Resource utilization and treatment costs of patients with severe hemophilia A: Real-world data from the ATHNdataset.

Hemophilia A is characterized by unpredictable spontaneous bleeds and chronic comorbidities. However, limited data exists at the national level into detailed management patterns related to patient clinical characteristics, representative real-world dosing and treatment frequency, and costs. To assess and characterize the US severe hemophilia A (SHA) population, including subgroups of patients, in terms of clinical and demographic characteristics, healthcare resource utilization received at hemophilia treatment centers (HTCs), and projected annual costs of treatment utilizing data from the ATHNdataset of the American Thrombosis and Hemostasis Network (ATHN). Adult male people with SHA (PwSHA) (FVIII < 1%) were identified in the ATHNdataset between January 2013 and September 2019. This retrospective cohort study described patients' demographic and clinical characteristics, clinical history, as well as the HTC-related health resource utilization (HRU), treatment utilization, and projected annual treatment costs of US PwSHA received over the most recent year. Results are reported for the overall population and for three mutually exclusive subpopulations of patients: PwSHA with a history of and/or current inhibitors, PwSHA without a history of inhibitors but with (or a history of) one or more transfusion-transmitted infections (hepatitis B virus [HBV], hepatitis C virus [HCV], or human immunodeficiency virus [HIV]), and PwSHA without a history of inhibitors or of transfusion-transmitted infections (HBV, HCV, or HIV). Of the overall PwSHA cohort (N = 3677), there was a high prevalence of HCV (24.1%) and HIV (13.7%), while the prevalence of HBV (4.9%) was lower. Note that 20.5% of PwSHA overall currently or ever had FVIII inhibitors. On average, PwSHA had 2.8 total HTC visits per year, including 0.9 comprehensive care visits, 1.1 telephone contact visits, 0.5 office visits, and 0.1 surgeries or other procedures. However, 23.3% of PwSHA were not seen at an HTC, and 33.8% of PwSHA did not have a comprehensive care visit during their most recent year of data. HTC-related HRU was similar between the overall cohort and across the patient subpopulations, although PwSHA and inhibitors had more frequent HTC visits (a mean of 3.6 visits annually vs. 2.5-2.8 in the other groups). Using reported treatment frequency and dosing, estimated mean annual hemophilia treatment costs varied by treatment and across the three subpopulations: extended half-life factor product ($893,609-934,301 by subpopulation), standard half-life factor product ($798,700-930,812), plasma-derived factor product ($613,220-801,061), and non-factor product treatment ($765,289-833,240). This study summarized recent sociodemographic and clinical characteristics, HTC-related HRU, and HA treatments and projected costs among adult PwSHA, including among key subpopulations of PwSHA. PwSHA experience substantial clinical and resource burden on a chronic basis, despite the care coordination efforts of ATHN-affiliated HTCs. These findings motivate further exploration of the drivers of resource utilization, observed differences across subpopulations and other disparities, and ongoing monitoring of clinical and treatment burden in the face of an evolving care landscape.

Health insurance coverage and switching among people with hemophilia A in the United States.

