A27Objectives: Cost effectiveness analyses play a critical role in determining coverage of novel drugs and devices. Increasingly, payers are demanding subgroup analyses to determine indications which would be covered by the national health system or insurance agency. MethOds: To understand and review trends in the use of subgroup cost effectiveness analysis, we analyzed NICE HTAs for products approved between 2011-2012. Manufacturer submissions for CEA were compared to final review and decision by HTA agency. Analogs were identified and case studies were developed to further understand the use of subgroup analyses and cost effectiveness models. Results: Decisions made by NICE in 2011-2012 show increasing trends towards the use of subgroup analysis for determining indications for coverage by national payer bodies. Between 2011-2012, 80% of the assessments included subgroup analyses. Approximately half of them included cost effectiveness analyses for various subgroups. Interestingly, the ICER values estimated by NICE for the same subgroups showed a large variation (1X-3X fold difference) compared to ICER values estimated by manufacturers. Selected case studies highlighted that for several products, NICE is recommending treatments only for subgroups whose ICER values are within the cost effectiveness threshold. cOnclusiOns: New products need robust broader population and subgroup analyses for insurance coverage.
Objectives: Many initiatives (e.g., PROTECT, EFSPI) are exploring quantitative methodologies to conduct benefit/risk assessments of medicines. Objectives of this study were to combine quantitative methodologies that can capture expert knowledge and decisionmakers insights to genuinely support real-world decisions. MethOds: Using the case study of efalizumab, approved by the EMA in 2004 for the treatment of plaque psoriasis and withdrawn in 2009, a pragmatic methodology was developed that combines advanced pharmacoepidemiology and MCDA for quantitative benefit/ risk assessment. Development involved application of: MCDA principles to ensure applicability to any therapeutic area, comparability across medicines, and portability over product cycle (re-evaluation); and advanced pharmacoepidemiology and Bayesian modeling to identify/generate most useful data. Overarching design was guided by ethical implications of criteria and data selection as well as applicability in real life settings including face validity, time constraints, complexity and transparency. Results: The hierarchical multicriteria model consists of two major domains: Benefits (favourable effects, covering the criteria Clinical efficacy/effectiveness and Patient Reported outcomes); and Risks (unfavourable effects -criterion Safety). The safety criterion is subdivided into three generic subcriteria (Adverse events, Serious AEs and Fatal AEs). The benefit criteria are subdivided into specific subcriteria that correspond to the most relevant outcomes for a treatment for plaque psoriasis. All performance are assigned relative to existing alternatives or placebo. Each subcriterion contributes to the output of the model, the Benefit/Risk Estimate, which is the sum of normalized weights for each subcriterion multiplied by the respective performance score. Pharmacoepidemiology data is provided in a standardized format for each subcriterion and includes meta-analytic comparative statistics based on clinical trials, observational data and Bayesian models. Uncertainty is explored in sensitivity analyses. cOnclusiOns: Integration of pragmatic MCDA modeling with advanced pharmacoepidemiology allows quantitative benefit/risk assessment that can be applicable and meaningful in real life regulatory settings.
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