Rਅਖਉਅਗ The National Haemophilia Program Standards, Evaluation and Oversight Systems in the United States of America Mark W. Skinner1, J. Michael Soucie2, Kathryn McLaughlin3 National Hemophilia Foundation, Washington, DC; 2Division of Blood Disorders, National Center on Birth Defects and Developmental Disabilities, Centers for Disease Control and Prevention, US Department of Health and Human Services, Atlanta, GA; 3Genetic Services Branch, Division of Children with Special Health Care Needs, Maternal and Child Health Bureau, Health Resources and Services Administration, US Department of Health and Human Services, Washington, DC, United States of America
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Title V funding has undergone many revisions since its passage in 1935: the most significant change relating to haemophilia occurred in 1981 with passage of the Omnibus Budget Reconciliation Act (OBRA '81), which converted the Title V program into a Maternal and Child Health (MCH) Block Grant program. In addition, with OBRA'81, 15 percent of the Title V appropriation for each fiscal year was set aside to support Special Projects of Regional and National Significance (SPRANS) as part of the MCH Block Grant3. Funding for federal haemophilia diagnostic and treatment centers (now known as the National Hemophilia Program) was included in the fifteen percent of the MCH Block Grant funds set aside for SPRANS programs; this appropriation remains to this day.
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The US National Hemophilia Program (NHP) is a collaborative relationship between the Maternal and Child Health Bureau (MCHB) of the Health Resources and Services Administration (HRSA), the Centers for Disease Control and Prevention (CDC) and the Medical and Scientific Advisory Council (MASAC) of the National Hemophilia Foundation (NHF). This article is written in three parts outlining the roles, collaborative and integrated working relationships of the three core entities (MCHB, CDC, NHF). We will discuss the birth and evolution of the modern day NHP and haemophilia treatment center (HTC) network, the evolution and role of surveillance programs, treatment guideline and standard setting, the impact of health care reform on access to comprehensive care and on-going initiatives to improve health outcomes for those living with haemophilia and related bleeding disorders. Although there is, at present, no formal HTC accreditation system in the US, we will describe a number of programs and systems that are already in place to promote and measure adherence to best practices, monitor patient outcomes and encourage continual operational and clinical improvement according to established standards. Finally, we will describe various initiatives that are underway to further develop evidencebased guidelines, which may lay the foundation for a future, formalised HTC self-audit evaluation or accreditation system.
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Introduction
Discussion History of the evolution of HTCs in the US Legislative Authority Providing federal support to facilitate Federal-State partnerships on public health issues for women and children, specifically those in rural areas and with special health care needs, has been longstanding at HRSA, beginning with the passage of Title V of the Social Security Act in 19351. Title V funding to States helped support the development of an infrastructure to treat patients with haemophilia in the US since 19752.
Evolution of HRSA's National Hemophilia Program (NHP) The MCHB SPRANS funding for the NHP is administered through the Division of Children with Special Health Care Needs (DCSHN) by the Genetic Services Branch (GSB). The NHP has two distinct programmatic parts: the Regional Hemophilia Network (RHN) and the National Hemophilia Program Coordinating Center (NHPCC). The RHN comprises the HTCs that receive the grant funding directly from MCHB. The NHPCC provides technical assistance to the RHN on care delivery and program evaluation to support participating HTCs in complying with NHP roles and responsibilities as discussed below. The main purpose of the RHN is to establish integrated and collaborative regional networks to promote the comprehensive care of individuals with haemophilia and related bleeding disorders or clotting disorders such as thrombophilia. The primary goal of the RHN grant program is to ensure that individuals with haemophilia and other bleeding disorders and their families have access to appropriate, quality care and other medical expertise and information in the context of a medical home model that provides family-centered, comprehensive, and culturally effective care4. Providing
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2000s, when specialists and clinicians who provide care to those living with haemophilia and others with rare bleeding disorders within the NHP acknowledged unmet needs within their community regarding collecting data and evaluating care for their patient populations with small numbers in the existing electronic medical record systems. During 2005 and early 2006, a broad range of key haemophilia stakeholders (e.g., haemophilia organizations, institutions, clinical disciplines, and experts in related fıelds of expertise) formed multiple clinical and operational working groups to assess the information needs of the community and develop a consensus approach to address them. The outcome of these deliberations led to the development of ATHN. ATHN's mission is "(…) to provide stewardship of a secure national database infrastructure to support standardised data collection, clinical outcomes analysis, research, advocacy, and public health reporting for the bleeding and clotting disorders community"8.
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Growth of the surveillance and data collection aspects CDC's Role CDC began its partnership with the US HTCs in the 1980s as part of public health efforts to prevent the spread of HIV infection resulting from use of treatment products made before the virus was identified. In 1991, in response to requests from constituents, Congress authorised CDC to expand its public health prevention activities with the aim of reducing the complications of bleeding disorders. Public health surveillance is the ongoing systematic collection, analysis, and interpretation of health data for purposes of improving health and safety9. In order to assess the public health needs of the haemophilia community, surveillance was conducted in six US states in 1993-1998. Data from this surveillance were used to assess the burden of haemophilia in the country and to identify sources of care and treatment practices. Data were also collected to measure rates and severity not only of infectious disease complications including hepatitis and HIV but also those of chronic joint disease, intracranial hemorrhage and other bleeding complications as well as mortality. Analyses of these population-based surveillance data revealed an estimated US haemophilia population of about 17,000, nearly 70% of which had received care in HTCs10. Although most of those not receiving care in HTCs had mild haemophilia, 34% of those with moderate and 14% of those with severe disease received care outside of the HTC network. When outcomes were compared, males with haemophilia who had received care from HTCs had lower mortality rates 11 and fewer hospitalizations for bleeding complications12.
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comprehensive care through a core team of specialists is integral to being considered a federally qualified HTC and all HTCs within the network are required to comply with the NHF MASAC Recommendation 132 - Standards and Criteria for Persons with Congenital Bleeding Disorders, in order to qualify for federal funding as discussed below5,6. Since OBRA '81, MCHB has awarded funding for haemophilia through a regional approach using grants. In 2012, HRSA adjusted the regional structure from 12 historical regions to 8 regions, along with the development and implementation of the NHPCC. This organizational change was made to move from a regional to a national focus in terms of data collection and evaluation, as well as to better align program resources with patient needs. The RHN is currently comprised of 132 HTCs including 8 regional core centers. The regional core centers are funded as grants, however, the NHPCC was awarded as a cooperative agreement which allows the MCHB Program Officer to adjust work plans and timelines to better suit successful completion of goals and objectives. The core centers distribute available funding to their subrecipient HTCs within their regions and the number of HTCs is variable, depending on the geographic size of the region. The core centers are responsible for ensuring that their subrecipient HTCs comply with the roles and responsibilities laid out in the funding opportunity announcement published by HRSA.
