18/8/2017 Efficacy - Wikipedia

Efficacy
From Wikipedia, the free encyclopedia

Efficacy is the ability to get a job done satisfactorily. The word comes from the same roots as effectiveness, and
it has often been used synonymously, although in pharmacology a distinction is now often made between
efficacy and effectiveness. The word efficacy is used in pharmacology and medicine to refer to both the
maximum response achievable from a pharmaceutical drug in research settings,[1] and to the capacity for
sufficient therapeutic effect or beneficial change in clinical settings.

Contents
1 Pharmacology
2 Medicine
3 See also
4 References

Pharmacology
In pharmacology, efficacy (Emax) is the maximum response achievable from an applied or dosed agent, for
instance, a small molecule drug.[1] Intrinsic activity is a relative term that describes a drug's efficacy relative to
a drug with the highest observed efficacy.[2] It is a purely descriptive term that has little or no mechanistic
interpretation.

In order for a drug to have an effect, it needs to bind to its target, and then to affect the function of this target.
The target of a drug is commonly referred to as a receptor, but can in general be any chemically sensitive site
on any molecule found in the body. The nature of such binding can be quantified by characterising how tightly
these molecules, the drug and its receptor, interact: this is known as the affinity. Efficacy, on the other hand, is a
measure of the action of a drug once binding has occurred. The maximum response, Emax, will be reduced if
efficacy is sufficiently low, but any efficacy greater than 20 or so gives essentially the same maximum
response.

The definition of efficacy has been discussed by.[3] The only way in which absolute measures of efficacy have
been obtained is by single ion channel analysis of ligand gated ion channels. It is still not possible to do this for
G protein-linked receptors.

In the case of the glycine receptor and the nicotinic acetylcholine receptor (muscle type), it has been proposed
by Sivilotti et al. that opening of the ion channel involves two steps after agonist is bound. Firstly a
conformation change to a higher affinity (but still shut) form, followed by the conformation change from shut
to open.[4][5] It was found that partial agonism results from deficiency in the first step, and that the opening and
shutting steps are essentially the same for both full and partial agonists. This has been confirmed and extended
by Sine and colleagues (2009).[6] The implication of this work[5] is that efficacy has to be defined by at least
two equilibrium constants (or, more generally, by four rate constants).

It is the combined influence of both affinity and efficacy which will determine how effectively a drug will
produced a biological effect, a property known as potency.

Medicine

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In medicine, efficacy is the capacity for beneficial change (or therapeutic effect) of a given intervention (for
example a drug, medical device, surgical procedure, or a public health intervention). Establishment of the
efficacy of an intervention is often done relative to other available interventions, with which it will have been
compared. Specifically, efficacy refers to "whether a drug demonstrates a health benefit over a placebo or other
intervention when tested in an ideal situation, such as a tightly controlled clinical trial."[7] These studies focus
to a primary parameter to be shown statistically different changes between placebo and intervention groups.
Comparisons of this type are called in 'explanatory' randomized controlled trials, whereas 'pragmatic' trials are
used to establish the effectiveness of an intervention regarding also non-specific parameters.

Effectiveness refers "how the drug works in a real-world situation," and is "often lower than efficacy because of
interactions with other medications or health conditions of the patient, sufficient dose or duration of use not
prescribed by the physician or followed by the patient, or use for an off-label condition that had not been
tested."[7][8]

See also
Efficiency (disambiguation)
Placebo (origins of technical term)
Potency (pharmacology)
Vaccine efficacy

References
1. Holford NHG & Sheiner LB (1981). "Understanding the dose-effect relationship: Clinical application of
pharmacokinetic-pharmacodynamic models". Clin. Pharmacokinet. 6 (6): 429453. PMID 7032803 (http
s://www.ncbi.nlm.nih.gov/pubmed/7032803).
2. Neubig, RR; Spedding, M; Kenakin, T; Christopoulos, A; International Union of Pharmacology
Committee on Receptor Nomenclature and Drug, Classification (December 2003). "International Union
of Pharmacology Committee on Receptor Nomenclature and Drug Classification. XXXVIII. Update on
terms and symbols in quantitative pharmacology.". Pharmacological reviews. 55 (4): 597606.
PMID 14657418 (https://www.ncbi.nlm.nih.gov/pubmed/14657418). doi:10.1124/pr.55.4.4 (https://doi.or
g/10.1124%2Fpr.55.4.4).
3. Colquhoun D (1998). "Binding, gating, affinity and efficacy. The interpretation of structureactivity
relationships for agonists and of the effects of mutating receptors" (https://www.ncbi.nlm.nih.gov/pmc/art
icles/PMC1565672). British Journal of Pharmacology. 125: 923948. PMC 1565672 (https://www.ncbi.
nlm.nih.gov/pmc/articles/PMC1565672) . PMID 9846630 (https://www.ncbi.nlm.nih.gov/pubmed/9846
630). doi:10.1038/sj.bjp.0702164 (https://doi.org/10.1038%2Fsj.bjp.0702164).
4. Burzomato V, Beato M, Groot-Kormelink P, Colquhoun D & Sivilotti LG (2004). "Single-channel
behavior of heteromeric alpha1beta glycine receptors: An attempt to detect a conformational change
before the channel opens". Journal of Neuroscience. 24: 1092410940. PMID 15574743 (https://www.nc
bi.nlm.nih.gov/pubmed/15574743). doi:10.1523/jneurosci.3424-04.2004 (https://doi.org/10.1523%2Fjne
urosci.3424-04.2004).
5. Lape R, Colquhoun D, Sivilotti L (2008). "On the nature of partial agonism in the nicotinic receptor
superfamily" (https://www.ncbi.nlm.nih.gov/pmc/articles/PMC2629928). Nature. 454: 722728.
PMC 2629928 (https://www.ncbi.nlm.nih.gov/pmc/articles/PMC2629928) . PMID 18633353 (https://w
ww.ncbi.nlm.nih.gov/pubmed/18633353). doi:10.1038/nature07139 (https://doi.org/10.1038%2Fnature07
139).
6. Mukhtasimova N, Lee WY, Wang HL, Sine SM (2009). "On the nature of partial agonism in the nicotinic
receptor superfamily" (https://www.ncbi.nlm.nih.gov/pmc/articles/PMC2712348). Nature. 459: 451454.
PMC 2712348 (https://www.ncbi.nlm.nih.gov/pmc/articles/PMC2712348) . PMID 19339970 (https://w
ww.ncbi.nlm.nih.gov/pubmed/19339970). doi:10.1038/nature07923 (https://doi.org/10.1038%2Fnature07
923).
7. Thaul, Susan (2012-06-25). How FDA Approves Drugs and Regulates Their Safety and Effectiveness
(CRS 7-5700, R41983) (https://fas.org/sgp/crs/misc/R41983.pdf) (CRS Report for Congress).
Washington, DC: Congressional Research Service (CRS). p. 4. Retrieved 22 March 2016.

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8. Porta, Miquel, Ed. (2008). A Dictionary of Epidemiology (5th ed.). Oxford, ENG: Oxford University
Press.

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