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. 2022 Apr 8;12(4):e058279.
doi: 10.1136/bmjopen-2021-058279.

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

Tobias B Polak  1   2   3 , David GJ Cucchi  4   5 , Jonathan J Darrow  6 , Matthijs M Versteegh  7
Affiliations

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

Tobias B Polak et al. BMJ Open. .

Abstract

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.

Keywords: general medicine (see internal medicine); health policy; therapeutics.

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Conflict of interest statement

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).

Figures

Figure 1
Figure 1
Flow diagram of the selection and retrieval of estimates of quality-adjusted life-years (QALYs) from NICE technology appraisals between 1 January 2010 and 31 December 2020. NICE, National Institute for Health and Care Excellence.
Figure 2
Figure 2
The added value of novel pharmaceuticals approved by NICE from 2010 to 2020 display of the distribution (boxplot) of added value in QALYs of novel pharmaceuticals per medical discipline that have received a positive coverage decision of NICE between 1 January 2010 and 31 December 2020, compared with their next-best alternative. Medical disciplines with fewer than eight appraisals were classified as ‘other’. NICE, National Institute for Health and Care Excellence.
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