FDA sets out framework for rare childhood diseases

The FDA has laid out plans to encourage the development of new therapies for rare diseases in children, including a quicker approval route, greater use of biomarkers in clinical trials, and more engagement with the private sector. With the new strategic plan the US regulator wants to tackle the unique challenges faced when developing new drugs and medical devices for rare paediatric diseases, such as the low number of patients who can participate in studies. "Not only is there the potential for children to respond differently to products as they grow but there are also additional ethical concerns for this patient population," said Jill Hartzler Warner, FDA's associate commissioner for special medical programmes in a blog.