A systematic review of pharmacoeconomic studies of rare disease drugs from a societal perspective
YANG Meng-yu
WANG Lin-ning
WANG Jia-xin
GU Xiao
GUO Xin-xin
LIU Yu-hong
SONG Yuan
LU Yun
Abstract：Objective:To systematically review pharmacoeconomic studies on the recognized rare diseases in China from a societal perspective and examine the current status of inclusion of treatment costs.Methods:A systematic search was conducted using the CEAR database to collect pharmacoeconomic studies on the treatment of 207 rare diseases recognized in China from a societal perspective,covering the period from database inception to November 2023.Two researchers independently screened literature,extracted data,and performed a qualitative systematic review.The quality of the included studies was assessed using the CHEERS 2022 reporting tool.Additionally,the studies were compared and summarized to evaluate the considerations on the societal impact of disease treatment.Results:Sixty-two pharmacoeconomic studies were included,covering 15 rare diseases recognized in China,with literature from 14 countries.Among these,26 studies conducted economic evaluation using SOC,BSC,placebo,or no treatment as control groups,while another 26 studies compared different drug interventions.Of all the studies,50 adopted a societal perspective exclusively,while the remaining 12 included both healthcare system or payer perspectives alongside the societal perspective.Most studies considered direct medical costs,direct non-medical costs,and indirect costs.Among the included studies,52 were model-based,and 60 applied uncertainty analysis to explore the robustness of their results.The reporting quality of most included studies was relatively high.Forty-two studies explicitly mentioned indirect costs such as productivity losses of patients and caregivers;however,only 35 detailed the inclusion methods,all of which employed the human capital approach,with varying levels of details in the calculation processes across studies.Conclusion:Current evidence suggests that there are relatively few pharmacoeconomic studies on rare disease medications from a societal perspective.The inclusion of indirect costs is not standardized,and improvements are needed in how costs are measured and included,and in the clarity and thoroughness of the evaluation process.
Keywords：rare diseasessystematic revieweconomic evaluationsocietal costindirect cost
Publication Date：2025-09-30
Online Publishing Date：2026-08-14(First online date of this platform, not the publication date of the document)
Pages：9( 2002-2010 )