- Clinical Trials: What You Need to Know
- Why do we need clinical trials?
- What happens before clinical trials?
- Some facts about clinical trials: Important points to keep in mind
- What are the phases of clinical trials?
- Phase 0 clinical trials: Exploring if and how a new drug may work
- Phase I clinical trials: Is the treatment safe?
- Phase II clinical trials: Does the treatment work?
- Phase III clinical trials: Is it better than what’s already available?
- Submission for FDA approval: New drug application (NDA)
- Phase IV clinical trials: What else do we need to know?
- Who sponsors and runs clinical trials?
- Should I think about taking part in a clinical trial?
- Answers to some common questions about clinical trials
- Other questions you should ask your research team
- What protects the study participants?
- What’s out there? Finding clinical trials
- How do I figure out which study is for me?
- What about cost? Will my insurance cover it?
- What would it be like to take part in a clinical trial?
- What if I’m not eligible for a clinical trial?
- Summing it all up
What happens before clinical trials?
One of the most important points that must be decided before a clinical trial can be done is whether it’s ethical to ask patients to volunteer for the experimental treatment. Has the study been designed, as much as possible, to make sure the people involved will be safe? Will the volunteers get a treatment that’s at least as good as, and maybe even better than, what they would get if they didn’t volunteer for the study? Scientific panels are set up to review and approve all clinical trials to make sure questions like these are answered before the researchers are allowed to sign up patients. Certain types of information are needed before these questions can be answered.
Pre-clinical (or laboratory) studies
Clinical trials are medical research studies involving people. But they’re done only after pre-clinical studies suggest that the proposed treatment is likely to be safe and will work in people.
Pre-clinical studies, also called laboratory studies, include:
Cell studies: These are often the first tests done on a new treatment. To see if it might work, researchers look for effects of the new treatment on cancer cells that are grown in a lab dish or a test tube. These studies may be done on human cancer cells or animal cancer cells.
Animal studies: Treatments that look promising in cell studies are next tested on cancers in live animals. This gives researchers an idea of how safe the new treatment is in a living creature.
Pre-clinical studies give a lot of useful information, but they don’t give all the answers that are needed. After all, humans and mice can be very different in the way they absorb, process, and get rid of substances. A treatment that works against cancer in a mouse may or may not work in people. And there could be side effects and other problems that didn’t show up when the treatment was studied in mice.
Cell studies and animal studies can be confusing when the news media reports them without making it clear that the studies were not done on people. Some reporters may not know how many more tests the treatment must go through before it can be used in humans. And rarely does anyone mention how many of the treatments being studied will fail one or more of these tests.
If the pre-clinical studies are completed and the treatment still seems promising, the FDA must give permission to test it in humans.
The investigational new drug (IND) application
Before a clinical trial can be started, the research must be approved. An investigational new drug or IND application or request must be filed with the FDA when researchers want to study a drug in humans. The IND application must contain certain information, as described below. The FDA reviews this information before human clinical trials start. Here’s some of the required information on an IND request:
Pre-clinical studies: Results from studies, including those on animals, allow the FDA to decide whether the product is reasonably safe for early testing in humans. This part may also include any experience with the drug in humans (if the drug has been used or studied in another country, for example).
Manufacturing information: This explains how the drug is made, who makes it, what’s in it, how stable it is, and more about the physical qualities of the drug. The FDA uses this information to decide whether the company can make batches of the drug that will always be exactly the same.
Clinical protocols and investigator information: Detailed outlines for the planned clinical studies, called the study protocols, are looked at to see if the study might expose subjects to unnecessary risks. (For more, see “The study protocol” in the section “How do I figure out which study is for me?”). Information on the clinical investigators who will supervise the study is reviewed to find out if they’re qualified to run clinical trials. Finally, the research sponsor must commit to getting informed consent from the research subjects, having the study reviewed by an institutional review board (IRB), and following all the rules required for studying investigational new drugs (see “Safeguards in institutions” in the section “What protects the study participants?”).
Last Medical Review: 09/21/2012
Last Revised: 09/21/2012