BACKGROUND: Hemophilia A (HA) is marked by substantial economic burden, including costs of ongoing treatment, increased monitoring, bleed events, and other health care utilization associated with managing the disease and comorbidities related to the disease. Gene therapies and other anticipated breakthrough treatments hold potential to substantially offset long-term traditional factor VIII (FVIII) prophylaxis in specific populations. Fragmentation of the US insurance system, however, may impact payers' approaches to coverage of new treatments, given concerns about patients "switching" insurance and the payer's ability to offset costs over time. OBJECTIVE: To assess insurance coverage and switching across payers among people with severe HA (SHA) using real-world data. METHODS: Adult men with SHA (FVIII measuring < 1%) in the American Thrombosis and Hemostasis Network dataset between January 2013 and September 2019 were identified. Patients' primary insurance category (ie, commercial, Medicaid, Medicare) and insurance switching over time were described. Outcomes included distribution of current primary insurance coverage by category and mean years of coverage per payer for commercially insured patients, including those with 2 or more commercial payers, and for those who switched insurance categories (eg, coverage by a commercial payer and government payer). RESULTS: Among the cohort of patients with SHA (N = 3,677), 51.9% had commercial primary insurance and 29.0% had coverage by Medicaid (including state-funded programs). The mean duration of follow-up in the database was 6.3 years for patients with at least 1 year of follow-up. Among patients who had ever been commercially insured, 74.9% had the same commercial payer for the entire follow-up period. The mean time covered by the same commercial insurance was 4.8 years. Only 7.5% of patients switched insurance categories (eg, from commercial to Medicaid). Among those who switched categories, patients averaged 3.9 years of commercial coverage, 4.0 years of Medicaid coverage, and 4.8 years of Medicare coverage during the follow-up period. CONCLUSIONS: Both commercially and government-insured patients with SHA typically maintain continuous coverage for extended periods, with limited switching between payers and insurance categories over time. These findings suggest that should breakthrough treatments be approved, payers would likely be able to realize substantial cost savings associated with avoiding long-term prophylactic therapies during the several years after treatment. DISCLOSURES: This study was funded by BioMarin Pharmaceutical Inc. Hinds, Chen, and Sammon are employees of BioMarin Pharmaceutical Inc. and own stock/stock options. Solari was an employee of BioMarin Pharmaceutical Inc. at the time of the study. Pezalla is CEO of Enlightenment Bioconsult, LLC. He, Cheng, and Recht are, or were at the time of this study, employees of American Thrombosis and Hemostasis Network (ATHN), which has received ATHNdataset licensing and other fees from BioMarin Pharmaceutical Inc. Research funding to Recht's employers has come from Bayer, BioMarin Pharmaceutical Inc., CSL Behring, Genentech, Grifols, Hema Biologics, LFB, Novo Nordisk, Octapharma, Pfizer, Sanofi, Spark, Takeda, and uniQure. Recht has also worked as a consultant for Catalyst Biosciences, CSL Behring, Genentech, Hema Biologics, Kedrion, Novo Nordisk, Pfizer, Sanofi, Takeda, and uniQure; sits on the board of directors of the Foundation for Women and Girls with Blood Disorders and of Partners in Bleeding Disorders; and is an employee of the Oregon Health & Science University. Data from this study were presented as a poster at AMCP Nexus 2021; October 18-21, 2021; Denver, CO.

Regional variation and cost implications of prescribed extended half-life factor concentrates among U.S. Haemophilia Treatment Centres for patients with moderate and severe haemophilia.

BACKGROUND: Extended half-life (EHL) factor VIII (FVIII) and IX (FIX) products are intended to decrease the burden of prophylaxis for patients with haemophilia A or B. Whether these newer concentrates have led to meaningful clinical practice change remains vague. AIM: To characterize the longitudinal use of standard (SHL) and EHL factor concentrates at haemophilia treatment centres (HTCs), using the ATHNdataset, a US database of 138 ATHN-affiliated HTCs. METHODS: Factor concentrate use among moderate and severe haemophilia A and B patients without inhibitors was analysed at three time points over 18 months. RESULTS: Use of EHL concentrates rose from 10% of patients to 22% during this study. EHL FVIII prophylaxis is prescribed to the minority of patients, 28%; EHL FIX now predominates for prophylaxis, 52%. Rates of prescribed EHL products varied significantly by age group and HTC region. Median prescribed prophylaxis for SHL compared to EHL products was FVIII 6240 and 5200 and FIX 6968 and FIX 3900 IU/kg/y, respectively. On-demand EHL use has grown but has minimal contribution to overall usage (2%). CONCLUSION: Haemophilia treatment centre region and patient age impact the rate of adoption of EHL products; however, EHL prescribing continues to rise nationally, particularly for EHL FIX. Careful attention to annual cost of prophylaxis is imperative as the decrease in median EHL prophylaxis consumption is not offset by the higher unit cost of these products. It is unclear how further growth in use of EHLs will be impacted by emerging non-factor replacement and gene therapies.

