Glossary

Basic research aims to gain new knowledge by observing, testing, and analyzing phenomena in a laboratory setting to better understand living systems. Although it does not directly seek to cure diseases, basic research is essential because it forms the foundation of clinical research. Many medical breakthroughs have resulted from discoveries in basic research. These discoveries pave the way for the development of treatments, technologies, diagnostic methods, and improvements in care.

A biomarker, or biological marker, is a measurable characteristic that provides information about a person’s health. Biomarkers can be used to detect a medical condition, monitor its progression, or evaluate the effects of a treatment. For instance, blood sugar levels are biomarkers used to monitor diabetes.  A genetic marker is a type of biomarker that corresponds to a specific DNA characteristic (e.g., a mutation). It can be used to identify a predisposition to a disease, to characterize a medical condition, or to better tailor certain treatments. 

The care trajectory refers to the sequence of healthcare services a person receives for diagnosis, treatment, and/or monitoring of a health condition. It may include multiple treatments and care services at different times and locations.

A caregiver is a person who provides ongoing or occasional support to one or more loved ones. The loved one may have a temporary or permanent physical, psychological, or other disability, and shares an emotional connection—whether or not they are related by family—with the caregiver.

Cell therapy involves administering or transplanting cells to replace or eliminate abnormal cells responsible for a medical condition or symptom. A stem cell transplant for an individual with multiple myeloma is an example of cell therapy.

Clinical research seeks, through clinical trials, to show that the possible new treatments are effective and safe for humans. It is based on previously obtained laboratory results (during the stages of basic research and preclinical research). Depending on the study treatment in question, clinical trials are carried out with participants who are healthy or who have a specific medical condition.

A cohort is a group of participants who share a medical condition and/or certain characteristics (such as an age group or a risk factor) and who receive the same health product as part of a clinical trial.  A single clinical trial may include several cohorts, for example: 

• One cohort receiving the investigational health product.
• One cohort receiving a placebo or standard treatment.

By comparing results across these groups, the research team can evaluate the safety and effectiveness of the health product being tested. 

A consent form is a document provided to individuals prior to their participation in a study. It clearly explains the purpose of the research, what participation involves, any potential risks or discomforts, the expected benefits, and the rights of the participants. The form also specifies how collected data or samples will be used and whom to contact with questions.  The form ensures that each person makes a free and informed decision with a clear understanding of the information provided. The form supplements discussions with the research team but does not replace them. 

A diagnostic tool helps medical personnel identify medical conditions. A diagnostic tool can be a questionnaire, a medical device, a software, etc.

In a clinical trial, efficacy refers to a health product’s ability to provide the anticipated benefit, such as relieving a symptom, treating a disease, or slowing its progression, to a group of patients most likely to benefit from the treatment.

Eligibility criteria are characteristics that determine whether a person can participate in a clinical trial. These may include factors such as age, type, and stage of the medical condition, or previous treatments. Eligibility criteria include:

• Inclusion criteria: characteristics that individuals must have in order to participate in the trial.
• Exclusion criteria: characteristics that individuals must not have to be included in the trial.

An experimental treatment (or a study treatment) is a treatment that has not yet been tested to determine its effectiveness and safety. All drugs, medical devices, and other treatments must be evaluated in clinical trials before they are made available to the general public.

Gene therapy involves inserting, modifying, or removing one or more genes in a patient's DNA to treat or prevent a medical condition. Depending on the strategy, the cells can be modified in vivo (directly in the patient's body) or ex vivo (by collecting the cells, modifying them in a laboratory, and then reinjecting them into the patient).

Good Clinical Practices (GCP) are a set of national and international rules and principles that govern how clinical trials involving humans must be planned, conducted, and monitored. The goal of these practices is to protect the safety, rights, and well-being of clinical trial participants while ensuring the quality and reliability of the obtained data and results.  These rules are recognized in Canada and in many other countries. They are enforced through inspections and regulatory oversight to ensure that clinical trials are carried out ethically, safely, and with scientific rigor. 

