A clinical trial is any study involving humans conducted to evaluate the safety and effectiveness of an active substance contained in the investigational medicinal product. Clinical trials aim to confirm the action of the substance administered to the patient and involve assessing parameters such as efficacy, safety profile, and adverse effects.
For the safety of patients, a new drug cannot be brought to the market based solely on laboratory and animal studies. A product tested only on animals is not considered a medication, and new drugs cannot be introduced to the market without the stage of clinical trials involving humans.
The primary role in clinical trials is played by study participants, both healthy and ill individuals. Without their involvement, participation, and genuine collaboration with physician-researchers and research centers, it would not be possible to determine whether the tested drug is safe and effective. In other words, it would not be possible to introduce increasingly advanced and effective medicines for sick individuals to the market.
Clinical trials are subject to very detailed procedures and rigorous control at every stage of their conduct. This is done to minimize the risks associated with conducting the study, particularly the risks that could result in harm to the study participant (patient).
The most significant risks to the patient are related to the possibility of adverse events and unwanted occurrences associated with the use of the investigational product or the study procedures.
Other risks may stem from a violation of the participant’s rights, such as if:
They are enrolled in the study without being provided with full information or giving informed consent to participate in the clinical trial.
Their rights to withdraw or protect personal data are not respected.
Sometimes, the patient can contribute to harm by not following medical recommendations. All parties involved in the study are responsible for minimizing the risks associated with participation in the trial. Qualifications of the physicians and the research team are crucial, as they need to be properly prepared to conduct each clinical trial.
Monitoring the progress of the study by the sponsor aims to ensure ongoing safety for the patients participating in the study.
Patient information is a document intended for potential study participants, providing a detailed description of all aspects of the clinical trial. This document can only be given to potential participants if it has been previously approved by the Office for Registration of Medicinal Products, Medical Devices, and Biocidal Products (URPL) and the Ethics Committee. The document serves to familiarize the potential participant with the study’s objectives, course, benefits, risks, obligations, participant’s rights, and other important matters that are crucial for making an informed decision about participating (or not participating) in a particular clinical trial.
The Informed Consent form is a document on which the participant places their signature and date if they decide to take part in the clinical trial. This happens after receiving comprehensive information from the conducting physician (investigator). The Informed Consent form and the Information for Clinical Trial Participants are often presented as a single combined document. The patient receives their copy of the Information and Consent. Obtaining the patient’s informed consent for the study is a necessary condition for subjecting the participant to any clinical trial procedure. The patient must always have an adequate amount of time to thoroughly review this document before signing it.
Participation in a clinical trial can provide the patient with additional or new therapeutic support.
A patient qualified for participation in a clinical trial receives the investigational product free of charge, as well as medical services resulting from the clinical trial. If the patient needs to travel for medical appointments related to their participation in the clinical trial, they have the right to receive reimbursement for travel expenses.
By choosing to participate in the study, the patient may gain therapeutic benefits for themselves and for other patients, such as potential treatment or symptom relief. Medications tested in clinical trials can help future patients with specific medical conditions, contributing to the advancement of medicine and the development of more effective drugs.
From the knowledge they receive from the researcher and the study information documents, the patient must consider potential benefits and possible adverse effects before making a decision to participate in the study.
They should contemplate the temporal and logistical commitments involved.
Thoroughly review the “participant information” and, in case of any doubts, ask questions to the investigator physician.
Adhere to the principles outlined in the “participant information.”
Before undergoing qualifying procedures, sign the “informed consent form” in the presence of the investigator physician, ensuring that all the information provided is clear.
Qualification procedures: Typically, the patient needs to arrive for the qualification assessment on an empty stomach and with information regarding their medical history, such as the results of laboratory, diagnostic, and imaging tests, a list of medications taken, and discharge summaries from hospital stays. All documents will be returned. During the qualification assessment, a medical interview, a physical examination, blood and urine samples for analysis, blood pressure, and pulse measurements are conducted. Depending on the investigational drug, other tests may also be performed to determine if the patient can participate in the study.
Clinical trial participants are individuals who meet the requirements outlined in the study protocol approved by the Office for Registration of Medicinal Products, Medical Devices, and Biocidal Products and positively reviewed by the Ethics Committee.
Potential participants can volunteer for the trials themselves or may be invited to participate in a clinical trial by a physician-researcher. After obtaining full information and obtaining the informed consent of the potential participant, the investigator assesses whether the protocol requirements are met. These requirements often relate to the stage of the disease, the presence of other medical conditions, medications being taken, and so on. The investigator decides to include the patient in the study if they meet the protocol requirements.
The rights of a patient participating in a clinical trial are described in, among other sources, the Good Clinical Practice guidelines. You can find this and other legal acts governing the rights of clinical trial participants HERE .
