Anshu Shrestha

Cost‐effectiveness is traditionally treated as a static estimate driven by clinical trial efficacy and drug price at launch. Prior studies suggest that cost‐effectiveness varies over the drug's lifetime. We examined the impact of “learning by doing,” one of the least studied drivers of changes in cost‐effectiveness across the product life cycle. We combined time‐series trends in effectiveness over time by cancer regimen using the Surveillance, Epidemiology, and End Results‐Medicare database. We estimated the time‐varying effects of treatments in colorectal and pancreatic cancer over their life cycle, including FOLFOX (leucovorin, 5‐fluorouracil, and oxaliplatin) and gemcitabine, on survival of patients. Mean prices over time by strength and dosage form were calculated using historical wholesale acquisition costs. We found consistent downward trends in the mortality hazard ratios, which suggest that effectiveness improves over time. In the case of first‐line FOLFOX for colorectal cancer, the implied incremental cost‐effectiveness ratio based on the observational data fell from $610,000 per life year gained in 2004 to $27,000 per life year gained in 2011. Cost‐effectiveness estimated at launch is unlikely to be representative of cost‐effectiveness over the drug's lifetime. In the drugs studied, the impact of time‐varying clinical effectiveness dominated the impact of changing prices overtime.

Introduction: Prophylaxis treatment is recommended for haemophilia patients, but associated real‐world economic costs and potential cost‐savings associated with improved disease management are not fully known. This study aimed to assess haemophilia A‐related resource use and cost by treatment type (prophylaxis versus non‐prophylaxis) and any associated cost‐savings.Methods: Truven MarketScan Commercial claims data (2004–2012) were used to identify haemophilia A‐related healthcare utilization, healthcare costs and patterns of prophylaxis and non‐prophylaxis treatment among 6‐ to 64‐year‐old males. We estimated bleeding‐related resource utilization and costs in three age groups (6–18, 19–44, 45–64) by treatment types and assessed the extent to which early initiation of prophylactic treatment can mitigate them. T‐tests and ordinary least squares regressions were used to compare unadjusted and demographics‐adjusted cost estimates.Results: Among children, overall haemophilia‐ and bleeding‐related non‐pharmacy costs were substantially lower for patients receiving prophylaxis (haemophilia‐related: $15,864 vs. $53,408; P < 0.001; bleeding‐related: $696 vs. $2013, respectively; P = 0.04). Among younger adults (19–44), haemophilia‐related non‐pharmacy costs were lower for patients receiving prophylaxis ($22,028 vs. $56,311, respectively; P = 0.001). Among children, these savings fully offset the incremental pharmacy cost due to prophylaxis. Among younger adults, the savings offset approximately 34% of the incremental pharmacy cost. No differences were found for older adults (45–64).Conclusion: These results suggest that initiating prophylaxis earlier in life may reduce the healthcare costs of bleeding events and their long‐term complications. Future studies should strive to collect more detailed information on disease severity and treatment protocols to improve estimates of disease burden.