Cost-Effectiveness of Apomorphine Sublingual Film as an "On-Demand" Treatment for "OFF" Episodes in Patients with Parkinson's Disease

Background: “On-demand” treatments approved in the United States (US) for “OFF” episodes in Parkinson’s disease (PD) include apomorphine hydrochloride injection (SC-APO), apomorphine sublingual film (APL), and levodopa inhalation powder (CVT-301). APL received US approval in 2020, and its cost-effectiveness has not been compared with SC-APO and CVT-301. Objective: To develop a cost-effectiveness analysis model comparing APL versus SC-APO and CVT-301 for treatment of patients with PD experiencing “OFF” episodes from a US payer perspective. Methods: The model estimated total costs and effectiveness for each comparator arm, informed from the treatments’ pivotal studies or literature, over a 10-year horizon. Total and incremental patient costs (in 2020 US dollars), total time spent without “OFF” episode symptoms, and quality-adjusted life years (QALY) gained were summarized and compared. Incremental cost-effectiveness ratios for APL versus SC-APO and CVT-301 were estimated and expressed as incremental patient costs per patient QALY gained and incremental cost per “OFF” hour avoided. Scenario analyses varying inputs and including caregiver costs were also conducted. Results: In the base case, APL had the lowest total “on-demand” treatment costs ($42,095) compared with SC-APO ($276,320; difference: –$234,225) and CVT-301 ($69,577; difference: –$27,482) over the 10-year horizon. APL was also associated with the highest utility, with incremental QALYs of 0.019 versus SC-APO and 0.235 versus CVT-301. APL was dominant over CVT-301 in terms of incremental cost per “OFF” hour, and dominant over both CVT-301 and SC-APO in terms of incremental cost per QALY gained. In all scenario analyses, APL was dominant against both SC-APO and CVT-301, confirming the robustness of the base-case results. Discussion: APL was dominant compared with both comparator arms, being less costly and more effective on average than SC-APO and CVT-301 in terms of QALYs. For SC-APO, cost-effectiveness of APL was driven by lower “on-demand” treatment costs and adverse event–related disutilities. For CVT-301, cost-effectiveness of APL was driven by lower “on-demand” treatment costs and substantially higher efficacy. Conclusions: From a US payer perspective, APL represents a cost-effective option compared with SC-APO and CVT-301 for treatment of “OFF” episodes in patients with PD.

Economic evaluation of advanced practice physiotherapy models of care: a systematic review with meta-analyses

Background The objective of this systematic review is to appraise evidence on the economic evaluations of advanced practice physiotherapy (APP) care compared to usual medical care. Methods Systematic searches were conducted up to September 2021 in selected electronic bibliographical databases. Economic evaluation studies on an APP model of care were included. Economic data such as health care costs, patient costs, productivity losses were extracted. Methodological quality of included studies was assessed with the Effective Public Health Practice Project tool and the Critical Appraisal Skills Programme checklist. Meta-analyses were performed and mean differences (MD) in costs per patient were calculated using random-effect inverse variance models. Certainty of the evidence was assessed with the GRADE Approach. Results Twelve studies (n = 14,649 participants) including four randomized controlled trials, seven analytical cohort studies and one economic modeling study were included. The clinical settings of APP models of care included primary, emergency and specialized secondary care such as orthopaedics, paediatrics and gynaecology. The majority of the included participants were adults with musculoskeletal disorders (n = 12,915). Based on low quality evidence, health system costs including salaries, diagnostic tests, medications, and follow-up visits were significantly lower with APP care than with usual medical care, at 2 to 12-month follow-up (MD: − 145.02 €/patient; 95%CI: − 251.89 to − 38.14; n = 7648). Based on low quality evidence, patient costs including travel and paid medication prescriptions, or treatments were significantly higher with APP care compared to usual medical care, at 2 to 6-month follow-up (MD: 22.18 €/patient; 95%CI: 0.40 to 43.96; n = 1485). Based on very low quality evidence, no significant differences in productivity losses per patient were reported between both types of care (MD: 450 €/patient; 95%CI: − 80 to 970; n = 819). Conclusions This is the first systematic review and meta-analysis on the economic evaluation of APP models of care. Low quality evidence suggests that APP care might result in lower health care costs, but higher patient costs compared to usual medical care. Costs differences may vary depending on various factors such as the cost methodology used and on the clinical setting. More evidence is needed to evaluate cost benefits of APP models of care.

