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What is a clinical trial ?
Tools indispensable to the setting up of trialsTo further the development of innovative therapies, AFM supports a number of key teams and organisations such as centres for the production of vectors (Généthon, Atlantic Bio GMP) and cells for clinical use, clinical investigation centres to host trials for rare diseases, gene and cell therapy units, DNA, cell and tissue banks for research, veterinary schools for pre-clinical studies, etc.
AFM has supported the development of another invaluable tool for clinical trials for a number of years : disease-linked databases, combining clinical and genetic information. These include the UMD-DMD (Universal mutation database - Duchenne muscular dystrophy) database, which contains detailed clinical data concerning almost 2,500 patients and provides a broad overview of the disease.
From molecule to drugThe term "drug" includes standard molecules produced by chemical synthesis or extracted from biological organisms and, more recently, products of cell and gene therapy. The origins of drugs evaluated in trials supported by AFM are varied. Some molecules are already available to the medical profession and are marketed to treat patients affected by a particular pathology. Additional work may subsequently suggest value for a neuromuscular disease. In other cases it is an interesting and novel molecule, i.e. it has never received market authorization. Here again, preclinical work may suggest a potential for the treatment of neuromuscular diseases. The research program will be longer, as it will have to go through all the development steps. In general, 7 to 12 years are required for a new molecule to become a marketable drug. This process involves a succession of steps which aim to assess patient safety and product efficacy.
Gene therapy products are pharmaceutical products which belong to a new category. They are also assessed according to criteria of quality, safety and efficacy. Just as " traditional " drugs, they go through stages in product development which is specific, however, to a vector carrying a " gene-drug " and not a chemical molecule. Pre-clinical studies, especially toxicologic studies, are particularly important and also constitute a vital precondition for clinical trials in humans. In parallel, it is necessary to develop production and control methods for vectors, usually of viral origin.
Stages in clinical trialsThe development of new therapies proceeds through several regulatory stages. The longest stage - the pre-clinical stage - lasts several years and costs are high.
Phase I
Number of people involved : between 10 and 100 people.
Goal : a small number of patients, or healthy volunteers, participate in these trials. The objective is to assess drug tolerance, body assimilation and identification of possible adverse effects.
Length of time: several weeks to several months.
Phase II
Number of people involved : a few dozen people.
Goal : this stage aims to investigate the efficacy of a drug and to define the smallest possible efficacious dose.
Length of time : several months to several years.
Phase III
Number of people involved : 300 to 30,000 people.
Goal : these trials, involving an important number of patients, are comparative trials : they help evalutate benefits vs. risks of a product. The new treatment is compared either to a placebo or to a reference drug. After this stage, the new product may receive market authorisation (MA).
Length of time : several years. |
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