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What is a clinical trial ?
Tools indispensable to the setting up of trialsAs they play a part during the course of clinical trials, the AFM promotes the development of tools necessary for the evaluation of these trials.
For example, databases which gather clinical and molecular data should enable researchers to identify eligibility between diseases and therapies and define the criteria to take into account when judging the effectiveness of a therapeutic product.
Thus, the Institute of Myology collaborates on databases which include information linked to the diagnosis and follow-up of patients affected with myasthenia, channelopathies, laminopathies, dysferlerlinopathies and sarcoglycanopathies. Moreover, the AFM finances the databases of the Cochin Institute and the Institute of Human Genetics at Montpellier. In 2005 the AFM took part in the first meeting of the French curators of mutation databanks.
Moreover, the AFM strongly supports those organisations carrying out preclinical work, a preliminary but essential step before trials in humans. For example, the national veterinary schools of Nantes and Maisons-Alfort house animal models of different rare diseases – myopathies, mucoplysaccharidoses, retinitis etc. 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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