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Advent of the NHPCC and its objectives During the discussions on how to organizationally restructure the NHP in 2011-2012, MCHB looked to other successful regional grant programs. Critical to the success of these other programs was the presence of a national coordinating center to compile and disseminate regional best practices; therefore, the decision was made to develop a similar funding opportunity for a NHPCC. The purpose of the NHPCC is to facilitate, coordinate, and evaluate (in terms of assisting in the development of regional and national evaluation plans) the activities carried out by the RHNs. Working collaboratively with the RHNs to develop a best practices model and establish a standard of care model will also be important activities. The American Thrombosis and Hemostasis Network (ATHN) ATHN was awarded funding as the NHPCC in June 2012. ATHN, as the NHPCC, is not an oversight or punitive organization, but one that provides technical assistance to the RHN on program evaluation, recruitment and retention of staff, as well as incorporation of genetics and public health into the NHP7. The genesis of ATHN dates back to the early
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Furthermore, similar to the rates of primary prophylaxis, inhibitor testing varied from 0% to 100% among the HTCs. Based on discussions with experts, a substantial barrier to regular inhibitor screening was the lack of capacity of most local laboratories to accurately perform the necessary testing in the presence of infused factor VIII, especially among patients who were receiving prophylactic therapy (unpublished CDC data). To address this barrier, the CDC Division of Blood Disorder's laboratory has developed and validated a modified testing method that can be used to screen for an inhibitor regardless of treatment status23. This method is being disseminated to local laboratories and its use for regular screening for inhibitors should become part of the standard of care for HTCs. The UDC has recently been updated (now called Community Counts) to include national monitoring of inhibitors through the use of both local and CDC testing of blood specimens collected as part of the surveillance. Using data from Community Counts will allow CDC to monitor the adoption of this important testing as a new standard of care. Current Status of the National Hemophilia Program and long term objectives CDC's Public Health Surveillance for the prevention of complications of bleeding and clotting disorders cooperative agreement is the funding mechanism through which its surveillance and prevention programs are funded. In 2011, the RHN applied for the CDC funding through ATHN. As part of the new cooperative agreement, the data elements collected through the new surveillance system were revised based on recommendations from an expert panel through a collaborative process between CDC scientists and a scientific committee comprised of clinicians organised and supported by ATHN. Community Counts, the new surveillance system has three components: 1) the HTC Population Profile; 2) Mortality Reporting; and 3) the Registry for Bleeding Disorders Surveillance. The first component collects a minimum set of demographic and clinical information on all of the patients with bleeding and clotting disorders who are receiving care within the RHN. The second component collects limited data about causes of death. The third component will be a more in-depth data collection project that will collect more complex data elements on health and complications, including collecting biologic samples for infectious disease and inhibitor surveillance. This collaboration marks an exciting new prospect for the haemophilia community as some data elements from the CDC's project will be utilised by the RHN and NHPCC to report as the national data source for several developmental Healthy People 2020 performance measures within the blood disorders and blood safety
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This population-based surveillance system was replaced in 1998 with the Universal Data Collection (UDC) surveillance system established in the HTC network in order to collect the more detailed data needed to inform and evaluate public health prevention programs13. For example, analyses of UDC data on height and weight and joint range of motion measurements lead to the recognition that increased body mass index (a measure of body adiposity) was associated with decreased joint mobility regardless of haemophilia severity14. Based on these findings it became clear that overweight and obesity, at epidemic levels in the general US population, was not only just as common among boys and men with haemophilia but also was contributing to preventable joint disease. Public health messages about the need to maintain a healthy weight to protect joints are part of the prevention strategies implemented because of these findings. Continued surveillance will be used to evaluate these prevention efforts. The UDC also included annual blood sample collection with infectious disease testing performed at CDC in order to assess the safety of treatment products. Results of this surveillance have provided evidence for the safety of these products as well as the need for continued vigilance15,16. UDC surveillance data have also been used to examine patterns of care within the HTC network. For example, the administration of treatment product on a regular basis to prevent bleeding episodes, called prophylaxis therapy, has been shown to prevent joint disease in young boys with haemophilia17. However, in an analysis of UDC data there was a full spectrum of variation from 0% to 100% of youth in individual HTCs who were receiving prophylaxis therapy18. These kinds of analyses can be useful both to monitor the adoption of standards of care as well as to gain an understanding of facilitators and barriers to the practices by studying the characteristics of HTCs with high and low adoption rates. Another example of using surveillance data is in the identification of an inhibitor, which is currently the most serious complication of haemophilia treatment. An inhibitor is an antibody to treatment product that develops in up to one-third of people with haemophilia and renders it ineffective in preventing or stopping bleeding episodes. People with an inhibitor are more likely to be hospitalised for a bleeding complication and treatment costs are far greater than those without an inhibitor19-21. A blood test is necessary for diagnosing an inhibitor. Earlier identification of inhibitors, accomplished through regular screening, leads to higher inhibitor eradication success22. From 2006-2010, only 46% of patients with severe haemophilia (those at highest risk of an inhibitor) had a screening test performed (unpublished CDC data).
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improvements for those living with high cost diseases or having pre-existing conditions such as haemophilia. The substantial growth in managed care plans and the many changes to private and public insurance programs made by the Affordable Care Act are having direct and indirect consequences for the bleeding disorders community. The Affordable Care Act provides for the establishment of essential health benefits that must be offered by new plans available in the insurance market. However, the choice of health care providers available to an insured within a plan is left to the insurance plan provider, subject to rules requiring access to a minimum percentage of the essential community providers in the plan service area27. These rules encourage, but do not require, insurers to include access to a federally funded HTC within their provider networks. To reduce reliance on expensive drugs and incentivize patients to choose lower-cost generic alternatives, if available, some plans are requiring higher cost-sharing (co-payments and coinsurance) for medications such as clotting factor concentrates. While the Affordable Care Act has the potential to increase patient access to needed treatments and services, it is too early to evaluate how this translates into outcomes for people with bleeding disorders. The Affordable Care Act also contained provisions placing a greater emphasis on evidence-based medicine and performance outcomes measures. Thus, establishing and bolstering evidence-based standards and recommendations to support haemophilia care and its delivery through a recognised HTC has taken on an increased importance. Over many years, the NHF and its MASAC have led the development of a range of standards and recommendations addressing clinical management, treatment safety, and care delivery for those living with a bleeding disorder. Issued in the form of recommendations, MASAC guidelines set the standard of care and are frequently referred to by an international array of physicians, medical schools, pharmacists, emergency room personnel, insurance companies, patients and others. MASAC is comprised of scientists, physicians, allied health professionals, patients and government liaisons from the relevant federal agencies within the US Department of Health and Human Services (CDC, Food and Drug Administration, MCHB and the National Institutes of Health). The full list of recommendations are available on the NHF website28. Adherence to MASAC recommendations is widely promoted, however, outside of the specific requirements with the NHP compliance with other MASAC recommendations is voluntary. Two MASAC recommendations are of particular note with regard to the oversight and management of US HTCs: 1) MASAC Recommendation 132 - Standards and Criteria for Persons with Congenital Bleeding Disorders29.
