What is a Clinical Trial?
A clinical trial is any study involving people that tests, discovers, or confirms the effects of an investigational product (potentially a new drug), a new therapeutic method, or new medical equipment or apparatus.
Is a Clinical Trial Safe?
Before a clinical trial involving people is conducted, the investigational drug undergoes a series of safety tests in laboratories to evaluate its safety and suitability for human use. While complications after taking the investigational product can occur, they are exceedingly rare, and the first administration in humans occurs under strict medical supervision and with the provision of necessary medical care. The first administration of the investigational drug is done with healthy volunteers, except for oncological drugs, which are administered only to patients.
To conduct a clinical trial, the approval of the President of the Office for Registration of Medicinal Products, Medical Devices and Biocidal Products is required. Such approval is granted following a detailed analysis of the study, clarification of any doubts, and registration of the study in the Central Register of Clinical Trials.
Who Ensures the Safe Conduct of Clinical Trials?
Doctors and medical personnel conducting the trial are obligated to follow detailed and strict principles for conducting clinical trials as defined by international guidelines and national legal regulations. In Poland, the entity overseeing clinical trials is the Office for Registration of Medicinal Products, which is subject to the Ministry of Health. International studies conducted in Poland may also be monitored by international supervisory bodies such as the FDA (Food and Drug Administration) and the EMA (European Medicines Agency). These institutions ensure that clinical trials are conducted properly in accordance with guidelines for conducting clinical trials, including those outlined in the ICH GCP (Good Clinical Practice) document. These principles are implemented in our country through the Minister of Health’s regulation regarding detailed requirements for Good Clinical Practice.
According to these guidelines, patient safety is always paramount and takes precedence over scientific or societal interests.
What is the Role of an Ethical Committee and What Does it Do?
Ethical Committees typically include not only medical experts but also professionals from other fields, such as philosophers, lawyers, nurses, and priests. The Ethical Committee evaluates clinical trials in terms of their validity, feasibility, the correctness of the study protocol, the appropriate selection of the principal investigator, the quality of the research facility, the analysis of the benefits and risks associated with the study, the completeness and quality of information provided to the patient, and the proper procedure for obtaining informed consent.
If a clinical trial does not receive a positive opinion from the Ethical Committee, it cannot proceed.
Who Typically Sponsors Clinical Trials?
Clinical trials in Poland are mainly sponsored by pharmaceutical companies, but they can also be sponsored by other medical, research, foundation, association, or individual institutions.
What Do the Phases in Clinical Trials Mean?
After laboratory tests of the investigational drug are completed, the next stage of research involves administering the drug to people and analyzing its effects on the human body. Clinical trials involving humans are divided into four phases:
Phase I: The investigational drug is administered to a person for the first time. A small group of people (20-80 individuals), typically healthy volunteers, participate in this phase. In Phase I, the safety of the drug is evaluated, a safe range of drug doses is established, and potential side effects are identified.
Phase II: The drug is administered to a group of patients who are the intended recipients of the medication. The new drug is then used in a larger group of people (100-300 individuals). The primary objectives of Phase II are to confirm the safety and efficacy of the drug, monitor possible side effects, determine optimal therapeutic doses, and collect data for safe use of the new drug.
Phase III: The purpose of this phase is to confirm the safety and efficacy of the new drug in a larger population of patients (1000-3000 individuals) and gather data for drug registration.
Phase IV: Phase IV studies involve the broadest group of people because the drug is already registered and available on the market. During this phase, further observations of the drug’s effects are carried out to obtain additional information about the benefits and risks of its use.
Why Participate in Clinical Trials?
Participating in a clinical trial can provide access to a new drug that may, but is not guaranteed to be, more beneficial than currently standard treatments. It offers the opportunity to be cared for by top-notch specialists in a university research center, often involving more frequent visits than standard medical procedures. Furthermore, by participating in clinical trials, you help other patients who may benefit from better treatment in the future.
It’s essential to keep in mind that the new drug (the investigational product) used during the study has not yet passed all tests confirming its effectiveness and safety.
Is Participation in a Clinical Trial Required to Receive Treatment?
No, participation in a clinical trial is not a prerequisite for receiving standard treatment. To access the standard treatment for a particular condition, there is no need to participate in a clinical trial. The decision to participate in a clinical trial is solely up to the patient, and the treating physician should not exert any pressure on the patient regarding participation.
A doctor should provide the patient with comprehensive information about the clinical trial.
Will I Be Guaranteed Treatment with the New (Investigational) Drug if I Decide to Participate in a Clinical Trial?
Not necessarily. In some trials, participants may receive a “placebo” or already registered standard treatment. In most clinical trials, the investigational drug is compared to a placebo. A placebo is a substance that has no effect on the health of the person who takes it.
Patients in a clinical trial are usually divided into groups, with each group receiving standard treatment. One group, in addition to standard treatment, will receive the investigational drug, while the other will receive a placebo.
What Is a Study Protocol?
A clinical trial protocol contains the study plan, detailing everything to be conducted within the study, and includes information for the safe conduct of the study. Clinical trials are designed to answer research hypotheses while ensuring participant safety. The protocol defines who can participate in the clinical trial, what criteria, especially regarding health status, participants should meet, and provides a schedule of procedures performed during the study. It describes the doses and frequency at which the investigational drug should be administered, outlines potential side effects, and explains how a physician should respond if side effects occur.
What Does Giving Informed Consent to Participate in a Study Mean?
Providing informed consent to participate in a study means that you agree to take part in the study with an understanding of the associated risks and potential benefits. Before giving informed consent, individuals who wish to participate in a clinical trial should be informed by the investigator/physician about the study’s main principles, the procedures to be performed, potential risks, and possible benefits. Based on the information provided by the investigator/physician, the patient decides whether they want to participate in the clinical trial. Before making such a decision, the patient should be sure they understand what their participation in the clinical trial will involve. If there are any doubts or if something is unclear, the patient should ask the physician for clarification.
It is the responsibility of the investigator/physician to obtain informed consent from the patient, which entails thoroughly informing the patient and addressing any doubts related to participation in the clinical trial.
A patient deciding to participate in the study will be asked to sign a document of informed consent. This document contains basic information about the study, such as the study’s purpose, duration, procedures performed as part of the study, and contact information for inquiries about the study. The informed consent document also describes the risks and benefits of participating in the study. Signing the informed consent document by the patient is not considered a contract, and the patient has the right to withdraw from the study at any time.
What should I know and consider before deciding to participate in a clinical trial?
A participant in a clinical trial should learn as much as possible about it. Some sample questions that may help and can be asked of the investigator/doctor include:
- What is the purpose of the clinical trial?
- Who can participate in the trial?
- Why do researchers believe that the new/investigational drug may be effective, and have there been previous studies on its effectiveness?
- What is the risk of using the investigational/new drug, what are the possible side effects, and what benefits can I expect from using the new drug compared to the therapy I’ve been using?
- Will participation in the trial affect my daily life?
- How long will the trial last?
- Are hospitalizations anticipated during the trial?
- Is there a follow-up period planned for after the completion of taking the investigational drug, and what does it involve? Are there visits to the hospital, and will any tests be conducted during those visits?
- Will I know if the treatment works for me?
- Will the results of the clinical trial be available to me?
- Who will be responsible for my medical care during my participation in the trial?