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The main objective of an experimental study is to assess whether the intervention is working well or not. There are three terms related to the outcome measure in experimental study:
1.Efficacy
2.Effectiveness
3.Efficiency
Could you give a brief explanation of these three terms? What would be the different in the study design in order to measure each of these three terms. -
According to this literature “Burches E, Burches M (2020) Efficacy, Effectiveness and Efficiency in the Health Care: The Need for an Agreement to Clarify its Meaning. Int Arch Public Health Community Med 4:035. doi.org/10.23937/2643-4512/1710035”
Efficacy is the capacity for beneficial change (or therapeutic effect) of a given intervention under ideal or controlled conditions. I think the efficacy is the outcome measurement of a randomized controlled trial.Effectiveness is doing “the right” things under ordinary circumstances to achieve the desired effect. Effectiveness links to the notion of external validity, in that it refers to patients who are visited by physicians in their everyday practice. I think the best example of effectiveness is the outcome of the intervention in the real-world setting, such as the study of the effect of new antidiabetic drugs on HbA1C level reduction in diabetic patients in the clinics. It can be evaluated through observational studies of real practice.
Efficiency is the ratio of the output to the inputs of any system. The input to the system can be physical inputs or financial inputs. While the output in the health sector divided into health services (visits, drugs, admissions) and health outcomes (by way of example: Preventable deaths, functional status, clinical outcomes such as blood pressure or blood sugar control).
Efficiency measures must also explicitly identify the inputs that are used to produce the output of interest. The study design to measure the efficiency should be a cross-sectional study. -
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A short explanation for the three terms can be explained by the drug developments/study. The three terms are used in a different kind of study during the development due to its different goals, tools, and messages the researcher wants to communicate.
‘Efficacy’ is a term used in the study under ‘ideal conditions’ such as in laboratory to answer the question of ‘What can the drugs do?’. For example, a study of how a phytochemical extract can inhibit the growth of tumour cells when managed in a test tube or ‘in vitro’, the result can be communicated by the efficacy of the extracts in inhibiting of the cells.
While, when the extracts showed expected sufficient ‘efficacy’, the next study can be to find the effectiveness of the extracts which produced in forms of drug and to be studied in ‘usual conditions’ or ‘real-life condition’. This is to answer the question of ‘What does the drug do?. The study can be a clinical trial in a selected healthcare setting or mass study in population. The drugs’ works would not only be between the extracts and the tumour cells anymore but involved by other factors such as conditions of the patient or dose management. The result can be expressed by the ‘Effectiveness’ of the drugs in the treatment of the patient with a tumour in different stages. The adverse outcomes are also the main interest in this kind of study.
‘Efficiency’ is mainly focused on resources/cost. The questions can be how does it takes/cost to make the drugs really work? For example, in a study, drug A show more effectiveness compared to drug B but it is more expensive. While the drug B require the patient to come to the hospital to get injected of the drugs and require for a one-night admission, the drug A which are in form of a tablet, the patient can take it at home following instructions and come to hospital only for following up. The evaluation considers the whole course which not only about the cost of the drugs but also the dose management, admission cost and other related costs/resources. When evaluated, drug B may have more ‘efficiency’ than drug A when considering the cost of the treatment comparing to the effectiveness of the drugs. The study can be to evaluate the ‘efficiency’ of the drugs for treatment considering many other aspects of health care or health system.
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Efficacy refers to whether the action provides clinical results under ideal condition (well-controlled condition).
Effectiveness refers to whether it can achieve results under usual circumstances (average clinical conditions).
This article has a infographic that explained the difference well.
Efficiency, as I have found, seems least confusing of the three terms, and refers to value of the intervention itself, relative its cost on society; that is, Return On Investment / Cost of Ownership.
For efficiency, I am looking at PubMed search, in particular, this article.
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In the experiment study, the criteria for good treatment are defined are examine for efficacy, effectiveness and efficiency. These three terms related to the outcome measure in experimental study, including of “Efficacy”, “Effectiveness” and “Efficiency” can measure as below.
1.Efficacy : This studies investigate the benefits and harms of an intervention under highly controlled conditions. It requires substantial deviations from clinical practice, including restrictions on the patient sample, control of the provider skill set and limitations on provider actions, and elimination of multimodal treatments.
2.Effectiveness : This studies (also known as pragmatic studies) examine interventions under circumstances that more closely approach real-world practice, with more heterogeneous patient populations, less-standardized treatment protocols, and delivery in routine clinical settings.
3.Efficiency :This studies provide the evidence base to make decisions about the introduction of innovative interventions while discouraging the use of inefficient interventions.
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