Book chapter
5 - Drug Discovery, Development, and Regulation
Brody's Human Pharmacology, pp 49-56
2025
Abstract
Drug discovery and development is a lengthy, highly regulated, expensive, and complex process involving individuals with wide-ranging expertise. Drug discovery begins with the identification and validation of drug targets such as enzymes and receptors. This is followed by the development of small molecules or biologics that interact with the target to produce the desired therapeutic effect. These molecules can be obtained from natural sources or through structure-based drug design which are then investigated using in vitro and in vivo models for efficacy and toxicity. Once preclinical studies are completed, an investigational new drug application is filed with the US Food and Drug Administration (FDA) to begin clinical trials. The clinical trials are divided into three phases; phase 1 involves the investigation of the safety and tolerability of the drug in approximately 20 to 100 healthy volunteers. Phase II assesses the efficacy of the drug in 100 to 300 patients, while phase III studies involve the assessment of efficacy in thousands of patients. A new drug or biologics license application is filed with the FDA after passing the clinical trials. The approved drug has a period of market exclusivity related to the patent. After the patent expires, other companies may file an abbreviated new drug application to manufacture a generic drug of the innovator drug. The generic drug has to be bioequivalent to the innovator drug. Although the overall approval rate for a new drug is low (13.8%), innovations such as improvements in biomarker identification, pharmacogenomics, and using adaptive clinical trials may help improve the success of clinical candidates.
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Details
- Title
- 5 - Drug Discovery, Development, and Regulation
- Creators
- Lance R. McMahonSamuel ObengChristopher R. McCurdyJames E. BarrettFelix J. Kim
- Publication Details
- Brody's Human Pharmacology, pp 49-56
- Publisher
- Elsevier Inc
- Edition
- Seventh Edition
- Resource Type
- Book chapter
- Language
- English
- Academic Unit
- Neurology
- Other Identifier
- 991022065621204721