Sickle cell disease patients’ willingness to participate in clinical trials for investigational treatments hinges on a number of factors, including the severity of their own disease, what’s known about the compound under study and whether the agent is more likely to help them than hurt them, patients and advocates told FDA at a Feb. 7 meeting.
The potential for rare but serious side effects, considerations about how the treatment might improve their health and how the trial might impact an individual’s current treatment plan also are important factors in