Original articleNumber needed to treat: easily understood and intuitively meaningful?: Theoretical considerations and a randomized trial
Introduction
Considerable proportions of health care resources are devoted first to detect and second to ameliorate risk factors such as hypertension, dyslipidaemia, osteoporosis, or certain types of life style. An important issue is then how to inform clinicians and patients about the benefits of interventions that delay or prevent chronic diseases. These benefits can be expressed in several ways such as absolute risk reduction (ARR), its reciprocal, the number needed to be treated (NNT), relative risk reduction (RRR), differences in event-free patients, the number of avoided events within a population, or as the gain in life expectancy.
First proposed by Laupacis and coworkers [1], the NNT has been recommended for communicating benefits from risk reductions 2, 3, 4, 5, 6. Advocates of evidence-based medicine claim that the “number needed to be treated (NNT) to prevent one event is the most useful measure of clinical effort … patients must expend in order to help them avoid bad outcomes” [7]. It is further claimed that NNT is “a currency for making decisions” [8], that it is “easily understood by clinicians” [1], and that it “has intuitive meaning” [9]. The use of NNT has been expanded to encompass harm (“number needed to harm”—NNH), screening (“number needed to screen”) [10], and education (“number needed to educate”) [11].
There is ample evidence that doctors 12, 13, 14, health care managers [15], and patients [16] are more likely to accept interventions where the benefits are presented in terms of RRR rather than ARR. Some authors claim that the RRR “may overstate the effectiveness of the treatment” [17], but the basis for judging what is “overstating” is unclear.
Although NNT is often used in medical publications and possibly also in clinical practice, there is little evidence that patients or doctors make more appropriate decisions when they use NNT rather than other measures of benefit [18]. By “appropriate” we mean decisions that are in line with those that patients and doctors would make when they have received and comprehended all relevant information.
In the following we will examine some theoretical properties of NNT, develop hypotheses about how lay people comprehend NNT, and test them empirically.
Section snippets
Theoretical properties of NNT
If the risk of an adverse event is Dc in a control group and Di in an intervention group at some point in time, the proportion (Dc − Di)/Dc is RRR while the difference (Dc − Di) is ARR. The reciprocal of ARR is NNT. In the following, NNT denotes the concept and nnt a specific value of it. The nnt expresses how many patients on average have been treated per event avoided at a specific point in time. When NNT sometimes is interpreted as ‘the number of patients needed to be treated for x years in
Methods
A random sample (n = 675) of noninstitutionalised Danes aged 20–74 was interviewed face to face through a so-called “Computer Assisted Personal Interviewing” by Gallup Inc, Denmark. When sampled individuals were not at home, up to two repeat calls were made. The interview questions for this paper were clustered with those for other customers of Gallup, but our items were placed at the beginning of the interview due to their complexity. For each week of interviewing, items from different
Results
In total, 675 subjects were interviewed. Their mean age was 44 years, and 51% were female, the same as for the Danish population of the same age range. The income distribution was similar to that of the Danish population, while the study sample had a lower proportion of individuals with elementary school only (21% vs. 37% in the general population). The health status was reported excellent among 29% of the respondents and less or poor among 10%. Heart disease was reported among 3.3% of the
Discussion
NNT has important shortcomings as a single measure of health benefit in that NNT measures benefit at an arbitrary point in time and fails to capture effects over time. Contrary to previous claims, NNT does not indicate the individual patient's probability of having benefit from a treatment when the aim of it is to delay an adverse event. The empirical study presented here lends little support to the claim that NNT is easily understood in that nnt had little influence on the proportions that
Acknowledgements
Financial support: Danish Heart Foundation. Comments from Gavin Mooney on the manuscript are gratefully acknowledged. Conflict of interest: none.
References (26)
- et al.
Completeness of reporting trial resultseffect on physicians' willingness to prescribe
Lancet
(1994) - et al.
Absolutely relativehow research results are summarized can affect treatment decisions
Am J Med
(1992) - et al.
Effect of ACE inhibitor trandolapril on life expectancy of patients with reduced left-ventricular function after acute myocardial infarction. TRACE Study Group
Trandolapril Cardiac Evaluation. Lancet
(1999) - et al.
Implications of trial resultsthe potentially misleading notions of number needed to treat and average duration of life gained
Lancet
(2000) - et al.
An assessment of clinically useful measures of the consequences of treatment
N Engl J Med
(1988) - et al.
The number needed to treata clinically useful measure of treatment effect
BMJ
(1995) - et al.
Basic statistics for clinicians3. Assessing the effects of treatment: measures of association
CMAJ
(1995) - et al.
Meaningful interpretation of risk reduction from clinical drug trials
Ann Pharmacother
(1993) - et al.
“Unqualified success” and “unmitigated failure”number-needed- to-treat-related concepts for assessing treatment efficacy in the presence of treatment-induced adverse events
Int J Epidemiol
(1996) - et al.
Perceptions of breast cancer risk and screening effectiveness in women younger than 50 years of age
J Natl Cancer Inst
(1995)
Evidence-based medicine. How to Practice and teach EBM
Using numerical results from systematic reviews in clinical practice
Ann Intern Med
Adjusting the number needed to treatincorporating adjustments for the utility and timing of benefits and harms
Med Decis Making
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