The Food and Drug Administration Approval Process
The Food and Drug Administration (FDA) ensures that all foods are safe and drugs are both safe and effective before they’re available to US consumers. The FDA’s Center for Drug Evaluation and Research (CDER) takes care of the drugs and medical devices side through a stringent process.
The FDA does not test drugs or medical devices. It simply reviews evidence from tests conducted by drug manufacturers to ascertain that a proposed drug or device’s benefits outweigh its known risks.
The CDER has a team of researchers comprising pharmacologists, statisticians, and physicians, among others. They can perform limited tests to confirm that a proposed drug is safe and meets the set standards. During discovery and development, drug manufacturers discover gaps in existing therapies and perform promising compound tests.
Once a drug has been developed, the first stage of the approval process is preclinical trials. First, tests are run in the laboratory. All prescription drugs are tested on animals. Animal tests reveal how a drug works and whether it can be tested on humans. Preclinical research tests for such things as toxicity.
If a drug passes the preclinical research stage, the manufacturer submits an Investigational New Drug (IND) Application. The CDER evaluates the findings to ensure the proposed drug is safe for human trials. The purpose of human trials is to find out whether the drug provides the intended health benefits. If the FDA approves the INDA, the drug moves to the second stage, clinical trials. This is where human trials are done in four phases.
Phase 1 involves 20 to 80 volunteers and can take up to one year. The goal is to ascertain the drug’s safety and profile (chemical components, mode of use, etc.). How the drug interacts with the body and affects processes like metabolism are reviewed.
Phase 2 involves 100 to 300 volunteers and tests for effectiveness. It can take up to two years. It further tests for safety and side effects.
If Phase 2 shows that the drug is effective, safe, and with acceptable side effects, it proceeds to Phase 3. The third phase involves up to 3,000 patient volunteers.
Phase 3 can be done in several hospital settings and on volunteers of different ages. The goal is to determine the drug’s effectiveness and uncover any additional side effects.
The FDA also considers other factors that may determine whether that particular drug is approved or not. It analyzes the target disease as well as existing treatments. In some instances, the target condition is life threatening and there’s no promising treatment on the market. In such instances, the FDA may approve a drug if its health benefits outweigh the risk but the risks would be considered unacceptable for a disease that is not as serious.
Other special considerations include Accelerated Approval, where promising treatments for life threatening diseases are approved more quickly. There’s also the Orphan Drug Designation program, where manufacturers of drugs that treat rare conditions receive financial incentives. Drugs and devices that fill such unmet medical needs can come to the market more quickly. For instance, in September 2020, Xywav, a treatment for idiopathic hypersomnia (narcolepsy, or daytime sleepiness), was fast-tracked and made available before the final approval. It was approved for mass use in August 2021.
The fourth and final phase is approval. Because of the FDA’s stringent approval process, it’s reported that only one in 1000 drugs that are tested in the laboratory is approved for human trials. Of drugs that enter clinical trials, only 12 percent make it to the market. What’s more, the entire process — discovery, research, development, and approval — can take up to 15 years. After approval, the FDA performs a continuous post-marketing assessment to ensure the drug continues to do what it was approved for.