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topic objective24. Healthy People is a national effort by the US Department of Health and Human Services to develop measures and benchmarks to track national progress, in 10-year cycles, on government efforts to improve the public health of the country25. With the new regional organization and the development of ATHN as the national coordinating center, the NHP is well placed to accomplish goals and activities that have not previously been possible given the lack of focus on working on a national level. Since June 2012, the RHN and the NHPCC has completed the first ever technical assistance needs assessment for HTC Staff, currently under analysis, which will provide insight to HRSA on the help needed by HTC staff both in the management of patient care and in navigating the federal grant process as well as informing HRSA how the NHPCC can begin to provide needed services. The program is also currently analyzing the results of the first national HTC patient needs assessment that was a requirement for both the RHNs and the NHPCC to accomplish, so that the haemophilia community can use the results to set national priorities for the program to help with the development of a national evaluation plan to track the improvement of the care received in the coming years. A third party evaluation consultant has been contracted by both the NHPCC and the regions to analyze the results of the needs assessment, on both a regional and national level. The results of the analysis will be utilised to set national priorities for the NHP through a multistakeholder process involving the regions, the NHPCC, consumer advocacy groups, and our federal partners. The national priorities, once determined, will form the basis of a national evaluation plan to track patient outcomes for those who receive care through the NHP. Results of the patient needs assessment will be widely disseminated within the federal program and haemophilia consumer advocacy groups and other stakeholders within the bleeding community. Finally, these major projects and increased requirements for data collection and evaluation plans will inform the development of standards of care and methods for dissemination of best practices, which will require the assistance and expertise of the National Hemophilia Foundation's MASAC, whose efforts are described below. The role of the NHF MASAC in standard setting Impact of the Affordable Care Act Federal enactment of the Patient Protection and Affordable Care Act26 on March 23, 2010 came with a promise to provide quality affordable health care for all Americans. Since then, the US health care system has been undergoing a major overhaul impacting both insurance availability and the delivery of care. The Affordable Care Act contains many long sought
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widely cited examples of best practice of model legislation are New Jersey and more recently Missouri31-33. As this article was written, a number of states are considering adoption of similar statutory or regulatory provisions. The National Association of Boards of Pharmacy recently featured a news article profiling the NHF model standards of service and is working with NHF to achieve uniformity in the regulation of this specialty area34. In 1997, New Jersey included language in state Health Maintenance Organization (HMO) regulations securing access to and reimbursement for care at HTCs. In 2000, legislation was enacted requiring HMO and managed care insurers to comply with standards in the provision of benefits to patients with haemophilia31. Insurance carriers offering managed care health benefit plans in New Jersey must contract with designated health care providers for the delivery of services for the home treatment of bleeding episodes associated with haemophilia, including the purchase of blood products and blood infusion equipment. The State maintains an on-line registry of designated haemophilia home care providers meeting statutory requirements32. Building on the New Jersey precedent, in 2011, Missouri enacted legislation33 creating standards of care for pharmacies dispensing blood-clotting therapies. The Missouri HealthNet Division (which oversees Medicaid) previously covered blood clotting products and services. The law specifically adds blood clotting equipment, supplies, and assessments in the home to the list of Missouri HealthNet benefits.
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Last revised in 2002, MASAC Recommendation 132 outlines comprehensive standards of care and lists services that should be provided by HTCs. The recommendation includes an extensive discussion of the delivery of integrated, multi-disciplinary, comprehensive care. The recommendation places an emphasis on early diagnosis and intervention to prevent disease complications. The composition, general and specific functions, availability and training of the Comprehensive C a r e Te a m d e p e n d o n p a t i e n t c e n s u s a n d individual circumstance. The staff and services required for proper HIV care for men and women, haepatitis-related services, care for women with bleeding disorders, the need for culturally sensitive outreach programs, and access to clinical research protocols are also addressed. Within the NHP, all HTCs are required and expected to comply with and follow the guidelines for elements and core services of the HTC-based model of care delineated in Recommendation 132. 2) MASAC Recommendation 188 - Standards of Service for Pharmacy Providers of Clotting Factor Concentrates for Home Use to Patients with Bleeding Disorders30. A varied range of pharmacy providers supply clotting factor concentrates to patients for treatment at home. The pharmacy providers are principally specialty pharmacies or factor programs administered through the HTC. When patients do not receive optimal service from these providers, there is potential for adverse health events that lead to poor outcomes and/ or increased costs30. Given challenges in achieving optimal service, MASAC adopted standards of service for pharmacy providers. MASAC acknowledged the necessity of cost efficiency in the provision of health care, yet emphasised that cost efficiency should not occur at the expense of quality patient care. The purpose of the recommendation is to establish minimum standards of service for pharmacy providers to meet the specific needs of individuals with bleeding disorders. The standards address pharmacy provider staff knowledge, availability of clotting factor concentrates and ancillary supplies, processing of prescription orders, hours of operation, access to pharmacy staff, delivery, recordkeeping, billing and product recall. Over 30 national specialty pharmacies and most US HTCs have voluntarily self-reported they have met or exceeded the standards of Recommendation 188. At the state level, various legislative and regulatory initiatives have been implemented to promote optimal access to home care services to further reinforce the importance of pharmacy standards. The most prominent and
Development of evidence-based clinical practice guidelines for Comprehensive Care NHF is acutely aware of the major ongoing changes in health care occurring in the US. In 2012, the NHF held a strategic summit to develop a plan for haemophilia care within the evolving US health care environment. The Summit report addressed the implications of the Affordable Care Act and provided a series of strategic recommendations35. Among these was a call for NHF to sponsor the production and maintenance of evidence-based clinical practice guidelines (CPGs). Health plans are taking note of the geographic variations in practice and standards of practice (e.g. prophylaxis regimens) and clinical behavior (e.g. use of comprehensive care services). Such variations suggest insufficient evidence to support practice or a lack of clinician concurrence about, or adherence to, evidence-based practice. Recognizing the value of establishing, promoting, and adhering to CPGs and associated standards of practice, the NHF and its MASAC leadership launched an initiative to develop a comprehensive set of evidence-based CPGs to support patient-centered clinical decision-making and optimize care.