Impact of the 340B Pharmacy Program on Services and Supports for Persons Served by Hemophilia Treatment Centers in the United States.

Purpose Hemophilia Treatment Centers (HTCs) provide integrated and comprehensive services to individuals affected with rare bleeding disorders, such as hemophilia and Von Willebrand disease. Through the 340 Drug Pricing Program, HTCs may use pharmacy income to support clinical staff and patient services. The objective of this study was to describe the impact of the 340B program funding on services and support provided by HTCs to persons affected by rare bleeding disorders. Description Federally designated comprehensive HTCs with established 340B programs were invited to participate in a mailed survey in 2014. Participants were requested to report on 340B program-funded staff and services in the calendar year 2013. Assessment The 31 of 37 HTCs responding served over 10,000 individuals, or one-third of the national HTC patient population. The majority of responding HTCs reported that 340B program income supported over 90% of staff such as nurses, social workers, and physical therapists. Conclusion The results from this survey of 31 centers with established programs demonstrates the HTCs' reliance on 340B program support for vital comprehensive services, that are otherwise non-reimbursable, and highlights the importance of the 340B program in sustaining the high quality of care and in increasing access for a geographically dispersed, medically vulnerable population.

The impact of electronic health records on workflow and financial measures in primary care practices.

OBJECTIVE: To estimate a commercially available ambulatory electronic health record's (EHR's) impact on workflow and financial measures. DATA SOURCES/STUDY SETTING: Administrative, payroll, and billing data were collected for 26 primary care practices in a fee-for-service network that rolled out an EHR on a staggered schedule from June 2006 through December 2008. STUDY DESIGN: An interrupted time series design was used. Staffing, visit intensity, productivity, volume, practice expense, payments received, and net income data were collected monthly for 2004-2009. Changes were evaluated 1-6, 7-12, and >12 months postimplementation. DATA COLLECTION/EXTRACTION METHODS: Data were accessed through a SQLserver database, transformed into SAS®, and aggregated by practice. Practice-level data were divided by full-time physician equivalents for comparisons across practices by month. PRINCIPAL FINDINGS: Staffing and practice expenses increased following EHR implementation (3 and 6 percent after 12 months). Productivity, volume, and net income decreased initially but recovered to/close to preimplementation levels after 12 months. Visit intensity did not change significantly, and a secular trend offset the decrease in payments received. CONCLUSIONS: Expenses increased and productivity decreased following EHR implementation, but not as much or as persistently as might be expected. Longer term effects still need to be examined.

Sedentary behaviour and physical inactivity assessment in primary care: the Rapid Assessment Disuse Index (RADI) study.

BACKGROUND: The emerging evidence of the effects of sedentary time on health outcomes suggests a need to better measure this exposure. Healthcare settings, however, are not equipped with a tool that can quickly assess the sedentary habits of their patient population. The purpose of this study was to validate a tool for rapidly quantifying and tracking the sedentary time and low levels of daily lifestyle physical activity among primary care patients. METHODS: The study examined the test-retest reliability and validity of the rapid assessment disuse index (RADI) among adult patients from a large primary care clinic. Patients completed RADI (comprised of 3 items: sitting, moving and stair climbing) twice, followed by accelerometer monitoring. Test-retest reliability was computed, and the correlation between survey responses and accelerometry was determined. A receiver operating characteristic curve was constructed and the area under the curve (AUC) was calculated. RESULTS: RADI was temporally stable (intraclass correlation coefficients 0.79), and a higher score was significantly correlated with greater sedentary time (ρ=0.40; p<0.01), fewer sedentary to active transitions (ρ=-0.42; p<0.01), and less light-intensity physical activity (ρ=-0.40; p<0.01). The ability of RADI to detect patients with high levels of sedentary time was fair (AUC=0.72). CONCLUSIONS: This brief assessment tool, designed to quickly identify patients with high levels of sitting and low daily physical activity, exhibits good reliability and moderate validity. RADI can assist in providing recommendations at the point of care pertaining to modifying sedentary behaviour.