Health Canada is the federal department responsible for protecting and improving the health of Canadians. The department regulates many health-related products, including food, cosmetics, health products, and pesticides. For health products, such as drugs, vaccines, natural health products, and medical devices, Health Canada ensures they are effective, safe, and high-quality. The department enforces laws and regulations, inspects research teams, evaluates products before they reach the market, monitors their safety after approval, and takes action if a problem arises. 

Health innovations involves designing, testing, and implementing new approaches to improve care, services, or the organization of the healthcare system. It may take the form of a new tool, a change in practice, or a solution tailored to a specific need.

A health product is any medical substance, biological product, or device used to prevent, diagnose, treat, or alleviate a health problem or improve well-being. This includes drugs, vaccines, and medical devices, such as thermometers, insulin pumps, and bandages. 

In vitro studies are experiments performed outside of the human or animal body in a controlled environment, such as a test tube or culture dish. These studies are used to examine biological mechanisms or the effects of molecules or medical compounds on human or animal cells, viruses, or bacteria in a laboratory setting. 

In vivo studies are conducted on humans or laboratory animals. These studies allow researchers to observe how a treatment works in a living organism and consider the interaction between organs and systems. 

The Institut national d’excellence en santé et en services sociaux (INESSS) is a public organization in Quebec that strives to enhance the quality of healthcare and the efficiency of social services. The organization evaluates the results of clinical trials of drugs, medical devices, and other health technologies to determine if they provide substantial health benefits and good economic value. Based on these evaluations, the INESSS advises the Minister of Health on which products should be covered by the public drug insurance plan and under what conditions.  INESSS also develops guides, recommendations, and tools to help health and social service professionals adopt best practices for the benefit of the population. 

Informed consent is a person's agreement to participate in a study after receiving and understanding all the important information. This information must be complete, accurate, and free of jargon to avoid confusion. It should cover the purpose of the research, its risks, potential benefits, and the rights of participants. 

Interventional studies involve testing health innovations, such as new drugs, vaccines, medical devices, or procedures, on humans. These studies evaluate the innovation's efficacy, safety, and potential adverse effects. These studies take place in several phases, each with a specific goal. If the results are favorable, the treatment may be approved by health authorities and made available to the public. 

Studies on laboratory animals are essential for a better understanding of the overall effect of a treatment on a living organism. Even if a treatment is designed to act on a specific part of the body or a particular biological system (such as the circulatory, respiratory, or nervous system), it may affect other parts of the body or other biological systems. Since some animals have biological systems similar to those of humans, particularly in terms of vital functions such as breathing, digestion, movement, and reproduction, testing a promising treatment in animals can therefore confirm its effects and safety before testing it in humans. As with clinical trials, studies on animals are highly regulated and require ethics board approval. In Canada, the Canadian Council on Animal Care (CCAC) plays an important role in the scientific community. It creates and establishes ethical practices for the use of animals in research, ensuring rigorous and valid scientific results.

The life sciences and health technologies (LSHT) sector includes all medical research and advances, as well as the development of health treatments, and products. The LSHT sector also includes all diagnoses, medical devices, prosthetics, and products designed to treat or assist people with a medical condition.

A market authorization allows the manufacturer of a health product to make it available to the public. This authorization is issued by Health Canada when a manufacturer presents strong scientific evidence of the safety, efficacy, and quality of the health product. After receiving market authorization, the manufacturer must continue to monitor the health product’s effects and provide updates on its safety and efficacy to Health Canada at set times.

Observational studies involve observing and collecting information about people, for example by analyzing health data or behaviors. These studies can help researchers better understand medical conditions and develop ideas about their causes or risk factors. No new treatment is administered and no intervention is carried out during an observational study. 