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Understanding Title V of the Social Security Act: A Guide to the Provisions of the Federal Maternal and Child Health Block Grant. Maternal and Child Health Bureau, Health Resources and Services Administration., Available at: http://www. amchp.org/AboutTitleV/Documents/UnderstandingTitleV. pdf. Accessed on 18/01/2014. Funding Opportunity Announcement: Regional Hemophilia Network. Announcement No. HRSA-12-133, CFDA# 93.110, FY 2012. Release Date: 11/16/2011; Due Date: 01/10/2012. Available at: https://grants3.hrsa.gov/2010/Web2External/ Platform/Interface/DisplayAttachment.aspx?dm_rtc=16&dm_ attid=89e6028e-37f2-4411-8334-acd06b02d5db&dm_ attinst=0. Accessed on 18/01/2014. NHF MASAC Recommendation 132 - Standards and Criteria for the care of Persons with Congenital Bleeding Disorders. National Hemophilia Foundation. Available at: http://www. hemophilia.org/NHFWeb/Resource/StaticPages/menu0/ menu5/menu57/masac132.pdf. Accessed on 20/01/2014. Funding Opportunity Announcement: Regional Hemophilia Network. Announcement No. HRSA-12-133, CFDA# 93.110, FY 2012. Release Date: 11/16/2011; Due Date: 01/10/2012. Available at: https://grants3.hrsa.gov/2010/Web2External/ Platform/Interface/DisplayAttachment.aspx?dm_rtc=16&dm_ attid=89e6028e-37f2-4411-8334-acd06b02d5db&dm_ attinst=0. Accessed on 18/01/2014. Funding Opportunity Announcement: National Hemophilia Program Coordinating Center. Announcement No. HRSA-12135, CFDA# 93.110, FY 2012. Release Date: 11/16/2011; Due Date: 12/30/2011. Available at: https://grants3.hrsa.gov/2010/ Web2External/Interface/FundingCycle/ExternalView. aspx?&fCycleID=EDA323E3-02AC-483A-ABE6-92474D2 335EF&txtAction=View+Details&submitAction=Go&View Mode=EU. Accessed on 18/01/14. Aschman DJ, Abshire TC, Shapiro AD, et al. A CommunityBased Partnership to Promote Information Infrastructure for Bleeding Disorders. Am J Prev Med 2011; 41 (6S4): S332-7. Available at: http://www.ajpmonline.org/article/S07493797%2811%2900680-5/fulltext. Accessed on 20/01/2014. Thacker SB, Berkelman RL. Public health surveillance in the United States. Epidemiol Rev 1988; 10: 164-90. Soucie JM, Jackson D, Evatt B. The occurrence of hemophilia in the United States. Am J Hematol 1998; 59: 288-94. Soucie JM, Nuss R, Evatt B, et al and the Hemophilia Surveillance System Project Investigators. Mortality among males with hemophilia: relations with source of medical care. Blood 2000; 96: 437-42. Soucie JM, Symons J, Evatt B, et al and the Hemophilia Surveillance System Project Investigators. Home-based factor infusion therapy and hospitalization for bleeding complications among males with hemophilia. Haemophilia 2001; 7:198-206. Soucie JM, McAlister S, McClellan A, et al. The universal data collection surveillance system for rare bleeding disorders. Am J Prev Med 2010; 38 (4 Suppl): S475-81. Soucie JM, Cianfrini C, Janco RL, et al. Joint range of motion limitations among young males with hemophilia: Prevalence and risk factors. Blood 2004; 103: 2467-73. Centers for Disease Control and Prevention. Blood safety monitoring among persons with bleeding disorders - United States, May 1998-June 2002. MMWR Morb Mortal Wkly Rep 2003; 51: 1152-4. Soucie JM, De Staercke C, Monahan PE, et al. Evidence for the Transmission of Parvovirus B19 in Patients with Bleeding Disorders Treated with Plasma-derived Factor Concentrates in the Era of Nucleic Acid Test (NAT) Screening. Transfusion 2013; 53: 1217-25. Manco-Johnson MJ, Abshire TC, Shapiro AD, et al. Prophylaxis versus episodic treatment to prevent joint disease in boys with severe hemophilia. N Engl J Med 2007; 357: 535-44.
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The initial phase of the CPG initiative addresses the comprehensive care model for haemophilia. In addition to recognizing the importance of a range of coordinated services to most patients with haemophilia, addressing the comprehensive care model as the first evidence-based CPG will establish a foundation upon which more specific CPGs addressing other aspects of care may be developed. The comprehensive care CPG will: - define comprehensive care as it relates to the haemophilia patient; - specify the coordinated set of diagnostic, therapeutic, and certain auxiliary and supplemental services that are most important for haemophilia patients across the U.S.; - identify the range of clinical and non-clinical members of a haemophilia comprehensive care team (including, e.g., social workers, dentists); - identify best-practices and evidence-based standards of comprehensive/coordinated care for haemophilia treatment centers (HTCs) and individual clinical practices. NHF foresees that the development of the comprehensive care CPG will provide a catalyst to promote harmonization of care delivery and reduce practice variations among the US HTC system. An agreed evidence-based benchmark would allow for both internal and external evaluation of adherence to best practices and benchmark available services with an HTC. Ultimately, it will serve as an important starting point for establishment of a US HTC self-audit or accreditation system.
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Keywords: Haemophilia Treatment Center, Program evaluation, Child Health Bureau (MCHB), Centers for Disease Control and Prevention (CDC), National Hemophilia Foundation (NHF).
Acknowledgments and disclosures
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Mark Skinner serves as a consultant to the National Hemophilia Foundation. CDC Disclaimer: The findings and conclusions in this report are those of the authors and do not necessarily represent the official position of the Centers for Disease Control and Prevention. The Authors declare no conflicts of interest.
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Katherine B. Oettinger. Title V of the Social Security Act: What it has meant for children. Social Security Bulletin. V23; No8, pgs 39-50, August, 1960. Available at: http://www. ssa.gov/policy/docs/ssb/v23n8/v23n8p39.pdf. Accessed on 19/01/2014. Understanding Title V of the Social Security Act: A Guide to the Provisions of the Federal Maternal and Child Health Block Grant. Maternal and Child Health Bureau, Health Resources and Services Administration. Available at: http://www.amchp. org/AboutTitleV/Documents/UnderstandingTitleV.pdf. Accessed on 18/01/2014.
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29) NHF MASAC Recommendation 132 - Standards and Criteria for the care of Persons with Congenital Bleeding Disorders. National Hemophilia Foundation. Available at: http://www. hemophilia.org/NHFWeb/Resource/StaticPages/menu0/ menu5/menu57/masac132.pdf. Accessed on 20/03/2014. 30) NHF MASAC Recommendation 188 - Standards of Service for Pharmacy Providers of Clotting Factor Concentrates for Home Use to Patients with Bleeding Disorders. National Hemophilia Foundation. Available at: http://www.hemophilia. org/NHFWeb/Resource/StaticPages/menu0/menu5/menu57/ masac188.pdf. Accessed on 20/03/2014. 31) New Jersey Statues Annotated. Section 1 of L. 2001, c. 121, codified at N.J.S.A. 26:2S-10.1, and sections 3 through 10 of L. 2001, c. 121, variously codified throughout Titles 17, 17B and 26. Available at: http://njlaw.rutgers.edu/cgi-bin/njstats/ showsect.cgi?title=26&chapter=2S§ion=10.1&actn=get sect. Accessed on 20/03/2014. 32) New Jersey Designated Hemophilia Providers. Available at: http://www.state.nj.us/dobi/division_insurance/managedcare/ mchemoprov.htm. Accessed on 20/03/2014. 33) Missouri Revised Statues. RSMo 338.400. Available at: http:// www.moga.mo.gov/statutes/C300-399/3380000400.HTM. Accessed on 20/03/2014. 34) States Look at Standards of Service for Pharmacies Serving Hemophilia Patients. NABP Newsletter. 2013; June-July: 12830. Available at: http://www.nabp.net/system/redactor_assets/ documents/618/Final_June-July_2013_Newsletter.pdf. Accessed on 20/03/2014. 35) National Hemophilia Foundation: Strategic Summit Report. October 2012. National Hemophilia Foundation. Available at: http://www.hemophilia.org/NHFWeb/MainPgs/MainNHF. aspx?menuid=119&contentid=2120. Accessed on 20/03/2014.