Accounting for response misclassification and covariate measurement error improves power and reduces bias in epidemiologic studies.

PURPOSE: To quantify the impact of ignoring misclassification of a response variable and measurement error in a covariate on statistical power, and to develop software for sample size and power analysis that accounts for these flaws in epidemiologic data. METHODS: A Monte Carlo simulation-based procedure is developed to illustrate the differences in design requirements and inferences between analytic methods that properly account for misclassification and measurement error to those that do not in regression models for cross-sectional and cohort data. RESULTS: We found that failure to account for these flaws in epidemiologic data can lead to a substantial reduction in statistical power, over 25% in some cases. The proposed method substantially reduced bias by up to a ten-fold margin compared to naive estimates obtained by ignoring misclassification and mismeasurement. CONCLUSIONS: We recommend as routine practice that researchers account for errors in measurement of both response and covariate data when determining sample size, performing power calculations, or analyzing data from epidemiological studies.

Reduction of 30-day postdischarge hospital readmission or emergency department (ED) visit rates in high-risk elderly medical patients through delivery of a targeted care bundle.

RATIONALE: Care coordination has shown inconsistent results as a mechanism to reduce hospital readmission and postdischarge emergency department (ED) visit rates. OBJECTIVE: To assess the impact of a supplemental care bundle targeting high-risk elderly inpatients implemented by hospital-based staff compared to usual care on a composite outcome of hospital readmission and/or ED visitation at 30 and 60 days following discharge. PATIENTS/METHODS: Randomized controlled pilot study in 41 medical inpatients predisposed to unplanned readmission or postdischarge ED visitation, conducted at Baylor University Medical Center. The intervention group care bundle consisted of medication counseling/reconciliation by a clinical pharmacist (CP), condition specific education/enhanced discharge planning by a care coordinator (CC), and phone follow-up. RESULTS: Groups had similar baseline characteristics. Intervention group readmission/ED visit rates were reduced at 30 days compared to the control group (10.0% versus 38.1%, P = 0.04), but not at 60 days (30.0% versus 42.9%, P = 0.52). For those patients who had a readmission/postdischarge ED visit, the time interval to this event was longer in the intervention group compared to usual care (36.2 versus 15.7 days, P = 0.05). Study power was insufficient to reliably compare the effects of the intervention on lengths of index hospital stay between groups. CONCLUSIONS: A targeted care bundle delivered to high-risk elderly inpatients decreased unplanned acute health care utilization up to 30 days following discharge. Dissipation of this effect by 60 days postdischarge defines reasonable expectations for analogous hospital-based educational interventions. Further research is needed regarding the impacts of similar care bundles in larger populations across a variety of inpatient settings.

Financial performance of primary care physician practices prior to electronic health record implementation.

While electronic health records (EHRs) are being widely implemented across the nation, few empirical data are currently available regarding their potential impact on financial performance and resource use. HealthTexas Provider Network is implementing a networkwide EHR, providing a unique opportunity to describe and evaluate fiscal effects. We conducted a retrospective, longitudinal observational study of financial performance related to inputs and income- and productivity-related outputs for the 33 primary care practices (July 2002-April 2006). Models for each outcome were constructed to test for a linear trend over time, adjusted for practice characteristics. F tests based on these models were used to determine the effect of each adjustor and to determine existence of a trend in each outcome. The observed staff per physician full-time equivalent (FTE) (3.6) was similar to staffing ratios reported for other primary care-only practices, while observation of 4692 work relative value units per physician FTE annually was higher than reported nationally. Significant monthly trends were identified for three of the outcome measures. During the pre-EHR baseline period, staffing ratios were equivalent to and physician productivity greater than reports available for these measures nationally or in other settings. Identification of time trends in three measures will allow these to be accounted for in the model used to evaluate the financial performance impact of EHR implementation.