Cost-effectiveness analysis of utidelone plus capecitabine for metastatic breast cancer in China

Abstract

Objective: To estimate the cost-effectiveness of utidelone plus capecitabine therapy compared to capecitabine alone in patients with metastatic breast cancer (MBC) resistant to anthracyclines and taxanes treatment in the Chinese context and provide a reference for the marketing of utidelone in China.

Methods: A Markov model was developed based on the NCT02253459 clinical trial to simulate the clinical course of patients with metastatic breast cancer who had received taxanes and anthracycline therapy. The quality-adjusted life years (QALYs) and Incremental Cost Effectiveness Ratio (ICER) were then analyzed to evaluate the benefits. Two-parametric Weibull distribution was conducted to fit PFS and OS curves by using R. Sensitivity analyses were performed to evaluate the stability of the model designed.

Results: The addition of utidelone increased the cost and QALYs by $13,370.25 and 0.1961, respectively, resulting in an increased ICER of $68,180.78 per QALY. The most sensitive influential parameter on ICER was the price of utidelone. At the threshold of willingness-to-pay (WTP) of $24,380 (3 per capita GDP of China), the cost of utidelone per 30?mg of less than $18.5, $33.7, and greater than $48.8 resulted in a 100%, 50%, and 0% possibility of cost-effectiveness, respectively. The addition of utidelone was not cost-effective when it was $115.4 per 30?mg—the price of its analog paclitaxel. In consideration of varied economics levels across China, cost-effectiveness could be achieved with the price of utidelone ranging from $5.2 to $35.9.

Limitations: The survival curves extended beyond the follow-up time horizon, of which data were generated not from the real analyses but from our established two-parameter Weibull survival model.

Conclusion: It is recommended that the price of utidelone would be less than $18.5 per 30?mg in order to obtain cost-effectiveness for metastatic breast cancer patients resistant to anthracyclines and taxanes treatment in China.     

Projecting total costs and health consequences of increasing mt-sDNA utilization for colorectal cancer screening from the payer and integrated delivery network perspectives

Abstract

Aims: To evaluate total costs and health consequences of a colorectal cancer (CRC) screening program with colonoscopy, fecal immunochemical tests (FIT), and expanded use of multitarget stool DNA (mt-sDNA) from the perspectives of Integrated Delivery Networks (IDNs) and payers in the United States.

Materials and methods: We developed a budget impact and cost-consequence model that simulates CRC screening for eligible 50- to 75-year-old adults. A status quo scenario and an increased mt-sDNA scenario were modeled. The status quo includes the current screening mix of colonoscopy (83%), FIT (11%), and mt-sDNA (6%) modalities. The increased mt-sDNA scenario increases mt-sDNA utilization to 28% over 10 years. Costs for both the IDN and the payer perspectives incorporated diagnostic and surveillance colonoscopies, adverse events (AEs), and CRC treatment. The IDN perspective included screening program costs, composed of direct nonmedical (e.g. patient navigation) and indirect (e.g. administration) costs. It was assumed that IDNs do not incur the costs for stool-based screening tests or bowel preparation for colonoscopies.

Results: In a population of one million covered lives, the 10-year incremental cost savings incurred by increasing mt-sDNA utilization was $16.2 M for the IDN and $3.3 M for the payer. The incremental savings per-person-per-month were $0.14 and $0.03 for the IDN and payer, respectively. For both perspectives, increased diagnostic colonoscopy costs were offset by reductions in screening colonoscopies, surveillance colonoscopies, and AEs. Extending screening eligibility to 45- to 75-year-olds slightly decreased the overall cost savings.

Limitations: The natural history of CRC was not simulated; however, many of the utilized parameters were extracted from highly vetted natural history models or published literature. Direct nonmedical and indirect costs for CRC screening programs are applied on a per-person-per modality basis, whereas in reality some of these costs may be fixed.

Conclusions: Increased mt-sDNA utilization leads to fewer colonoscopies, less AEs, and lower overall costs for both IDNs and payers, reducing overall screening program costs and increasing the number of cancers detected while maintaining screening adherence rates over 10 years.     

Budget impact analysis of niraparib and olaparib for maintenance treatment of platinum-sensitive,recurrent ovarian cancer in the US

Aims: This study aimed to evaluate the budget impact of niraparib and olaparib in patients with platinum-sensitive, recurrent ovarian cancer from a US third party payer perspective.

Materials and methods: A budget impact model was constructed to assess the additional per member per month (PMPM) costs associated with the introduction of niraparib and olaparib, two poly ADP-ribose polymerase ribose polymerase (PARP) inhibitors recently approved to be used in platinum-sensitive, recurrent ovarian cancer patients with and without a gBRCA mutation. The model assessed both pharmacy costs and medical costs. Pharmacy costs included adjusted drug costs, coinsurance, and dispensing fees. Medical costs included costs associated with disease monitoring and management of adverse events from the treatment. Epidemiological data from the literature were used to estimate the target population size. The analysis used 1-year time frame, and patients were assumed on treatment until disease progression or death. All costs were computed in 2017 USD. One-way sensitivity analyses were conducted to evaluate the model robustness.

