Incremental benefits of novel pharmaceuticals in the United Kingdom 63 4♣ In our review period, eight (4.3%) positive coverage decisions were granted to drugs contributing more than the equivalent of two life years in perfect health. Both dinutuximab beta to treat neuroblastoma and nusinersen used to treat children with spinal muscular atrophy led patients to accumulate 5.2 incremental QALYs. On the other hand, 50 (27%) drugs contributed no more than the equivalent of one month in perfect health over the best alternative therapeutic option (≤ 0.082 QALY) (Table 3). Eight drugs were estimated to provide lower QALY gains than their next best alternative. Government decision makers may nevertheless be willing to pay for such products thanks to the uncertainty around point estimates, together with strategic pricing by manufacturers. For example, one drug, venetoclax, was estimated to be inferior to its direct comparator (ibrutinib) in the treatment of chronic lymphocytic leukemia. Although this negative point estimate was considered most plausible by the evidence review group, there was still considerable uncertainty remaining as the group also provided higher estimates (an incremental benefit of 0.51 when idelalisib was the comparator) and lower estimates (-1.75 when treatment effects of venetoclax were assumed to be waning faster than expected) under varying assumptions. Venetoclax was offered at a lower price than ibrutinib, and NICE concluded that the new drug was likely a cost-effective use of NHS resources in the treatment of lymphocytic leukemia.18
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