Correspondence: Mark W. Skinner National Hemophilia Foundation 1155 23rd Street NW #3A Washington, DC 20037, USA e-mail: [email protected]
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18) Soucie JM, McAlister S, McClellan A, et al. The universal data collection surveillance system for rare bleeding disorders. Am J Prev Med 2010; 38 (4 Suppl): S475-81. 19) Soucie JM, Symons J, Evatt B, et al and the Hemophilia Surveillance System Project Investigators. Home-based factor infusion therapy and hospitalization for bleeding complications among males with hemophilia. Haemophilia 2001; 7: 198-206. 20) Guh S, Grosse SD, McAlister S, et al. Healthcare expenditures for males with haemophilia and employer-sponsored insurance in the United States, 2008. Haemophilia 2012; 18: 268-75. 21) Guh S, Grosse SD, McAlister S, et al. Health care expenditures for Medicaid-covered males with haemophilia in the United States, 2008. Haemophilia. 2012; 18: 276-83. 22) DiMichele DM. Immune tolerance in haemophilia: the long journey to the fork in the road. Brit J Haematol 2012; 159: 123-34. 23) Miller CH, Rice AS, Boylan B, et al, the Hemophilia Inhibitor Research Study Investigators. Comparison of clot-based, chromogenic, and fluorescence assays for measurement of factor VIII inhibitors in the U.S. Hemophilia Inhibitor Research Study (HIRS). J Thromb Haemost 2013: 11; 1300-9. 24) Healthy People 2020. Blood Disorders and Blood Safety Topic Objectives. Available at: http://www.healthypeople.gov/2020/ topicsobjectives2020/overview.aspx?topicid=4. Accessed on 20/01/2014. 25) HHS News Press Release: HHS announces the nation's new health promotion and disease prevention agenda. OASH Press Office. Released December 2, 2010. Available at: http:// www.healthypeople.gov/2020/about/DefaultPressRelease.pdf. Accessed on 20/03/2014. 26) Pub L. 111-148, 111th United States Congress. Washington, DC. March 23, 2010. 27) HHS Centers for Medicare and Medicaid Services. Letter to Issuers in the Federally-facilitated Marketplace. March 14, 2014. Available at: http://www.cms.gov/CCIIO/Resources/ Regulations-and-Guidance/Downloads/2015-final-issuerletter-3-14-2014.pdf. Accessed on 26/03/2014. 28) NHF MASAC Recommendations. National Hemophilia Foundation. Available at: http://www.hemophilia.org/NHFWeb/ MainPgs/MainNHF.aspx?menuid=157&contentid=347. Accessed on 20/03/2014.
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Title V funding has undergone many revisions since its passage in 1935: the most significant change relating to haemophilia occurred in 1981 with passage of the Omnibus Budget Reconciliation Act (OBRA '81), which converted the Title V program into a Maternal and Child Health (MCH) Block Grant program. In addition, with OBRA'81, 15 percent of the Title V appropriation for each fiscal year was set aside to support Special Projects of Regional and National Significance (SPRANS) as part of the MCH Block Grant3. Funding for federal haemophilia diagnostic and treatment centers (now known as the National Hemophilia Program) was included in the fifteen percent of the MCH Block Grant funds set aside for SPRANS programs; this appropriation remains to this day.
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The US National Hemophilia Program (NHP) is a collaborative relationship between the Maternal and Child Health Bureau (MCHB) of the Health Resources and Services Administration (HRSA), the Centers for Disease Control and Prevention (CDC) and the Medical and Scientific Advisory Council (MASAC) of the National Hemophilia Foundation (NHF). This article is written in three parts outlining the roles, collaborative and integrated working relationships of the three core entities (MCHB, CDC, NHF). We will discuss the birth and evolution of the modern day NHP and haemophilia treatment center (HTC) network, the evolution and role of surveillance programs, treatment guideline and standard setting, the impact of health care reform on access to comprehensive care and on-going initiatives to improve health outcomes for those living with haemophilia and related bleeding disorders. Although there is, at present, no formal HTC accreditation system in the US, we will describe a number of programs and systems that are already in place to promote and measure adherence to best practices, monitor patient outcomes and encourage continual operational and clinical improvement according to established standards. Finally, we will describe various initiatives that are underway to further develop evidencebased guidelines, which may lay the foundation for a future, formalised HTC self-audit evaluation or accreditation system.
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Introduction
Discussion History of the evolution of HTCs in the US Legislative Authority Providing federal support to facilitate Federal-State partnerships on public health issues for women and children, specifically those in rural areas and with special health care needs, has been longstanding at HRSA, beginning with the passage of Title V of the Social Security Act in 19351. Title V funding to States helped support the development of an infrastructure to treat patients with haemophilia in the US since 19752.
Evolution of HRSA's National Hemophilia Program (NHP) The MCHB SPRANS funding for the NHP is administered through the Division of Children with Special Health Care Needs (DCSHN) by the Genetic Services Branch (GSB). The NHP has two distinct programmatic parts: the Regional Hemophilia Network (RHN) and the National Hemophilia Program Coordinating Center (NHPCC). The RHN comprises the HTCs that receive the grant funding directly from MCHB. The NHPCC provides technical assistance to the RHN on care delivery and program evaluation to support participating HTCs in complying with NHP roles and responsibilities as discussed below. The main purpose of the RHN is to establish integrated and collaborative regional networks to promote the comprehensive care of individuals with haemophilia and related bleeding disorders or clotting disorders such as thrombophilia. The primary goal of the RHN grant program is to ensure that individuals with haemophilia and other bleeding disorders and their families have access to appropriate, quality care and other medical expertise and information in the context of a medical home model that provides family-centered, comprehensive, and culturally effective care4. Providing
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2000s, when specialists and clinicians who provide care to those living with haemophilia and others with rare bleeding disorders within the NHP acknowledged unmet needs within their community regarding collecting data and evaluating care for their patient populations with small numbers in the existing electronic medical record systems. During 2005 and early 2006, a broad range of key haemophilia stakeholders (e.g., haemophilia organizations, institutions, clinical disciplines, and experts in related fıelds of expertise) formed multiple clinical and operational working groups to assess the information needs of the community and develop a consensus approach to address them. The outcome of these deliberations led to the development of ATHN. ATHN's mission is "(…) to provide stewardship of a secure national database infrastructure to support standardised data collection, clinical outcomes analysis, research, advocacy, and public health reporting for the bleeding and clotting disorders community"8.
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Growth of the surveillance and data collection aspects CDC's Role CDC began its partnership with the US HTCs in the 1980s as part of public health efforts to prevent the spread of HIV infection resulting from use of treatment products made before the virus was identified. In 1991, in response to requests from constituents, Congress authorised CDC to expand its public health prevention activities with the aim of reducing the complications of bleeding disorders. Public health surveillance is the ongoing systematic collection, analysis, and interpretation of health data for purposes of improving health and safety9. In order to assess the public health needs of the haemophilia community, surveillance was conducted in six US states in 1993-1998. Data from this surveillance were used to assess the burden of haemophilia in the country and to identify sources of care and treatment practices. Data were also collected to measure rates and severity not only of infectious disease complications including hepatitis and HIV but also those of chronic joint disease, intracranial hemorrhage and other bleeding complications as well as mortality. Analyses of these population-based surveillance data revealed an estimated US haemophilia population of about 17,000, nearly 70% of which had received care in HTCs10. Although most of those not receiving care in HTCs had mild haemophilia, 34% of those with moderate and 14% of those with severe disease received care outside of the HTC network. When outcomes were compared, males with haemophilia who had received care from HTCs had lower mortality rates 11 and fewer hospitalizations for bleeding complications12.
