Glossaire

General expression which covers the packaging, outer packaging and leaflet of medicines.

Individual receiving healthcare.

Entities legally mandated for the financing and/or reimbursement of healthcare. This includes national, regional or local authorities responsible of assessment (e.g. HTA bodies) and decision-making for pricing and reimbursement of health technologies, public or private entities in charge of their reimbursement (national health system or private insurance).

Medicines for children under 18 years old. 

Personalised medicine, also known as precision medicine, is an approach to healthcare that tailors medical treatment and prevention strategies to the individual characteristics of each patient. This includes factors such as genetic makeup, lifestyle, and environmental influences. By considering these unique aspects, personalised medicine aims to optimise the effectiveness of treatments, minimise side effects, and improve patient outcomes.

Organised set of rules and activities designed to monitor the safety of an authorised medicine and detect any change to its risk-benefit balance. It includes detection, assessment, understanding and prevention of medicine related safety problems.

The phenotype refers to the observable characteristics or traits of an organism. These traits result from the interaction of its genetic makeup (genotype) with the environment. These characteristics can include physical features, such as height, eye color, and hair texture, as well as physiological traits, behavior, and disease susceptibility. The phenotype is the product of gene expression, where the genes within an organism's genome are expressed and interact with each other and the environment to produce a specific phenotype.

A plasmid is a small, often circular DNA molecule, typically found in bacteria. In gene therapy, the gene of interest is typically added into a plasmid, which is then delivered to the target cell. Several methods, such as non-viral vectors and electroporation are used to facilitate plasmid delivery.

See Personalised medicine.

Precision-Safety-Accuracy are the three main concerns when we talk about gene therapy. The ideal gene therapy solution must:

1. accurately produce the one and only planned modification
2. precisely modify the interested type of cells/tissues
3. be safe for patients

Any study conducted before a clinical study to provide the proof of concept of a treatment. This might involve studying a medicine, procedure or treatment in cell or animal models. A preclinical study is a sub-type of non-clinical study. A non-clinical study is a wider expression to refer to any study that is not clinical, and that addresses safety widely before the start of a clinical trial.

Prime editing is a genome editing technique that combines the precision of CRISPR-Cas9 with a modified Cas9 enzyme. This modified enzyme is fused to a reverse transcriptase to make highly accurate changes to DNA, by converting a genetic instruction from RNA to DNA and inserting that genetic information at a specific and programmable location in the genome. It allows scientists to insert, delete, or replace specific DNA sequences in the genome with minimal errors, offering great potential for treating genetic diseases.

An investigator who leads the investigators team conducting a clinical trial at a clinical trial site.