TL;DR:
- Clinical Trials Definition: Research on human subjects to study a drug’s clinical effects, safety, dosage, metabolism, and side effects.
- Safety Measures: Volunteers are monitored by specialists, protected by regulations (e.g., FDA, EMA), and can withdraw anytime without penalty.
- Preparation Process: Drugs are pre-tested via lab studies and animal trials before human testing begins with low doses.
- Trial Phases:
- Phase 1: Tests safety in small healthy groups or patients.
- Phase 2: Evaluates safety and effectiveness in hundreds, often blind trials.
- Phase 3: Confirms safety, efficacy, and compares to existing treatments with thousands; may last years.
- Phase 4: Post-approval monitoring.
- Benefits for Volunteers: Free care, innovative therapies, medications, and contribution to medical advancement. Common "guinea pig" fear is a myth.
There are many diseases and ailments that have long been described and diagnosed, but there are still no drugs to effectively cure or eliminate recurrences. This is, for example, type II diabetes, rheumatoid arthritis, or ulcerative colitis. That is why it is so important to constantly work on the development of medicine and develop new preparations. In order for them to be available to all those struggling with diseases, they must undergo clinical trials and be marketed. What are these trials and how are they conducted? Are they safe? What are the benefits of volunteers taking part in them?
What are clinical trials?
Clinical trials are a specific type of research and procedures conducted with human subjects to discover, determine, and confirm the pharmacological, or clinical effects of a substance, treatment, or therapy. During the procedures, possible side effects and the occurrence of reactions with other drugs or foods are also identified. The entire process of metabolism of the preparation, its absorption and excretion is studied. During the tests, the safety of the substance, the exact dosage and the effectiveness of the action in relation to the indications for the use of the drug are determined.
Safety, health and welfare of volunteers
The guiding principle of all clinical trials is the health and welfare of the volunteers participating in the procedure. At every stage they remain under the close care of a staff of specialists, and their parameters and general health are regularly monitored. The guiding principle of medicine "primum non nocere" – first, do no harm – applies here. Therefore, the patient's health takes precedence over the science and testing of a substance during clinical trials. In addition, patients participating in clinical trials are protected by a number of independent institutions, such as the Food And Drug Administration (FDA), and the European Medicines Agency (EMA). Every step of the ongoing research is therefore monitored and documented in detail, and reported strictly in accordance with specific guidelines.
How do we know what is in the preparation administered to volunteers? Is it safe?
Before any human clinical trials occur, a substance undergoes a series of thorough preclinical studies, laboratory procedures and tests. Once a drug product is determined in its formulation, in vitro tests are carried out, which are performed under laboratory conditions on living cells isolated from the body. The next stage is animal testing. This is done to evaluate the effects of the formulation on a living, functioning organism. If these procedures are successful, then the medicinal product is approved for clinical trials with humans, where it starts with the lowest possible doses, while studying reactions in detail.
Recruitment of willing participants in clinical trials. How does it proceed?
Clinical trials involve individuals who have volunteered to participate. They must meet certain conditions, which constitute predisposition to the research in question. For example, in a clinical study of a preparation for skin problems, a person whose skin is in perfect condition and does not need any medications will not be able to participate. A willing person who meets the criteria for a clinical research project is given detailed information about the procedures that will affect him. She is presented with a plan for the entire experiment, the various stages and information on the indications for the product. If the person is still willing to take part in the research after taking this knowledge, then he or she signs a special informed consent form. The volunteer for the duration of the clinical trials is provided with full insurance, anonymity and full access to information about his or her condition, which he or she will be able to obtain at every stage of the procedures. At any time during the experiment, the patient has the right to withdraw from the study without giving a reason and without suffering any consequences.
Is a volunteer a "guinea pig"?
