Drug repositioning: from the discovery of a useful pharmacological effect to making the treatment available to the patient

THERAPIE(2023)

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摘要
The repositioning on a new indication of a drug already on the market can be an opportunity to respond quickly to a therapeutic need not yet covered, in particular in the context of rare or neglected diseases, or even a health emergency. However, at each step, difficulties may arise that will prevent the repositioned drug from being available. Beyond chance or a systematic strategy for detecting a useful pharmacological effect, the implementation of preclinical and clinical stages is sometimes made difficult by the difficulty of access to the molecule and its pharmaceutical file. Furthermore, relevant clinical results will not always be sufficient to guarantee obtaining a marketing authorization or satisfactory treatment methods for patients. In addition to the description of these various brakes, the round table was the occasion to propose recommendations able to remove them, in particular the creation of a structure in public-private partnership, endowed with sufficient financing in order to be able to propose an individualized accompaniment projects up to and including the marketing request.
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关键词
Repositioning, Marketing authorization, Rare diseases, Assistive device, Therapeutic need
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