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.