Healthcare Resource Utilization and Costs of Advanced Systemic Mastocytosis Among Medicare Fee for Service Beneficiaries

Systemic mastocytosis (SM) is a rare mast cell neoplasm driven by KIT D816V mutation. Advanced SM (AdvSM) includes three disease subtypes: aggressive SM, SM with associated hematological neoplasm, and mast cell leukemia. 1 Advanced disease onset often develops in patients above the age of 60. 1 Patients with AdvSM experience a range of severe symptoms including organ damage and shortened survival. 2 There is limited research quantifying the impact of AdvSM on healthcare resource utilization (HCRU) and costs, particularly within the Medicare population. This retrospective study compared direct HCRU and costs in Medicare beneficiaries with AdvSM to a matched cohort of beneficiaries without SM. This study used Centers for Medicare and Medicaid Services-sourced 100% Medicare Fee for Service (FFS) claims (Parts A/B/D) to identify newly diagnosed SM patients with >2 medical claims for SM (ICD-10-CM Dx codes: D47.02 OR C94.30 OR C94.31 OR C94.32 OR C96.21) between 01/01/2017 and 12/31/2018. The index date was the date of first observed SM diagnosis code. A claims-based algorithm was used to determine AdvSM subtype. Patients were required to have continuous enrollment in Medicare Parts A/B/D for 12 months pre- and post-index. AdvSM patients were direct matched (1:1) to a non-SM control cohort on age, sex, race, index year, Medicare-Medicaid dual eligibility, and Charlson Comorbidity Index score. HCRU and costs were assessed pre- and post-index. Chi-square and t-tests were used to evaluate differences in outcomes between AdvSM and non-SM patients. Medical costs are reported in 2021 USD. After matching, there were 339 AdvSM and 339 non-SM patients. Mean [SD] age was 68.2 [13.2] among AdvSM and 68.5 [13.9] among non-SM patients. More than 25% of patients were <65 years old at index and qualified for Medicare due to a preexisting disability. Majority of patients were female (59%) and White (91%). Compared to non-SM patients, AdvSM patients had more specialist and emergency department (ED) visits during the baseline period. Baseline prevalence of asthma (26% vs. 16%, p=0.0009) and any malignancy (60% vs. 23%, p<0.0001) was higher among AdvSM patients compared to controls, and lower for hypertension (70% vs. 81%, p=0.0006), and diabetes with and without complications (28% vs. 52%; 16% vs. 33%; both p<0.0001). During the 12 months post-index, all cause total healthcare expenditures (Parts A/B/D) were significantly higher for AdvSM patients than for non-SM comparator patients (mean [SD]: $123,412 [$180,386] vs. $47,988 [$64,693]; p<0.0001). Pharmacy costs (Part D) were a key driver of the total, accounting for 41% ($50,494 [$140,561]) of the total costs for AdvSM patients and 19% ($9,221 [$22,270]) of the total for non-SM patients. Inpatient hospital stays made up 24% of AdvSM patient costs and 26% of non-SM patient costs. More AdvSM