Unfortunately, there is still a misconception in many social circles that clinical trials are a form of human testing of preparations of unknown origin that can cause a number of colossal side effects. This is a myth. The most important link in the course of the entire clinical trials is the patient, who is subject to full protection, both health and legal. The very idea of clinical trials also aims to do good and develop medicine for people, in order to make available innovative means to treat conditions that determine the lives of a huge number of patients, preventing them from enjoying life and functioning without pain.
The course of clinical trials
Clinical trials are subject to the rules of so-called Good Clinical Practice. They are controlled simultaneously by several independent institutions, and their start must be approved by the Bioethics Committee. Clinical trials are conducted in three main stages. The first stage is a preliminary assessment of the safety of the product. In the case of a trial of a vaccine or a new treatment regimen, a group of dozens of healthy people usually take part. If a new drug is being studied, for example, for an incurable disease, people with a specific condition enter the study. This stage of the activity begins with the administration of progressively higher doses of the drug to patients under close monitoring of the body's response. This provides preliminary information on whether the preparation has therapeutic value. The first phase of research usually takes place in the centers of scientific institutions, pharmaceutical companies or special research centers. The second phase of clinical trials is to further check the safety of the substance's use and determine the presence of possible side or toxic effects. This phase is also aimed at obtaining a scientific answer to the question of whether the tested preparation works in a given group of patients. Here, several hundred volunteers are usually involved. A so-called "blind" method is used, which involves keeping a secret from both the one administering the drug and the one taking it whether the current dose contains the test substance or a simple placebo. Such measures allow for reliable, authoritative and subjective results as to the actual therapeutic properties of the preparation. At this stage, the dosage regimen of the drug, the exact parameters as to the absorption, excretion and metabolism of the substance, as well as possible types of side effects, are finally established. The second stage will be completed when it is determined that the benefits of taking the preparation far outweigh the possible side effects. This conclusion must be supported by a series of detailed documentation. The third stage of clinical trials is the final confirmation of the efficacy of the drug's predetermined dosage regimens and a detailed description of the contraindications to its use. This is also the point at which the safety and efficacy of the test substance in the average patient must be documented and proven. In the third phase of clinical trials, all the documentation collected on the drug's effect on a given condition is compared with the effectiveness of other substances in the same group. At this stage, up to several thousand volunteers from different countries take part in the study. It can last up to several years, due to the testing of the effectiveness and safety of the preparation in both short-term and long-term treatment. If the results of these prove the substance's effectiveness and is more favorable than similar drugs already on the market, then the product can be registered and placed on the general market. Sometimes, in connection with clinical trials, a fourth stage is also distinguished, which involves monitoring the effects of the marketed substance. These are also the methods leading to the improvement of the product, the determination of further indications for use, or the release of further versions with a different dose, or, for example, in a flavored version, depending on the nature of the preparation.
Credibility of clinical trials. Is it worth taking part in them?
Reliable results of clinical trials are supervised by the aforementioned institutions and government drug registration authorities. A number of programs have also been established, and plenty of opinions are available from people who have actively participated as volunteers in such procedures. Clinical trials carry a number of advantages. First of all, participation in them allows you to benefit from innovative technologies and specialized therapies under the guidance of qualified medical staff. It's a chance to win against a disease that many people have been struggling with for years without success. In addition, the tests are free, as is all the health care associated with them and monitoring of the condition. Also free are the medications the patient takes throughout the research. In addition to access to first-line therapy and the chance to resolve ailments, the research participant gains his individual contribution to science and medicine. Thus, he contributes to improving the livelihood and health of society as a whole.
Conclusion
Clinical trials are vital for developing safer, more effective treatments to fight diseases. They follow strict protocols to ensure volunteer safety and produce reliable results. By participating, you not only access cutting-edge care but also contribute to advancements in medicine. These trials are not experiments on "guinea pigs," but a controlled, ethical process aimed at improving lives. If you value progress and want to be part of meaningful change, clinical trials are worth considering. Medicine moves forward because of brave volunteers like you, and together, we can shape a healthier future.