Results: In a hypothetical plan of 1,000,000 members, 206 patients were estimated to be potential candidates for niraparib or olaparib maintenance treatment after applying all epidemiological parameters. At listed 30-day supply WAC prices of $14,750 for niraparib and $13,482 for olaparib, budget impacts of these two drugs were $0.169 PMPM and $0.156 PMPM, respectively, most of which were contributed by pharmacy costs. Sensitivity analyses suggested that assumptions around market share, platinum-sensitive rate after first treatment, and WAC prices affected results the most.

Limitations: In this model, it was assumed that adopting niraparib and olaparib would not affect utilization of existing medications. Also, the estimated clinical parameters from clinical trials could differ from real-world data.     

An evaluation of consumer participation in hospital breast services

Community expectations and research demonstrate that consumers play an important role in shaping services for women with breast cancer. Consumer contribution has been mandated recently in Victoria, Australia, to ensure the inclusion of consumer involvement in the planning and decision‐making processes within health organizations. As part of the redevelopment of breast services in Victoria, Southern Health has been funded to plan local improvements to care co‐ordination for women diagnosed with breast cancer in the southern metropolitan area of Melbourne. The establishment of effective consumer participation in breast services is an integral aspect of this project and a range of initiatives has been undertaken to achieve meaningful consumer involvement including the appointment of a consumer advisor; appointment of staff with extensive knowledge in women's health and community development to the project; establishment of a consumer reference group; and plans made to improve the receptivity of health service systems to consumer input. A preliminary evaluation of this ongoing project has indicated that a productive role for consumers in service practice review and policy and planning activities has been established and some change and engagement of staff has occurred. There is still work to be done to promote the involvement of a wider range of health professionals and to increase the level of trust between consumers and staff.     

晚期乳腺癌患者的临床治疗及其护理效果

目的探讨晚期乳腺癌患者的临床治疗及其护理效果。方法选取我院近年收治的晚期乳腺癌患者共52例,采用药物治疗、手术治疗、内分泌治疗,并采取针对性护理干预,比较患者的术后并发症情况。结果患者并发症发生率达到34.6%,其中以皮下积液为主,另存在术侧上肢水肿以及皮瓣坏死2类并发症。所有患者术后经过护理干预后均痊愈而顺利出院。结论对晚期乳腺癌患者的临床治疗可多元化,临床护理也应从多角度展开,注重并发症的预防及护理干预,提升治疗的有效性。     

大肠癌结肠镜检查与分析

目的探讨结肠镜检查大肠癌患者的发病情况及发病率。方法本组资料源于我院2013年1月至2014年6月结肠镜检查并经病理活检确诊的58例大肠癌患者。所有受检者均在电子结肠镜下观察,对有溃疡、肿块、糜烂、狭窄等肿瘤病灶部位取活检行病理诊断,明确组织类型。结果 58例大肠癌患者就诊时主要症状腹痛15例,肉眼血便10例,腹泻3例,腹部包块2例,贫血1例;所有患者粪便潜血实验阳性46例;直肠39例,左半结肠9例,右半结肠10例;溃疡型22例,肿块型27例,浸润型9例;腺癌56例,黏液腺癌2例。结论兴山县大肠癌肠镜检出率相对较高,发病部位以直肠为主,本地区应大力开展大肠癌早期诊断、早期治疗工作。     

宫颈癌新辅助化疗的临床疗效

目的探讨宫颈癌术前应用新辅助化疗的临床疗效。方法 68例宫颈癌术前采用2个疗程的新辅助化疗后进行手术,观察近期疗效。结果 68例患者化疗2个疗程后的临床总有效率为70.1%。经过术前辅助化疗后,进行广泛性子宫切除加盆腔淋巴结清扫手术,全部患者均顺利切除病灶。结论术前新辅助化疗对宫颈癌Ⅰb1~Ⅱb期患者治疗安全有效,可缩小肿瘤体积,提高手术切除率及疗效,进一步改善患者的生活质量。     

Confirming the timing of phase-based costing in oncology studies: a case example in advanced melanoma

Aims: The utilization of healthcare services and costs among patients with cancer is often estimated by the phase of care: initial, interim, or terminal. Although their durations are often set arbitrarily, we sought to establish data-driven phases of care using joinpoint regression in an advanced melanoma population as a case example.

Methods: A retrospective claims database study was conducted to assess the costs of advanced melanoma from distant metastasis diagnosis to death during January 2010–September 2014. Joinpoint regression analysis was applied to identify the best-fitting points, where statistically significant changes in the trend of average monthly costs occurred. To identify the initial phase, average monthly costs were modeled from metastasis diagnosis to death; and were modeled backward from death to metastasis diagnosis for the terminal phase. Points of monthly cost trend inflection denoted ending and starting points. The months between represented the interim phase.