than non-SM patients had ≥1 inpatient hospitalization (58% vs. 42%; p=0.0001), and length of hospital stay was significantly longer for AdvSM patients (13.1 [26.95] days) than for comparator patients (5.22 [12.66]; p<0.0001). Mean [SD] number of ED visits per patient was over twice as high for AdvSM than for non-SM patients (3.7 [12.0] vs. 1.6 [3.6]; p=0.0026). AdvSM patients had more than 5 times as many oncology/hematology and allergy/immunology physician office visits per patient versus non-SM controls (10.9 [21.5] vs. 2.0 [7.2]; 2.3 [7.4] vs. 0.1 [0.7]; both p<0.0001). AdvSM patients were higher utilizers of epinephrine (29.2% vs. <3% non-SM patients; p<0.0001), oral and systemic corticosteroids (54.0% vs. 38.1%, p<0.0001; 51.9% vs. 41.6%, p=0.0071), chemotherapy (12.09% vs. 6.78%; p=0.0181), and omalizumab (4.7% vs. 0.0%, p<0.0001). AdvSM Medicare FFS patients were more resource intensive and had 2.5 times higher per-patient healthcare costs in the 12 months following SM diagnosis compared to a matched cohort of non-SM patients. These costs were driven by significantly higher rates of inpatient stays, more frequent ED and physician outpatient visits, and higher utilization of multiple medications. Notably, AdvSM patients in this analysis included a larger proportion of patients <65 years old with preexisting disability compared to the broader Medicare population (~25% vs. 14%), suggesting that AdvSM patients may be more likely to qualify for Medicare due to disability rather than age compared to the overall Medicare population. Further research in this area is warranted. Disclosures Sullivan: Blueprint Medicines: Current Employment, Current equity holder in publicly-traded company, Divested equity in a private or publicly-traded company in the past 24 months. Cohen: Blueprint Medicines: Current Employment, Current equity holder in publicly-traded company, Divested equity in a private or publicly-traded company in the past 24 months. Norregaard: Blueprint Medicines: Current Employment, Current equity holder in publicly-traded company. Nguyen: Blueprint Medicines: Current Employment. Sloan: Blueprint Medicines: Current Employment, Current equity holder in publicly-traded company, Current holder of individual stocks in a privately-held company, Current holder of stock options in a privately-held company. Petrilla: Blueprint Medicines: Other: Allison Petrilla is an employee of Avalere Health, which received consulting fees from Blueprint Medicines for this study.. Silverstein: Blueprint Medicines: Other: Alison Silverstein is an employee of Avalere Health, which received consulting fees from Blueprint Medicines for this study.. Murunga: Blueprint Medicines: Other: Anne Murunga is an employee of Avalere Health, which received consulting fees from Blueprint Medicines for this study.. Schinkel: Blueprint Medicines: Other: Jill Schinkel is an employee of Avalere Health, which received consulting fees from Blueprint Medicines for this study..

