Clinical studies represent an additional therapeutic opportunity for the patient as, in addition to standard treatments, they allow the patient to receive drugs that are not yet registered, and therefore, not yet used in everyday clinical practice. Clinical studies are also the only reliable and safe tool, able to establish the efficacy and safety of a new compound.

Clinical studies

To ensure the safety of the patients taking part in the study, a research group made up of doctors, specialized nurses, computer scientists and statisticians draw up a protocol, in which they define the criteria for inclusion and exclusion of patients who can take part in the study (chosen to guarantee the patients’ safety), information on the drug to be tested and the objectives of the study. The protocol is therefore approved by the Ethics Committees of the Hospitals that take part in the experimentation and which must, in fact, assess whether the study is ethical or not for the patient.

The patient receives all the information regarding the study , which includes, what the new drug being tested is, the reasons why the study was designed, and the possible risks or side effects. All this information is collected in a form, called informed consent, that the patient, must complete and sign if willing to take part in the study. The patient has the right to withdraw from the study at any time, without having to provide any explanation.

Registration process for a new drug

The process that leads to the registration of a new drug is very rigorous and long.

Before entering the clinical phase, a compound, created in the laboratory, is tested in vitro (i.e. on tumor cells, which have been isolated from human tumors and grown in the laboratory) and in vivo (i.e. on animals). This is a very important phase, as it allows us to study the mechanism of action of the new drug and its metabolism (i.e. how it is eliminated from the body and in which organs it accumulates in, to hypothesize its side effects).

When the compound, synthesized in the laboratory, passes the preclinical phase, it enters the clinical phase.

The steps that a drug must pass in the clinical phase to be registered are:

Phase I

It aims to define the dose to be used in humans. We start by testing the lowest dose (chosen on the basis of the data obtained in studies conducted in animals) in a few patients (usually three). Patients are not enrolled at the same time, but one at a time, and wait at least 20-30 days before the next patient is enrolled. This allows us to detect the possible appearance of side effects and, therefore, it is an additional safety measure for patients.

If with the tested dose there are no adverse effects, the dose is increased, until the maximum tolerated dose is determined in humans and, therefore, the dose to be used is chosen in the subsequent clinical development.

Phase II

It aims to evaluate the effectiveness of the new treatment and to further confirm safety.

Phase III

It aims to demonstrate that the new treatment is better than the standard one. If, at the end of clinical development, evidence of efficacy and an acceptable safety profile are found, the marketing of the new treatment to the competent authority may be required. In Europe, this is the European Medicines Agency (EMA). Each European state has its own regulatory agency which, for Italy, is represented by AIFA, the Italian Drug Agency.

If you are interested in participating in a clinical trial, ask your oncologist to check the currently available protocols for treating your illness by connecting to the sites:
LUCE Network