The process of developing a new drug involves five steps. These steps are ordered so that each step builds on information gained during the earlier steps. Each step is described briefly below.
Step 1: Discovery and Development
- Knowledge of a disease process helps researchers identify potential candidates for development as a drug to treat or prevent a disease
- Thousands of potential candidates might be studied to identify those that are most promising
- No human study participants are involved at this stage
Step 2: Preclinical Research
- These studies do NOT include human study participants
- May involve the study of cell cultures (In Vitro research) or animal models (In Vivo research)
Step 3: Clinical Research
- These studies involve human study participants
- They are called clinical trials. Before starting to do clinical research, the company developing the drug must submit an Investigational Drug Application (IND) to the Food and Drug Administration (FDA)
- Research cannot begin until the FDA approves the IND
- INDs are considered proprietary and are not typically made available to the public
- Clinical research usually involves four distinct phases, with each phase having a different goal
This website will focus on findings from clinical research. Click here to find out about the four phases of clinical research.
Step 4: FDA Review
- Once the drug developer has evidence that they believe demonstrates that a drug is safe and effective, it can submit a New Drug Application (NDA) to the FDA
- For vaccines, the vaccine developer submits a Biologics License Application (BLA) instead of a NDA
- Within the FDA, the Center for Biologics Evaluation and Research (CBER) is responsible for reviewing BLAs and approving new vaccines for use in the US
- The FDA usually requires data from two large, controlled clinical trials before they will approve a drug for marketing
Step 5: FDA Post-Market Safety Monitoring
- Knowledge concerning the safety and effectiveness of new medications continues to evolve AFTER a drug is approved for marketing.
- The FDA monitors reports of safety concerns (e.g., medication side effects) through the MedWatch program.
- More information about the MedWatch program can be accessed at: https://www.fda.gov/safety/medwatch-fda-safety-information-and-adverse-event-reporting-program
You can find more detailed information about the drug development process at https://www.fda.gov/patients/learn-about-drug-and-device-approvals/drug-development-process, which was the primary source of the information contained on this page.