The FDA Encourages New Treatments for Sickle Cell Disease

The FDA continues to work with stakeholders including patients, academics and companies developing treatments to improve therapeutic options for people living with SCD. A growing number of new products are in the pipeline, and researchers are exploring new treatment approaches. “These potential treatments are in different stages of development, including early and late clinical trials,” Farrell says. (Clinical trials are voluntary human research studies designed to answer specific questions about the safety and effectiveness of potential new treatments or to study new ways of using existing treatments.) Companies developing sickle cell products can ask the FDA to grant a “fast track” designation, a type of regulatory status that seeks to bring new products to market faster. “This allows for early and more frequent interactions with the FDA to discuss any issues that come up during development, such as manufacturing and trial design,” Farrell explains. If preliminary data show potential promise over existing therapies, companies also can request “breakthrough therapy designation” to speed the development and review of products. Abstract source