TL;DR:
- Clinical trials assess the safety and effectiveness of new therapies and are essential for drug registration and market approval.
- Trials follow Good Clinical Practice, prioritizing patient safety, rights, and reliable results.
- There are four phases:
- Phase I: Safety and dosage testing on healthy volunteers.
- Phase II: Efficacy and further safety testing on specific patient groups.
- Phase III: Large-scale efficacy and safety testing for short- and long-term use.
- Phase IV: Monitoring of marketed drugs' safety and effectiveness.
- Factors to choose a clinical trial center include trained staff, strong monitoring, compliance with standards, and patient support like reimbursement and home visits.
- Supervision involves bioethics committees and regulatory bodies.
- Benefits: Access to experimental therapies, no treatment costs, continuous health monitoring, and contribution to advancing medical knowledge.
Clinical trials make it possible to assess the effectiveness and safety of new therapeutic substances. As a result, they are the foundation of modern medicine, a prerequisite for patients' access to modern therapies, as well as significantly expanding the professional knowledge of doctors. Clinical trials are a fundamental part of registering a new drug and bringing it to market. Virtually any person who meets certain criteria for the substance being tested can undergo them. However, many patients who are beginning their adventure with clinical trials do not know which center will be the best choice. We explain what to look out for.
What are clinical trials based on?
Clinical trials are conducted in various locations around the world. They are supervised by the relevant regulatory authorities. In order for clinical trials to be conducted effectively, they require precisely defined methodology, principles and scale of diagnosis, method of recruiting patients, method of verification and exercise of control over the course of clinical trials. In Poland, supervision over the course of research is exercised by the Office for Registration of Medicinal Products, Medical Devices and Biocidal Products, as well as by the Bioethics Commissions.
What makes clinical trials safe?
The most important thing in clinical trials is the safety of the patient and safeguarding his or her rights. This is why all research must be performed on the basis of Good Clinical Practice. The main principles relate to respecting the patient's welfare, rights and safety – overriding the interests of science or society. The data obtained, on the other hand, must be reliable and accurate.
How are clinical trials conducted?
Clinical trials are conducted in four phases, each of which must be successful in order to proceed to the next phase.
Phase I consists of a preliminary assessment of the safety of the test substance. Among a group of dozens of healthy volunteers, its metabolism, absorption, excretion, toxicity and interactions with other ingested drugs and substances are analyzed. Once this phase of the study is completed, the dosage of the drug in question can be determined.
Phase II identifies whether the drug works on a specific group of patients, and whether it is safe for them. The relationship between the dose of the drug and the substance's effect is also analyzed. This results in the determination of the dose used in subsequent stages of the study. All the time, the drug's efficacy and safety are also evaluated. Data related to the drug's absorption, metabolism and excretion according to gender and age are analyzed in detail in this phase of the study. Specialists also compare the effects of the new drug and placebo.
This is possible to do, among other things, using a blinded method that provides an objective assessment of the effect. Neither the patient nor the researcher knows whether the patient is given the substance under study or a placebo. A positive evaluation of Stage II is obtained when the benefit/risk ratio of the drug used, is significantly higher.
Phase III is aimed at confirming the efficacy of the investigational drug in the treatment of a given condition. This phase involves testing the relationship between the safety of a substance and its effectiveness with short-term and long-term use. The study involves a group of up to several thousand patients, and can last from one year to several years. As in Phase II, a double-blind method and random selection of study participants is used here. After a positive Phase III result, the drug can be registered and marketed. A drug dossier is submitted to the medicinal product registration authority, which includes all the data collected during the Phase I to Phase III trials.
Phase IV applies to registered drugs and marketed drugs, i.e. those that are available for sale. This phase determines whether a drug is safe in all manufacturer's recommendations and for all groups of patients. This phase also verifies the results obtained in previous phases.
What should a clinical trial center be like?
The most important element that affects the quality of the study is properly conducted monitoring during the trial. The best center in clinical research consists of a team of specialists who excel in interpersonal skills and a high level of responsibility. On the other hand, regular staff training in accordance with international standards allows them to gain valuable, up-to-date knowledge. Reputable centers also ensure that all members involved in a given trial participate in training and the Sponsor's Standard Operating Procedures – if required. Clinical trial sites should ensure that trials are documented and reported according to protocol, standard operating procedures, good clinical practice and relevant regulations. Organize and fully supervise payment to investigators and study personnel, and provide up-to-date information and payment statements. Professionals ensure management of the reimbursement process for patients' travel to appointments and (sometimes) their accommodation, in accordance with Polish legal regulations and clients' requirements. Specialists have the necessary experience in processing confidential information and protecting personal data. Together with business partners, the centers also offer to perform nursing procedures in clinical trials at the Patient's home. At the same time, they have specialized tools for scheduling, monitoring and staff training. With home visits, Patients do not have to travel to and from the research center. You can learn more about center capabilities on the aesthetic medicine section of the OT.CO Clinic website. The speed of data entry is also an important part of evaluating cooperation with the center. It's also an element that provides oversight of Patient safety.
Who ensures compliance with Good Clinical Practice?
Compliance with the principles of Good Clinical Practice is supervised by the internal audit units of the companies that conduct the trials, as well as by the government authorities in charge of drug registration and inspection. All clinical trials must have a scientific rationale and a plan. This document is subjected to thorough ethical and substantive review by independent bioethics committees. For broader context, see how plastic surgery principles are similarly governed by rigorous oversight. Not only the content of the information addressed to the patient is evaluated, but also its form – it should be as comprehensible to the patient as possible.
Participation in clinical trials offers a number of tangible benefits for patients, participants, researchers, centers, etc. First and foremost, it is the opportunity to use a therapy that is unavailable or difficult to access on the market. As a result, it provides an opportunity for the patient to have a new and better effect of the therapy used. In addition, the patient does not incur any costs related to treatment and diagnosis. All expenses related to participation in the clinical trial can be reimbursed. Participation in a clinical trial also guarantees close monitoring of the Patient's health status prior to treatment with the investigational medicinal product – during the trial and after the administration of the drug.
This allows the patient's health status to be recognized on an ongoing basis, and the safety and effect of therapy to be continuously monitored. Without the Patient's participation in clinical trials, it is not possible to register the medicinal product, allow it to be marketed and ensure its availability – despite the high effectiveness of the treatment. Every Patient, therefore, by his or her participation in the study, contributes to improving the level of public health. For researchers, on the other hand, participation in a clinical trial provides an opportunity to gain unique knowledge and accumulate new experience in the medical field. This is made possible by access to new therapies, increased ability to use modern medical technology.
At the same time, researchers gain the opportunity for greater collaboration through interdisciplinary cooperation and access to additional training and materials. Clinical trials also provide many benefits for market participants, in the form of, among other things, development of know-how. All the data and information that is received during clinical trials is an important contribution to the development of medical knowledge regarding the efficacy and safety of pharmacotherapy. It's also an opportunity to create new treatment standards, as new forms of treatment are implemented and new, more effective drugs are provided as a result of the trials.
Conclusion
Clinical trials drive medical progress, ensuring treatments are both safe and effective. By following strict protocols and Good Clinical Practice, they protect patients while advancing healthcare knowledge. Choosing the right research center ensures a positive experience, with professional teams and robust oversight. Participation offers patients innovative therapies, while aiding researchers in developing groundbreaking treatments. Clinical trials are essential for registering new drugs and improving public health. Their success depends on careful collaboration, precise execution, and trust in the process. Ultimately, these trials benefit all, shaping the future of medicine and transforming lives worldwide.
