When to wait for more evidence?: real options analysis in proton therapy.

Purpose. Trends suggest that cancer spending growth will accelerate. One method for controlling costs is to examine whether the benefits of new technologies are worth the extra costs. However, especially new and emerging technologies are often more costly, while limited clinical evidence of superiority is available. In that situation it is often unclear whether to adopt the new technology now, with the risk of investing in a suboptimal therapy, or to wait for more evidence, with the risk of withholding patients their optimal treatment. This trade-off is especially difficult when it is costly to reverse the decision to adopt a technology, as is the case for proton therapy. Real options analysis, a technique originating from financial economics, assists in making this trade-off. Methods. We examined whether to adopt proton therapy, as compared to stereotactic body radiotherapy, in the treatment of inoperable stage I non-small cell lung cancer. Three options are available: adopt without further research; adopt and undertake a trial; or delay adoption and undertake a trial. The decision depends on the expected net gain of each option, calculated by subtracting its total costs from its expected benefits. Results. In The Netherlands, adopt and trial was found to be the preferred option, with an optimal sample size of 200 patients. Increase of treatment costs abroad and costs of reversal altered the preferred option. Conclusion. We have shown that real options analysis provides a transparent method of weighing the costs and benefits of adopting and/or further researching new and expensive technologies. The Oncologist 2011;16:1752-176

Mindfulness-based cognitive therapy versus treatment as usual in adults with ADHD: A trial-based economic evaluation

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Clinical biomarker innovation: when is it worthwhile?

Contains fulltext : 208980.pdf (publisher's version ) (Open Access

Linked Pharmacometric-Pharmacoeconomic Modeling and Simulation in Clinical Drug Development

Market access and pricing of pharmaceuticals are increasingly contingent on the ability to demonstrate comparative effectiveness and cost-effectiveness. As such, it is widely recognized that predictions of the economic potential of drug candidates in development could inform decisions across the product life cycle. This may be challenging when safety and efficacy profiles in terms of the relevant clinical outcomes are unknown or highly uncertain early in product development. Linking pharmacometrics and pharmacoeconomics, such that outputs from pharmacometric models serve as inputs to pharmacoeconomic models, may provide a framework for extrapolating from early-phase studies to predict economic outcomes and characterize decision uncertainty. This article reviews the published studies that have implemented this methodology and used simulation to inform drug development decisions and/or to optimize the use of drug treatments. Some of the key practical issues involved in linking pharmacometrics and pharmacoeconomics, including the choice of final outcome measures, methods of incorporating evidence on comparator treatments, approaches to handling multiple intermediate end points, approaches to quantifying uncertainty, and issues of model validation are also discussed. Finally, we have considered the potential barriers that may have limited the adoption of this methodology and suggest that closer alignment between the disciplines of clinical pharmacology, pharmacometrics, and pharmacoeconomics, may help to realize the potential benefits associated with linked pharmacometric-pharmacoeconomic modeling and simulation

Cost-effectiveness of Magnetic Resonance (MR) Imaging and MR-guided Targeted Biopsy Versus Systematic Transrectal Ultrasound-guided Biopsy in Diagnosing Prostate Cancer: A Modelling Study from a Health Care Perspective

