Does the use of efficacy or effectiveness evidence in cost-effectiveness analysis matter?

Abstract

25 cost-effectiveness studies were included. Ten (40.0%) studies were effectiveness-based, yet 15 (60.0%) studies were efficacy-based. Of 17 studies using endpoints that could be compared to WTP threshold, 7 out of 8 (87.5%) effectiveness-based studies yielded favorable cost-effectiveness results, whereas 4 out of 9 (44.4%) efficacy-based studies yielded favorable cost-effectiveness results. The adjusted odds ratio was 15.12 (95% confidence interval; 0.59 to 388.75) for effectiveness-based versus efficacy-based achieving favorable cost-effectiveness findings. More asthma cost-effectiveness studies used efficacy-based evidence. Studies using effectiveness-based evidence trended toward being more likely to disseminate favorable cost-effective findings than those using efficacy. Health policy decision makers should pay attention to the type of clinical evidence used in cost-effectiveness studies for accurate interpretation and application.

Limitations of traditional health technology assessment methods and implications for the evaluation of novel therapies

“…Differences in baseline characteristics likely translate to differences in treatment costs and cost-offsets, which would in turn affect ICERs and, therefore, should routinely calculate ICERs by baseline risk. Compared with CEAs that utilize risk and efficacy estimates based on real world data (RWD), those based on clinical trial evidence often generate higher ICERs 25,26 . Several agencies have recommended using RWD to assess baseline risk and efficacy in CEA 2,[27][28][29] .…”

“…For example, despite the often debilitating effects of a migraine 39 , adherence and persistence to commonly prescribed prophylactic oral medications for episodic and chronic migraine are poor (<80%) and long-run (12)(13)(14)(15)(16)(17)(18)(19)(20)(21)(22)(23)(24)(25)(26) week) discontinuation rates are high ( 45%) in both randomized control trials (RCT) and real-world/community settings 40 . In two large RCTs, discontinuation rates for less frequently (every 12 weeks) administered injected onabotulinumtoxin A at 24 weeks was relatively low (10.4%) 41,42 .…”

Why published studies of the cost-effectiveness of PCSK-9 inhibitors yielded such markedly different results

“…better) than the RCT-based CE model. A review of 25 studies of asthma treatments, which compared CE models using RWD vs RCT data, found that studies which relied on RWD were twice as likely to conclude that a treatment was cost-effective 12 . Additionally, the UK's National Institute of Clinical Excellence Decision Support Unit recently published a report outlining the need to use RWD when estimating absolute treatment effects 13 .…”