Monday, February 17, 2014
There are two key takeaways
from FDA's fifth Patient-Focused Drug Development workshop. The first is
that there are opportunities for drugs that treat debilitating chronic effects
of the disease, which are not the primary focus of current development
programs. The second is that patients want better information about potential
risks and benefits of participating in trials.
Sickle cell disease is a
hereditary disorder in which red blood cells make abnormal hemoglobin that can
distort them into a sickle shape. Symptoms and severity vary widely, even for
patients with similar genotypes.