Expanding Access to Cell and Gene Therapies

Cell and gene therapies have become very effective for treating patients with hematologic malignancies, hemoglobinopathies, and immune deficiencies, but most patients are being treated at academic medical centers. It’s important to make these therapies available to more patients. For hospitals without specialized cell processing facilities the blood bank or hospital pharmacy is well suited to handle these products. The administration of in vivo gene therapies which does not involve the infusion of cells and is similar to the intravenous administration of drugs, can be handled by pharmacies. Providing cellular therapies is more difficult since most are cryopreserved. To provide these therapies a hospital must have access to −80 °C or liquid nitrogen freezers for storage of the cellular therapy, the ability to thaw and quickly administer the therapy, and a system to document their administration. The difficulties associated with providing these therapies at more medical centers and how to overcome these issues are discussed.