Incremental benefits of novel pharmaceuticals in the United Kingdom 59 4♣ INTRODUCTION Before a novel treatment is allowed on the market, its clinical benefit is assessed by regulatory agencies such as the US Food and Drug Administration (FDA) and the European Medicines Agency (EMA). However, clinical benefit evaluations do not provide insight into issues deemed relevant by payers, such as comparative effectiveness, cost effectiveness, or lifetime benefit. Therefore, several countries have created independent health technology assessment bodies to conduct drug value assessments, commonly referred to as cost effectiveness analyses.11 Through these value assessments, publicly-funded experts help to clarify the incremental clinical benefit and incremental costs of selected new therapies according to their approved indications, which professional societies may then rely on when revising treatment guidelines to include the new drug. Despite the increased focus on incremental drug value, surprisingly little attention has been devoted to understanding the magnitude and distribution of their clinical benefits across disease areas. The limited scholarship in this area can be explained in part by the fact that, until recently, it has been difficult to compare the benefit of drugs intended to treat widely divergent diseases or conditions. However, the emergence of official government drug value assessments over the past two decades, rigorously conducted following a consistent set of health economic modelling guidelines, now makes such comparisons feasible. These assessments utilize the Quality Adjusted Life Year (QALY), a common metric of patient health. One QALY, for example, represents the equivalent of one additional year of life in perfect health, or some longer period of time in less-thanperfect health.12,13 Although the QALY has long been available as a measure and is frequently used in individual economic evaluations,14 the QALY can, in combination with forecasts over the lifetime of patients from health economic models, be used to compare health benefits across medical disciplines in a consistent and transparent manner. QALYs are primarily used to calculate incremental cost-effectiveness ratios (ICER), which signals the efficiency with which a health technology produces health by dividing incremental costs by incremental benefits expressed as QALYs. However, it is often overlooked that the QALY part of an ICER is, in and of itself, a parameter that provides relevant insights into the size of forecasted health benefit. In the case of the UK, QALYs are produced following specific modelling guidance by the National Institute for Health and Care Excellence (NICE), enhancing their comparability across diseases. NICE is a non-departmental public body that assesses the value of novel drugs and the impact on the English National Health System (NHS) of adopting them. Since NICE was established in 1999, drug manufacturers have been invited to submit evidence on the health benefits and costs
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