The importance of natural histories for rare diseases

Abstract

Developing a new drug for a disease that affects relatively few people poses unique challenges that do not apply to medical products for more prevalent diseases. One relatively new strategy to help address the special needs of the orphan drug development process is through better understanding the natural progression of rare diseases. This information is then used to facilitate the product development and approval process. National Organization for Rare Disorders (NORD) is actively engaged with the National Institutes of Health and FDA in collaborating on programs to advance the development of increased and improved natural history data. One of the unique contributions that NORD is making is that it is bringing the patient voice to the process of data generation.