Efficacy and effectiveness are two concepts related to assessing the health care innovations. These concepts were defined by famous British Clinical Epidemiologist, Archie Cochrane. Although, they sound and look same, there are some major differences between these two terms. Before looking at the differences, we must understand that the distinction between efficacy and effectiveness should be viewed as a spectrum, not a strict dichotomy.
Table of Contents
Now, let’s have a look the differences:
Efficacy |
Effectiveness |
Efficacy is the extent to which an intervention does more good than harm under ideal circumstances | Effectiveness assesses whether an intervention does more good than harm when provided under usual/practical circumstances |
Efficiency measures the degree of success of an intervention in ideal conditions | Effectiveness measures the degree of success of an intervention under normal or usual circumstances |
While assessing the efficacy of the study/intervention, the research question must be explanatory | While assessing the efficacy of the study/intervention, the research question must be pragmatic |
Efficacy measures how well an intervention produces its desired result under perfect/ideal conditions | Effectiveness measure the real-world success, easiness and difficulties of an intervention |
Efficacy describes how the intervention works in ideal, controlled or laboratory setting. | Effectiveness describes how the intervention works in real-world setting where different variables/factors cannot be controlled |
Efficacy checks the success in ideal condition | Effectiveness checks the success in real world |
It asks the question: Can a treatment/intervention work under ideal circumstances? | It asks the question: Does the treatment/intervention work in real world? |
Efficacy studies provide a ground base for effectiveness studies. This is because efficacy studies show the effect of an intervention in an ideal condition. This will provide a base for effectiveness study to see the result in real world setting. | Effectiveness studies can be valuable to test whether and how much the results of efficacy trials are applicable to practical life. |
Characteristic differences of efficacy studies and effectiveness studies: |
|
Efficacy studies/trial are also known as explanatory trial | Effectiveness studies/trial are also known as pragmatic trial |
Efficacy trials are used to study or see if the intervention has a specific effect | Effectiveness trials are used to study or capture the multi-factorial nature of interventions and inform the result of intervention in practical setting. |
Efficacy trials have strict inclusion and exclusion criteria for study population | Effectiveness trials have limited exclusion criteria and it involves more heterogeneous population |
Efficacy studies are usually conducted in large populations/large tertiary care which have more facilities, infrastructure and better technical equipment. | Effectiveness studies are usually conducted in more practical situations which are applicable to diverse population. |
Intensive resources are dedicated to these studies | Less resources are dedicated to these studies |
These studies use features such as randomization and blinding | These studies can be randomized but they are rarely blinded |
It uses placebo | It may rarely use placebo |
These studies do everything possible to maximize the chance of showing an effect of treatment/intervention | These studies allow the real-world factors to take place and try harder to duplicate the situations that will be encountered in real-world |
In these studies, compliance is closely monitored and ensured | In these studies, compliance is not monitored closely and given less value |
It has high internal validity | It has high external validity |
The study is done following the protocol strictly | The study does not strictly limit to the protocols, rather is gives space for practical contexts |
It usually has fewer missing data | It usually has more missing data |
The outcome measures of efficacy studies are more objective | The outcome measures of effectiveness studies are more subjective |
Study process is strictly defined | Study process is loosely defined |
It has less bias and confounders as they are minimized | It has more bias and confounders. Moreover, the effect of bias and confounders is also substantial. |
These studies/trials can establish the cause-effect relationship | These studies/trials can rarely establish cause-effect relationships |
References and for More Information:
https://www.ncbi.nlm.nih.gov/pmc/articles/PMC3726789/
https://www.managedcaremag.com/archives/2013/1/effectivenessefficacy-difference-too-often-ignored
https://jamanetwork.com/journals/jamapediatrics/article-abstract/348184
https://onlinelibrary.wiley.com/doi/pdf/10.1111/j.1365-2796.2006.01707.x
http://pediaa.com/difference-between-efficacy-and-effectiveness/
http://www.physiologicalpt.com/physiological/2014/09/09/efficacy-vs-effectiveness
https://www.nature.com/articles/ctg201313
https://www.jospt.org/doi/pdf/10.2519/jospt.2003.33.4.163
https://journals.sagepub.com/doi/pdf/10.1177/070674370204700607
https://www.healthcare-economist.com/2016/01/25/efficacy-vs-effectiveness-vs-efficiency/
https://rwe-navigator.eu/clarify-the-issues/understanding-effectiveness-vs-efficacy-intervention/
https://writingexplained.org/efficacy-vs-efficiency-difference
https://pdfs.semanticscholar.org/ef59/c29198efb1eb8704073049005538c8592aa9.pdf
https://forum.wordreference.com/threads/effectiveness-vs-efficacy.1903546/
https://thetranslationalscientist.com/issues/0716/efficacy-vs-effectiveness/
https://www.nps.org.au/australian-prescriber/articles/efficacy-effectiveness-efficiency
https://www.insightsquared.com/2013/08/effectiveness-vs-efficiency-whats-the-difference/