Cost per treatment responder analysis of atogepant compared to rimegepant for the preventive treatment of episodic migraine.

OBJECTIVE: To estimate the number needed to treat and cost per additional responder for atogepant and rimegepant versus placebo for the preventive treatment of episodic migraine (EM) in the United States. BACKGROUND: Migraine has an enormous impact on a person's daily activities and quality of life, and results in significant clinical and economic burden to both individuals and society. It is important to understand the comparative efficacy and economic value of oral calcitonin gene-related peptide receptor antagonists (gepants) for preventive treatment of EM. Currently, atogepant and rimegepant are US Food and Drug Administration approved for preventive treatment of migraine (rimegepant for EM and atogepant for EM and for chronic migraine). In the absence of head-to-head trials, we utilized an indirect treatment comparison on efficacy data from clinical trials conducted for the preventive treatment of EM. We estimated number needed to treat, a valuable metric used in clinical practice to compare treatment efficacy, and cost per additional responder, which can be used to establish the cost effectiveness of a treatment. METHODS: An indirect treatment comparison was conducted to compare the efficacy of atogepant 60 mg once daily and rimegepant 75 mg once every other day as preventive treatments for EM using published data from the registrational trials of atogepant (ADVANCE) and rimegepant (BHV3000-305). The efficacy outcome of interest was ≥50% reduction from baseline in mean monthly migraine/headache days (≥50% responder rate), which was variably defined for a base case and two scenario analyses. Number needed to treat and cost per additional responder versus placebo were calculated and compared between both treatments (weeks 9-12 in the base case analysis; weeks 1-12 and 9-12 for atogepant and during weeks 9-12 for rimegepant in the scenario analyses). RESULTS: In the base case analysis, ≥50% responder rates were 64.9% (95% confidence interval [CI], 53.9-74.5) for atogepant and 51.8% (95% CI, 42.9-60.6) for rimegepant, compared to 44.1% (95% CI, 39.4-49.0) for placebo. The median number needed to treat versus placebo in the base case scenario was 4.8 (95% CI, 3.1-9.0) for atogepant compared to 13.0 (95% CI, 5.9-75.1) for rimegepant. The cost per additional responder versus placebo in the base case scenario was estimated to be $15,823 (95% CI, $11,079-$29,516) for atogepant compared to $73,029 (95% CI, $32,901-$422,104) for rimegepant. Results of the two scenario analyses were consistent with the base case analysis. CONCLUSIONS: Atogepant had substantially lower numbers needed to treat and costs per additional responder versus placebo than rimegepant for the preventive treatment of EM across all evaluated scenarios. These analyses suggest that atogepant may be more cost effective than rimegepant for the preventive treatment of EM. Limitations include differences in inclusion/exclusion criteria and in reporting of the ≥50% responder rates between trials.

Cost-effectiveness of overactive bladder treatments from a US commercial and payer perspective.

Aim: The cost-effectiveness of treatment options (anticholinergics, ß3-adrenoceptor agonists, onabotulinumtoxinA, sacral nerve stimulation and percutaneous tibial stimulation [the latter two including new rechargeable neurostimulators]) for the management of overactive bladder (OAB) were compared with best supportive care (BSC) using a previously published Markov model. Materials & methods: Cost-effectiveness was evaluated over a 15-year time horizon, and sensitivity analyses were performed using 2- and 5-year horizons. Discontinuation rates, resource utilization, and costs were derived from published sources. Results: Using Medicare and commercial costs over a 15-year time period, onabotulinumtoxinA 100U had incremental cost-effectiveness ratios (ICERs) gained of $39,591/quality-adjusted life-year (QALY) and $42,255/QALY, respectively, versus BSC, which were the lowest ICERs of all assessed treatments. The sensitivity analyses at 2- and 5-year horizons also showed onabotulinumtoxinA to be the most cost-effective of all assessed treatments versus BSC. Conclusion: OnabotulinumtoxinA 100U is currently the most cost-effective treatment for OAB.

Cost-effectiveness of pegfilgrastim versus filgrastim primary prophylaxis in women with early-stage breast cancer receiving chemotherapy in the United States.

BACKGROUND: Prophylaxis with granulocyte colony-stimulating factor reduces the risk for febrile neutropenia (FN) in patients receiving myelosuppressive chemotherapy. OBJECTIVE: We estimated the incremental cost-effectiveness of primary prophylaxis (starting in cycle 1 of chemotherapy) with pegfilgrastim versus filgrastim in women with early-stage breast cancer receiving myelosuppressive chemotherapy in the United States. METHODS: A decision-analytic model was constructed from a health payer's perspective with a lifetime study horizon. The model considered direct medical costs and outcomes related to reduced FN and potential survival benefits due to reduced FN-related mortality and on-time receipt of full-dose chemotherapy. Sensitivity analyses were conducted. RESULTS: Pegfilgrastim was cost-saving and more effective (ie, dominant strategy) than 11-day filgrastim. The incremental cost-effectiveness ratio (ICER) for pegfilgrastim versus 6-day filgrastim was $12,904 per FN episode avoided. Adding the survival benefit due to reduced FN mortality and receipt of optimal chemotherapy dose yielded an ICER of $31,511 per quality-adjusted life year (QALY) gained and $14,415 per QALY gained, respectively. The most influential factors included inpatient FN case-fatality rate, cost of pegfilgrastim and filgrastim, baseline probability of FN, relative risk for FN between filgrastim and pegfil-grastim, and cost of administration of filgrastim. CONCLUSION: Pegfilgrastim was cost-saving compared with 11-day filgrastim and cost-effective compared with 6-day filgrastim from a health payer's perspective for the primary prophylaxis of FN in these women with early-stage breast cancer receiving myelosuppressive chemotherapy.

