Gene therapies can reduce treatment burden and costs for patients with blood disorders

Katie Koziara
Katie Koziara May 18, 2022

Gene therapies can reduce treatment burden and costs for patients with blood disorders.

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A new analysis found potential gene therapies for blood disorders like hemophilia, beta thalassemia, and sickle cell disease hold promise in dramatically reducing existing treatment burdens for patients, which could result in as much as $730,000 in savings to the health care system and increase a patient’s income by as much as $9,500 in the year following treatment.

Blood disorders often require lifelong treatment involving routine drug infusions or blood transfusions. They can also be associated with frequent visits to physician offices, infusion centers or even the emergency room. Patients with these disorders often experience debilitating pain, disability, reduced life expectancy and quality of life.

Gene therapies currently in development hold tremendous promise to completely transform the treatment paradigm for these patients, not only improving quality of life but leading to significant savings to the health care system and patients.


Today, there are more than 500 medicines in development for disorders of the blood, including many gene therapies. While many gene therapies are administered just one time with long-lasting or even potentially curative effects, the full value they provide to patients, caregivers and the entire health care system may only be realized over the lifetime of the patient. Addressing the unique challenges presented by 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 sheets here.

Topics: Medicines in Development, Personalized Medicine