TY - JOUR
T1 - Incremental benefits of novel pharmaceuticals in the UK
T2 - a cross-sectional analysis of NICE technology appraisals from 2010 to 2020
AU - Polak, Tobias B.
AU - Cucchi, David G. J.
AU - Darrow, Jonathan J.
AU - Versteegh, Matthijs M.
N1 - Funding Information: Competing interests TBP is employed part-time by myTomorrows, which facilitates expanded access programs for the pharmaceutical industry. TBP holds stock in myTomorrows (<0.1%). TBP has received funding for research from the Dutch government (grant EMCLSH20012). The research of TBP is conducted independently and TBP is contractually free to publish any results for all the conducted work. None of the recent work concerns the topic in this analysis. DGJC received speaker fees from Takeda. MMV is director of the institute of Medical Technology Assessment (iMTA). iMTA conducts cost-effectiveness research funded by international governments, pharmaceutical industry, and med-tech industry. All research is conducted independently and iMTA is contractually free to publish any results for all conducted work. None of the recent work concerns the topic in this analysis. JJD receives funding from Arnold Ventures, the Greenwall Foundation, the Kaiser Permanente Institute for Health Policy, West Health, and the Novo Nordisk Foundation (grant for a scientifically independent Collaborative Research Programme; grant NNF17SA0027784). Publisher Copyright: © 2022 BMJ Publishing Group. All rights reserved.
PY - 2022/4/8
Y1 - 2022/4/8
N2 - OBJECTIVES: To 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 PARTICIPANTS: This 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 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 (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. CONCLUSIONS: In 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.
AB - OBJECTIVES: To 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 PARTICIPANTS: This 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 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 (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. CONCLUSIONS: In 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.
KW - general medicine (see internal medicine)
KW - health policy
KW - therapeutics
UR - http://www.scopus.com/inward/record.url?scp=85127880901&partnerID=8YFLogxK
U2 - https://doi.org/10.1136/bmjopen-2021-058279
DO - https://doi.org/10.1136/bmjopen-2021-058279
M3 - Article
C2 - 35396306
SN - 2044-6055
VL - 12
SP - e058279
JO - BMJ Open
JF - BMJ Open
IS - 4
M1 - e058279
ER -