PRM27 Evaluating European Data Sources for Assessing Real-World Effectiveness of New Anti-Diabetic Drugs

Abstract

medical device, whilst crucial for late-stage reimbursement decisions, is often neglected by developers early on due to lack of data. The headroom method has been proposed as a way to integrate economic evaluation into early go/no-go decisions. By estimating the maximum reimbursable price (MRP) for a new device idea and comparing this with forecasted developer's costs, R&D resources can be channelled toward innovations for which future returns appear feasible. The aim of this study was to evaluate the method by applying it retrospectively to a large and diverse set of case studies, and comparing predicted 'headroom' with actual market success, within the UK setting. METHODS: The method was applied systematically to twenty devices/diagnostics invented in the past, retrieved from the UK national horizon scanning centre (NHSC)'s 2000-2009 database; literature searches were date restricted to mimic the information available at the 'concept stage' of development. Each case study was followed up to observe the product's actual market success or failure compared with the estimated 'headroom' and the development decision this would have engendered, in order to explore the method's predictive value. RESULTS: Headroom correlated with subsequent market success in 85% of cases. Headroom is most easily elicited where the change proposition of the innovation is straightforward (e.g. direct replacement technologies); diagnostic equipment posed particular difficulties. When the numerical headroom assessments were considered alongside unquantifiable factors identified relating to the clinical and market context, the method offered a good indication of future market potential. CONCLUSIONS: Despite the strong advocacy of early economic evaluation, the literature lacks a critical appraisal of any such method. This study for the first time explores the potential implications of basing development decisions on the headroom method, thereby assessing its potential value to stakeholders. OBJECTIVES:Clinical trials remain the gold standard for proving efficacy but restrictive enrolment criteria and overly controlled study environments limit generalizability. Increasingly, payers and health care providers are interested in real world evidence to support the case for pharmaceutical innovations. We review A465