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The Determinants of Cost-Effectiveness Potential: An Historical Perspective on Lipid-Lowering Therapies

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Abstract

Background

The concept of cost effectiveness emerged in an attempt to link the prices of new healthcare technologies to the immediate value they provide, with payers defining the acceptable cost per unit of incremental effect over the alternatives available. It has been suggested that such measures allow developers to assess potential market profitability in an early stage of development, but may result in discouraging investment in efficient research if not used appropriately.

Objective

The objective of this study is to identify the pattern of the factors determining cost effectiveness and assess the evolution of cost-effectiveness potential for drugs in development using lipid-lowering therapy as a case study.

Methods

The study is based on observational clinical and market data covering a 20-year period (from 1990 to 2010) in the UK. Real-life clinical data including total cholesterol laboratory test results were extracted from the Clinical Practice Research Datalink (CPRD) and are used to illustrate how the clinical effectiveness of existing standard care changed over time in patients managed in clinical practice. Prescription Cost Analysis (PCA) data were extracted and the average price of the drug mix used was computed throughout the study period. Using this information, the maximum clinical benefit and cost savings to be had were estimated for each year of the analysis using a cost-effectiveness model. Subsequently, the highest price a new technology providing the maximum clinical effectiveness possible (i.e. eliminating cardiovascular risk from high cholesterol levels) could achieve under current cost-effectiveness rules was calculated and used as a measure of the potential cost effectiveness of drugs in development.

Results

The results in this study show that the total cholesterol values of patients managed in clinical practice moved steadily towards recommended clinical targets. Overall, the absolute potential for incremental health-related quality of life decreased by approximately 78 %, contracting from 0.36 QALYs to 0.08 QALYs, which resulted in a saving of approximately 15 % of the costs related to cardiovascular events. The price of the drug mix used in the management of high blood cholesterol varied considerably across the years: the weighted average monthly price (in year 2007 values) started at approximately £14, peaked around £26 and progressively decreased to its minimum at £6.85 in 2010. As a consequence, the maximum price allowed by current cost-effectiveness rules for a new technology achieving the clinical target was found to decrease by a minimum of 80 % between 1990 and 2010.

Conclusion

The analysis supports the hypothesis that the potential for cost effectiveness of new therapies is dependent on factors specific to each disease area and furthermore to sub-populations within disease areas. Despite a clinical need still existing, the results suggest that no more technologies are likely to be developed in certain disease areas based on their low perceived cost-effectiveness potential. This occurs without considering the immediate and future value of the effectiveness lost, which may depend on the technical difficulty of materializing future advancements, and ignores the permanent character of such a decision. The analysis suggests that a single, static and arbitrary cost-effectiveness threshold may not be sufficient to capture the drug-development dynamics occurring at the disease level and successfully direct research to the disease areas that are most valued by society.

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Notes

  1. In this process, we followed the framework proposed by Refoios Camejo and colleagues [19] and assumed there was a physiologically defined clinical effectiveness ceiling (\( E_{D} \hbox{Max} \)) representing the maximum absolute clinical effectiveness that could be attained, i.e. when cure or maximum risk reduction was obtained. The maximum incremental clinical effectiveness a new technology could achieve (\( IE_{d} \hbox{Max} \)) was then computed by \(IE_{d} {\text{Max}} = E_{D} {\text{Max}} - E_{c} \), where \( E_{c} \) is the clinical effectiveness of available standard care.

  2. The cost effectiveness of a new drug can be represented by the ratio of the incremental differences (of costs and benefits) between the new drug and existing comparators. A drug will be considered cost effective if the cost-effectiveness ratio is lower than the cost-effectiveness threshold in place. The maximum price (\( P_{D} \hbox{Max} \)) achievable for reaching \( E_{D} \hbox{Max} \) can then be computed taking in consideration the price of the comparator (\( P_{c} \)). When computed over time, \( P_{D} \hbox{Max} \) allows assessing the evolution of the potential for cost effectiveness and can roughly be used as an indicator of the prospects for clinical development in a particular disease area.

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Acknowledgments

Whilst conducting this study, R. Refoios Camejo was supported by the Portuguese Fundação para a Ciência e a Tecnologia (FCT), and C. McGrath was an employee of Pfizer when this study was planned and the data were extracted and analysed. Clare McGrath is now an employee of AstraZeneca. However, ideas expressed in this paper are entirely those of the authors and do not necessarily represent the views of FCT, Pfizer or AstraZeneca. Access to CPRD data was funded by Pfizer, but the publication of the study was not contingent on Pfizer’s approval or any censorship of the manuscript. We thank the reviewers of the CPRD Independent Scientific Advisory Committee for their insightful comments on the study protocol.

Author Contributions

All authors contributed extensively to the work presented in this paper. R. Refoios Camejo designed the study, extracted the data, conducted the analyses and prepared the manuscript; C. McGrath participated in the design of the study and contributed to the writing of the manuscript; M. Miraldo scrutinized the whole analytical procedure, ensuring the quality and validity of the analysis, and participated in the writing of the manuscript; and F. Rutten participated in the design of the study, contributed to the writing of the manuscript, and ensured the overall quality of the study. R. Refoios Camejo acts as guarantor for the overall content.

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Authors and Affiliations

  1. Institute of Health Policy & Management, Erasmus Medical Centre, Erasmus University, P.O. Box 1738, 3000 DR, Rotterdam, The Netherlands

    Rodrigo Refoios Camejo & Frans Rutten

  2. HTA Policy and Payer Evidence, AstraZeneca, Macclesfield, UK

    Clare McGrath

  3. Healthcare Management Group, Imperial College Business School, London, UK

    Marisa Miraldo

Authors
  1. Rodrigo Refoios Camejo
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  2. Clare McGrath
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  4. Frans Rutten
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Corresponding author

Correspondence to Rodrigo Refoios Camejo.

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Refoios Camejo, R., McGrath, C., Miraldo, M. et al. The Determinants of Cost-Effectiveness Potential: An Historical Perspective on Lipid-Lowering Therapies. PharmacoEconomics 31, 445–454 (2013). https://doi.org/10.1007/s40273-013-0041-x

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