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Living with Dementia > Research > Phases
The development of new medical treatments is an extensive and complicated process. The U.S. Food and Drug Administration estimates that it takes about eight-and-a-half years, on average, to thoroughly research and test a new treatment before approving it for the market. A successful treatment passes through rigorous examination in research laboratories, animal testing, and finally, tests in humans. The data collected is carefully reviewed before the FDA approves the drug for market. Below is a detailed description of each step along the way.
First Steps in Treatment Development
In the initial stages of development, the FDA plays a very limited role. The agency is not charged with the responsibility of developing new drugs; rather, its role is to review the treatments developed by research entities such as pharmaceutical companies and universities.
Laboratory Testing – Treatment development always begins with a look into the most basic levels of how the body functions. This fundamental research can be achieved in three ways:
- Assays – Test-tube experiments in which compounds are added to biological enzymes, cell cultures, or other physiological environments to determine if the compound produces the desired effect.
- Computer simulations – New technology allows scientists to observe the intricate geometry of the body’s highly developed biological molecules, and how to create treatments that react better with those molecules. Any compounds made based on simulations must still go through true biological testing (assays), though.
- Natural compound testing – Compounds made by microscopic organisms are tested to see if they produce the desired effects.
Animal Testing – If the laboratory testing shows at least some of the researcher’s desired results, the compound moves on to tests in animals. Typically, two or more species are used to monitor differing treatment behaviors. Animal tests allow researchers to understand how the body handles the treatment; important information from animal tests includes the occurrence of any side effects, how the animal responds to different dosages, how the compound is absorbed in the blood, how quickly it is metabolized and if its byproducts exhibit their own toxicity, and finally, how quickly the compound and its byproducts are excreted from the human body. All of this information is essential in helping researchers better plan for human testing, and possibly product labeling.
Human Testing: Clinical Trials
After the Investigational New Drug Application is approved by the FDA, it is also reviewed by a local oversight body known as the Institutional Review Board (IRB), a panel of scientists and other experts who oversee clinical research in hospitals and research institutions. The IRB must approve the clinical trial protocol, which describe the type of people who may participate in the trial, the schedule of tests and procedures, the medications under study, and other important details. Once the protocol is approved, human testing begins.
Phase I Trials – These trials are usually conducted in a small group of healthy volunteers (20-80 people) to determine the most common side effects and how the drug is metabolized and excreted by the human body.
Phase II Trials – If phase I studies reveal that the drug is reasonably non-toxic, then phase II trials begin, typically with 100-300 volunteers who have the disease that the treatment is supposed to treat. Phase II trials emphasize effectiveness, compared to phase I trials which placed a premium on safety. This phase aims to acquire the first data about whether the drug works in people with a certain disease.
Phase III Trials – If phase II trials show evidence of effectiveness, phase III trials begin, using a large group of volunteers with the disease (1,000-3,000). Phase III trials further test the product’s efficacy and continues to monitor side effects. Often, the product is compared to a standard treatment, if one already exists, or to a placebo (inactive substance).
Phase IV Trials - Sometimes, Phase IV trials are conducted after the product is approved and on the market to learn more about the treatment’s long-term risks and optimal use.
Final Stages
At this stage in the process, the treatment has undergone years of study and researchers have been collecting data on the treatment’s behavior, safety, and efficacy.
New Drug Application (NDA) – All that data is collected into a formal application, the NDA, which is submitted for approval by the FDA. The FDA decides whether or not to file it for review within 60 days; the only reasons they will turn it down are because of significant flaws in the data collected or lack of support for the claims made about the treatment. If the treatment is accepted into the queue for review, the FDA will examine the drug’s safety, effectiveness, product labeling, and manufacturing facilities, among other details. There are three different outcomes from here:
- Approved – The treatment is approved and the manufacturer can put it on the market.
- Approvable – The treatment is not approved outright; more information is needed to prove the treatment’s safety or efficacy.
- Not Approvable – The treatment cannot be approved without a substantial amount of new information in strong support of the safety or efficacy of the drug.
Understanding the development of treatments is crucial if you ever consider taking part in a clinical study for any treatment. Each phase in clinical testing has its own risks and you should only volunteer for a clinical trial once you understand the risks of the trial and your rights as a volunteer.
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