.
Recommendation 8
Clinicians should reassess the patient at every visit to determine if additional care is necessary, the condition is worsening, or the patient has recovered. Patients reporting significant recovery should be discharged.
Gene expression profiling (GEP) testing using 12-gene recurrence score (RS) assay (EndoPredict®), 58-gene RS assay (Prosigna®), and 21-gene RS assay (Oncotype DX®) is available to aid in chemotherapy ...decision-making when traditional clinicopathological predictors are insufficient to accurately determine recurrence risk in women with axillary lymph node-negative, hormone receptor-positive, and human epidermal growth factor-receptor 2-negative early-stage breast cancer. We examined the cost-effectiveness of incorporating these assays into standard practice. A decision model was built to project lifetime clinical and economic consequences of different adjuvant treatment-guiding strategies. The model was parameterized using follow-up data from a secondary analysis of the Anastrozole or Tamoxifen Alone or Combined randomized trial, cost data (2017 Canadian dollars) from the London Regional Cancer Program (Canada) and secondary Canadian sources. The 12-gene, 58-gene, and 21-gene RS assays were associated with cost-effectiveness ratios of $36,274, $48,525, and $74,911/quality-adjusted life year (QALY) gained and resulted in total gains of 379, 284.3, and 189.5 QALYs/year and total budgets of $12.9, $14.2, and $16.6 million/year, respectively. The total expected-value of perfect information about GEP assays' utility was $10.4 million/year. GEP testing using any of these assays is likely clinically and economically attractive. The 12-gene and 58-gene RS assays may improve the cost-effectiveness of GEP testing and offer higher value for money, although prospective evidence is still needed. Comparative field evaluations of GEP assays in real-world practice are associated with a large societal benefit and warranted to determine the optimal and most cost-effective assay for routine use.
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