Ties Hoomans

Value of Information and Value of Implementation: Application of an Analytic Framework to Inform Resource Allocation Decisions in Metastatic Hormone-Refractory Prostate Cancer

OBJECTIVE In a budget-constrained health-care system, decisions about investing in strategies to promote implementation have to be made alongside decisions about health-care provision and research funding. Using a Bayesian decision-theoretic approach, an analytic framework has been developed to inform these separate but related decisions, establishing the expected value of both perfect information (EVPI) and perfect implementation (EVPIM). We applied this framework to inform decision-making about resource allocation to metastatic hormone-refractory prostate cancer (mHRPC) in the UK. METHODS Based on available evidence on the cost-effectiveness of all plausible treatments for mHRPC, we determined which treatment option(s) were cost-effective and explored the uncertainty surrounding this decision. Given the decision uncertainty and the variation in care provided by health-care professionals, we then determined the EVPI and EVPIM. Finally, we performed sensitivity analyses to explore the influence of alternative assumptions regarding various decision parameters on the efficiency of resource allocation. RESULTS Depending on the cost-effectiveness threshold (lambda), we identified mitoxantrone plus prednisone/prednisolone and docetaxel plus prednisone/prednisolone (3 weekly) as the optimal treatments for mHRPC. Given current clinical practice, there appears to be considerable scope for improving the efficiency of health-care provision: the EVPI (estimated to be over pound13 million) indicates that acquiring further information could be cost-effective; and the EVPIM (estimated to be over pound4 million) suggests that investing in strategies to implement the treatments regimens being identified as optimal is potentially worthwhile. Through sensitivity analyses, we found that the EVPI and EVPIM are mainly driven by lambda, the number of treatment options being considered, the current level of implementation, and the size of the eligible patient population. CONCLUSION The application demonstrates that the framework provides a simple and useful analytic tool for decision-makers to address resource allocation problems between health-care provision, further research, and implementation efforts.

Methodological quality of economic evaluations of new pharmaceuticals in The Netherlands.

AbstractBackground: In the Netherlands, decisions about the reimbursement of new pharmaceuticals are based on cost effectiveness, as well as therapeutic value and budget impact. Since 1 January 2005, drug manufacturers are formally required to substantiate the cost effectiveness of drugs that have therapeutic added value in comparison with existing ones through pharmacoeconomic evaluations. Dutch guidelines for pharmacoeconomic research providemethods guidance, ensuring consistency in both the evidence and the decision-making process about drug reimbursement. Aim: This study reviewed the methodological quality of all 21 formally required pharmacoeconomic evaluations of new pharmaceuticals between 1 January 2005 and 1 October 2008, and verified whether these evaluations complied with pharmacoeconomic guidelines. Methods: Data on the quality of the pharmacoeconomic evaluations were extracted from the pharmacoeconomic reports published by the Dutch Health Care Insurance Board (CVZ). The Board’s newsletters provided information on the advice to, and reimbursement decisions made by, the Dutch Minister of Health. All data extraction was carried out by two independent reviewers, and descriptive analyses were conducted. Results: The methodological quality was sound in only 8 of the 21 pharmacoeconomic evaluations. In most cases, the perspective of analysis, the comparator drugs, and the reporting of both total and incremental costs and effects were correct. However, drug indication, form (i.e. cost utility/cost effectiveness) and time horizon of the evaluations were frequently flawed. Moreover, the costs and effects of the pharmaceuticals were not always analysed correctly, and modelling studies were often non-transparent. Twelve drugs were reimbursed, and nine were not. Conclusions: The compliance with pharmacoeconomic guidelines in economic evaluations of new pharmaceuticals can be improved. This would improve the methodological quality of the pharmacoeconomic evaluations and ensure consistency in the evidence and the decision-making process for drug reimbursement in the Netherlands.

Implementing guidelines into clinical practice: what is the value?

RATIONALE AND OBJECTIVE In budget-constrained health systems, decision makers need to consider both the costs and effects of introducing and actively implementing clinical guidance. We aim to demonstrate how, as an alternative to conventional methods, a total net benefit approach to economic evaluation can be used to inform decision making about guidelines and specific implementation strategies, like education or financial incentives. METHODS Aside from providing more detail on the decision framework, we describe how to collect and analyse the relevant data for calculating the total net benefit of guideline use and the value of implementation. We illustrate the process of decision analysis for a stylized example on improving diabetes care in the UK. For the analysis, economic evidence on intensified glycemic control and that on audit and feedback to promote control is combined with information on diabetes practice. RESULTS Our illustration demonstrates that the total net benefit of guideline use and the value of implementation can vary substantially, depending on the clinical intervention chosen, the health system being studied and the specific implementation strategies. This also holds for the threshold value for cost-effectiveness, the duration of guideline usage or validity, the size of the patient population served, and the trends and ceiling rates in the implementation of clinical guidance. CONCLUSIONS In comparison with conventional methods for health economic evaluation, a total net benefit approach allows for the explicit consideration of the current (or future) use of guidelines or guideline recommendations, the cost of implementation and the scope of clinical practice. Decisions made on the basis of the total net benefit of all plausible combinations of clinical guidance and implementation strategies provide optimal patient care and an efficient use of resources.

