A drug which is effective in the treatment of cancer.

In a controlled clinical trial, the patients are placed into groups (see Randomization) called arms, which are compared to one another. The patients in one group all receive the same intervention. Different scenarios develop, depending on the health conditions under study and the aim of the study: each group might receive a different intervention or one group might receive the intervention and the other does not. The researchers evaluate the effects of such interventions on biomedical or health-related outcomes separately in each group and compare the effects among the groups (see Control group).

The storage of human biological samples, such as blood, urine, tissue.

Any data that provide the characterization of the biological, genetic, biochemical and/or physiological properties, compositions, or activities of analysed biological materials.

Is a parameter of a biological state or condition that can be useful to evaluate the health status of a person, select the appropriate intervention or monitor the effect of a treatment. Examples of biomarkers used for health monitoring are platelets, neutrophils and lymphocytes. Examples of biomarkers used for cancer diagnosis or during follow-ups are: CA-125 (ovarian cancer), CEA (colorectal cancer), PSA (prostate cancer), BRCA1/BRCA2 (breast and ovarian cancer), HER2 (breast cancer), etc

A study in which the patients do not know if they are receiving the intervention studied or the control. In a double blind clinical trial the researchers themselves do not know what intervention the patient is receiving. Blindness can be broken if the patient’s condition requires it.

A disease in which abnormal cells undergo an uncontrolled division and can invade neighbouring structures (tissues, blood). Cancer is a generic name for a large group of diseases that can affect any part of the body.

The basic microscopic unit of all living organisms. A cell is surrounded by a membrane and has several small structures inside it, among which its nucleus.

The repository of genetic information (such as DNA) and the control center of the cell. It is present both in animal and plant cells.

A pharmacological anticancer treatment aimed at stopping the growth of cancer cells, either by killing the cells or by stopping them from cell division.

A threadlike structure made of protein and a single molecule of DNA present in the cell nucleus. Chromosomes carry information of the genome. The human species has 23 chromosome pairs, designated 1 to 22, plus sex chromosomes (X and Y for the female and male, respectively).

A study involving human volunteers (also called participants) which is intended to add medical knowledge and improve healthcare. There are two main types of clinical studies: clinical trials (also called interventional studies) and observational studies.

A study in which participants receive specific interventions according to the research plan or protocol designed by the researchers. These interventions may be medical products (such as drugs), radiation, the use of other medical devices or procedures or lifestyle interventions, such as diets. The researchers use clinical trials to study the safety and efficacy of interventions by measuring specific outcomes (such as adverse events for safety and benefits for efficacy) in the participants.

In a controlled clinical trial, this is the group that does not receive the intervention under study and may receive either a different intervention, or a placebo (see Placebo), or no intervention at all.

A clinical trial in which the intervention under study is compared to the control group (see Control group).

A process leading to the identification of an illness or problem (for example presence of cancer, cancer type and characteristics, etc.). It is made after examining signs and symptoms in patients.

An extremely long molecule which is the main component of chromosomes. DNA is the material that transfers genetic characteristics in all life forms. DNA consists of two nucleotide strands coiled around each other in a ladder like arrangement.

The capacity of producing a desired result or effect.

Traditionally defined as a state of genuine uncertainty on the value of the different interventions under consideration. It is required when deciding whether to conduct a new clinical trial to study an intervention. Indeed, the ethics of clinical research requires equipoise.

Is a multidisciplinary group of experts (scientists, doctors, nurses, and other professionals) and lay people, who evaluate the ethical and scientific quality of clinical studies involving human subjects, in accordance with national and international law. Clinical studies have to be approved by an Ethics Committee before they can begin. The members of Ethics Committees have no link with the research team applying for approval and thus have no conflict of interest about the approval.

See Ethics Committee.

A portion of DNA that determines the expression of a characteristic.

Information about specific genes, including variations and gene expression, found in an individual or in a certain type of tissue through genetic testing (see genetic testing). A genetic profile may be used to diagnose a disease, to gather information  on the risk of developing cancer, to choose the most appropriate cancer treatment for a patient or to learn how the disease may progress or respond to treatment with drugs or radiation.

A test examining the DNA and looking for changes in it, sometimes called mutations or variants. Genetic testing is useful in many areas of medicine.

The study of the microscopic structure of tissues

Observations, results or other findings that may occur during an exam or data analysis but are not directly related to the goals of the exam. The patients can declare in advance whether they want to be informed of these results if risky or silent conditions are identified.

