Clinical Research: What You Need to Know

A clinical study is research conducted on individuals who have a specific disease, with the aim of better understanding the frequency, causes, and effectiveness of different treatments for that disease, in order to improve the duration and quality of life for current and future patients. 
Clinical studies have made and continue to make significant contributions to knowledge and progress in the fight against many diseases, including cancer. Many of the most effective therapies commonly used in oncology today are the result of long research paths that, like drugs, begin in the laboratory (preclinical research, which studies the mechanisms behind cancers to develop and evaluate new treatment, diagnosis, and prevention strategies) and then move on to the patient’s bedside (clinical research). 
In oncology, most clinical studies aim to verify the efficacy and tolerability of treatments or diagnostic methods. 
In the case of drugs, the most common questions are: 

• Is it well tolerated by the patient? 
• Does it have therapeutic effects? 
• Does it achieve better results than previous drugs used? 
• What is the most effective dose? 
• What is the best way to administer it? 

The Protocol 
The first step in conducting a clinical study is drafting the protocol, which is a document that contains a detailed description of the study itself and governs its conduct. 
Among other things, the protocol contains: 

• The reasons for carrying out the study (scientific rationale) 
• The characteristics of the patients who may participate (eligibility criteria) 
• The treatment plan with which patients will be treated 
• The technical methods for conducting the study itself 
• The methods for managing and analyzing the data 

All patients who meet the characteristics described in the clinical study’s inclusion criteria can participate. 
It is the referring oncologist who evaluates the eligibility criteria for the individual patient and proposes participation in the study. 
It is important to note that eligibility criteria are not intended to discriminate, but rather to identify the most suitable individuals for conducting the research, ensuring scientific rigor and protecting the participants' health. 

Ethics Committee 
The Ethics Committee is the regulatory body established by law to protect patients participating in clinical studies. It is composed of doctors from various specialties, nurses, religious figures, legal experts, representatives from patient associations, and other experts. Its primary role is to assess whether the study is in the patient's best interest, including evaluating its scientific relevance and rigor. 
No clinical study can be started unless the corresponding protocol has been approved by the ethics committee. 
For more information regarding the Ethics Committee of our foundation, you can consult the dedicated section on our website. 

Informed Consent 
Informed consent pertains to any medical act, except in emergency situations, and represents the patient's authorization to receive medical treatment after being fully informed by healthcare personnel. 
Informed consent is therefore necessary for participation in a clinical study. 
The patient’s voluntary agreement requires that the doctor clearly and comprehensively informs them about the objective and procedures of the study, potential risks and benefits, and the patient's ability to freely decline participation or withdraw at any time. 

Confidentiality of Data 
Researchers are required to maintain the confidentiality of participants’ identity and medical information. 

Each patient should have the opportunity to consider whether a clinical study is appropriate for their condition. 
The decision to participate or not starts with two key pieces of information: the diagnosis and prognosis based on the standard treatment. The choice is then made based on the information the doctor provides about the clinical study and its potential effects. 
Since prognosis provides only statistical information that does not take into account the specificities of each individual patient, and because clinical studies address questions that have not yet been answered, it is not easy to predict in advance whether a patient should participate in a clinical trial. 
Only the final data of the clinical study will show whether the new treatment is superior to previous treatments. 
In any case, the patient always has the right to make the choice in full freedom, guided by the explanations provided by their doctor. 

Regarding costs, it is the responsibility of those in charge of the trial to ensure that all exams and visits required by the protocol are either free of charge or covered by a very minimal fee. 
The patient can decide not to participate in the clinical study, and the treatment offered instead of the experimental one must be the best available at the time. 
The patient can choose to withdraw from the study at any time, and even in this case, they will receive the best alternative treatment available. Similarly, the doctor can decide to halt the clinical trial if it is determined that the experimental therapy is not benefiting the patient, or if a more effective treatment is identified during the course of the study. 

When a study is proposed, different phases may be described: 
• Phase I: Focuses on defining the safety of a new drug that has reached the clinic and determining the best way to administer it. 
• Phase II: Clarifies the activity of a drug in a specific group of patients. 
• Phase III: Determines whether a new treatment is superior to the standard of care. 
• Phase IV: Conducted once the drug is already available in pharmacies; it involves long-term surveillance studies regarding the outcomes of treatments over time or the evaluation of rare toxicities. 

Experimental Studies: These are studies in which a drug, technique, or equipment still experimental (i.e., not yet accepted in clinical practice) is used. 
Observational Studies: These studies are useful for description and analysis. For example, they help assess the relationship between new treatment targets and prognosis, the outcomes of treatments, or simply the presence or absence of a biological target in a specific group of patients. In these studies, the patient’s involvement is typically minimal, but they are equally important for the information they provide. 

A study is randomized when participants are assigned to two groups randomly: the experimental group (with new drugs, techniques, instruments, etc.) and the observation/control group (standard treatment, which based on previous clinical studies has already shown to achieve the best results). 
The purpose of random assignment is to ensure the comparability of the treatment groups with respect to other potentially confounding factors, so that any differences in outcomes can be attributed solely to the effect of the treatment.