Critical need for establishing value that justifies the current rising costs of cell and gene therapy

Published: 9 July 2021
Richard T Maziarz, MD

Autologous and allogeneic hematopoietic cell transplantation (HCT) remain valuable tools in the treatment of patients with advanced hematologic malignancies, some solid tumor cancers, immune deficiency syndromes and genetic inherited disorders. The confirmation of the allograft graft versus malignancy effect observed after the administration of donor leukocyte infusions to patients with relapsed chronic myelogenous leukemia has been highlighted by many as the birth of cell therapy. With the emergence of clinical cellular immune oncology over the past three decades, cellular and now gene therapies have expanded dramatically, with indications beyond the scope of HCT. The cost of care of HCT has been heavily scrutinized and since become a barometer for new cellular therapy interventions. Cell and gene therapy innovations are changing the landscape of care for many individuals, but it remains unclear if access to care will be limited by the high costs. Value frameworks may provide the appropriate tools to determine and guide valuation and pricing of these emerging agents.

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