Experimental treatment |What is good to know

What is a experimental treatment?

It is a test in which the effectiveness and risks of any intervention are verified. Anything that can change the course of events: e.g. a surgery, a medicine, an examination, an advice, a brochure. doctor, including therapies, are shown to cause a specific effect in an experimental condition. The adjective clinical indicates that the trial is performed on the man, sick or healthy. The clinical trial is usually preceded by a pre-clinical phase, using in vitro or in vivo models. In vitro testing is conducted on cell cultures, in vivo testing on laboratory animals.

What does experimental therapy mean?

This is a new treatment on which there is not enough scientific data to demonstrate its effectiveness and risks.

Who is it conducted on?

In order to obtain reliable data, a clinical trial must be carried out on a group of individuals with similar characteristics. Usually among the parameters are considered age (children, adults, elderly), sex (from some experiments are excluded women for possible risks on the fetus), diagnosis, phase of disease, the presence of other diseases. The inclusion criteria list the parameters that allow you to participate in the study, while the exclusion criteriaFeatures that must be present for a subject to be involved in a study. The inclusion criteria generally refer to gender, age, diagnosis and severity of the clinical condition. On the other hand, the exclusion criteria are the parameters that prevent their participation.

What happens during a clinical trial?

A clinical trial consists of several phases that are different depending on the type of trial in question. The study protocol describes both the type of study and the phases in detail.

What types of clinical trials are there?

The aims of a clinical trial can be multiple: the effectiveness and side effects of a therapy, the usefulness of a new examination in diagnosing a disease or a certain measure in preventing its onset (for example, refraining from smoking) can be verified.  In clinical therapy studies, the effectiveness of a new drug, a new therapy, a surgical or radiotherapy approach is tested. Diagnostic trials evaluate the usefulness of a sensitivity testProbability of a positive outcome of a diagnostic test if the patient is suffering from a disease. Together with specificity, it measures the discriminatory capacity of a diagnostic test. A very sensitive test will be positive in a very high number of cases where the disease is actually present; a very sensitive test will be positive in a very low number of cases where the disease is actually present.

The sensitivity of a test is related to its ability to detect a change (from disease not present to disease present). (ability to demonstrate the presence of a certain disease) and specificityProbability that a diagnostic test will fail if the patient is NOT suffering from a disease. Together with sensitivity, it measures the discriminatory capacity of a diagnostic test. A very specific test will be negative in a very high number of cases where the disease is not actually present; a very specific test will be negative in a very low number of cases where the disease is not actually present. (ability to detect a disease only when it is actually present).  Screening studies assess the ability of an examination to determine a disease before it causes symptoms.  In quality of life trials, the impact of the disease on the daily life of the patient and his or her mental sphere is assessed.

What are the benefits of participating in a clinical trial?

It is a way of taking care of one’s own health; it allows access to new therapies before they are accessible to many; it allows care to be taken by experts in the field and active participation in the progress of scientific research.

What are the risks of participating in a clinical trial?

The proposed treatment can lead to serious unpleasant side effects, which can also put the patient’s life at risk. Participation in a study often takes longer than traditional treatment, requires more examinations and more time in hospital.

What does “screening” mean?

Screening or early diagnosis involves undergoing tests to detect a particular disease before it causes symptoms. For some of these diseases there is no possibility of treatment and recovery. For this reason, the doctor must inform the patient about the advantages and disadvantages of early diagnosis tests and he must decide whether he wants to know in advance that he has a disease that can be diagnosed, but not always treated.

Can you stop participating in a clinical trial at any time?

At any time and for any reason, the patient may decide to stop participating in a trial.

What are side effects?

Side effects and adverse drug reactions are the unwanted consequences of medications, which can occur immediately after treatment or over long distances. The most common side effects are nausea, vomiting, headaches, skin rash.

What questions should I ask myself before participating in a clinical trial?

Before you decide to take part in a study, it is a good idea to read the documentation provided carefully and have your doctor explain what is not clear.

Some questions may facilitate dialogue with your doctor:

-What is the purpose of the study?
-Why do researchers believe that the proposed treatment will be useful?
-Has the treatment already been tested?
-If so, what were the results of the other studies?
-What are the side effects of the treatment?
-How long does the study take?
-Will I have to be hospitalised?
-Who finances the study?
-If the proposed treatment is effective, will it be provided to me free of charge even after the trial is over?
-Will I be reimbursed for any expenses incurred?

What does informed consent mean?

Informed consent is an act that is formally expressed through the signing of a document containing information on the proposed search. By signing it, the patient declares to have read and understood the information contained in it.

Who finances the clinical trials?

Clinical trials can be funded by foundations, public or private research institutes, hospitals, pharmaceutical companies, patient organizations.

What is the study protocol?

The study protocol is the research project that describes in detail the aims, the modalities of the experimentation, the criteria for inclusion and exclusion, the examinations to be carried out and their deadlines, the parameters on which the effectiveness will be evaluated and the statistical methods with which the results will be analyzed.

What does controlled clinical study mean?

It is a type of trial in which participants are divided into two groups: one receives medical intervention or medication, the other nothing or placebo. This type of design allows to compare the course of the disease in the two groups and verify the effectiveness of the treatment under examination.

What is placebo?

Placebo is an inactive, biologically inert treatment; it is administered as a pill, powder or liquid substance to patients belonging to the control groupIn a controlled clinical trial, a group of subjects who take the standard treatment or placebo instead of the experimental treatment. In a case-control study, a group that does not have the condition in the study. Serves to verify the possibility that the effects observed in the group receiving treatment are induced not by the active drug, but by the expectations of the patient.

What does bias mean?

It’s a systematic error in a study. It can be a selection bias, when, due to the incorrect division of the participants in the control group and in the active one, the control patients have a less serious form of disease, or a measurement bias, when the measurement parameters are not uniform among the patients studied.

What does analysis according to the principle of intention to treat mean?

It is a type of analysis of results in which not only those who have actually taken the treatment, but all those who have been assigned to that type of treatment are evaluated. Subjects may stop treatment early either because of side effects or because of the disease that may have taken a more severe course. Failure to take these results into account may lead to erroneous conclusions.

What does “quality of life” study mean?

In quality of life trials, the impact of medical intervention or therapy on the daily life of the patient and his or her mental sphere is assessed.