A document describing the details of a clinical study (aim, intervention under study, duration…) that the clinician gives a person who is invited to participate in the study. The information sheet has several purposes, including that of helping to ensure that the patient receives appropriate and complete information, in order to decide whether to take part in a study; it is also a “take home” reminder of the elements of the clinical investigation, providing contact information, in case additional questions or concerns arise.

The legal document a person has to sign to express consent to participate in any clinical study (either observational or interventional).

The process by which potential participants in a study receive the information needed to decide whether or not to participate. As a first step, a potential participant meets one of the researchers, usually a doctor, who explains, in a one-to-one talk, the details of the study (aims, process, interventions, procedures), and the possible benefits and risks associated with participation. After this discussion, the doctor provides the same details in a written form (see Information sheet). If the patient decides to participate in the study, she/he has to sign the informed consent form.

In the field of medicine it is related to any kind of treatment, procedure, or other action taken to prevent or treat disease, or improve health in other ways.

See Clinical trials

A change in the structure of a gene, resulting in a variant structure that might be transmitted to subsequent generations. It is caused by the alteration, deletion, or insertion of the DNA.

See Observational study.

See Observational study.

A study in which researchers observe the “natural history” of  a patient’s disease according to routine clinical practice (standard of care), including diagnostic procedures and therapeutic interventions. Differently from interventional studies, the standard of treatment provided to the patient is not modified during participation in the study. Data on standard therapy and its outcomes, as well as information on other patients and on the disease, are collected during the study to evaluate the association between outcomes and treatment and patient-related characteristics. This kind of study is also called non-interventional or non-experimental.

A specific result or effect of an intervention that can be measured or ascertained. Examples of outcomes include survival, quality of life, side effects of treatments, pain intensity, and tumor size.

The appearance of an organism resulting from the interaction of the genotype and the environment.

An inactive substance, a tablet or a capsule, for example, that does not contain an active drug ingredient. For example, placebo pills may contain sugar. Placebos can be used in clinical trials (see Clinical trial) as control intervention when there is no standard treatment for the disease. In blinded clinical trials (see Blind study), placebos must look like the active treatment under study, so that participants (and researchers) can’t discern them from the active treatment.

A doctor’s judgment of the likely or expected development of a disease or of the chances of recovering.

Large complex molecules that play many critical roles in the body, such as structure, function and regulation of tissues and organs.

A document that lays out the research plan before conducting the study. The protocol is designed to describe the study methods and procedures, potentially allowing for its replication. The protocol should clearly detail the following information: the reason for conducting the study, the definition of the target population (the eligibility criteria), the number of participants to be included, the description of the interventions being studied, the schedule of tests and diagnostic procedures, drugs and their dosages, the length of the study and the participants’ data. Sometimes, the protocol is prepared by the researchers with the collaboration of patients or their representatives. Each clinical study protocol has to be registered in ad hoc institutional databases (USA www.clinicaltrials.gov , EU www.clinicaltrialsregister.eu/ ).

The process by which participants in clinical trials are assigned by chance (randomly) to separate groups (see Arm), which will receive different interventions. Neither the researcher nor the participant can choose the intervention assigned to the group. By randomly assigning the participants to the intervention groups, the comparison between the intervention effects are fairer, because the groups are similar, according to the characteristics of the patients or of the disease – both known and unknown. The only difference consists in the administered interventions. Therefore, at the end of the clinical trial, any difference which should emerge in terms of efficacy and toxicity, in favour of one particular intervention, can be attributed exclusively to that particular intervention and not to pre-existing differences among the groups.

This is a controlled clinical study in which the participants are divided by chance (randomly – see Randomization) into separate groups, which receive the different interventions to be compared (see Control group). Randomized trials are based on the equipoise assumption (see Equipoise). Randomized clinical trials can compare the effects of drugs, surgical techniques, medical devices, diagnostic procedures or any other medical/clinical intervention.

A molecule that has structural similarities to DNA. RNA is a single-stranded molecule. It is involved in processes of coding and decoding the regulation and expression of genes.

A test carried out to detect potential health disorders or diseases in people who do not have any disease symptoms. The goal of this could be the early detection of a disease and its most effective treatment, or monitoring, in order to reduce the risk of the insurgence of the disease. Three examples for cancer screening tests involve breast, colorectal and uterine cancer.

An undesirable effect of a drug or medical intervention. Some common side effects of cancer treatments are nausea, vomiting, weakness, decreased blood cell counts, hair loss, and mouth sores.

A clinical study in which every participant receives the same intervention.

A type of molecular drug that blocks the growth of cancer cells by interfering with specific targeted molecules involved in tumor growth rather than by simply interfering with all the rapidly dividing cells (e.g. traditional chemotherapy).

See Target therapy.

See Cancer.