What Is the Net Added Value of Personalised Medicine? A Systematic Literature Review
Author(s)
Vellekoop H1, Huygens S1, Versteegh M2, Corro Ramos I3, Szilberhorn L4, Zelei T4, Nagy B4, Koleva-Kolarova R5, Tsiachristas A6, Wordsworth S6, Rutten-van Mölken M7
1institute for Medical Technology Assessment, Rotterdam, Netherlands, 2Institute for Medical Technology Assessment, Erasmus School of Health Policy & Management, Erasmus University, Rotterdam, ZH, Netherlands, 3institute for Medical Technology Assessment, Eindhoven, NB, Netherlands, 4Syreon Research Institute, Budapest, Hungary, 5University of Oxford, Oxford, OXF, UK, 6University of Oxford, Oxford, UK, 7Erasmus University Rotterdam, Rotterdam, Netherlands
OBJECTIVES Amidst high expectations about the benefits of personalised medicine (PM) as well as concerns about the potentially high costs of implementing PM, we sought an overview of the available evidence on the added value of PM. METHODS A systematic literature review was conducted of economic evaluations of PM published between 2009 and 2019. Various data items were extracted for included studies, with key items being patient-level incremental quality-adjusted life-years (ΔQALYs) and incremental costs (Δcosts). Δcosts were expressed in 2020 international dollars (Int $). ΔQALYs and Δcosts were combined with estimates of the national cost-effectiveness thresholds to calculate incremental net monetary benefit (ΔNMB). ΔQALYs, Δcosts and ΔNMB were subsequently used as dependent variables in regression analysis, with ‘PM category’, ‘type of treatment’ and ‘industry sponsorship’ as independent variables. Random effects estimation was used, with studies nested under the country in which the evaluation was set. RESULTS Out of 4,774 studies identified, 139 were included, providing cost-effectiveness outcomes for 314 PM interventions. Most interventions were evaluated in the US and the UK (48% and 15%, respectively), using a healthcare perspective (81%). 59% of interventions were for neoplasms and 73% were pharmaceutical treatments. The least common PM category was ‘therapy that involves genetic modification (gene therapy)’ (6%), while ‘testing to identify likely (non-)responders to treatment and subsequent therapeutic choice’ was most common (31%). Median (mean) ΔQALYs, Δcosts and ΔNMB were 0.03 (0.39), Int$ 588 (Int$ 85,899) and Int$ -24 (Int$ -71,409), respectively. Regression analysis showed that while gene therapies were associated with higher ΔQALYs than other PM categories, they were also associated with higher costs and much lower ΔNMB. CONCLUSIONS PM adds health, but its costs tend to result in zero to negative net monetary benefit.
Conference/Value in Health Info
2021-11, ISPOR Europe 2021, Copenhagen, Denmark
Value in Health, Volume 24, Issue 12, S2 (December 2021)
Code
POSB75
Topic
Economic Evaluation
Topic Subcategory
Cost-comparison, Effectiveness, Utility, Benefit Analysis
Disease
Genetic, Regenerative and Curative Therapies, Multiple Diseases, Personalized and Precision Medicine
Explore Related HEOR by Topic