Mean and Mean difference are the two key statistical measures used in the statistical analysis. Both are essential for meta-analysis as well. Mean and Mean difference are used for the interpretation of a large set of values into a single number which explains the heterogeneity and variation among the individual values. However, one of a common challenge in meta-analysis is the unavailability of this data (mean and standard deviation).
Q & A Forum
Meta Analysis
Q: What is the difference Safety and Efficacy in drug Research?
Safety:
Safety is the detection, evaluation, understanding, and prevention of adverse effects from pharmaceuticals, nutraceuticals, and cosmetics in humans and animals. Safety is the key parameter in a drug development process as a new drug or chemical can cause serious health hazards including carcinogenicity, mutagenicity, teratogenicity, and allergic reaction.
There are many parameters used to evaluate the safety of chemical compounds or other substances concerning drug approval.
Safety studies are performed in preclinical and clinical studies to find out the unwanted side effects at various dose levels. The dose at which a drug does not cause any adverse effects or produce tolerable side effects is referred to as a safe dose level.
The safe dose is generally found in the preclinical stage to prevent adverse effects in the clinical stage. [1]
Efficacy:
Efficacy is the term used to address the effectiveness of a drug or other substances to treat or prevent a specific disease or any other health ailments and the ability of the drugs to activate the receptor target which causes biological action. For example, the effectiveness of a drug substance to treat the symptoms of Alzheimer’s disease or prevention of Alzheimer’s in the future.
Efficacy is also referred to as therapeutic action in pharmacological terms.[2] [3]
Difference between safety and efficacy:
- Safety is the parameter used to prevent the occurrence of unwanted health hazards caused by the drugs or chemicals, while efficacy is the term that denotes the effectiveness of the drug substance in treating various health ailments.
Example: The safety and efficacy data of the drug tolcapone from Olanow CW, Watkins PB [4] study.
Efficacy of Tolcapone:
- Tolcapone is the selective & reversible inhibitor of the catechol-O-methyltransferase (COMT). It increases the concentration of levodopa in the brain. Thus, it enhances the dopaminergic activity in brain.
- The effective dose of the drug is 100 and 200 mg per 3 times a day.
- It is used as adjunct drug with levodopa for the antiparkinsonian activity by reducing the daily dose required for Parkinson’s disease.
Safety of Tolcapone:
- The adverse effects of the drug in this study is generally mild to moderate in this article.
- The study reported adverse effects are dyskinesia and other dopaminergic- related side effects.
- The 26% of the patients had diarrhoea.
- The lever enzymes elevated in the patients.
- One case reported severe liver injury by tolcapone.
- This review article states 3 deaths reported from hepatic dysfunction in post-marketing surveillance [4]
Refernce:
- Samara, C., et al. (2023). Safety surveillance during drug development: Comparative evaluation of existing regulations. Advances in Therapy, 40(5), 2147–2185. https://doi.org/10.1007/s12325-023-02492-3
- Waller, D. G., et al. (2018). Principles of pharmacology and mechanisms of drug action. In Medical Pharmacology and Therapeutics (5th ed., pp. 3–31). https://doi.org/10.1016/B978-0-7020-7167-6.00001-4
- Lynch, S. S. (2022). Drug efficacy and safety. University of California San Francisco School of Pharmacy. https://www.msdmanuals.com/professional/clinical-pharmacology/concepts-in-pharmacotherapy/drug-efficacy-and-safety
- Olanow CW, Watkins PB. Tolcapone: An efficacy and safety review. Clin Neuropharmacology. 2007;30(5):287-294. doi:10.1097/WNF.0B013E318038D2B6.