What are clinical trials for, why participate, what is important to know before participating and a host of other useful information.
In order to be made available to patients or obtain a new indication in addition to those already confirmed, a drug must be adequately tried: to do this, specific research studies are conducted in accordance with universally recognized and accepted procedures. Clinical trials provide information on a drug's safety and efficacy.
Different reasons may lead to the decision to take part in a clinical trial. By participating in a clinical study, patients have the opportunity to be examined very carefully, to have their health monitored and to learn more details about their conditions. There is also the opportunity to access treatments that are under experimentation and therefore not yet available. Reasons not directly linked to the patient's personal conditions include the possibility to contribute to the advance of medical research in areas that are still unexplored, helping to make new, better treatments available to the future generations.
All clinical trials clearly indicate who can participate through the list of inclusion and exclusion criteria, which are necessary for the findings to be reliable. These criteria may include age, gender, type and grade of disease, the patient's history and other medical conditions. Far from being random, the criteria are selected very carefully in order to find reliable answers to the scientific questions that the researchers intend to investigate, as well as to ensure an adequate level of patient safety.
Clinical trials are necessary to develop a drug, but because of their very purpose – the need to learn more about the agent's effects – are not exempt from risks. For example, the study treatment may turn out to be not particularly effective - or at least not equally effective for all participants - or adverse events may occur. This is why it is essential for patients to ask their doctor about the characteristics of the trial in which they are participating, so that they have all the information they need to make this decision in full awareness, before signing the Informed Consent form agreeing to participate.
A protocol is a study plan on which all clinical trials are based. The plan is carefully designed to protect the participants' health, while aiming to answer the clinical questions for which it is being conducted. The protocol describes what characteristics patients must have to be eligible for participation and how the trial will be carried out, including method of administration, doses and duration of treatment with the study drug.
The drug needs to be constantly evaluated, during the entire clinical development phase and subsequently at the time of marketing, in order to monitor negative side effects and protect patient safety.
During the clinical trial, the study drug is carefully observed: in particular, the investigators collect all reports of adverse events, i.e. any negative clinical event that occurs in a patient treated with the study drug. Adverse events do not necessarily have a causal relationship with the study treatment.
Among adverse events, the Sponsor of a clinical trial must record and evaluate adverse reactions, i.e. undesired harmful reactions correlated with the drug under experimentation, regardless of the dose administered.
A clinical trial may be started only if the Ethics Committee and/or the Competent Authority have determined that the expected benefits, in terms of treatment and public healthcare, justify the risks, and may only continue if compliance with this requirement is constantly monitored. For this reason, during the clinical trial any adverse reactions identified as serious and unexpected are urgently notified to the Competent Authority and to the Ethics Committee. The Competent Authority, the Ethics Committee and the study Sponsor all cooperate to protect the safety of the participating patients.
It is essential to speak with the doctor and understand all the characteristics of the research study, starting from its objectives, why it is being conducted, and how it will be carried out: tests and treatments that the patient will receive, the risk-benefit ratio of the treatment and its impact on activities of daily life, whether hospital stays will be required, and duration of follow-up once the treatment is completed. It is very important to know that all the information gathered as part of the clinical trial may be shared with the patient's General Practitioner.
In most cases, the purpose of the clinical trial is to compare the efficacy and safety of a study drug against one that is already available or against an inactive agent called placebo. In the majority of clinical trials, each patient is assigned to a group that may be receive the study drug or the control treatment. The choice is most often made on the basis of a random criterion: this is why the trial is described as randomized. Not even the doctor is allowed to know which treatment the patient is receiving: this is the meaning of "masked study". Of course, for the entire period of the trial the patient will be constantly in contact with the centre where the study is conducted, but will not be able to learn the results of the research until it is completed. Once the findings of the clinical trial have been reviewed, they are published in scientific journals, during conferences or on the portals www.clinicaltrials.gov and www.clinicaltrialsregister.eu.
For every medical act, patients are entitled and required to ask for extremely detailed explanations on the treatment they will receive and on how their personal data will be used. This guiding principle is even more important when patients participate in a clinical trial: before this happens, it is the doctor's duty to provide the patient with all the information concerning the treatments and tests he or she will receive. In the case of children, the doctor informs the parents or guardian and obtains the informed consent from them.
Clinical trials can be funded by various organizations: foundations, universities, groups of doctors, volunteer associations, pharmaceutical companies and public institutions. The trial centres are located throughout the country, in particular in hospitals, and this is where patients are enrolled. Information about the centres involved in the study and on enrolment procedures are provided in the protocol, which doctors must share with the patients. The protocol contains the details of the treatments to be administered to the patients, the dosage and method of administration, the frequency of visits, and other detailed information. It is also very important to understand the study design, i.e. whether the control group (which includes the patients who will not be treated with the study drug) will receive placebo or the standard treatment for that disease.
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