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.
Who Is Eligible to Be in a Research Study?
Almost anyone can be in some type of research study. Each study has certain requirements about health, medications or age depending on what specific questions are being asked. You must meet the requirements of a particular study to be an eligible volunteer
What Is Involved in Participating in a Research Study?
- Participating in a research study is much like a regular visit to a clinic or doctor’s office, but with even greater personal attention. The study subject may be referred by their doctor or may have heard about the study elsewhere.
- Preliminary screening for the study is usually done over the phone. Basic criteria of age, symptoms, and medical history are reviewed and the details of the study are discussed. If the caller seems to qualify for the study and is interested, they are asked to come in for the initial, or screening, visit.
- The screening visit is done in the a clinic, office or hospital. After reviewing the information gathered over the phone, the informed consent form is signed by the subject and the supervising physician. A copy is given to the subject. A physical exam, blood, and other tests may be done. Following this, in most studies, there is a period, usually a few weeks, where baseline information is collected, for example severity and frequency of symptoms.
- At the end of the screening period the patient returns to the clinic for the randomization visit. If the patient’s baseline information shows that they qualify for the study, they are then randomized (usually by computer) to receive placebo or active drug.
- During the treatment period the subjects are taking the study medication on a regular basis, and recording their symptoms. There are regular visits with the study coordinator during the treatment period. At the end of the treatment period, medication use and symptoms are reviewed. Possible side effects from the study medication are recorded. After completion of the treatment period, many studies have a follow up period to assess how symptoms and possible side effects have changed. There may be one additional visit or a telephone call to assess how the subject has been doing since stopping the study drug.
What Are the Risks of Participating in a Study?
Risks vary from study to study. Researchers expect certain results but since the treatment is new and is still being studied it is impossible to say exactly what the risks may be. If a side effect or adverse event does occur, it is generally temporary and will go away as soon as the treatment is stopped.
Why Think About Participating in a Research Study
- To help yourself, as you might have a beneficial effect from the study drug.
- You will receive a great deal of personal medical attention generally at no cost to you.
- To help others, as a great deal of information is gathered during studies, making new treatments available.
Deciding to Participate in a Research Study
- Think it over carefully, weigh possible benefits against risks.
- Make sure all your questions are answered by the study personnel.
- Discuss the study with your own doctor to see what their feelings may be about it.
- If you decide to enter a study, do not do so just out of curiosity. It is important to make a commitment to try to finish the study, unless you develop serious problems.
- Remember participation in a research study is always voluntary.
- You may refuse to participate, or withdraw your consent at any time, and for any reason, without jeopardizing your future care at this institution or your relationship with your doctor.
- If you are a patient with an illness, you do not have to participate in research in order to receive treatment.
Resources for Studies
- Current On-going Studies at the UNC Center for Functional GI & Motility Disorders
- International Foundation for Functional GI Disorders: www.iffgd.org
- IBS Self Help Group: www.ibsgroup.org