What Does the Economic Burden of Acute Myeloid Leukemia Treatment Look Like for the Next Decade? An Analysis of Key Findings, Challenges and Recommendations.

Acute myeloid leukemia (AML) is conventionally treated with chemotherapy in eligible patients. Potentially curative regimens are associated with significant toxicity, and the major cost drivers in AML historically have been hospitalization and hematopoietic stem cell transplantation. The past several years have seen a dramatic increase in the number of treatment options, including oral therapies and drugs targeted to biological pathways implicated in AML. Major current and future drivers of cost in AML include hospitalization and medical costs, stem cell transplantation for eligible patients, and medication costs. It is likely that hospitalization and medical costs will decline as more AML treatment moves to the outpatient setting. Stem cell transplantation costs may increase, if more patients are eligible for improved procedures, although the overall cost of transplantation could decrease if new procedures reduce the need for hospitalization. Medication costs are likely to increase, with various branded drugs available and in development. From a broader perspective, another driver of cost is the proportion of patients with AML who can undergo treatment. Patients who may previously have been unable to tolerate chemotherapy are more likely to be treated with the range of less intensive, more tolerable options now available. The effectiveness of newer AML treatment options also suggests that, overall, there may be more patients staying alive and on treatment longer than in the past. While certain advances, such as increased use of oral and outpatient therapies, could potentially reduce costs, the overall economic impact of AML is likely to increase as more patients are eligible for novel therapies across several phases from induction to maintenance to relapsed/refractory disease. While these novel therapies have the potential to deliver value in the form of improved efficacy, safety, and convenience, payers will need to determine how to cover a longer, more complex AML treatment pathway.