Does presenting outcome postponement (average survival gain) provide a more understandable measure of therapeutic benefit compared to traditional metrics like hazard ratio or relative risk reduction in cardiovascular trials?
Presenting trial results as outcome postponement (average survival gain) may be more easily understood by patients and physicians than traditional relative risk reductions or hazard ratios.
Background: The impact of randomised controlled trials (RCTs) depends heavily on the presentation of the findings.Objective: Classically, RCT findings are presented in the form of absolute risk reduction (ARR), number needed to treat (NNT) to prevent one adverse outcome, and relative risk reduction (RRR) or hazard ratio (the most favourable means for drug marketing). However, the estimation of average survival gain (i.e. outcome postponement between a trial intervention and comparator) is an alternative and informative means of presenting the findings of RCTs.Study selection: Recent cardiovascular RCTs evaluating ezetimibe added to simvastatin, evolocumab, canakinumab, ticagrelor, rivaroxaban, ivabradine, LCZ 696 (sacubitril/valsartan), and transfemoral aortic valve replacement are analysed and discussed.Findings: The average survival gains ranged between 4.9 days on a composite end point with ticagrelor versus clopidogrel in randomised patients with acute coronary syndrome and 117 days of life expectancy obtained with TAVR versus standard therapy in patients with severe aortic stenosis deemed ineligible for surgery.Conclusions: Using outcome postponement as an additional measure of treatment effect is likely to be more easily understood than hazard ratio or RRR by both patients and physicians and could help when evaluating drugs.
Ennezat et al. (2018) studied this question.