Intractable Rare Dis Res. 2017;6(1):29-34. (DOI: 10.5582/irdr.2016.01094)

From promising molecules to orphan drugs: Early clinical drug development.

Dooms M


Phase-1 (also known as "First-in-Man") clinical trials initiate the early clinical development of possible new medicines. Patient participation in this early phase of clinical trials is 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% of the orphan drug applications were authorized. 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 (EMA), the original rare indication was extended to another rare disease and no phase 1 trials were needed. Phase 1 studies need to be performed in a sufficient number of volunteers even for medicinal products intended for a very limited number of patients.

KEYWORDS: Rare diseases, orphan drugs, exploratory clinical trial, phase-1, first-in-man

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