Outcome postponement as a potential patient centred measure of therapeutic benefit: examples in cardiovascular medicine

Pierre Vladimir Ennezat, Thierry Le Jemtel, Shona Cosgrove, Jesper Hallas, Morten Rix Hansen

Research output: Contribution to journalComment/debateResearchpeer-review

Abstract

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.

Original languageEnglish
JournalActa Cardiologica
Volume75
Issue number1
Pages (from-to)10-19
ISSN0001-5385
DOIs
Publication statusPublished - Jan 2020

Fingerprint

Randomized Controlled Trials
Medicine
Numbers Needed To Treat
clopidogrel
Risk Reduction Behavior
ivabradine
Simvastatin
Acute Coronary Syndrome
Life Expectancy
Marketing
Pharmaceutical Preparations
Physicians
Ticagrelor

Keywords

  • Cardiovascular therapeutic trials
  • number needed to treat
  • outcome postponement
  • survival gain
  • therapeutic benefit

Cite this

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abstract = "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.",
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Outcome postponement as a potential patient centred measure of therapeutic benefit : examples in cardiovascular medicine. / Ennezat, Pierre Vladimir; Le Jemtel, Thierry; Cosgrove, Shona; Hallas, Jesper; Hansen, Morten Rix.

In: Acta Cardiologica, Vol. 75, No. 1, 01.2020, p. 10-19.

Research output: Contribution to journalComment/debateResearchpeer-review

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AU - Ennezat, Pierre Vladimir

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AU - Cosgrove, Shona

AU - Hallas, Jesper

AU - Hansen, Morten Rix

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N2 - 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.

AB - 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.

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