Drug Development Process According to FDA

Throughout the 20th and 21st centuries, modern medicine has saved countless lives. Thanks to drugs like insulin, penicillin, and the polio vaccine, many deadly diseases are kept at bay. However, pharmaceutical companies do not begin distributing drugs at the slightest hint that it might cure or mitigate these diseases. In fact, there is a long and tedious process of experimentation, testing, and trials that most drugs have to go through in order to be deemed effective for treatment and safe for the public. Said process is referred to as the Drug Development Process and is commonly recognized as a five-step procedure: discovery and development, preclinical research, clinical research, FDA review, and FDA post-market safety monitoring.

According to the FDA, the initial stage of the Drug Development Process consists of discovery and development. Laura Lansdowne’s article, “Target Identification & Validation in Drug Discovery” provides deeper insight into this stage. She claims that drug discovery begins when pharmaceutical scientists find an adequate “target” – a biological entity that both factors into disease and can be modified by therapeutic measures. Essentially, it is the act of identifying a “cause” of the disease so that it may be treated with the soon-to-be-developed drug. What follows is target validation, where numerous experiments are performed on the aforementioned target to confirm that it truly plays a role in the disease. Afterward, the actual development of the drug commences. Scientists are now looking for drugs that can affect the target. In this stage, over ten thousand compounds are screened and tested in a brute-force trial-and-error strategy. Drugs that pass testing are then further developed and modified by scientists in hopes of improving its effects on the target.

Drugs that advance through discovery and development go through what is called preclinical research. In preclinical research, the potential dangers of a drug are evaluated in toxicology studies. These studies are usually conducted with one of two methods: in vitro, which are experiments done in test tubes and Petri dishes, and in vivo, which are experiments done on animals such as mice, rabbits, and monkeys. As the FDA states, “preclinical studies must provide detailed information on dosing and toxicity levels.” This is especially important because these drugs will be tested on humans in clinical research, where hazardous drugs can lead to injury or death.

Before clinical research begins, however, scientists must fill out the Investigational New Drug application, in which they include information such as manufacturing details and data on the drug from previous research. Afterward, clinical research begins with Phase I, where the drug in question is tested on a small group of 20 to 100 people with the target disease. This phase lasts several months, where scientists try to determine how safe the drug is and what the appropriate dosage is to administer. The next phase, referred to as Phase II, consists of tests with hundreds of participants. Its purpose now is to test how effective the drug is at treating the disease and to further explore side effects that might arise in patients. Phase III is similar to Phase III, with the most significant difference being the scale of the experiment. The number of participants in this stage can reach thousands and the duration can last up to four years.

With clinical research finished, drug developers only have to pass the FDA review team before they are approved to issue the drug. In this stage, developers submit a New Drug Application (NDA) along with all data gathered from previous steps. After 6 to 10 months, the FDA Review Team will either approve or reject the new drug for distribution. However, in some cases, the FDA might call for drug developers to address certain issues about the drug, which can delay approval time.

Assuming that the drug is approved, the FDA still has to make sure that the drug is safe when it is on the market. For example, healthcare professionals can submit Form FDA 3500 to the FDA if they find any issues with the drug, and consumers can do the same by reporting a Form FDA 3500B. Manufacturers and importers of the drug are also involved, as they are required to submit Form FDA 3500A if “any of their devices may have caused or contributed to a death or serious injury” (“Mandatory Reporting Requirements: Manufacturers, Importers, and Device User Facilities”). Furthermore, the FDA actively monitors health databases for any problems that may arise. This is done with what is called the Sentinel System, which uses statistics, data science, and more to find issues in real-time (“FDA's Sentinel Initiative”).

Ultimately, the Drug Development Process is not a simple one. With discovery and development, preclinical research, clinical research, FDA review, and post-market monitoring, digesting every fine detail of every step can be challenging. However, it is reassuring to know that there are rules and regulations set to protect American consumers from potentially deadly drugs that were advertised to treat their illnesses.

Works Cited

1. Center for Devices and Radiological Health. “Mandatory Reporting Requirements.” U.S. Food and Drug Administration, FDA, www.fda.gov/medical-devices/postmarket-requirements-devices/mandatory-reporting-requirements-manufacturers-importers-and-device-user-facilities.
2. Center for Drug Evaluation and Research. “FDA's Sentinel Initiative.” U.S. Food and Drug Administration, FDA, www.fda.gov/safety/fdas-sentinel-initiative.
3. “The Drug Development Process.” U.S. Food and Drug Administration, FDA, www.fda.gov/patients/learn-about-drug-and-device-approvals/drug-development-process.
4. “Reporting Serious Problems to FDA.” U.S. Food and Drug Administration, FDA, www.fda.gov/safety/medwatch-fda-safety-information-and-adverse-event-reporting-program/reporting-serious-problems-fda.
5. “Step 3: Clinical Research.” U.S. Food and Drug Administration, FDA, www.fda.gov/patients/drug-development-process/step-3-clinical-research.
6. “Step 4: FDA Drug Review.” U.S. Food and Drug Administration, FDA, www.fda.gov/patients/drug-development-process/step-4-fda-drug-review.
7. Lansdowne, Elizabeth. “Target Identification & Validation in Drug Discovery.” Drug Discovery from Technology Networks, www.technologynetworks.com/drug-discovery/articles/target-identification-validation-in-drug-discovery-312290.
8. “Understanding the Drug Discovery Process.” Compound Interest, 5 Apr. 2017, www.compoundchem.com/2016/01/16/drug-discovery/.