Chapter 4 58 ABSTRACT Objectives: To evaluate the incremental value of new drugs across disease areas receiving favorable coverage decisions by the United Kingdom’s National Institute for Health and Care Excellence (NICE) over the past decade. Design, setting, and participants: This cross-sectional study assessed favorable appraisal decisions of drugs between January 1st, 2010 and December 31st, 2020. Estimates of incremental benefit were extracted from NICE’s evidence review groups reports. Primary outcome measure: Incremental benefit of novel drugs relative to the best alternative therapeutic option, expressed in Quality Adjusted Life Years (QALYs). Results: 184 appraisals of 129 drugs provided QALYs. The median incremental value was 0.27 QALY (interquartile range[IQR]: 0.07-0.73). Benefits varied across drug-indication pairs (range: -0.49-5.22 QALYs). The highest median benefits were found in hematology (0.70 QALY, IQR: 0.551.22) and oncology (0.46 QALY, IQR: 0.20-0.88), the lowest in ophthalmology (0.09, IQR: 0.04-0.22) and endocrinology (0.02, IQR: 0.01-0.06). Eight appraisals (4.3%) found contributions of more than two QALYs, but one in four (50/184) drug-indication pairs provided less than the equivalent of one month in perfect health compared to existing treatments. Conclusions: In our review period, the median incremental value of novel drugs approved for use within the English NHS, relative to the best alternative therapeutic option, was equivalent to three to four months of life in perfect health, but data were heterogeneous. Objective evaluations of therapeutic value help patients and physicians to develop reasonable expectations of drugs and delivers insights into disease areas where medicinal therapeutic progress has had the most and least impact.
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