Treatment Patterns and Economic Burden Among Elderly Patients with Acute Myeloid Leukemia Treated with Hypomethylating Agents: A SEER-Medicare Analysis

Introduction: Previously, we assessed the economic burden of newly diagnosed acute myeloid leukemia (AML) among Medicare patients and found that the economic burden of relapse is high, at approximately 1.2 and 1.6 times the monthly per-patient costs associated with early and late post-remission therapy, respectively (Tabah A, et al. Blood 2020:136(suppl 1):45). A notably high proportion of patients (55%) received ≥ 1 cycle of a hypomethylating agent (HMA). Therefore, the objective of this study was to assess the economic burden of AML among a subgroup of elderly patients who received only HMA monotherapy during induction and then achieved disease remission. Healthcare resource utilization (HCRU) and costs associated with various phases (ie, induction, post-remission therapy, and post-relapse) were assessed. Methods: A retrospective analysis was conducted using the Surveillance, Epidemiology, and End Results (SEER)-Medicare database, which comprised Medicare claims (parts A, B, and D from 2007 through 2016) and the US National Cancer Institute's SEER database (cancer diagnoses from 2007 to 2015). Included patients had an AML diagnosis in the SEER registry, were ≥ 65 years of age at the AML diagnosis date, had initiated HMA (and no other active treatment) in the outpatient (OP) setting during the first induction cycle post-AML diagnosis, and had an International Classification of Diseases, Ninth/Tenth Revision (ICD-9/10) diagnosis code for AML remission following the initiation of induction therapy. Patients were excluded if they had another blood malignancy (including a history of myelodysplastic syndromes), had received hematopoietic stem cell transplantation, or were enrolled in a clinical trial. The induction therapy period was defined as the first initiation of HMA post diagnosis (index date) to the end of the cycle during which a patient had a code for AML remission. The 6-month period prior to the index date was defined as the baseline period. The post-remission phase ended at the earliest of relapse or end of follow-up (ie, death, end of eligibility, or end of available data [December 31, 2016]). The post-relapse phase was from the date of first AML relapse ICD-9/10 code after remission to the end of follow-up. Patient characteristics during the baseline period were summarized descriptively. HCRU and costs (adjusted to 2019 US dollars) associated with induction and post-remission therapy were assessed during days that were part of a treatment cycle. The average per-patient monthly HCRU and costs were reported for the induction, post-remission, and post-relapse phases. Results: A total of 71 patients with newly diagnosed AML (azacitidine: n = 31; decitabine: n = 40) received HMA induction therapy and achieved remission. The median age at AML diagnosis was 78.8 years, 50.7% of patients were male, and 85.9% were White. The mean ± standard deviation (median) time from index date to the end of follow-up was 16.0 ± 12.3 (14.0) months. A total of 63.4% of patients (n = 45) received post-remission therapy. Among all patients, 43.7% relapsed and 85.9% died by the end of follow-up. OP visits were the most common type of visit across all phases with 95.6% of patients having ≥ 1 OP visit during post-remission therapy and 83.9% during the post-relapse phase. Inpatient (IP) visits were highest in the post-relapse phase with 77.4% of patients having ≥ 1 IP visit. The monthly mean per-patient healthcare costs were highest for the post-relapse phase, followed by post-remission therapy, and induction (Table). Costs associated with the OP setting were the greatest contributor to the induction costs (48.9%) while costs associated with the IP setting were the drivers of the costs in the post-remission therapy (56.2%) and post-relapse (72.8%) phases. Conclusions: The economic burden of AML treated with HMA induction therapy was highest in the post-relapse phase, at approximately 1.7 and 1.6 times the monthly per-patient costs during the induction and post-remission therapy phases, respectively. In addition, IP costs made up nearly two-thirds of total monthly per-patient costs in the post-relapse phase, up from approximately 44% and 56% of the induction and post-remission therapy phases, respectively. Treatment options that extend the post-remission phase would reduce the high economic burden associated with AML relapse. Figure 1 Figure 1. Disclosures Brunner: Celgene, Forty Seven Inc, Jazz: Other: Advisory Board; Novartis, Celgene, Takeda, AstraZeneca: Research Funding. Huggar: Bristol Myers Squibb: Current Employment, Current equity holder in publicly-traded company. Copher: Bristol Myers Squibb: Current Employment, Current equity holder in publicly-traded company. Zhou: Sanofi: Research Funding; Analysis Group: Current Employment, Other: Employee of Analysis Group which received consulting fees for this project . Zichlin: Analysis Group, Inc.: Consultancy, Current Employment; GlaxoSmithKline: Research Funding. Anderson: Analysis Group, which received consultancy fees from GSK: Consultancy, Current Employment. Downes: nalysis Group (employment), Bristol Myers Squibb (consultancy): Consultancy, Current Employment. McBride: BMS: Current Employment.

A Cost–Consequence Analysis of Preemptive SLCO1B1 Testing for Statin Myopathy Risk Compared to Usual Care

