The process of conducting the study depends on each individual study, and is carried out by a study team consisting of doctors, nurses, and other health professionals as needed. Their task is checking the health of the subjects before starting the study and monitoring them over time, giving specific instructions for the duration of the study, and staying in touch with the patient even after the study is completed.
A clinical trial includes human volunteers and serves to answer a specific medical question. Carefuly conducted clinical studies are the fastest and safest method to discover new treatments or ways to improve human health. Interventional studies investigate whether experimental treatment (or standard treatment applied in a new way) is safe and effective under controlled conditions, while observational studies investigate the health problems of large populations of people in natural conditions.
Each individual study has specific criteria which define the patients who may participate in it. The use of inclusion and exclusion criteria is an important principle of medical research that ensures the reliability of the final results. The factors that allow the patient to participate are the inclusion criteria, while the patient who has some of the exclusive criteria is not a candidate for participation in the study. These factors may include age, sex, type and stage of the disease, previous treatment, and other medical conditions / illnesses. Some studies include patients with a specific disease, while other studies include healthy individuals. It is important to note that the inclusion and exclusion criteria are not defined to prevent the inlcusion of patients in the study on a personal level, but to identify groups of patients who have the highest chance of treatment success and to ensure the safety of therapy. Also, well-defined inclusion and exclusion criteria allow the study team to get a clear answer to the question posed by the study.
Well-designed and conducted clinical studies are the best approach for patients who want to have an active role in their own health care, and who want to gain access to the newest types of treatments before they are widely available. Also, clinical study participants can help other similar patients by supporting scientific research. There are also risks, such as the development of side effects (this also applies to any other standard drugs and therapies), treatment may be ineffective for the individual patient, and the protocol itself may be time-consuming for some patients (traveling to the study center, more therapy, complex dosage regimen…), although this is not the rule.
Ethical and legal health care regulations apply to clinical studies as well. In addition, specific regulations reagrding for clinical studies apply, in order to ensure that the safety of subjects included in the study is not compromised. As stated in the question about the study protocol, the study is conducted according to a well-defined detailed plan. Over time, study team reports research findings at scientific conferences, in medical journals, and to various government agencies. The identity of participants is always kept secret and there is no mention of the patient’s name or any other characteristic by which he/she can be identified.
It is always a good idea to gather enough information about the clinical study before deciding to participate, and to clarify any concerns you may have with the doctor who is conducting the study. All important issues are also clarified in informed consent, which must be signed by the patient before the study starts. Some of the important questions that should be answered are:
- What is the purpose of the study?
- Who will be included into the study?
- Why do researchers believe that the new drug being tested could be effective?
- What diagnostic tests and treatment will be performed/given to the patient?
- What are the possible risks, side effects, and benefits of the new drug?
- How can participating in a clinical study affect my daily life?
- How long will the study take?
- Will hospitalization be required?
- Who will pay for the treatment in the study? Will my expenses be covered?
- Is there any form of patient follow-up after the study is completed?
- How do I know if a study drug is working? Will I get the final results of the study?
- Which study team will take care of me?
No special preparation is required for the first interview. Make sure you bring your complete medical documentation. The study doctor will answer all your questions during the first interview, so it is a good idea to write down everything that is not clear. If necessary, the patient can bring a trusted person with him/her for support.
Yes, as before inclusion in the study. This is especially important since most clinical studies focus on relatively short-term treatment of a specific disease or condition, and comprehensive health care is not provided. Also, the collaboration of the family physician and the study team ensures that the medications the patient regularly takes are not in conflict with the study medications.
Yes, the patient may choose to leave the study at any time. It is only necessary to inform the leading study physician, who will record the reason for the patient’s withdrawal from the study.
Clinical studies can be sponsored by a variety of organizations or individuals, such as physicians, healthcare institutions, groupations, volunteer groups, and pharmaceutical companies, as well as some government agencies. Likewise, studies can be conducted in a variety of medical institutions, such as hospitals, colleges, offices and clinics.
Each study is based on a protocol which covers the clinical study plan in detail. The protocol is carefully designed to ensure the safety of participants (patients) and the relevance of the final results. The protocol defines, among other things, what characteristics a patient must have in order to be included in a particular clinical study, the schedule of diagnostic tests, procedures related to the administration of study drug(s) and drug doses, and the duration of the study. In accordance to the protocol, patients regularly undergo clinical examinations by a competent physician to monitor their health status and determine the safety and efficacy of the study drug used.
Placebo is an inactive tablet, liquid or powder that has no therapeutic effect. As noted in the previous question, in most clinical studies the new drug is compared with the current standard treatment or (often if there is no adequate standard treatment) placebo, to investigate the effectiveness of the new drug. In most cases, neither the patient nor the doctor knows whether the patient is receiving a new drug or a placebo to avoid unconsciously affecting the results.
Many studies are conducted in such a way that one group of patients receives a new experimental drug or other inovative treatment, while the other group receives the standard treatment or placebo. The latter group is referred to as the control group and is used to compare the results of the study drug with the current standard.
The most common are therapeutic studies, which examine new forms of treatment, new drug combinations, or a new approach to treating a disease. There are also prevention studies (looking for a better way to prevent disease in healthy people or to prevent disease recurrence; they may include administering medications, vitamins, vaccines, and minerals, or lifestyle changes), diagnostic studies (exploring new tests and methods in the diagnosis of specific diseases or conditions), screening studies (exploring the best way to detect diseases at early stage), and quality of life studies (exploring ways to improve the quality of life of individuals with chronic diseases).
Clinical trials are conducted in phases. The preclinical phase of testing is the initial stage when in vitro (in laboratory) studies and in vivo (on animals) studies are performed. This is followed by the clinical phase of the trial, which can be divided into four segments:
Phase 1. Researchers are testing a new drug on a small group of healthy individuals (20-80) to analyze the safety of drug administration for the first time, in order to determine the safe dose of the drug and identify possible side effects.
Phase 2. The study drug is administered to a slightly larger group of patients (100-300) to analyze the efficacy of the drug and to continue monitoring of side effects.
Phase 3. The study drug is administered to a large group of patients (1000-3000) to confirm efficacy, monitor side effects, compare the effect of the new drug versus standard therapy, and to gather data that allows the drug to be safely administered.
Phase 4. These are so called post-marketing studies, which gather additional information of the risks and benefits of the drug and determine the optimal use of the drug.