Research Testing of New Drugs
Drug trials are not like regular therapy, because the medical team is obliged to give medication according to a protocol. The protocol is developed before opening the trial to patients, by the sponsoring company and regulatory bodies. The protocol specifies all details of treatment with the experimental drug: who is eligible to get it, what other medications are allowed during the testing, what dose or doses of drug should be used, how fast it should be started, under what conditions it should be stopped, what tests are done, how long the drug can be used, how and when doctor visits must take place, and so on.
The patient and the physician always have the right to quit a trial at any point, but they do not have the right to alter the rules of the protocol. In effect, license is granted to use the drug only under very specific conditions. This is different from use of a regular prescription drug, where the doctor and patient can tailor, adjust, or change the drug regimen to obtain the best effect in an individual patient.
Many, but not all, drug trials require the use of a placebo. A placebo, sometimes called a "sugar pill", is an inactive pill made to look like the study drug. Some patients in the trial receive the real drug and some receive the placebo. Neither the patient nor the medical team know who is getting a placebo, although the information is made available by the drug company in case of an emergency that requires knowledge of treatment.
The placebo is required by FDA rules of testing to eliminate drugs that really do not work, but just make people think that they work because of excessive optimism, desire to please, or more attention paid to people during a trial. Most trials have provisions to provide the active drug to all participants at some point (usually after a test phase), since it is unfair to volunteer without ever having a chance to try the drug.
The main concern in all drug trials is safety of the study participants. If there is a conflict between following a protocol and keeping an individual patient safe or well, then the safety issue must be addressed first. All protocols are reviewed by an objective body, called an "Institutional Review Board" or IRB, that verifies that the drug appears to be safe, the protocol is well-designed, and all possible safeguards are in place in case of unexpected problems.
Every drug has side effects, and new drugs can have unexpected side effects. Each participant will be asked to read and sign a document of "informed consent" which lists the reasons for doing the testing, what you can expect to happen during the testing, what are the possible benefits, what are the risks, and what to do if you have problems.
No one should ever be forced to participate in drug trials. Just because you are eligible, does not mean that you need to participate. You should be offered alternative treatments, or when none exist, no treatment at all. If the trial seems right for you, however, it may provide a useful treatment to you years earlier than you would otherwise be able to get it.