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comprehensive care through a core team of specialists is integral to being considered a federally qualified HTC and all HTCs within the network are required to comply with the NHF MASAC Recommendation 132 - Standards and Criteria for Persons with Congenital Bleeding Disorders, in order to qualify for federal funding as discussed below5,6. Since OBRA '81, MCHB has awarded funding for haemophilia through a regional approach using grants. In 2012, HRSA adjusted the regional structure from 12 historical regions to 8 regions, along with the development and implementation of the NHPCC. This organizational change was made to move from a regional to a national focus in terms of data collection and evaluation, as well as to better align program resources with patient needs. The RHN is currently comprised of 132 HTCs including 8 regional core centers. The regional core centers are funded as grants, however, the NHPCC was awarded as a cooperative agreement which allows the MCHB Program Officer to adjust work plans and timelines to better suit successful completion of goals and objectives. The core centers distribute available funding to their subrecipient HTCs within their regions and the number of HTCs is variable, depending on the geographic size of the region. The core centers are responsible for ensuring that their subrecipient HTCs comply with the roles and responsibilities laid out in the funding opportunity announcement published by HRSA.
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Advent of the NHPCC and its objectives During the discussions on how to organizationally restructure the NHP in 2011-2012, MCHB looked to other successful regional grant programs. Critical to the success of these other programs was the presence of a national coordinating center to compile and disseminate regional best practices; therefore, the decision was made to develop a similar funding opportunity for a NHPCC. The purpose of the NHPCC is to facilitate, coordinate, and evaluate (in terms of assisting in the development of regional and national evaluation plans) the activities carried out by the RHNs. Working collaboratively with the RHNs to develop a best practices model and establish a standard of care model will also be important activities. The American Thrombosis and Hemostasis Network (ATHN) ATHN was awarded funding as the NHPCC in June 2012. ATHN, as the NHPCC, is not an oversight or punitive organization, but one that provides technical assistance to the RHN on program evaluation, recruitment and retention of staff, as well as incorporation of genetics and public health into the NHP7. The genesis of ATHN dates back to the early
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Furthermore, similar to the rates of primary prophylaxis, inhibitor testing varied from 0% to 100% among the HTCs. Based on discussions with experts, a substantial barrier to regular inhibitor screening was the lack of capacity of most local laboratories to accurately perform the necessary testing in the presence of infused factor VIII, especially among patients who were receiving prophylactic therapy (unpublished CDC data). To address this barrier, the CDC Division of Blood Disorder's laboratory has developed and validated a modified testing method that can be used to screen for an inhibitor regardless of treatment status23. This method is being disseminated to local laboratories and its use for regular screening for inhibitors should become part of the standard of care for HTCs. The UDC has recently been updated (now called Community Counts) to include national monitoring of inhibitors through the use of both local and CDC testing of blood specimens collected as part of the surveillance. Using data from Community Counts will allow CDC to monitor the adoption of this important testing as a new standard of care. Current Status of the National Hemophilia Program and long term objectives CDC's Public Health Surveillance for the prevention of complications of bleeding and clotting disorders cooperative agreement is the funding mechanism through which its surveillance and prevention programs are funded. In 2011, the RHN applied for the CDC funding through ATHN. As part of the new cooperative agreement, the data elements collected through the new surveillance system were revised based on recommendations from an expert panel through a collaborative process between CDC scientists and a scientific committee comprised of clinicians organised and supported by ATHN. Community Counts, the new surveillance system has three components: 1) the HTC Population Profile; 2) Mortality Reporting; and 3) the Registry for Bleeding Disorders Surveillance. The first component collects a minimum set of demographic and clinical information on all of the patients with bleeding and clotting disorders who are receiving care within the RHN. The second component collects limited data about causes of death. The third component will be a more in-depth data collection project that will collect more complex data elements on health and complications, including collecting biologic samples for infectious disease and inhibitor surveillance. This collaboration marks an exciting new prospect for the haemophilia community as some data elements from the CDC's project will be utilised by the RHN and NHPCC to report as the national data source for several developmental Healthy People 2020 performance measures within the blood disorders and blood safety
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This population-based surveillance system was replaced in 1998 with the Universal Data Collection (UDC) surveillance system established in the HTC network in order to collect the more detailed data needed to inform and evaluate public health prevention programs13. For example, analyses of UDC data on height and weight and joint range of motion measurements lead to the recognition that increased body mass index (a measure of body adiposity) was associated with decreased joint mobility regardless of haemophilia severity14. Based on these findings it became clear that overweight and obesity, at epidemic levels in the general US population, was not only just as common among boys and men with haemophilia but also was contributing to preventable joint disease. Public health messages about the need to maintain a healthy weight to protect joints are part of the prevention strategies implemented because of these findings. Continued surveillance will be used to evaluate these prevention efforts. The UDC also included annual blood sample collection with infectious disease testing performed at CDC in order to assess the safety of treatment products. Results of this surveillance have provided evidence for the safety of these products as well as the need for continued vigilance15,16. UDC surveillance data have also been used to examine patterns of care within the HTC network. For example, the administration of treatment product on a regular basis to prevent bleeding episodes, called prophylaxis therapy, has been shown to prevent joint disease in young boys with haemophilia17. However, in an analysis of UDC data there was a full spectrum of variation from 0% to 100% of youth in individual HTCs who were receiving prophylaxis therapy18. These kinds of analyses can be useful both to monitor the adoption of standards of care as well as to gain an understanding of facilitators and barriers to the practices by studying the characteristics of HTCs with high and low adoption rates. Another example of using surveillance data is in the identification of an inhibitor, which is currently the most serious complication of haemophilia treatment. An inhibitor is an antibody to treatment product that develops in up to one-third of people with haemophilia and renders it ineffective in preventing or stopping bleeding episodes. People with an inhibitor are more likely to be hospitalised for a bleeding complication and treatment costs are far greater than those without an inhibitor19-21. A blood test is necessary for diagnosing an inhibitor. Earlier identification of inhibitors, accomplished through regular screening, leads to higher inhibitor eradication success22. From 2006-2010, only 46% of patients with severe haemophilia (those at highest risk of an inhibitor) had a screening test performed (unpublished CDC data).
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improvements for those living with high cost diseases or having pre-existing conditions such as haemophilia. The substantial growth in managed care plans and the many changes to private and public insurance programs made by the Affordable Care Act are having direct and indirect consequences for the bleeding disorders community. The Affordable Care Act provides for the establishment of essential health benefits that must be offered by new plans available in the insurance market. However, the choice of health care providers available to an insured within a plan is left to the insurance plan provider, subject to rules requiring access to a minimum percentage of the essential community providers in the plan service area27. These rules encourage, but do not require, insurers to include access to a federally funded HTC within their provider networks. To reduce reliance on expensive drugs and incentivize patients to choose lower-cost generic alternatives, if available, some plans are requiring higher cost-sharing (co-payments and coinsurance) for medications such as clotting factor concentrates. While the Affordable Care Act has the potential to increase patient access to needed treatments and services, it is too early to evaluate how this translates into outcomes for people with bleeding disorders. The Affordable Care Act also contained provisions placing a greater emphasis on evidence-based medicine and performance outcomes measures. Thus, establishing and bolstering evidence-based standards and recommendations to support haemophilia care and its delivery through a recognised HTC has taken on an increased importance. Over many years, the NHF and its MASAC have led the development of a range of standards and recommendations addressing clinical management, treatment safety, and care delivery for those living with a bleeding disorder. Issued in the form of recommendations, MASAC guidelines set the standard of care and are frequently referred to by an international array of physicians, medical schools, pharmacists, emergency room personnel, insurance companies, patients and others. MASAC is comprised of scientists, physicians, allied health professionals, patients and government liaisons from the relevant federal agencies within the US Department of Health and Human Services (CDC, Food and Drug Administration, MCHB and the National Institutes of Health). The full list of recommendations are available on the NHF website28. Adherence to MASAC recommendations is widely promoted, however, outside of the specific requirements with the NHP compliance with other MASAC recommendations is voluntary. Two MASAC recommendations are of particular note with regard to the oversight and management of US HTCs: 1) MASAC Recommendation 132 - Standards and Criteria for Persons with Congenital Bleeding Disorders29.