Economic evaluation of implementation strategies in health care

Economic evaluations can inform decisions about the efficiency and allocation of resources to implementation strategies—strategies explicitly designed to inform care providers and patients about the best available research evidence and to enhance its use in their practices. These strategies are increasingly popular in health care, especially in light of growing concerns about quality of care and limits on resources. But such concerns have hardly motivated health authorities and other decision-makers to spend on some form of economic evaluation in their assessments of implementation strategies. This editorial addresses the importance of economic evaluation in the context of implementation science—particularly, how these analyses can be most efficiently incorporated into decision-making processes about implementation strategies.

Value for Money in Changing Clinical Practice: Should Decisions about Guidelines and Implementation Strategies Be Made Sequentially or Simultaneously?:

Decisions about clinical practice change, that is, which guidelines to adopt and how to implement them, can be made sequentially or simultaneously. Decision makers adopting a sequential approach first compare the costs and effects of alternative guidelines to select the best set of guideline recommendations for patient management and subsequently examine the implementation costs and effects to choose the best strategy to implement the selected guideline. In an integral approach, decision makers simultaneously decide about the guideline and the implementation strategy on the basis of the overall value for money in changing clinical practice. This article demonstrates that the decision to use a sequential v. an integral approach affects the need for detailed information and the complexity of the decision analytic process. More importantly, it may lead to different choices of guidelines and implementation strategies for clinical practice change. The differences in decision making and decision analysis between the alternative approaches are comprehensively illustrated using 2 hypothetical examples. We argue that, in most cases, an integral approach to deciding about change in clinical practice is preferred, as this provides more efficient use of scarce health-care resources.

Modeling the value for money of changing clinical practice change: A stochastic application in diabetes care

Background:Decision making about resource allocation for guideline implementation to change clinical practice is inevitably undertaken in a context of uncertainty surrounding the cost-effectiveness of both clinical guidelines and implementation strategies. Adopting a total net benefit approach, a model was recently developed to overcome problems with the use of combined ratio statistics when analyzing decision uncertainty. Objective:To demonstrate the stochastic application of the model for informing decision making about the adoption of an audit and feedback strategy for implementing a guideline recommending intensive blood glucose control in type 2 diabetes in primary care in the Netherlands. Methods:An integrated Bayesian approach to decision modeling and evidence synthesis is adopted, using Markov Chain Monte Carlo simulation in WinBUGs. Data on model parameters is gathered from various sources, with effectiveness of implementation being estimated using pooled, random-effects meta-analysis. Decision uncertainty is illustrated using cost-effectiveness acceptability curves and frontier. Results:Decisions about whether to adopt intensified glycemic control and whether to adopt audit and feedback alter for the maximum values that decision makers are willing to pay for health gain. Through simultaneously incorporating uncertain economic evidence on both guidance and implementation strategy, the cost-effectiveness acceptability curves and cost-effectiveness acceptability frontier show an increase in decision uncertainty concerning guideline implementation. Conclusions:The stochastic application in diabetes care demonstrates that the model provides a simple and useful tool for quantifying and exploring the (combined) uncertainty associated with decision making about adopting guidelines and implementation strategies and, therefore, for informing decisions about efficient resource allocation to change clinical practice.

The Authorsʼ Reply

Dear Editor, In response to our article, van Luijn questions whether there is a role for economic evaluation in decision making about coverage or payment for new medicines. Our answer: yes, economic evaluations can provide information about the efficiency of pharmaceuticals and nondrug technologies that can help decision makers, including insurers and payers, decide about the best use of healthcare resources. In effect, evidence suggests increased use of economic evaluation information, complementary to the information fromclinical studies and other evaluative approaches such as budget impact analyses, in decision making. Performing economic evaluations requires decision makers, manufacturers, providers and others involved in evidence and decision-making processes to invest effort in collecting and synthesizing data on the effect of technologies on outcomes and costs. As relevant data may be readily available from existing research, these efforts need not be demanding or costly. This and the fact that using economic evaluation information in making decisions can improve health and healthcare make investments in economic evaluations likely cost effective. The findings of our study reinforce the need for economic evaluations to be methodologically sound so that consequent coverage or payment decisions are credible and appropriate. To enhance the use of economic evaluation in specific circumstances or decision contexts, for example, when only indirect evidence exists on health technologies, or when heterogeneity is expected in outcomes and patient groups, methods for evaluation will continue to be advanced. If any such undertaking is done with consideration to the needs of decision makers and others, it would be more likely that economic evaluations are found useful and are applied in decision-making practice.

PHP20 COST-EFFECTIVE PHARMACEUTICAL CARE IN THE NETHERLANDS? A REVIEW OF THE PHARMACOECONOMICS AND ITS ROLE IN THE ASSESSMENT AND REIMBURSEMENT OF NEW DRUGS

pathways for biopharmaceuticals are well established, it is only in the past decade that regulatory development has evolved to secure guidelines and controls for stem cell based therapies, creating evaluation challenges for payers as these agents enter the market. METHODS: Conventional pharmaceuticals are generally assessed using a “value based” approach, in which their value is assessed by evaluating the positive and negative differentiation from established references. As no true direct reference will be available for allogenic products, potential references will be identified to support the “value based” approach and identify market access challenges. RESULTS: Skin replacement therapies are the only commercialized allogenic products to date (e.g. TransCyte and Apligraf), and serve as interesting case studies to evaluate how these types of therapies may be valued. Biologic agents faced a similar dilemma when first launched and demonstrated the importance of identifying appropriate indirect references to support these novel drugs. CONCLUSIONS: In the evolving regulatory and P&R environment, a greater unknown in regenerative based medicine surrounds the process of pricing and market access. Learning from biologics and skin replacement therapies can infer challenges and opportunities for these new therapies.