Clinical trials

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:

1. Clinical and biological tolerance: determining the maximum dose tolerated by the body.
2. Pharmacodynamics: defining the effects on the body and determining the minimum dose with therapeutic activity.
3. 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.