Imagine choosing from among medicines and treatments that made bold claims about managing or curing serious conditions without knowing which could be taken seriously. If drugs and treatments were not studied and held to consistent standards, it would be up to patients to make a guess based on word of mouth, advertising messages, simple observations, and folk wisdom. Thanks to clinical trials, we don’t have to guess.
The clinical trial process is regulated by the U.S. Food and Drug Administration (FDA), and it includes medicines, therapies, devices, and procedures. All modern health care treatments eventually go through clinical trials – human research studies – as the final steps toward demonstrating their safety and effectiveness and evaluating them for side effects. Clinical trials result in safer and more effective ways of treating medical conditions.
About clinical trials
Health researchers are always looking for new and better ways to identify, diagnose, treat, manage, and prevent medical conditions. Whether publicly or privately funded, research often begins with pre-clinical testing, meaning tests done in a laboratory without human participants.
When researchers find a possible treatment, they first test it in computer models, bacterial cultures, and/or on isolated human cells. If the treatment seems to work, the testing usually advances to animals, to learn how it affects them and how it might affect humans. This pre-clinical testing may take months or years.
The treatments that make it to clinical trials already have shown promise and a reasonable risk-to-benefit ratio. The standards for entering a clinical trial are so strict that only about five in 5,000 new treatments that begin the road toward clinical trials are approved for human testing by the FDA.
Hear from UAB Medicine clinical researcher Meredith Fitz-Gerald about how clinical trials enhanced the care her husband received.
Once a treatment is optimized in the laboratory, a public or private entity (called a sponsor) may ask the FDA for an Investigational New Drug (IND) application, which authorizes new treatments to enter the health care market. The FDA decides whether it is reasonably safe for the company to move forward with testing the treatment in humans. The clinical trial process usually takes several more years.
By the time a treatment from a clinical trial is offered as a beneficial option to patients, its potential is clear, and it has been determined to be relatively safe. The plan for a clinical trial must gain approval from the FDA and be overseen by an institutional review board (IRB), a panel of scientists and non-scientists at research institutions.
Depending on their complexity and who is sponsoring the study, clinical trials can be conducted at hospitals, universities, physician offices, and community clinics. Many trials take place at multiple sites, each having a principal investigator. UAB Medicine is able to host many groundbreaking clinical trials due to having the required resources and specialists. This is beneficial to our patients, providers, and researchers. Clinical trials are a critical part of our entire system.
Anne Kendrick was part of the first clinical trial of any drug for her rare genetic kidney disease. Learn how she gained hope for the future during five years as a participant.
The four phases
Clinical trials advance through three phases to test a treatment, find the appropriate dosage, and look for side effects. After the first three phases, if researchers find a treatment to be safe and effective, the FDA approves it for patient use and continues to monitor its effects in a fourth phase.
- Phase I trials test an experimental treatment on a small group of often healthy people (usually numbering 20-80) to judge its safety and side effects and to find the correct drug dosage.
- Phase II trials involve more people (100-300). While the emphasis in phase I is on safety, the emphasis in phase II is on effectiveness. This phase seeks to obtain data on whether the treatment works in people who have a certain disease or condition. These trials also continue to study safety, including short-term side effects.
- Phase III trials gather more information about safety and effectiveness, studying different populations and different dosages, and using the treatment in combination with other drugs. The number of participants usually ranges from several hundred to about 3,000 people. If the FDA agrees that the trial results are positive, it will approve the experimental drug or device.
- Phase IV trials take place after the FDA approves the treatment for use and are done to monitor for longer-term effects.
Review the basics of clinical trials and learn how to take the next step.
Patient participation
Participating in a clinical trial is a way to help advance health care for everyone, and it also may be a way to receive treatments that are not yet available elsewhere. Treatment received as part of clinical trials is often provided at no cost, and participants may even be paid for their time.
However, participants may have to commit to extended periods of time and endure some side effects. Although many steps are taken to limit the risk to participants, it is up to patients to decide if any particular clinical trial is right for them.
Sarah Hays has participated in multiple clinical trials for Parkinson’s disease, to help researchers learn more about the condition. She hopes to contribute to a cure that will benefit future generations.
Clinical trials offer hope for people whose conditions have no treatment or haven’t responded to the standard treatments. They also offer another way for institutions such as UAB Medicine to change the world.
UAB Medicine participates in hundreds of clinical trials at any given time. Search active clinical trials by type and by keyword.
Sources: National Institutes of Health, clinicaltrials.gov, and cancer.gov