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Real-World Evidence Regarding Newer Treatment Options, Overall Survival, and Healthcare Costs in Patients with Multiple Myeloma

TOP - February 2017, Vol 10, No 1 - Hematologic Cancers

Limited real-world evidence is available to describe the recent trends in multiple myeloma (MM) treatment costs and outcomes. This study assessed trends in novel therapy use, total healthcare costs, and survival outcomes among patients with newly diagnosed MM in the United States since 2000. Novel agents approved for use in 2015 were not included in the analysis.

Patients aged ≥18 years with 2 or more medical claims with MM between January 2000 and September 2015 were identified in administrative claims databases. The first medical claim for MM was the index date. Controls were selected from a pool of patients without MM and matched 1:1 with MM patients on index year, age, sex, and geographic region. Total healthcare costs were defined as the sum of health plan– and patient-paid costs for inpatient admissions, outpatient services, and outpatient prescriptions. MM treatment-related drug costs included outpatient prescription costs and infusion costs from medical services associated with MM treatment.

After exclusions, 18,260 patients were matched to controls. The proportion of patients with MM using newer therapies (ie, pomalidomide, carfilzomib, bortezomib, lenalidomide, or thalidomide) continuously increased—from 8.7% in 2000 to 61.3% in 2014. The total per-patient per-month all-cause healthcare costs increased from $3263 in 2000 to $14,656 in 2014 in newly diagnosed patients; these increases were primarily driven by costs of outpatient services. Hospitalization costs accounted for 21.5% of the total costs in 2000, which increased to 32.7% in 2014. Treatment-related drug costs for MM accounted for 10.6% of the total costs in 2000, 23.6% in 2009, and 28.5% in 2014.

Patients diagnosed with MM after 2010 had significantly higher utilization rates of newer drugs and significantly better survival outcomes than patients diagnosed before 2010. In fact, patients diagnosed in 2012 were 1.25 times more likely to survive 2 years than those diagnosed in 2006. Overall survival outcomes improved over the study period, with the 2-year survival gap between patients with MM and their matched controls decreasing at an annual rate of 3%.

These findings are aligned with clinical data, suggesting that the increase in novel MM treatment options over the past 15 years has led to substantial survival gains. Although total healthcare costs among patients with newly diagnosed MM have increased steadily since 2000 because of increases in all healthcare cost categories, the contribution of drug costs has remained stable since 2009 despite new novel therapies coming to market.

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Last modified: April 27, 2020