Selected rights of clinical trial participants (based on the guidelines of the Ministry of Health):
1) Participation in a clinical trial is entirely voluntary.
2) The patient has the right to refuse participation in a clinical trial without any consequences. In case of refusal, the patient does not lose their rights to treatment considered standard and appropriate in their case.
3) The patient must have enough time to thoroughly review the written study information and the informed consent form before deciding to participate in the trial.
4) The decision to participate in a clinical trial must be fully informed. If the information for the patient is unclear, they have the opportunity to ask questions to the proposing physician. The patient information is assessed by independent ethics committees, among other things, for its understandability by the average patient.
5) The patient has the right to receive a copy of the written study information and the informed consent form signed by them and the physician.
6) The patient can withdraw from the trial at any time if they believe it is in their best interest. However, they should inform their doctor about their withdrawal and attend a scheduled follow-up visit to assess their health status after leaving the trial.
7) Withdrawing from the trial during its course does not entail any consequences. It is the doctor’s responsibility to ensure further treatment following the principles of medical ethics.
8) The patient has the right to receive information from the physician about their health status at any stage of the clinical trial and to access documentation concerning them.
9) The patient has the right to be informed about any new data that could affect their decision regarding further participation in the trial. For example, they should receive new information regarding the risks associated with the use of the investigational drug.
10) The patient has the right to seek compensation if they have suffered permanent harm directly related to the use of the drug or medical procedure required by the study protocol, in accordance with the regulations of the Minister of Finance regarding mandatory liability insurance for researchers and sponsors.
11) The patient has the right to seek compensation if they have suffered permanent harm directly related to the use of the drug or medical procedure required by the study protocol, in accordance with the regulations of the Minister of Finance regarding mandatory liability insurance for researchers and sponsors.
12) The costs of treating adverse events are covered by the study sponsor, typically a pharmaceutical company.
13) Participation in a clinical trial is free of charge for the patient. The costs of investigational drugs, specialized tests, and medical care are borne by the study sponsor.
14) The patient has the right to confidentiality of the part of the clinical trial that would allow their identification and to exclude personal data from any publications. The patient has the right to protect their personal data.
15) Both during the trial and after its completion, the patient has the right to report any changes in their well-being to the attending physician.
Insurance in clinical trials
Currently, in clinical trials, the rules for liability insurance of the researcher and sponsor for damages caused in connection with the conduct of the clinical trial apply. The conditions, scope of insurance, and minimum insurance guarantee sums are specified in the regulation of the Minister of Finance of April 30, 2004, regarding the mandatory liability insurance of the researcher and sponsor. Liability insurance covers the civil liability of the researcher and sponsor for causing bodily injury, health impairment, or death to a clinical trial participant as a result of the action or omission of the insured or persons for whom they are responsible during the insurance protection period and in connection with the conduct of the clinical trial.
Starting from April 14, 2023, the Clinical Trials Compensation Fund began operating at the Patient Rights Ombudsman’s Office. More information about the Fund can be found on the Patient Rights Ombudsman’s website.
The patient does not incur costs for visits, procedures, or medications in the clinical trial – they are provided free of charge to the study participant.
A patient qualified to participate in a clinical trial receives the investigational product, medical services arising from the clinical trial, and reimbursement of costs incurred in connection with participation in the clinical trial, such as travel to the study center, free of charge. This is provided to all eligible participants or their legal representatives, and it is documented before the start of the study.
The patient can withdraw from the study at any time if they believe it is in their best interest. However, they should inform their doctor about their withdrawal. They should also attend a scheduled follow-up visit, which is meant to assess their health status after ending their participation in the study.
Withdrawing from the study during its course does not entail any consequences. It is the doctor’s responsibility to ensure further treatment following the principles of medical ethics.
Receiving compensation (or gratuities as described in Pharmaceutical Law) for participating in a clinical trial is possible when the study is conducted on a group of healthy, adult volunteers who can independently give informed consent. The need for compensation arises because a healthy study participant, who is administered an investigational drug, and is subjected to various inconveniences such as blood draws, does not derive any therapeutic benefits.
In clinical trials, it is possible to provide compensation to patients for the expenses incurred by them in connection with their participation in the study (the level of compensation depends on the specific study). Information about the compensation paid to study participants is included in the application for permission submitted to the President of the Office for Registration of Medicinal Products, Medical Devices, and Biocidal Products and the relevant ethics committee. The level of reimbursement for travel expenses or expenses incurred by the patient during the study depends on the sponsor. Such detailed information regarding expenses and their reimbursement should appear in the participant information form. The method or form of reimbursement of incurred costs can vary, such as a bank transfer to the patient’s account performed by an external company. Patients should be properly informed about the principles of cost reimbursement.