Chapter 4: Treating Rare Diseases: Business Model for Orphan Drug Development Check Access

The 1983 Orphan Drug Act (ODA) in the USA, and similar policies enacted in several other global pharmaceutical markets since then, has not only stimulated tremendous focus, product development and launch of new medicines to address a broad range of orphan and rare diseases, but has also had significant impact for the overall delivery of biopharmaceutical products, in general, to patients and the world’s population. Key areas of contribution underlying orphan drug development's impact cover the biopharmaceutical spectrum, including compelling discoveries in the life sciences, novel drug technologies and platforms, as well as creative approaches and innovation in clinical development and commercialisation. The significance of orphan drug development's evolution is underscored by the continued growth in orphan designations and approvals, and its increasing contribution to, and share of, the biopharmaceutical industry's performance and value creation. The favourable economics of orphan drug development, and its impact on science and health care, support the outlook that this sector will continue to be an important contributor to ongoing biopharmaceutical innovation.