RESULTS FOR : 'Orphan drugs'
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Biotechnology-based ORPHAN DRUGS achievements and challenges. Fall 03

For many patients suffering from rare diseases there are simply no medicines available to treat their condition. With an estimated 20-30 million such patients in Europe alone, rare diseases collectively represent a significant unmet medical need. Individual disease groups, however, may have few sufferers worldwide. Historically, pharmaceutical companies had no incentive to develop drugs for such rare conditions because the chances of recovering the necessary investments from such small patient populations were very slim, especially for biotechnologybased medicines, which require specialised development and up scaling, as well as a large investment in manufacturing processes and a dedicated plant.