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.
Sickled cells have shortened lifespans, leading to anemia, and can clump in small blood vessels, causing pain, hypoxia and tissue damage. Vessel blockage can lead to an acute pain crisis (vaso-occlusive crisis) that requires hospitalization and narcotics, and can take days to resolve.
Common treatments include hydroxyurea, transfusions and bone marrow transplant.
Only bone marrow transplant is potentially curative. It requires a suitable donor and can lead to graft-versus-host disease (GvHD) or infections.
At FDA's meeting on Feb. 7, some panelists said hydroxyurea reduced their frequency of hospitalization,