De la Paz Manuel Posada, Taruscio Domenica, Groft Stephen C. (eds.) Rare Diseases Epidemiology: Update and Overview

2nd Ed. — Springer, 2017. — 675 p. — (Advances in Experimental Medicine and Biology). — ISBN10: 3319671421, 13 978-331967142.The fields of rare diseases research and orphan products development continue to expand with more products in research and development status. In recent years, the role of the patient advocacy groups has evolved into a research partner with the academic research community and the bio-pharmaceutical industry. Unique approaches to research and development require epidemiological data not previously available to assist in protocol study design and patient recruitment for clinical trials required by regulatory agencies prior to approval for access by patents and practicing physicians.