Results: A total of 1,671 patients with advanced melanoma who died met the eligibility criteria. Initial phase was identified as the 5-month period starting with diagnosis of metastasis, after which there was a sharp, significant decline in monthly cost trend (monthly percent change [MPC]?=?–13.0%; 95% CI?=?–16.9% to –8.8%). Terminal phase was defined as the 5-month period before death (MPC?=?–14.0%; 95% CI?=?–17.6% to –10.2%).

Limitations: The claims-based algorithm may under-estimate patients due to misclassifications, and may over-estimate terminal phase costs because hospital and emergency visits were used as a death proxy. Also, recently approved therapies were not included, which may under-estimate advanced melanoma costs.

Conclusions: In this advanced melanoma population, optimal duration of the initial and terminal phases of care was 5 months immediately after diagnosis of metastasis and before death, respectively. Joinpoint regression can be used to provide data-supported phase of cancer care durations, but should be combined with clinical judgement.     

Real-world utilization of molecular diagnostic testing and matched drug therapies in the treatment of metastatic cancers

Aims: To assess the frequency of biopsies and molecular diagnostic testing (human DNA/RNA analysis), anti-cancer drug use (genomically-matched targeted therapy [GMTT], unmatched targeted therapy [UTT], endocrine therapy [ET], and chemotherapy [CT]), and medical service costs among adults with metastatic cancer.

Methods: Adults diagnosed with metastatic breast, non-small cell lung (NSCLC), colorectal, head and neck, ovarian, and uterine cancer (2010Q1–2015Q1) were identified in the OptumHealth Care Solutions claims database and followed from first metastatic diagnosis for ≥1 month and until the end of data availability. Utilization was assessed for each cancer cohort (all and patients aged ≥65 years); per-patient-per-month (PPPM) medical service costs were assessed for all patients. Testing frequency estimates were applied to Surveillance, Epidemiology, and End Results Program data to estimate the number of untested patients (2010–2014).

Results: Patients with metastatic cancer (n?=?8,193; breast [n?=?3,414], NSCLC [n?=?2,231], colorectal [n?=?1,611], head and neck [n?=?511], ovarian [n?=?275], and uterine [n?=?151]) were 63 years old (mean), with 11.1–22.2 months of observation. Biopsy and molecular diagnostic testing frequencies ranged from 7% (uterine) to 73% (ovarian), and from 34% (head and neck) to 52% (breast), respectively. Few were treated with GMTT (breast, 11%; NSCLC, 9%; colorectal, 6%). Treatment with UTT ranged from 0.7% (uterine) to 21% (colorectal). Biopsy, diagnostic testing, and anti-cancer drug therapy were less frequent for those ≥65 years. Medical service costs (PPPM, mean) ranged from $6,618 (head and neck) to $9,940 (ovarian). The estimated number of untested new patients with metastatic cancer was 636,369 (all) and 341,397 (≥65).

Limitations: In addition to the limitations of claims analyses, diagnostic testing frequency may be under-estimated if patients underwent testing prior to study inclusion.

Conclusions: The low frequency of molecular diagnostic testing suggests there are opportunities to better inform management of patients with advanced cancer, particularly decisions to treat with GMTT.     

The economic burden of common adverse events associated with metastatic colorectal cancer treatment in the United States

Aims: Adverse events (AEs) associated with treatments for metastatic colorectal cancer (mCRC) may compromise the course of treatment, impact quality-of-life, and increase healthcare resource utilization. This study assessed the direct healthcare costs of common AEs among mCRC patients in the US.

Methods: Adult mCRC patients treated with chemotherapy or targeted therapies were identified from administrative claims databases (2009–2014). Up to the first three mCRC treatment episodes per patient were considered and categorized as with or without the AE system/organ category during the episode. Total healthcare costs (2014 USD) were measured by treatment episode and reported on a monthly basis. Treatment episodes with the AE category were matched by treatment type and line of treatment to those without the AE category. Adjusted total cost differences were estimated by comparing costs during treatment episodes with vs without the AE category using multivariate regression models; p-values were estimated with bootstrap.

Results: A total of 4158 patients with ≥1 mCRC treatment episode were included (mean age?=?59 years; 58% male; 60% with liver and 14% with lung metastases; 2,261 [54%] with a second and 1,115 [27%] with a third episode). On average, two treatment episodes were observed per patient with an average length of 166 days per episode. Adjusted monthly total cost difference by AE category included hematologic ($1,480), respiratory ($1,253), endocrine/metabolic ($1,213), central nervous system (CNS; $1,136), and cardiovascular ($1,036; all p?Limitations: Claims do not include information on the cause of AEs, and potentially less severe AEs may not have been reported by the physician when billing the medical service. This study aimed to assess the association between costs and AEs and not the causation of AEs by treatment.

Conclusions: The most costly AEs among mCRC patients were hematologic, followed by respiratory, endocrine/metabolic, CNS, and cardiovascular.