Journal of Genetic Syndromes & Gene Therapy
Open Access

From promising molecules to orphan drugs: Early clinical drug development

4^th World Congress on Rare Diseases and Orphan Drugs

June 11-12, 2018 | Dublin, Ireland

Marc Dooms

Center for Clinical Pharmacology, Belgium

Posters & Accepted Abstracts: J Genet Syndr Gene Ther

Abstract :

Phase-1 (also known as â�?�?First-in-Manâ�?) clinical trials initiate the early clinical development of possible new medicines. By raising the dose of the investigational compound in healthy volunteers pharmacokinetic and â�?�?dynamic parameters are recorded alongside the safety profile of the new substance in humans. Patient participation in this early phase of clinical trials will be rather limited. After successful phase -1 trials, further phase -2 and phase -3 clinical trials in patients may lead to a marketing authorization. In the first 15 years of the European Union Orphan Drug Directive 4-5- percent of the orphan drug applications were authorizated. However, for many of these orphan drugs no phase -1 studies were required as these products were already well known pharmaceutical substances with a clearly defined pharmacological profile. Furthermore, for 19 orphan drugs, already authorized by the European Medicines Agency, the original rare indication was extended to another rare disease and no phase -1 trials were needed. For all the other orphan drugs clinical development started with regular phase -1 studies in human volunteers. dooms@evonet.be