San Francisco, CA—The introduction of the 3 targeted therapies—lenalidomide, decitabine, and azacitidine—for myelodysplastic syndrome (MDS) has resulted in a 74% increase in median survival and generates more than $100 billion in value resulting from survival gains for current and future patients, reported Joanna P. MacEwan, PhD, Associate Research Economist, Precision Health Economics, San Francisco, CA, at ASH 2014.
Despite improvement in cancer survival since the 1970s, critics have argued that newer cancer treatments offer minimal survival gains, at high costs. Noting the lack of economic analyses, Dr MacEwan and colleagues studied the value of MDS treatments relative to their cost.
Building on an existing analytic framework using data on survival and prescription drug claims, the researchers conducted 3 subanalyses, including survival analysis, the value of survival gains, and value appropriation.
Cox proportional hazards models were applied to estimate the increase in survival associated with the introduction of new therapies for MDS in 38,085 patients with MDS diagnosed between 2001 and 2011. The results showed that patients diagnosed with MDS since 2006 (when all 3 therapies were available) had an approximate 10% lower mortality risk than patients diagnosed between 2001 and 2003 (hazard ratio, 0.901; P <.10).
The median survival in patients with MDS who received lenalidomide, decitabine, or azacitidine (approximately 25%) increased from 33 months to 57.5 months, representing a 74% improvement.
“The annual value of survival gains associated with these innovative treatments—roughly $208,000 per year—equaled the estimated amount a patient would be willing to pay for the higher survival profile associated with new therapies,” Dr MacEwan suggested.
This value was estimated to exceed the cost of therapy, with an annual net benefit of $68,200 to patients with MDS and a lifetime benefit of $238,700. The present value of survival gains for all current and future cohorts was determined to be $101.5 billion.
Based on estimates of patent expiration and price decreases after the patent expirations, the investigators concluded that 85% of the value of survival gains created by novel therapies ($86.5 billion) will accrue to patients with MDS, with the remaining 15% accruing to the drugs’ manufacturers.