Lexique

Defining Efficacy

Defining Efficacy

The highest level of evidence of efficacy of therapeutic innovation or prevention is given by a randomized controlled trial. This experimental methodology provides the most rigorous demonstration of the effect of innovation on the endpoints measured at the beginning and end of the protocol. Are there profits on (biological psychological clinics, socio-professional and/or survival) health criteria, quality of life (generic and/or specific), safety (benign and/or serious incidents) or utility (cost/effectiveness, cost/benefit)? What are the chances that a treatment works? Without proof of its effectiveness, the added value of an innovation remains subject to endless discussions in doubt. His chances of repayment by the community may be compromised. The Blog aims to increase awareness of clinical studies involving the highest level of evidence according to the classification of Evidence Based Medicine (Sackett et al., 2000; Boutron et al., 2012).


The general message

Efficacy is not satisfaction. A real health benefit, ie a value on the health and quality of life, should be confirmed by a randomized controlled trial (RCT). Pay attention to messages confusing the two.

What it means for Health Professionals

Demonstrate the efficacy of a health innovation means providing irrefutable evidence of its effect on one or more endpoints on the basis of a sufficient number of people and a significant statistical power. An observation is not an evidence. This demonstration generally requires a RCT.

What it means for Researchers

Demonstrate the efficacy of a health innovation means providing irrefutable evidence of its effect on one or more criteria on the basis of a sufficient number of people and a given statistical power (Cucherat, 2013). Observe a case in not demonstrate. Demonstrating the efficacy of a NPI requires a RCT.

What it means for Policymakers

Demonstrate the efficacy of a health innovation means providing irrefutable evidence of its effect on one or more criteria on the basis of a sufficient number of people and a given statistical power (Cucherat, 2013). Show is not proven. Demonstrating efficacy through a randomized controlled trial is needed.


References

Boutron I, Ravaud P, Moher D (2012). Randomized clinical trials of non pharmacological treatments. Boca Raton: CRC Press Taylor and Francis.

Cucherat M (2013). Site web.

Sackett, D.L., Straus S.E., Richardson W.S., Rosenberg W., Haynes R.B. (2000). Evidence-Based Medicine: how to practice and teach EBM. Second ed. London: Churchill Livingstone.


To reference this Blog en Sante © article.

Ninot G (2014). Definition of efficacy. Blog en Sante, L11.

© Copyright 2014 Grégory Ninot. All rights reserved.