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topic objective24. Healthy People is a national effort by the US Department of Health and Human Services to develop measures and benchmarks to track national progress, in 10-year cycles, on government efforts to improve the public health of the country25. With the new regional organization and the development of ATHN as the national coordinating center, the NHP is well placed to accomplish goals and activities that have not previously been possible given the lack of focus on working on a national level. Since June 2012, the RHN and the NHPCC has completed the first ever technical assistance needs assessment for HTC Staff, currently under analysis, which will provide insight to HRSA on the help needed by HTC staff both in the management of patient care and in navigating the federal grant process as well as informing HRSA how the NHPCC can begin to provide needed services. The program is also currently analyzing the results of the first national HTC patient needs assessment that was a requirement for both the RHNs and the NHPCC to accomplish, so that the haemophilia community can use the results to set national priorities for the program to help with the development of a national evaluation plan to track the improvement of the care received in the coming years. A third party evaluation consultant has been contracted by both the NHPCC and the regions to analyze the results of the needs assessment, on both a regional and national level. The results of the analysis will be utilised to set national priorities for the NHP through a multistakeholder process involving the regions, the NHPCC, consumer advocacy groups, and our federal partners. The national priorities, once determined, will form the basis of a national evaluation plan to track patient outcomes for those who receive care through the NHP. Results of the patient needs assessment will be widely disseminated within the federal program and haemophilia consumer advocacy groups and other stakeholders within the bleeding community. Finally, these major projects and increased requirements for data collection and evaluation plans will inform the development of standards of care and methods for dissemination of best practices, which will require the assistance and expertise of the National Hemophilia Foundation's MASAC, whose efforts are described below. The role of the NHF MASAC in standard setting Impact of the Affordable Care Act Federal enactment of the Patient Protection and Affordable Care Act26 on March 23, 2010 came with a promise to provide quality affordable health care for all Americans. Since then, the US health care system has been undergoing a major overhaul impacting both insurance availability and the delivery of care. The Affordable Care Act contains many long sought
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widely cited examples of best practice of model legislation are New Jersey and more recently Missouri31-33. As this article was written, a number of states are considering adoption of similar statutory or regulatory provisions. The National Association of Boards of Pharmacy recently featured a news article profiling the NHF model standards of service and is working with NHF to achieve uniformity in the regulation of this specialty area34. In 1997, New Jersey included language in state Health Maintenance Organization (HMO) regulations securing access to and reimbursement for care at HTCs. In 2000, legislation was enacted requiring HMO and managed care insurers to comply with standards in the provision of benefits to patients with haemophilia31. Insurance carriers offering managed care health benefit plans in New Jersey must contract with designated health care providers for the delivery of services for the home treatment of bleeding episodes associated with haemophilia, including the purchase of blood products and blood infusion equipment. The State maintains an on-line registry of designated haemophilia home care providers meeting statutory requirements32. Building on the New Jersey precedent, in 2011, Missouri enacted legislation33 creating standards of care for pharmacies dispensing blood-clotting therapies. The Missouri HealthNet Division (which oversees Medicaid) previously covered blood clotting products and services. The law specifically adds blood clotting equipment, supplies, and assessments in the home to the list of Missouri HealthNet benefits.
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Last revised in 2002, MASAC Recommendation 132 outlines comprehensive standards of care and lists services that should be provided by HTCs. The recommendation includes an extensive discussion of the delivery of integrated, multi-disciplinary, comprehensive care. The recommendation places an emphasis on early diagnosis and intervention to prevent disease complications. The composition, general and specific functions, availability and training of the Comprehensive C a r e Te a m d e p e n d o n p a t i e n t c e n s u s a n d individual circumstance. The staff and services required for proper HIV care for men and women, haepatitis-related services, care for women with bleeding disorders, the need for culturally sensitive outreach programs, and access to clinical research protocols are also addressed. Within the NHP, all HTCs are required and expected to comply with and follow the guidelines for elements and core services of the HTC-based model of care delineated in Recommendation 132. 2) MASAC Recommendation 188 - Standards of Service for Pharmacy Providers of Clotting Factor Concentrates for Home Use to Patients with Bleeding Disorders30. A varied range of pharmacy providers supply clotting factor concentrates to patients for treatment at home. The pharmacy providers are principally specialty pharmacies or factor programs administered through the HTC. When patients do not receive optimal service from these providers, there is potential for adverse health events that lead to poor outcomes and/ or increased costs30. Given challenges in achieving optimal service, MASAC adopted standards of service for pharmacy providers. MASAC acknowledged the necessity of cost efficiency in the provision of health care, yet emphasised that cost efficiency should not occur at the expense of quality patient care. The purpose of the recommendation is to establish minimum standards of service for pharmacy providers to meet the specific needs of individuals with bleeding disorders. The standards address pharmacy provider staff knowledge, availability of clotting factor concentrates and ancillary supplies, processing of prescription orders, hours of operation, access to pharmacy staff, delivery, recordkeeping, billing and product recall. Over 30 national specialty pharmacies and most US HTCs have voluntarily self-reported they have met or exceeded the standards of Recommendation 188. At the state level, various legislative and regulatory initiatives have been implemented to promote optimal access to home care services to further reinforce the importance of pharmacy standards. The most prominent and
Development of evidence-based clinical practice guidelines for Comprehensive Care NHF is acutely aware of the major ongoing changes in health care occurring in the US. In 2012, the NHF held a strategic summit to develop a plan for haemophilia care within the evolving US health care environment. The Summit report addressed the implications of the Affordable Care Act and provided a series of strategic recommendations35. Among these was a call for NHF to sponsor the production and maintenance of evidence-based clinical practice guidelines (CPGs). Health plans are taking note of the geographic variations in practice and standards of practice (e.g. prophylaxis regimens) and clinical behavior (e.g. use of comprehensive care services). Such variations suggest insufficient evidence to support practice or a lack of clinician concurrence about, or adherence to, evidence-based practice. Recognizing the value of establishing, promoting, and adhering to CPGs and associated standards of practice, the NHF and its MASAC leadership launched an initiative to develop a comprehensive set of evidence-based CPGs to support patient-centered clinical decision-making and optimize care.
