The predictive value of a positive phase 2 ASH abstract for peer-reviewed publication and progression to phase 3

Cucchi, David G. J.; Polak, Tobias B.; Ossenkoppele, Gert J.; Rowe, Jacob M.; Estey, Elihu H.

doi:10.1182/blood.2021013988

Cited by 1 publication

(2 citation statements)

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“…QALYs are a useful tool for comparison, but the measure omits important health-related variables, such as the extent to which a patient remains unable to live out a ‘normal’ life expectancy or achieve complete health. Other factors, such as lack of fundamental understanding of disease pathologies,20 21 or the abundance or absence of sufficient research funding may also limit health gains 22. Our figures evaluate the net present health-related benefits of drugs that are considered cost-effective by NICE over the past decade.…”

Section: Discussionmentioning

confidence: 99%

“…Other factors, such as lack of fundamental understanding of disease pathologies, 20 21 or the abundance or absence of sufficient research funding may also limit health gains. 22 Our figures evaluate the net present health-related benefits of drugs that are considered cost-effective by NICE over the past decade. In combination with indices measuring health needs, such as the Global Burden of Disease, 23 as well as cost-effectiveness/cost-saving data of novel drugs that might produce similar QALYs as already available therapies, our findings can help provide context for the allocation of research funding and thereby shape health policy.…”

Section: Discussionmentioning

confidence: 99%

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Incremental benefits of novel pharmaceuticals in the UK: a cross-sectional analysis of NICE technology appraisals from 2010 to 2020

et al. 2022

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ObjectivesTo evaluate the incremental value of new drugs across disease areas receiving favourable coverage decisions by the UK’s National Institute for Health and Care Excellence (NICE) over the past decade.Design, setting, and participantsThis cross-sectional study assessed favourable appraisal decisions of drugs between 1 January 2010 and 31 December 2020. Estimates of incremental benefit were extracted from NICE’s evidence review groups reports.Primary outcome measureIncremental benefit of novel drugs relative to the best alternative therapeutic option, expressed in quality-adjusted life-years (QALYs).Results184 appraisals of 129 drugs provided QALYs. The median incremental value was 0.27 QALY (IQR: 0.07–0.73). Benefits varied across drug-indication pairs (range: −0.49 to 5.22 QALY). The highest median benefits were found in haematology (0.70, IQR: 0.55–1.22) and oncology (0.46, 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 1 month in perfect health compared to existing treatments.ConclusionsIn our review period, the median incremental value of novel drugs approved for use within the English National Health System, relative to the best alternative therapeutic option, was equivalent to 3–4 months of life in perfect health, but data were heterogeneous. Objective evaluations of therapeutic value helps 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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Section: Discussionmentioning

confidence: 99%