There is a well-validated association between SLCO1B1 (rs4149056) and statin-associated muscle symptoms (SAMS). Preemptive SLCO1B1 pharmacogenetic (PGx) testing may diminish the incidence of SAMS by identifying individuals with increased genetic risk before statin initiation. Despite its potential clinical application, the cost implications of SLCO1B1 testing are largely unknown. We conducted a cost–consequence analysis of preemptive SLCO1B1 testing (PGx+) versus usual care (PGx−) among Veteran patients enrolled in the Integrating Pharmacogenetics in Clinical Care (I-PICC) Study. The assessment was conducted using a health system perspective and 12-month time horizon. Incremental costs of SLCO1B1 testing and downstream medical care were estimated using data from the U.S. Department of Veterans Affairs’ Managerial Cost Accounting System. A decision analytic model was also developed to model 1-month cost and SAMS-related outcomes in a hypothetical cohort of 10,000 Veteran patients, where all patients were initiated on simvastatin. Over 12 months, 13.5% of PGx+ (26/193) and 11.2% of PGx− (24/215) participants in the I-PICC Study were prescribed Clinical Pharmacogenetics Implementation Consortium (CPIC) guideline-concordant statins (Δ2.9%, 95% CI −4.0% to 10.0%). Differences in mean per-patient costs for lipid therapy prescriptions, including statins, for PGx+ compared to PGx− participants were not statistically significant (Δ USD 9.53, 95% CI −0.86 to 22.80 USD). Differences in per-patient costs attributable to the intervention, including PGx testing, lipid-lowering prescriptions, SAMS, laboratory and imaging expenses, and primary care and cardiology services, were also non-significant (Δ− USD 1004, 95% CI −2684 to 1009 USD). In the hypothetical cohort, SLCO1B1-informed statin therapy averted 109 myalgias and 3 myopathies at 1-month follow up. Fewer statin discontinuations (78 vs. 109) were also observed, but the SLCO1B1 testing strategy was 96 USD more costly per patient compared to no testing (124 vs. 28 USD). The implementation of SLCO1B1 testing resulted in small, non-significant increases in the proportion of patients receiving CPIC-concordant statin prescriptions within a real-world primary care context, diminished the incidence of SAMS, and reduced statin discontinuations in a hypothetical cohort of 10,000 patients. Despite these effects, SLCO1B1 testing administered as a standalone test did not result in lower per-patient health care costs at 1 month or over 1 year of treatment. The inclusion of SLCO1B1, among other well-validated pharmacogenes, into preemptive panel-based testing strategies may provide a better balance of clinical benefit and cost.

Cost-minimisation analysis of a treat-and-extend regimen with anti-VEGFs in patients with neovascular age-related macular degeneration

Purpose Although intraocular anti-vascular endothelial growth factors (anti-VEGFs) are effective as treatment of neovascular age-related macular degeneration (nAMD), the (economic) burden on the healthcare system is considerable. A treat-and-extend (T&E) regimen is associated with a lower number of injections without compromising the effectiveness and can therefore help optimise nAMD treatment. This study investigates the per-patient costs associated with nAMD treatment, when using aflibercept, bevacizumab, or ranibizumab with a T&E regimen. Methods In this cost-minimisation model, the per-patient costs in the Netherlands were modelled using a healthcare payers’ perspective over a 3-year time horizon with the assumption that efficacy of treatments is similar. Additionally, the break-even price of the different anti-VEGFs was calculated relative to the cheapest option and injection frequency. Results The injection frequency varied from 14.2 for aflibercept to 27.4 for bevacizumab in 3 years. Nonetheless, bevacizumab remains the cheapest treatment option (€14,215), followed by aflibercept (€18,202) and ranibizumab (€31,048). The medication covers the majority of the per-patient costs for aflibercept and ranibizumab, while administration covers the majority of the per-patient costs for bevacizumab. The break-even prices of aflibercept and ranibizumab are respectively €507 and €60.58 per injection. Brolucizumab was included in the scenario analysis and was more expensive than aflibercept (€20,446). Brolucizumab should reduce to 13.8 injections over 3 years to be as costly as aflibercept. Conclusion Bevacizumab is the cheapest anti-VEGF treatment. The list prices of all anti-VEGFs should reduce to be as costly as bevacizumab. Aflibercept is the second-choice treatment and so far brolucizumab is not.

Extended Roles in Primary Care When Physiotherapist-Initiated Referral to X-Ray Can Save Time and Reduce Costs

ABSTRACT Background We evaluated an extended role for the physiotherapist in primary care in referring patients to plain X-ray. Methods This prospective cohort study was set in a single region in Sweden. It included 20 physiotherapists who were educated in a one-day training in performing referral to X-ray, along with 107 patients with musculoskeletal disorders who were referred to X-ray. We evaluated that referral quality, patient and physiotherapist satisfaction, and calculated health care and patient costs. Results All referrals fulfilled the basic requirements of quality, and 78% were classified as good, fulfilling all criteria. Both patients and physiotherapists were satisfied with the extended role for the physiotherapist that decreased waiting time to diagnosis and to adequate treatment. Costs were reduced for patients (by €53/patient) and health care (by €6286.2/107 patients). The cost to visit a physician was twice that of a physiotherapist visit. Conclusions An extended role for physiotherapists in primary care in referring patients to X-ray was effective and safe for patients and reduced costs for patients and for health care. Physiotherapists in primary care were able to refer patients to X-ray after one day of training, and the extended role freed up 45 minutes of physician time for each patient with a musculoskeletal disorder in need of an X-ray.

