Special treatment for rare diseases

Since relatively few people suffer from rare diseases – fewer than 200,000 in the US - the development of new drugs for these diseases can be difficult to gain approval for. Dr Nicola Davies examines US Food and Drug Administration guidance to aid drug developers working in the area of rare diseases.

Facilitating drug development for rare diseases

Liz Kalina, senior health communications strategist of Shire, a global specialty biopharmaceutical company , estimates that of the approximately 7,000 known rare diseases, only a small percentage have approved treatments. “The development process for a rare disease drug is vastly different due to the complexity of the disease areas, the limited patient numbers, and the unknown environment related to any rare disease. The challenges begin in early research and continue through development, launch, and post-marketing,” she explains.