New Drug Research, Development, and Approval

The journey of a medicine from lab to shelf is a time-consuming and expensive process, which usually takes about 15 years and $1.5 billion on average. The new drug discovery begins with basic research to understand the background of disease and identify the potential target. Through the preclinical study, researchers will select the promising molecule for Investigational New Drug (IND) and New Drug Application (NDA). Once FDA approved, the drug will be sold for disease treatment. Regulatory authorities may need phase 4 trials for post-marketing safety surveillance.

Overview of the traditional process of new drug development Fig.1 Overview of the traditional process of new drug development (Coloma, 2013)

New Drug Research

The pharmaceutical industry develops a variety of new drugs every year. For new drug discovery, the first work is to understand the processes behind a disease, which often at a cellular or molecular level. Through the basic research, the potential target was identified. The next step is to find a molecule or compound that acts on this target. After a promising compound was selected, safety and efficacy tests need to be conducted using cells and animal models. If it through this pre-clinical testing stage, the company will submit the new drug (IND) applications for clinical trials.

New Drug Development

The next stage is to confirm that the candidate drugs have an effect and that they are safe in human. In phase 1 trial, the new drug will be tested in a small group of healthy volunteers. Researchers will closely supervise the condition of volunteers and determine that the compound is safe enough to progress to phase 2 trials. The aim of phase 2 studies is to examine the efficacy of a compound in volunteer patients, usually 100-500 patients. Based on the phase 2 trial, the most effective drug dose, delivery method (oral or intravenous), and dosing interval will be confirmed. The drugs through phase 2 will then be tested in phase 3 trials, often 1,000 to 5,000 across multiple international sites. The purpose of this stage is to reconfirm the phase 2 findings in a larger population. Also, the best dosage regimen will be identified in phase 3 studies.

Overview of the clinical trials Fig.2 Overview of the clinical trials

New Drug Approval

For the drugs that make it to through phase 3, the company will submit marketing authorizations. The regulatory authorities such as European Medicines Agency (EMA) in the UK and Food and Drug Administration (FDA) in the US. The submission usually contains the drug information obtained during preclinical and clinical studies, including the chemical makeup, manufacturing process, drug toxicity and pharmacology, pharmacokinetics, clinical results, and proposed labeling. If a license is granted, the clinical trials may also continue. Phase 4 trials for post-marketing safety surveillance (pharmacovigilance) will be conducted to assure long-term safety and effectiveness of the drug, vaccine, device, or diagnostic test.


  1. Coloma, P., 2013. Phase 0 clinical trials: theoretical and practical implications in oncologic drug development. Open Access Journal of Clinical Trials, p.119.
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