Value of survival gains in chronic myeloid leukemia

Although clinical trial data have quantified patient survival gains associated with tyrosine kinase inhibitors in chronic myeloid leukemia, the overall value of these benefits is unknown.

To estimate the total value of survival gains associated with first- and second-line TKI therapy in chronic myeloid leukemia (CML) and the fraction of tyrosine kinase inhibitor (TKI)- related survival-gain value retained by patients and drug companies.

This retrospective study identified CML patient data from the Surveillance, Epidemiology and End Results registry, dasatinib clinical trials, and insurance claims data sets.

Multivariate Cox proportional hazard models were used to estimate improvements in CML survival associated with the introduction of first-line imatinib therapy. Survival gains associated with second-line dasatinib treatment were identified via retrospective analyses and published clinical outcomes. An economic model was developed to calculate the social value of survival gains derived from first- and second-line TKI treatment. TKI costs were used to estimate the fraction of survival gain value retained by patients and drug companies.

The introduction of TKIs in 2001 was associated with a hazard ratio of 0.833 (P <.01). Cost analyses indicate that the TKI drug class in CML therapy has created more than $143 billion in social value. Approximately 90% of this value is retained by patients and society, while approximately 10% is recouped by drug companies.

These estimates indicate that the introduction of TKI drugs to treat CML has generated significant social value as a result of survival gains, the vast majority of which has accrued to patients.