Cost-effectiveness of cinacalcet in secondary hyperparathyroidism in the United States.

OBJECTIVE: Cinacalcet has been used in controlling secondary hyperparathyroidism (SHPT) in dialysis patients since 2004, but its full economic evaluation has not been conducted from the US perspective. This study assesses the cost-effectiveness of cinacalcet and low-dose vitamin D for the treatment of SHPT in dialysis patients compared with flexible vitamin D. METHODS: A lifetime patient-level simulation model was developed using ADVANCE trial data, including biomarker levels: parathyroid hormone, calcium, and phosphorus. The impact of the biomarkers on mortality, cardiovascular events, fractures, and parathyroidectomy were estimated from literature: Block, an observational study; Cunningham, a combined analysis of four randomized trials of cinacalcet; and Danese, a study investigating the effect of duration in recommended targets. Baseline event rates were derived from the large dialysis organizations registries. One-way and probabilistic sensitivity analyses (PSA) were conducted. RESULTS: The cost-effectiveness ratio for cinacalcet compared with standard of care (vitamin D and phosphate binders) was $54,560 and $72,456/quality-adjusted-life-year (QALY) gained or an incremental cost of $3155 and $2638 per year alive for the Block and Danese variants, respectively. In the Cunningham variant, cost-effectiveness ratio for cinacalcet was $5064/QALY gained or a cost saving of $1068 per year. The difference in the results of the Cunningham variant vs other variants can be explained by the favorable impact of cinacalcet on outcomes, specifically cardiovascular events observed in the Cunningham study. The PSA showed 98% likelihood for cinacalcet to be cost-effective at $100,000/QALY threshold. LIMITATIONS: Observational data assessing effects on clinical outcomes, trial restriction to use calcium-containing phosphate binders, no utility data in SHPT dialysis population, and insufficient evidence on long-term impact of cinacalcet and vitamin D on biochemical markers. CONCLUSIONS: Cinacalcet treatment is cost-effective for treatment of SHPT in the US. Due to cost offsets, cinacalcet can reduce annual costs in some scenarios.

Cost-effectiveness of pegfilgrastim versus 6-day filgrastim primary prophylaxis in patients with non-Hodgkin's lymphoma receiving CHOP-21 in United States.

OBJECTIVES: Prophylaxis with granulocyte-colony stimulating factor (G-CSF) reduces the risk of febrile neutropenia (FN) in patients receiving myelosuppressive chemotherapy. Randomized clinical trials have shown that pegfilgrastim, a 2nd-generation G-CSF, is at least as effective as the 1st-generation G-CSF filgrastim. In the meta-analysis of trials pegfilgrastim performed better than filgrastim with respect to FN risk. The incremental cost-effectiveness of primary prophylaxis (starting in cycle 1 and continuing in subsequent cycles of chemotherapy) with pegfilgrastim versus filgrastim used for 6 days (as is often used in clinical practice) was estimated in patients with aggressive non-Hodgkin's lymphoma (NHL) receiving myelosuppressive chemotherapy in the United States. METHODS: A decision-analytic model was constructed from a health insurer's perspective with a life-time study horizon. The model considered direct medical costs and outcomes related to reduced FN and potential survival benefits due to reduced FN-related mortality. Inputs for the model were obtained from the medical literature. Sensitivity analyses were conducted across plausible ranges in parameter values. RESULTS: The incremental cost-effectiveness (ICER) of pegfilgrastim versus 6-day filgrastim primary prophylaxis was $2167/FN episode avoided. Adding survival benefit from avoiding FN mortality yielded an ICER of $5532/LY gained or $6190/QALY gained. When the potential benefit of optimized chemotherapy was included, the ICER was $1494/LY gained or $1677/QALY gained. The most influential factors included cost of pegfilgrastim, relative risk of FN between pegfilgrastim and filgrastim, FN case-fatality rate, cost of filgrastim and baseline FN risk. CONCLUSIONS: Pegfilgrastim is cost-effective in primary prophylaxis of FN compared to 6 days per cycle of filgrastim, in patients with NHL receiving myelosuppressive chemotherapy (e.g., cyclophosphamide + doxorubicin + vincristine + prednisolone [CHOP-21]) chemotherapy. Study limitations included lack of direct evidence linking G-CSF use with a reduction in FN-related mortality and limited data that show a relationship between relative dose intensity (RDI) and cancer-specific patient survival.

Cost effectiveness of contraceptives in the United States.

BACKGROUND: The study was conducted to estimate the relative cost effectiveness of contraceptives in the United States from a payer's perspective. METHODS: A Markov model was constructed to simulate costs for 16 contraceptive methods and no method over a 5-year period. Failure rates, adverse event rates and resource utilization were derived from the literature. Sensitivity analyses were performed on costs and failure rates. RESULTS: Any contraceptive method is superior to "no method". The three least expensive methods were the copper-T intrauterine device (IUD) (US$647), vasectomy (US$713) and levonorgestrel (LNG)-20 intrauterine system (IUS) (US$930). Results were sensitive to the cost of contraceptive methods, the cost of an unintended pregnancy and plan disenrollment rates. CONCLUSION: The copper-T IUD, vasectomy and the LNG-20 IUS are the most cost-effective contraceptive methods available in the United States. Differences in method costs, the cost of an unintended pregnancy and time horizon are influential factors that determine the overall value of a contraceptive method.