Real-World Treatment Patterns and Outcomes in Patients Receiving Second-Line Therapy for Advanced/Metastatic Esophageal Squamous Cell Carcinoma.

INTRODUCTION: Currently available second-line (2L) therapies for advanced/metastatic esophageal squamous cell carcinoma (adv/met ESCC) include the taxanes paclitaxel and docetaxel. In clinical trials, such therapies have provided only modest improvements in survival. Few studies have assessed outcomes in routine clinical practice in the USA. We compared real-world clinical outcomes in the US for patients receiving taxane or non-taxane 2L therapy for adv/met ESCC. METHODS: The Flatiron Health database was used to identify patients diagnosed with adv/met ESCC (1 January 2011-31 January 2019) who received 2L therapy; index date was date of adv/met diagnosis. Baseline variables and treatment regimens received were identified. Overall survival (OS; 2L start until death or last recorded medical activity) and duration of therapy (DoT; start of 2L therapy until last administration date of 2L therapy) in patients receiving taxane vs. non-taxane-based therapies in the 2L setting were estimated by Kaplan-Meier method. RESULTS: There were no clear differences in baseline characteristics between patients who received 2L taxane therapy (n = 37) and 2L non-taxane therapy (n = 49). Median (95% CI) 2L OS was significantly longer with 2L taxanes (7.3 [5.9-11.5] months) vs. 2L non-taxanes (5.1 [2.9-7.6] months); median (95% CI) 2L DoT was 2.1 (1.8-3.0) months vs. 3.3 (2.6-6.7) months, respectively. CONCLUSION: Survival was generally poor in patients receiving 2L therapy for adv/met ESCC and was longer in patients receiving 2L taxanes than 2L non-taxane therapy. Efficacious, tolerable therapies for ESCC in the 2L setting are urgently needed.

Budget impact analysis of tapentadol extended release for the treatment of moderate to severe chronic noncancer pain.

BACKGROUND: Opioids are commonly used to manage chronic pain. Although traditional µ-opioids are effective in reducing pain, they are often associated with opioid-induced side effects (OISEs) that can limit treatment effectiveness. Studies have shown that tapentadol extended release (ER) has a lower incidence of gastrointestinal adverse events than oxycodone controlled release (CR) at equianalgesic doses. OBJECTIVE: A model was developed to estimate the budget impact of placing tapentadol ER on a hypothetical US health plan formulary of Schedule II long-acting opioids. METHODS: We estimated annual direct health care costs for patients who received 6-month therapy with long-acting formulations of tapentadol, oxycodone, morphine, hydromorphone, oxymorphone, or fentanyl. Costs included medications, copayments, OISE management, and switching/discontinuation. Published estimates of incidence/prevalence, OISEs, and pain management resources and costs were used. The base case analysis assumed a 10% formulary share of tapentadol ER with a 10% decrease of oxycodone CR. The resulting per-member per-month (PMPM) formulary cost differences and results of a 1-way sensitivity analysis are reported. RESULTS: In a health plan of 500,000 members, 2600 (0.52%) are estimated to experience chronic pain annually. Adding tapentadol ER to the formulary was associated with an annual budget savings of $148,945 ($0.0248 PMPM). This savings was achieved through a decrease in both pharmacy costs ($144,062; $0.0240 PMPM) and medical costs ($4883; $0.0008 PMPM). Cost decreases were driven by lower daily average consumption and fewer OISEs with tapentadol ER versus oxycodone CR, leading to reduced resource utilization over 6 months of treatment. Sensitivity analyses showed results were most sensitive to drug acquisition costs. CONCLUSIONS: Our results suggest that replacing 10% of oxycodone CR's formulary share with tapentadol ER would decrease the overall budget of a health plan with 500,000 members. Placing tapentadol ER on a health plan formulary may result in a reduction in both pharmacy and medical costs.