Potential gene therapies bring hope to patients with sickle cell disease

A recent analysis highlights how potential gene therapies have the potential to dramatically reduce income disparities for patients with sickle cell disease, leading to as much as $21,000 more in average income in the year following gene therapy administration alone.

Sickle cell disease is a rare blood disorder that disproportionately affects Black Americans. The disease causes crescent-shaped blood cells to clog blood vessels, which prevents the normal flow of nutrition and oxygen throughout the body and can lead to serious complications, including pain crisis and acute chest syndrome.

Patients with sickle cell disease are hospitalized more than once a year, on average, and visit the emergency room 2 to 3 times a year, most commonly due to pain crisis. For people living with sickle cell disease, 50% report pain on half of their days and 30% report pain most of the time.

50% to 60% of patients reported the disease negatively impacted their employment status, forced them to stop working completely or take a leave of absence, or led to unpaid time off or reduced work hours. As a result, patients are estimated to earn $750,000 less over a lifetime, representing a significant burden on the Black and African American community disproportionately impacted by this disease.

There are 30 medicines in development for sickle cell disease. That list includes gene therapies in the late stages of development that have shown evidence in clinical trials of an almost complete reduction in painful vaso-occlusive crisis as well as acute chest syndrome in the years following a one-time administration of therapy.

Reducing these serious complications can help restore the quality of life in people with sickle cell disease and enable people to maintain more consistent and reliable employment, dramatically reducing income disparities. And the full value of gene therapies may only be realized over a patient’s lifetime. That’s why these groundbreaking therapies requires a rethinking of the way we pay for medicines to account for the long-term value these therapies provide.

Learn more by viewing the report and fact sheet here.