Sickle cell disease (SCD) is a rare but debilitating condition that profoundly affects patients and their families. Until 2019, few approved therapies specifically targeted SCD—and those that existed were often insufficient to prevent or relieve painful occlusive crises. Over the past six years, however, four new therapies have been approved, and three are currently available, including novel gene therapies. But access to life-altering therapy depends on many factors, including financial barriers. 

This paper explores the following barriers that may limit access to SCD therapies across the United States, particularly within the cell and gene therapy (CGT) delivery journey:

• Geographic access to qualified treatment centers
• Hospital and manufacturing capacity constraints
• The long lead time to receive CGT treatment
• Required supplemental services such as fertility preservation
• Suggestions to improve insurance coverage