Patient costs for prevention of mother-to-child HIV transmission and antiretroviral therapy services in public health facilities in Zimbabwe

Zimbabwe has made large strides in addressing HIV. To ensure a continued robust response, a clear understanding of costs associated with its HIV program is critical. We conducted a cross-sectional evaluation in 2017 to estimate the annual average patient cost for accessing Prevention of Mother-To-Child Transmission (PMTCT) services (through antenatal care) and Antiretroviral Treatment (ART) services in Zimbabwe. Twenty sites representing different types of public health facilities in Zimbabwe were included. Data on patient costs were collected through in-person interviews with 414 ART and 424 PMTCT adult patients and through telephone interviews with 38 ART and 47 PMTCT adult patients who had missed their last appointment. The mean and median annual patient costs were examined overall and by service type for all participants and for those who paid any cost. Potential patient costs related to time lost were calculated by multiplying the total time to access services (travel time, waiting time, and clinic visit duration) by potential earnings (US$75 per month assuming 8 hours per day and 5 days per week). Mean annual patient costs for accessing services for the participants was US$20.00 [standard deviation (SD) = US$80.42, median = US$6.00, range = US$0.00–US$12,18.00] for PMTCT and US$18.73 (SD = US$58.54, median = US$8.00, range = US$0.00–US$ 908.00) for ART patients. The mean annual direct medical costs for PMTCT and ART were US$9.78 (SD = US$78.58, median = US$0.00, range = US$0.00–US$ 90) and US$7.49 (SD = US$60.00, median = US$0.00) while mean annual direct non-medical cost for US$10.23 (SD = US$17.35, median = US$4.00) and US$11.23 (SD = US$25.22, median = US$6.00, range = US$0.00–US$ 360.00). The PMTCT and ART costs per visit based on time lost were US$3.53 (US$1.13 to US$8.69) and US$3.43 (US$1.14 to US$8.53), respectively. The mean annual patient costs per person for PMTCT and ART in this evaluation will impact household income since PMTCT and ART services in Zimbabwe are supposed to be free.

DRG-based payment system and management accounting changes in an Indonesian public hospital: exploring potential roles of big data analytics

Purpose This study aims to provide insights into management accounting changes (MACs) and potential roles of big data analytics (BDA) in accelerating the MACs in an Indonesian public hospital as a response towards the adoption of the diagnosis-related groups (DRG)-based payment system. Design/methodology/approach A mixed-method approach was used to collect and analyse data from a referral public hospital in Indonesia. First, a BDA simulation was carried out to reveal its usefulness in predicting and evaluating patient costs, and finally improving the cost recovery rate (CRR) of each DRG case. This part formulated and tested the mathematical models that predict patient cost, the CRR and determinants (length of stay/LOS, severity/SEV, patient age/AGE and gender/SEX). For this purpose, data of the top ten inpatient cases of 2018 were collected and analysed. Second, semi-structured interviews with senior staff and doctors were carried out to understand cost control strategies implemented in the hospital and the management and doctors’ perceptions regarding the application of tested mathematical models for cost control. Old institutional economics and new institutional sociology were used to gain insight about how and why management accounting practices changed in the hospital. Findings The findings show that the absence of detailed per-case/patient cost information has not only hindered further evolvement of MACs but also stimulate tensions between managerial and medical worlds in the studied Indonesian public hospital. The simulation of BDA in this study was not only discovering the determinants of case cost recovery but also enabling the prediction of CRR of patients immediately after admission. The application of BDA and casemix accounting in the hospital will potentially become catalysts of discussion and mutual learning between managerial and medical staff in controlling patient costs. Originality/value This paper provides a more comprehensive picture of the potential roles of BDA in cost control practices. The study assesses the feasibility of BDA application in the hospital and evaluates the potential roles and acceptance of BDA application by both management and doctors.