The words “efficacy” and “effectiveness” are used to qualitatively and quantitatively describe the performance of a particular drug or vaccine. Most media outlets and the general public often use these two terms interchangeably. However, technically, they do not mean the same thing.
Drug and Vaccine Development: Understanding the Difference Between Efficacy and Effectiveness
Note that efficacy pertains to the performance of a drug or vaccine based on clinical studies and, thereby, under ideal and controlled circumstances. Consider a particular type of vaccine as an example. A vaccine with a 90 percent efficacy in a clinical trial means that its administration produced a 90 percent reduction in disease cases within the vaccinated group compared to the placebo or unvaccinated group.
On the other hand, effectiveness corresponds to the performance of drugs and vaccines in real-world settings. Determining how effective a particular drug or vaccine meets the desired outcome requires medium-term to long-term post-market surveillance and observational studies. Hence, regularly collecting and processing surveillance data for extended periods is critical to measuring and understanding effectiveness.
The Reliability and Limitation of Efficacy Value
Remember that determining the efficacy value of a specific drug or vaccine requires rigorous clinical trials. Failure to meet a predetermined percentage value would prompt developers to improve further or abandon the entire undertaking. On the other hand, it is imperative for researchers, laboratories, or companies to publish the results of clinical trials with satisfactory efficacy values to build public confidence.
However, despite the rigor of the entire drug and vaccine development process, it is important to note that the efficacy value does not always correspond to effectiveness because laboratory conditions are different from the real world. In clinical trials, participants are carefully selected based on their health status, gender and age, and other pertinent health-related backgrounds. These participants represent a subsection of a large and diverse population.
Determining the Effectiveness of a Drug or Vaccine
There are several factors that can reduce or affect the effectiveness of a drug or vaccine. These include age, underlying medical conditions, other medications they are taking, and the manner in which the particular drug or vaccine is stored and administered. These factors are present in real-world conditions, and researchers need to take note of them through continued and often long-term post-market surveillance.
Some developers opt to do separate effectiveness trials without making the particular product available in the general market. However, it is more cost-efficient to conduct these trials alongside mass distribution. More often than not, satisfactory results of efficacy trials are enough to secure necessary permits or licenses from the government. But developers need to conduct post-market surveillance as part of the overall development process.
Conclusion and Takeaway
To understand the difference between efficacy and efficiency, it is essential to note that both describe whether a particular drug, vaccine, or other medical intervention does more good than harm. However, efficacy is specifically defined as performance under ideal and controlled conditions, while effectiveness pertains to performance under real-world settings.
Note that efficacy or fastidious trials test for efficacy while effectiveness or pragmatic trials test for effectiveness. It is also worth highlighting the fact that although both trials aim to measure and determine the performance of a specific medical intervention, both address different research questions. Efficacy trials aim to answer if a drug or vaccine can work, while effectiveness trials aim to determine if it does work.
FURTHER READINGS AND REFERENCES
- Ernst, E., and Pittler, M. H. 2006. “Efficacy or Effectiveness?” Journal of Internal Medicine. 260(5): 488-490. DOI: 1111/j.1365-2796.2006.01707.x
- Kim, S. Y. 2013. “Efficacy versus Effectiveness.” Korean Journal of Family Medicine. 34(4): 227. DOI: 4082/kjfm.2013.34.4.227
- Singal, A. G., Higgins, P. D. R., and Waljee, A. K. 2014. “A Primer on Effectiveness and Efficacy Trials.” Clinical and Translational Gastroenterology. 5(1): e45. DOI: 1038/ctg.2013.13