Clinical research refers to all studies conducted on humans (healthy or sick individuals). It primarily focuses on advancing knowledge of diseases, developing new treatments or medical devices, diagnostic methods to ensure better patient care. Highly regulated, it adheres to a specific study protocol and is only carried out under certain conditions:
Observational studies:
They aim to improve our understanding of the disease and its progression over time. These studies are conducted as part of patient follow-up in reference centers.
Interventional studies or clinical trials:
They provide scientific evidence of the effectiveness and safety of a new medication, a new medical device, or a new approach to disease management. This step is necessary for a new molecule to become a medication or for a new medical device to be commercialized.
In the context of developing new medications, clinical trials go through five stages:
Phase 1
First human administration
Phase 1 involves the initial administration of the molecule in humans. It is carried out on individuals who do not have the disease (healthy subjects). Its purpose is to study:
- Clinical and biological tolerance: determining the maximum dose tolerated by the body.
- Pharmacodynamics: defining the effects on the body and determining the minimum dose with therapeutic activity.
- Pharmacokinetics: determining how the molecule behaves in the body based on the mode of administration (oral, intravenous, etc.).
Phase 2
First administration in patients
Phase 2 involves the initial administration of the molecule to patients. This phase consists of two steps (phase 2a and phase 2b). It involves only a limited number of individuals and a short treatment period.
Phase 3
Therapeutic efficacy
Phase 3, also known as the “pivotal study,” is the final phase before the molecule is commercialized as a medication. It assesses the therapeutic efficacy of the molecule and its tolerance under conditions close to real-life use.
This step involves a large number of patients over an extended treatment period, with administration conditions resembling those of the future medication.
Marketing Authorization (AMM)
All data collected during the various phases (from phase 1 to phase 3) of a molecule’s development enable the submission of a Marketing Authorization request (AMM) to the French National Agency for Medicines and Health Products Safety (ANSM). When authorization is granted, the molecule is recognized as a medication and can be marketed.
Phase 4
Surveillance and evaluation of side effects
Phase 4 is a surveillance phase during which side effects are recorded and analyzed to assess patient safety. This long-term study of the medication (lasting several years) helps identify rare adverse effects or delayed complications.
