Clinical trial design and management

Article type: Research Article

Authors: Baiardi, Paola | Santilli, Veronica

Affiliations: Consorzio per Valutazioni Biologiche e Farmacologiche (CVBF), Pavia, Italy | Division of Immunology and Infectious Disease, "Bambino Gesu" Children's Hospital, Rome, Italy

Note: [] Corresponding author. E-mail: pbaiardi@cvbf.net

Abstract: The randomised, controlled trial (RTC) is considered the gold standard for the assessment of the effect of any therapeutic intervention. However, rare diseases carry several issues in terms of population size, life-threatening nature of the condition and lack of other available treatments, which basically render this approach not always feasible. Alternative methodological approaches are being developed to better suit the need of an orphan drug development.

Keywords: Randomised, controlled trial, population size, rare disorder

DOI: 10.3233/PPL-2010-0267

Journal: Pharmaceuticals, Policy and Law, vol. 12, no. 1-2, pp. 57-62, 2010