Understanding Pharmaceutical Research Studies
Medical researchers are constantly looking for new or better ways to treat illness or disease. If they discover something that may be helpful, it cannot be put into general use until years of careful testing has been done. Research studies are what link medical research to a drug becoming available to physicians and patients. Research studies may also be called clinical trials, drug trials or drug studies.

What are Research Studies?
Research studies are designed to test the effect of a medication or treatment in a group of volunteers, measure a drug’s ability to treat the medical condition, monitor the drug’s safety, and possible side effects.
Pharmaceutical companies or other health organizations may sponsor research studies by providing funding and designing the protocol, which is a set of detailed guidelines. A study that is conducted at several different locations is called a multi-center study.
Trained doctors, nurses and researchers conduct research studies. The study coordinator is in charge of the day-to-day running of the study. The principal investigator (usually a physician) has overall responsibility for carrying out the protocol.
How Are Study Subjects’ Rights and Safety Protected?
The Food and Drug Administration (FDA) is the government agency that is responsible for research studies. It regulates the conduct of research studies, enforces the laws on the use of drugs, and must approve all new drugs before they are available to the general public.
In every university or medical center, the Institutional Review Board (IRB) reviews any study that may be done in that location. The IRB is composed of physicians and lay people. They review the study protocol to make sure patients’ rights are protected and that there are no unnecessary risks in the study. Any physician awarded a research study must get approval from the IRB before beginning the study.
Participants are required to sign an “informed consent” form, which is also signed by the investigator (the doctor conducting the study). It details the nature of the study, the risks involved and what will happen throughout the study. It informs study subjects that they have a right to leave the study at any time and who to call if they have questions. Finally, since the patient is under a doctor’s supervision, the same laws and ethics that normally regulate the medical profession protect the study subject.
What Are the Different Types of Pharmaceutical Research Studies?
There are three phases, or steps, in doing research studies. All three of these steps must be successfully completed and all results known before a new drug can be approved for public use.

Phase I studies are done on healthy volunteers who agree to take the study drug to help the doctors determine how safe the drug is and if there are any side effects. Studies are also done to determine how the drug is absorbed, metabolized and excreted. Usually a small number of subjects (20-100) participate in Phase I studies. Approximately 70% of new drugs will pass this phase.
Phase II studies measure the effect of the new drug in patients with the disease or disorder to be treated. The main purpose is to determine safety and effectiveness of the new drug. Usually several hundred patients participate. These studies are usually “Double-blinded, randomized and controlled”. In controlled studies, the effect of the active drug is compared to the effect of a placebo (inactive or “sugar” pill). In double blinded studies neither the investigator nor the study subject knows who is getting active drug and who is receiving placebo medication. One third of studied drugs complete both Phase I and II.
Phase III studies also use patients with the disorder to be treated by the new drug. These studies are done to gain a more thorough understanding of the effectiveness, benefits and side effects of the study drug. These studies use a large numbers of subjects, several hundred to several thousand. Of the new drugs that enter Phase III studies, 70 to 90% of drugs successfully complete this phase. If the results show a good effect and safety profile, the company will submit the data and request FDA approval for marketing the drug.