Hemophilia Management via Data Collection and Reporting: Initial Findings from the Comprehensive Care Sustainability Collaborative

BACKGROUND: Despite being a rare disorder, hemophilia represents a significant driver of health care resource utilization and requires expert hematologic and multidisciplinary services to achieve optimal outcomes. Since their inception nearly 40 years ago, hemophilia treatment centers (HTCs) have served as centers of excellence in providing the intensive care and ancillary services necessary for this unique patient base; however, the full capabilities of these centers may be underused in the current framework of managed care, largely because of a lack of communication and information shared between payers and HTC stakeholders. PROGRAM DESCRIPTION: In an effort to enact tangible change toward improving the quality of care for bleeding disorders, the National Hemophilia Foundation developed an ongoing initiative among 18 leading clinicians and managed care decision makers called the Comprehensive Care Sustainability Collaborative (CCSC). The primary aim of the CCSC is to develop a framework for quality improvement pilot programs that can be replicated across the United States between payers and HTCs to facilitate cost-effective hemophilia management by integrating the HTC comprehensive care model. OBSERVATIONS: After CCSC committee members shared perspectives on care delivery, quality, and value, actionable data points were reviewed at length in order to develop meaningful metrics to facilitate information sharing between HTC and payer stakeholders. The following pragmatic measures will be reported by HTCs and payers via a series of pilot programs (reporting group is indicated in brackets): (a) patient classification by diagnosis (type, severity, and inhibitor status) [HTC]; (b) total cost of clotting factor [payer]; (c) prescribed factor dose/dispensed dose/patient weight (± range) [payer and HTC]; (d) emergency department visits/hospitalizations [payer and HTC]; (e) home infusion of clotting factor (%) [HTC]; (f) total cost per patient [payer]; and (g) patient contacts (e.g., clinic visits, follow-ups, telemedicine, and e-mail) [HTC]. IMPLICATIONS: Routine information sharing between HTCs and payer stakeholders is paramount to improving the quality and reducing the cost of hemophilia care, and the CCSC initiative provides a unique forum for such dialogue. Over the course of several consensus meetings, the CCSC has rigorously developed a set of quality improvement and cost management metrics. These metrics will be used in a first-of-its-kind series of pilot projects that are anticipated to forge innovative collaboration between payers and HTCs so as to improve outcomes in the management of bleeding disorders. DISCLOSURES: The preparation of this article was funded as part of the Comprehensive Care Sustainability Collaborative (CCSC) initiative, which is jointly sponsored by the National Hemophilia Foundation (NHF) and Impact Education, LLC, and supported via a charitable donation from Shire. Tarantino and Pindolia are members of the CCSC and were part of the NHF CCSC group that developed the metrics included in this article. Both authors received honorariums from the NHF for content development and expert review of the manuscript. Both authors contributed equally to the concept and design of this article and to analysis and manuscript preparation

Evaluating biologics and their biosimilars using a distributed research network to demonstrate real-world outcomes.

BACKGROUND: The Biologics and Biosimilars Collective Intelligence Consortium (BBCIC) was initiated in anticipation of biosimilars entering the U.S. market. OBJECTIVE: To present descriptive and comparative analyses of innovator compounds and biosimilars to address patient and physician concerns and potentially facilitate greater acceptance and adoption of biosimilars. METHODS: The BBCIC leverages Sentinel\u27s distributed research network (DRN) and the collaboration includes 18 founding member organizations from managed care integrated delivery networks, pharmacy benefit managers, and industry sponsors. Initial descriptive analysis (DA) studies were conducted to characterize the DRN data. Primary outcome measures included: (1) febrile neutropenia hospitalizations among first cycle granulocyte-colony stimulating factor (G-CSF) prophylaxis for breast and lung cancer (2) adverse events following erythropoietin stimulating agent (ESA) use in hemodialysis; (3) major adverse cardiac and hypoglycemia events following long-acting insulin (LAI) use in Types 1 and 2 diabetes and; (4) hospitalizations for serious infections following anti-inflammatory (AI) use for rheumatoid arthritis, psoriatic and gastrointestinal conditions. RESULTS: (1) G-CSF prophylaxis can be discerned from treatment and rare safety events were identified; (2) DRN ESA data lack granularity and future studies will be conducted using the Medicare ESRD data set; (3) major adverse cardiac and hypoglycemia events associated with LAIs are comparable to other observational trials; and (4) serious infection outcomes are comparable to other AI observational trials. In addition, it was identified that methods workgroups are needed to define best practices for switching patterns in comparative effectiveness research (CER) studies, International Classification of Diseases, Ninth Revision, Clinical Modification (ICD-9-CD) to ICD-10-CM mapping will be needed, and monitoring of data partner capture of National Drug Code and Healthcare Common Procedure Coding System codes is necessary. CONCLUSIONS: Outcomes comparable to those in the published literature were demonstrated for G-CSFs, LAIs, and AIs. ESA data do not contain needed granularity and future research will use alternative data sources. Following sufficient biosimilar exposures, CER studies will be initiated to assess the safety and effectiveness of biosimilars compared with innovators. In preparation for the planned CER studies, workgroups are identifying best practices to be implemented