Cost Savings of Expedited Care with Upfront Next-Generation Sequencing Testing versus Single-Gene Testing among Patients with Metastatic Non-Small Cell Lung Cancer Based on Current Canadian Practices.

This study assessed the total costs of testing, including the estimated costs of delaying care, associated with next-generation sequencing (NGS) versus single-gene testing strategies among patients with newly diagnosed metastatic non-small cell lung cancer (mNSCLC) from a Canadian public payer perspective. A decision tree model considered testing for genomic alterations using tissue biopsy NGS or single-gene strategies following Canadian guideline recommendations. Inputs included prevalence of mNSCLC, the proportion that tested positive for each genomic alteration, rebiopsy rates, time to test results, testing/medical costs, and costs of delaying care based on literature, public data, and expert opinion. Among 1,000,000 hypothetical publicly insured adult Canadians (382 with mNSCLC), the proportion of patients that tested positive for a genomic alteration with an approved targeted therapy was 38.0% for NGS and 26.1% for single-gene strategies. The estimated mean time to appropriate targeted therapy initiation was 5.1 weeks for NGS and 9.2 weeks for single-gene strategies. Based on literature, each week of delayed care cost CAD 406, translating to total mean per-patient costs of CAD 3480 for NGS and CAD 5632 for single-gene strategies. NGS testing with mNSCLC in current Canadian practice resulted in more patients with an identified mutation, shorter time to appropriate targeted therapy initiation, and lower total testing costs compared to single-gene strategies.

Total cost of testing for genomic alterations associated with next-generation sequencing versus polymerase chain reaction testing strategies among patients with metastatic non-small cell lung cancer.

BACKGROUND: To assess the total cost of testing associated with next-generation sequencing (NGS) versus polymerase chain reaction (PCR) testing strategies among patients with metastatic non-small cell lung cancer (mNSCLC) from a Medicare and US commercial payer's perspective. MATERIALS AND METHODS: A decision tree model considered testing for genomic alterations in EGFR, ALK, ROS1, BRAF, KRAS, MET, HER2, RET, NTRK1 among patients with newly diagnosed mNSCLC using (1) liquid or tissue biopsy NGS tests, (2) exclusionary mutation (KRAS) test followed by sequential PCR tests, (3) sequential PCR tests, or (4) hotspot panel PCR tests. The alteration test sequence followed clinical guideline recommendations. Inputs based on literature, expert opinion, or assumptions included prevalence of mNSCLC, proportion of patients using each testing strategy (50% NGS [90% tissue, 10% liquid], 10% exclusionary, 10% sequential, 30% hotspot), proportion testing positive for each genomic mutation, rebiopsy rates, and costs for testing and associated medical care. Time to appropriate targeted therapy initiation and total costs were calculated for NGS, each PCR testing strategy, and all PCR strategies combined. RESULTS: Among a hypothetical plan of 1,000,000 members (75% commercial, 25% Medicare), 1,119 patients were estimated to have mNSCLC and be eligible for testing. Estimated mean time to appropriate targeted therapy was 2 weeks for NGS and 6 weeks for PCR (sequential: 9 weeks, exclusionary: 8 weeks, hotspot: 3 weeks). Mean per patient costs were $4,932 for NGS and $6,605 for PCR (exclusionary: $5,563, sequential: $6,263, hotspot: $7,066). Per patient costs were higher from a commercial perspective (NGS: $6,225; PCR: $8,430) relative to Medicare (NGS: $2,099; PCR: $2,646); nevertheless, NGS was the least costly testing strategy across plan types. CONCLUSION: NGS was associated with the fastest time to appropriate targeted therapy initiation and lowest total cost of testing compared to PCR testing strategies for newly diagnosed patients with mNSCLC.

Treatment adherence and persistence with long-acting somatostatin analog therapy for the treatment of acromegaly: a retrospective analysis.

BACKGROUND: Many patients with acromegaly require medical treatment that includes somatostatin analogs (SSAs). Long-acting SSA formulations are widely used, due in part to increased patient convenience and increased treatment adherence vs daily medications. Although medication compliance can be poor in patients with chronic conditions, adherence and persistence with these SSAs in patients with acromegaly has not been evaluated. This analysis utilized claims data to estimate treatment adherence and persistence for lanreotide depot and long-acting octreotide in this population. METHODS: This retrospective analysis used the MarketScan® database (~100 payors, 500 million claims in the US), which was searched between January 2007 and June 2012 to identify patients with acromegaly taking either lanreotide depot or long-acting octreotide. Patients switching treatments were excluded. Treatment adherence was assessed using medication possession ratio (MPR; number of doses dispensed in relation to dispensing period; ≥80% is considered adherent), injection count, and treatment time. Persistence was estimated by Kaplan-Meier analyses and Cox proportional hazards modeling. A washout period, defined as no acromegaly-related prescription activity 180 days prior to the index date, was employed to minimize effects of prior therapy and focus on patients more likely to be treatment-naïve. RESULTS: Altogether 1308 patients with acromegaly receiving a single SSA for treatment (1127 octreotide, 181 lanreotide) who had not switched treatments were identified. Mean MPR in patients with a 180-day washout (n = 663) was 89% for those receiving octreotide (n = 545) and 87% for those receiving lanreotide (n = 118). Median number of days on therapy was 169 (95% CI 135-232) for octreotide patients and 400 (95% CI 232-532) for lanreotide patients. The point estimate of the Cox proportional hazard ratio for stopping treatment was 1.385 for octreotide vs lanreotide (95% CI 1.079-1.777), suggesting a 38.5% increased risk for stopping octreotide before lanreotide. CONCLUSIONS: Treatment adherence was similarly good for both injectable SSA treatments studied, at 87% or greater. Persistence was greater with lanreotide than octreotide and the risk of discontinuing therapy was lower with lanreotide than octreotide. Further studies to determine factors leading to these differences in persistence or to predict discontinuation of therapy may aid in clinical management of these patients.

A mixed treatment comparison to compare the efficacy and safety of botulinum toxin treatments for cervical dystonia.

A systematic pair-wise comparison of all available botulinum toxin serotype A and B treatments for cervical dystonia (CD) was conducted, as direct head-to-head clinical trial comparisons are lacking. Five botulinum toxin products: Dysport(®) (abobotulinumtoxinA), Botox(®) (onabotulinumtoxinA), Xeomin(®) (incobotulinumtoxinA), Prosigne(®) (Chinese botulinum toxin serotype A) and Myobloc(®) (rimabotulinumtoxinB) have demonstrated efficacy for managing CD. A pair-wise efficacy and safety comparison was performed for all toxins based on literature-reported clinical outcomes. Multi-armed randomized controlled trials (RCTs) were identified for inclusion using a systematic literature review, and assessed for comparability based on patient population and efficacy outcome measures. The Toronto Western Spasmodic Torticollis Rating Scale (TWSTRS) was selected as the efficacy outcome measurement for assessment. A mixed treatment comparison (MTC) was conducted using a Bayesian hierarchical model allowing indirect comparison of the interventions. Due to the limitation of available clinical data, this study only investigated the main effect of toxin treatments without explicitly considering potential confounding factors such as gender and formulation differences. There was reasonable agreement between the number of unconstrained data points, residual deviance and pair-wise results. This research suggests that all botulinum toxin serotype A and serotype B treatments were effective compared to placebo in treating CD, with the exception of Prosigne. Based on this MTC analysis, there is no significant efficacy difference between Dysport, Botox, Xeomin and Myobloc at week four post injection. Of the adverse events measured, neither dysphagia nor injection site pain was significantly greater in the treatment or placebo groups.