Item does not contain fulltextBACKGROUND: The current diagnostic strategy using transrectal ultrasound-guided biopsy (TRUSGB) raises concerns regarding overdiagnosis and overtreatment of prostate cancer (PCa). Interest in integrating multiparametric magnetic resonance imaging (MRI) and magnetic resonance-guided biopsy (MRGB) into the diagnostic pathway to reduce overdiagnosis and improve grading is gaining ground, but it remains uncertain whether this image-based strategy is cost-effective. OBJECTIVE: To determine the cost-effectiveness of multiparametric MRI and MRGB compared with TRUSGB. DESIGN, SETTING, AND PARTICIPANTS: A combined decision tree and Markov model for men with elevated prostate-specific antigen (>4 ng/ml) was developed. Input data were derived from systematic literature searches, meta-analyses, and expert opinion. OUTCOME MEASUREMENTS AND STATISTICAL ANALYSIS: Quality-adjusted life years (QALYs) and health care costs of both strategies were modelled over 10 yr after initial suspicion of PCa. Probabilistic and threshold analyses were performed to assess uncertainty. RESULTS AND LIMITATIONS: Despite uncertainty around the presented cost-effectiveness estimates, our results suggest that the MRI strategy is cost-effective compared with the standard of care. Expected costs per patient were euro 2423 for the MRI strategy and euro 2392 for the TRUSGB strategy. Corresponding QALYs were higher for the MRI strategy (7.00 versus 6.90), resulting in an incremental cost-effectiveness ratio of euro 323 per QALY. Threshold analysis revealed that MRI is cost-effective when sensitivity of MRGB is >/= 20%. The probability that the MRI strategy is cost-effective is around 80% at willingness to pay thresholds higher than euro 2000 per QALY. CONCLUSIONS: Total costs of the MRI strategy are almost equal with the standard of care, while reduction of overdiagnosis and overtreatment with the MRI strategy leads to an improvement in quality of life. PATIENT SUMMARY: We compared costs and quality of life (QoL) of the standard "blind" diagnostic technique with an image-based technique for men with suspicion of prostate cancer. Our results suggest that costs were comparable, with higher QoL for the image-based technique

A Survey Method For The Assessment Of The Dental Health And Treatment Needs Of A Population

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Uncertainty on the effectiveness and safety of rivaroxaban in premenopausal women with atrial fibrillation: empirical evidence needed

Contains fulltext : 182213.pdf (publisher's version ) (Open Access)BACKGROUND: Novel anticoagulations (NOACs) are increasingly prescribed for the prevention of stroke in premenopausal women with atrial fibrillation. Small studies suggest NOACs are associated with a higher risk of abnormal uterine bleeds than vitamin K antagonists (VKAs). Because there is no direct empirical evidence on the benefit/risk profile of rivaroxaban compared to VKAs in this subgroup, we synthesize available indirect evidence, estimate decision uncertainty on the treatments, and assess whether further research in premenopausal women is warranted. METHODS: A Markov model with annual cycles and a lifetime horizon was developed comparing rivaroxaban (the most frequently prescribed NOAC in this population) and VKAs. Clinical event rates, associated quality adjusted life years, and health care costs were obtained from different sources and adjusted for gender, age, and history of stroke. A Monte Carlo simulation with 10,000 iterations was then performed for a hypothetical cohort of premenopausal women, estimated to be reflective of the population of premenopausal women with AF in The Netherlands. RESULTS: In the simulation, rivaroxaban is the better treatment option for the prevention of ischemic strokes in premenopausal women in 61% of the iterations. Similarly, this is 98% for intracranial hemorrhages, 24% for major abnormal uterine bleeds, 1% for minor abnormal uterine bleeds, 9% for other major extracranial hemorrhages, and 23% for other minor extracranial hemorrhages. There is a 78% chance that rivaroxaban offers the most quality-adjusted life years. The expected value of perfect information in The Netherlands equals 122 quality-adjusted life years and 22 million Euros. CONCLUSIONS: There is a 22% risk that rivaroxaban offers a worse rather than a better benefit/risk profile than vitamin K antagonists in premenopausal women. Although rivaroxaban is preferred over VKAs in this population, further research is warranted, and should preferably take the shape of an internationally coordinated registry study including other NOACs

The potential health gain and cost savings of improving adherence in chronic myeloid leukemia

Contains fulltext : 204847.pdf (publisher's version ) (Open Access)Healthcare costs are rising due to an increase in chronic diseases, including chronic myeloid leukemia (CML) due to improved survival. In CML care, patient adherence and physician adherence are key elements. We assessed the potential health gain and cost savings when both are improved, using a decision analytic model that integrated various sources of evidence. The current situation was compared to a theoretical situation in which either patient or physician adherence is improved, in terms of costs and quality-adjusted life years (QALYs). Current patient adherence rate is 74%, improvement to 100% resulted in 0.1031 QALYs gained and a saving of euro17,509 per patient over a 25-year period. Improving physician adherence from 72% to 100%, resulted in 0.0380 QALYs and euro7606. Enhancement of either adherence results in substantial health gain and cost savings. Regarding the rising healthcare costs, new strategies should focus on improving adherence to keep healthcare affordable in the future