A Decade of Health Innovation: The Impact of New Medicines on Patient Health and the Implications for NICE's Size of Benefit Multiplier.

ABSTRACT

OBJECTIVES: This study aimed to estimate the incremental health benefits of pharmaceutical innovations approved between 2011 and 2021 and the share that would surpass the National Institute for Health and Care Excellence (NICE) "size of benefit" decision weight thresholds. METHODS: We identified all US-approved drugs between 2011 and 2021. Health benefits, in terms of quality-adjusted life-years (QALYs) for each treatment, were extracted from published cost-effectiveness analyses. Summary statistics by therapeutic area and cell/gene therapy status identified the treatments with the largest QALY gains. RESULTS: The Food and Drug Administration approved 483 new therapies between 2011 and 2021 and of these 252 had a published cost-effectiveness analysis meeting our inclusion criteria. The average incremental health benefits produced by these treatments were 1.04 QALYs (SD = 2.00) relative to standard of care, with wide variation by therapeutic area. Pulmonary and ophthalmologic therapies produced the highest health benefits with 1.47 (SD = 2.17, n = 13) and 1.41 QALYs gained (SD = 3.53, n = 7), respectively; anesthesiology and urology had the lowest gains (< 0.1 QALYs). Cell and gene therapies produced an average health benefit that was 4 times greater than noncell and gene therapies (4.13 vs 0.96). Among the top treatments in terms of incremental QALYs gained, half (10 of 20) were oncology therapies. Three of 252 treatments (1.2%) met NICE's threshold for a "size of benefit" multiplier. CONCLUSIONS: Treatments for rare disease, oncology, and cell and gene therapies produced some of the highest level of health innovation relative to previous standard of care, but few therapies would have qualified for NICE's "size of benefit" multiplier as currently constructed.