SICKLE CELL DISEASE | Limited Progress in Sickle Cell Disease Highlights Barriers to Treatment Access
A new study from the United States suggests that limited progress in sickle cell disease (SCD) can be attributed to barriers in treatment access and challenges with recently approved therapies.
According to the findings, there has been limited improvement in outcomes for SCD patients, in large part due to barriers in accessing first-line therapies such as hydroxyurea. Although several new agents were approved for SCD between 2017 and 2019, many have since been withdrawn or failed to demonstrate mortality benefits.
The approval of gene therapy has generated hope for long-term solutions. The high cost of these treatments, however, remains a major obstacle to widespread access, raising concerns about the extent to which patients will be able to benefit from these advances.
The authors stressed the urgent need for equitable investment in research, treatment availability, and advocacy to ensure that individuals living with SCD are not left behind.
Source: MedPage Today