The patient experience describes the entire experience of an individual with a medical condition: their symptoms, quality of life, use of the healthcare system, and more. The patient experience is personal and may differ from one person to another, and even between individuals with the same medical condition.

Pharmacoeconomic value is an estimate of the economic benefit of a health product (for example, the high cost of a health product is compared to the cost associated with not having that product). The purpose of estimating pharmacoeconomic value is to justify coverage of a health product by Régie de l’assurance maladie du Québec (RAMQ).

A placebo is a substance that does not contain any drugs but has the same appearance as a study treatment (same shape, size, color, and taste if it is an oral treatment) and is administered the same way (orally, by injection, etc.). For some people, simply receiving a placebo treatment improves their medical condition. This is known as the “placebo effect”. In a clinical trial, the use of a placebo in one of the groups allows researchers to evaluate the actual efficacy of the study treatment. This use must be approved by a research ethics board. Participants are informed when a placebo is used, but they usually do not know whether they are receiving the placebo or the study treatment.

Preclinical research is based on the knowledge gathered during basic research. It is conducted by academic or pharmaceutical centers, generally using animal or human cells (in vitro) and laboratory animals (in vivo). Once preclinical research confirms that a health product has promising and safe effects in animals, and if the necessary approvals are obtained, clinical research can begin with a first clinical trial on humans.

Research ethics boards are independent bodies that ensure respect for the dignity, safety, well-being, and rights of participants in clinical trials. Ethics boards approve and monitor clinical trials closely, and may suspend or cancel all or part of a trial at any time. In Québec, research ethics committees may collaborate with ministries, academic, or health institutions such as university faculties, integrated health and social services centres (CISSS), integrated university health and social services centres (CIUSSS), or university hospital centres (CHU). They are composed of members with diverse areas of expertise.

The research protocol gathers all the information related to the conduct of the clinical trial. It explains why the trial is being conducted (background, hypothesis, and objectives), how it will be conducted, and how the safety of the participants will be ensured.

Risk factors are individual and environmental characteristics that increase the likelihood of a person developing a medical condition. The presence of risk factors does not mean that a person will necessarily develop this medical condition. For example, people with a mutation in the BRCA2 gene will not necessarily develop breast cancer, even if their risk is higher than average.

Safety refers to the absence of toxicity or major health risks posed by a health product. Safety is assessed by identifying and measuring side effects or adverse events to ensure the benefits of a treatment outweigh the risks. After a health product is marketed, its safety continues to be monitored to detect and address any potential issues. 

A side effect is a reaction to a health product that differs from the intended effect. It can be expected or unexpected, mild or severe, temporary or permanent, harmful, neutral, or positive. An adverse effect or an adverse side effect is a harmful and unwanted side effect resulting from the intake of a health product. The onset of side effects varies from person to person and from treatment to treatment. It is important to ask medical staff about the possible side effects of a treatment.

The sponsor takes ownership of and responsibility for all stages of a clinical trial. In most cases, the sponsor also funds the clinical trial. The sponsor may be a pharmaceutical or biotechnology company, a university, a healthcare institution, a volunteer group, a private organization, a government health agency, a physician, or a member of the medical staff.

A standard treatment is a therapy that has been approved for market use, has proven efficacy and safety, and is commonly used to treat a specific medical condition. In some clinical trials, an experimental treatment is compared to a standard treatment to evaluate its effectiveness and safety.

A health product is used to treat, prevent, or diagnose a medical condition or symptom. Its therapeutic indication shows how it should be used and the population for which it is intended. For example, ibuprofen is commonly indicated to reduce fever, relieve pain, or decrease inflammation. This information is provided for illustrative purposes only and does not replace advice from a healthcare professional.

Therapeutic value is an estimate of the benefit a health product can have for people with a medical condition or symptom. In Quebec, the Institut national d’excellence en santé et en services sociaux (INESSS) assesses therapeutic value based on two criteria:

• The presence and significance of a health need to be met.
• The health product’s ability to benefit the public.