US regulatory framework for rare plasma protein diseases

Article type: Research Article

Authors: Weinstein, Mark^{; ;} | Jain, Nisha^{; ;}

Affiliations: US Food and Drug Administration, Rockville, MD, USA | Center for Biologics Evaluation, Research, Rockville, MD, USA | Office of Blood Research and Review, Rockville, MD, USA

Note: [] Corresponding author: Mark Weinstein, FDA/CBER/OBRR, 1401 Rockville Pike, Rockville, MD 20852, USA. E-mail: [email protected]

Abstract: FDA encourages the development of products that demonstrate promise for the treatment of rare plasma protein disorders. This is accomplished through a program of regulatory provisions, financial incentives, clinical trials designed to address small patient populations, and measures to increase the number of patients under study.

Keywords: Orphan drug act, rare plasma protein disorders

DOI: 10.3233/PPL-2009-0241

Journal: Pharmaceuticals, Policy and Law, vol. 11, no. 4, pp. 397-402, 2009