What is a clinical trial?
Clinical trials are the way doctors discover better ways to diagnose and treat disease. Participants must volunteer to be in the trials. Patients who participate in clinical trials may have access to new medicines not available to the general public. They are also helping other people and medicine in general, because they will be a part of learning something new. They may help prove a new medicine works. Even if something does not work, that is important information, because doctors will know not to use the particular medicine for the particular disease.
The FDA (Food and Drug Administration) requires that all medications be proven both safe and effective. There is a lengthy process for testing medications that begins in the laboratory, long before any people will be taking the new medicine. A new drug must be tested on cells in a laboratory, and on animals. An IND Application (Investigational New Drug) must be filed with the FDA. This must include the information from testing the drug on animals. The drug must appear to be relatively safe for humans. A study protocol must be designed to test the drug while minimizing the risk to participants. An Institutional Review Board must approve the study. This Board could be at the medical center where the trial is to take place, or it may be a larger, regional Board.
In a cancer trial, every patient will be given treatment of some kind. In a mesothelioma trial, the standard treatment might be chemotherapy with pemetrexed and cisplatin, and that might be compared with another two-drug combination, or with surgery and chemotherapy, as examples.
If these trials prove safety and efficacy (effectiveness), the FDA will say that the treatments can be given to the general public. Phase IV testing means that the FDA continues to look at the treatment in case rare side effects occur that will only be seen when thousands of people have used the medication.