Are we on the brink of a cure for sickle cell disease? NICE recently announced the approval of a one-off gene therapy sickle cell for severe sickle cell disease.

Epidemiology of Sickle Cell Disease

Sickle cell disease affects a significant number of individuals in the United States. Estimates suggest there are approximately 120,156 cases of SCD in the US, with 87% of these cases from the US-born population and 13% from immigrants, particularly from the Caribbean, Western Africa, and Middle and Southern Africa. The disease is most prevalent among Black or African American newborns. Approximately one in every 365 Black newborns in the US has SCD. The birth prevalence among non-Hispanic Black newborns is about 28.54 per 10,000 live births.

Health Economic Impact

Sickle cell disease imposes a substantial economic burden. It is estimated to result in $1.5 billion in lost wages and productivity each year in the US. On an individual level, this translates to over $650,000 in lost wages over the average working life of a person with SCD. Employed individuals with SCD miss an average of seven weeks of work per year due to pain. They also experience significant distraction and lost productivity when working while in pain, leading to an estimated $15,000 annual loss per employed patient. The disease also severely impacts daily activities. Approximately 75% of patients report impairment in completing everyday tasks such as caring for children, running errands, and doing housework.

Gene Therapy Approval and Its Implications

The approval of the one-off gene therapy sickle cell, CASGEVY (exagamglogene autotemcel), by NICE provides a potential cure for certain patients with severe SCD, particularly those aged 12 years and older. This therapy is significant as it offers an alternative to donor stem cell transplants. The approval also addresses healthcare inequalities. SCD disproportionately affects individuals from African, Caribbean, Middle Eastern, or South Asian family backgrounds. By making this gene therapy available, NICE aims to reduce these inequalities.

The gene therapy carries a high cost and presents higher cost-effectiveness estimates than what NICE typically considers. However, the potential long-term benefits—including reduced pain crises, improved quality of life, and increased life expectancy—justify the investment. Models from the Clinical and Economic Impact Analysis (CEIA) Consortium suggest that gene therapies can be life-altering, reducing morbidity and improving long-term employment prospects and overall quality of life.

In summary, the approval of this gene therapy sickle cell is a significant advancement in addressing the complex health and economic challenges posed by sickle cell disease. It has the potential to improve health outcomes, reduce healthcare costs over time, and address the socioeconomic disparities associated with SCD. However, challenges related to accessibility, insurance coverage, and the high costs of these therapies remain. These issues need to be tackled to ensure equitable access to this treatment.