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Understanding Title V of the Social Security Act: A Guide to the Provisions of the Federal Maternal and Child Health Block Grant. Maternal and Child Health Bureau, Health Resources and Services Administration., Available at: http://www. amchp.org/AboutTitleV/Documents/UnderstandingTitleV. pdf. Accessed on 18/01/2014. Funding Opportunity Announcement: Regional Hemophilia Network. Announcement No. HRSA-12-133, CFDA# 93.110, FY 2012. Release Date: 11/16/2011; Due Date: 01/10/2012. Available at: https://grants3.hrsa.gov/2010/Web2External/ Platform/Interface/DisplayAttachment.aspx?dm_rtc=16&dm_ attid=89e6028e-37f2-4411-8334-acd06b02d5db&dm_ attinst=0. Accessed on 18/01/2014. NHF MASAC Recommendation 132 - Standards and Criteria for the care of Persons with Congenital Bleeding Disorders. National Hemophilia Foundation. Available at: http://www. hemophilia.org/NHFWeb/Resource/StaticPages/menu0/ menu5/menu57/masac132.pdf. Accessed on 20/01/2014. Funding Opportunity Announcement: Regional Hemophilia Network. Announcement No. HRSA-12-133, CFDA# 93.110, FY 2012. Release Date: 11/16/2011; Due Date: 01/10/2012. Available at: https://grants3.hrsa.gov/2010/Web2External/ Platform/Interface/DisplayAttachment.aspx?dm_rtc=16&dm_ attid=89e6028e-37f2-4411-8334-acd06b02d5db&dm_ attinst=0. Accessed on 18/01/2014. Funding Opportunity Announcement: National Hemophilia Program Coordinating Center. Announcement No. HRSA-12135, CFDA# 93.110, FY 2012. Release Date: 11/16/2011; Due Date: 12/30/2011. Available at: https://grants3.hrsa.gov/2010/ Web2External/Interface/FundingCycle/ExternalView. aspx?&fCycleID=EDA323E3-02AC-483A-ABE6-92474D2 335EF&txtAction=View+Details&submitAction=Go&View Mode=EU. Accessed on 18/01/14. Aschman DJ, Abshire TC, Shapiro AD, et al. A CommunityBased Partnership to Promote Information Infrastructure for Bleeding Disorders. Am J Prev Med 2011; 41 (6S4): S332-7. Available at: http://www.ajpmonline.org/article/S07493797%2811%2900680-5/fulltext. Accessed on 20/01/2014. Thacker SB, Berkelman RL. Public health surveillance in the United States. Epidemiol Rev 1988; 10: 164-90. Soucie JM, Jackson D, Evatt B. The occurrence of hemophilia in the United States. Am J Hematol 1998; 59: 288-94. Soucie JM, Nuss R, Evatt B, et al and the Hemophilia Surveillance System Project Investigators. Mortality among males with hemophilia: relations with source of medical care. Blood 2000; 96: 437-42. Soucie JM, Symons J, Evatt B, et al and the Hemophilia Surveillance System Project Investigators. Home-based factor infusion therapy and hospitalization for bleeding complications among males with hemophilia. Haemophilia 2001; 7:198-206. Soucie JM, McAlister S, McClellan A, et al. The universal data collection surveillance system for rare bleeding disorders. Am J Prev Med 2010; 38 (4 Suppl): S475-81. Soucie JM, Cianfrini C, Janco RL, et al. Joint range of motion limitations among young males with hemophilia: Prevalence and risk factors. Blood 2004; 103: 2467-73. Centers for Disease Control and Prevention. Blood safety monitoring among persons with bleeding disorders - United States, May 1998-June 2002. MMWR Morb Mortal Wkly Rep 2003; 51: 1152-4. Soucie JM, De Staercke C, Monahan PE, et al. Evidence for the Transmission of Parvovirus B19 in Patients with Bleeding Disorders Treated with Plasma-derived Factor Concentrates in the Era of Nucleic Acid Test (NAT) Screening. Transfusion 2013; 53: 1217-25. Manco-Johnson MJ, Abshire TC, Shapiro AD, et al. Prophylaxis versus episodic treatment to prevent joint disease in boys with severe hemophilia. N Engl J Med 2007; 357: 535-44.
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The initial phase of the CPG initiative addresses the comprehensive care model for haemophilia. In addition to recognizing the importance of a range of coordinated services to most patients with haemophilia, addressing the comprehensive care model as the first evidence-based CPG will establish a foundation upon which more specific CPGs addressing other aspects of care may be developed. The comprehensive care CPG will: - define comprehensive care as it relates to the haemophilia patient; - specify the coordinated set of diagnostic, therapeutic, and certain auxiliary and supplemental services that are most important for haemophilia patients across the U.S.; - identify the range of clinical and non-clinical members of a haemophilia comprehensive care team (including, e.g., social workers, dentists); - identify best-practices and evidence-based standards of comprehensive/coordinated care for haemophilia treatment centers (HTCs) and individual clinical practices. NHF foresees that the development of the comprehensive care CPG will provide a catalyst to promote harmonization of care delivery and reduce practice variations among the US HTC system. An agreed evidence-based benchmark would allow for both internal and external evaluation of adherence to best practices and benchmark available services with an HTC. Ultimately, it will serve as an important starting point for establishment of a US HTC self-audit or accreditation system.
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Keywords: Haemophilia Treatment Center, Program evaluation, Child Health Bureau (MCHB), Centers for Disease Control and Prevention (CDC), National Hemophilia Foundation (NHF).
Acknowledgments and disclosures
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Mark Skinner serves as a consultant to the National Hemophilia Foundation. CDC Disclaimer: The findings and conclusions in this report are those of the authors and do not necessarily represent the official position of the Centers for Disease Control and Prevention. The Authors declare no conflicts of interest.
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References 1)
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Katherine B. Oettinger. Title V of the Social Security Act: What it has meant for children. Social Security Bulletin. V23; No8, pgs 39-50, August, 1960. Available at: http://www. ssa.gov/policy/docs/ssb/v23n8/v23n8p39.pdf. Accessed on 19/01/2014. Understanding Title V of the Social Security Act: A Guide to the Provisions of the Federal Maternal and Child Health Block Grant. Maternal and Child Health Bureau, Health Resources and Services Administration. Available at: http://www.amchp. org/AboutTitleV/Documents/UnderstandingTitleV.pdf. Accessed on 18/01/2014.
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29) NHF MASAC Recommendation 132 - Standards and Criteria for the care of Persons with Congenital Bleeding Disorders. National Hemophilia Foundation. Available at: http://www. hemophilia.org/NHFWeb/Resource/StaticPages/menu0/ menu5/menu57/masac132.pdf. Accessed on 20/03/2014. 30) NHF MASAC Recommendation 188 - Standards of Service for Pharmacy Providers of Clotting Factor Concentrates for Home Use to Patients with Bleeding Disorders. National Hemophilia Foundation. Available at: http://www.hemophilia. org/NHFWeb/Resource/StaticPages/menu0/menu5/menu57/ masac188.pdf. Accessed on 20/03/2014. 31) New Jersey Statues Annotated. Section 1 of L. 2001, c. 121, codified at N.J.S.A. 26:2S-10.1, and sections 3 through 10 of L. 2001, c. 121, variously codified throughout Titles 17, 17B and 26. Available at: http://njlaw.rutgers.edu/cgi-bin/njstats/ showsect.cgi?title=26&chapter=2S§ion=10.1&actn=get sect. Accessed on 20/03/2014. 32) New Jersey Designated Hemophilia Providers. Available at: http://www.state.nj.us/dobi/division_insurance/managedcare/ mchemoprov.htm. Accessed on 20/03/2014. 33) Missouri Revised Statues. RSMo 338.400. Available at: http:// www.moga.mo.gov/statutes/C300-399/3380000400.HTM. Accessed on 20/03/2014. 34) States Look at Standards of Service for Pharmacies Serving Hemophilia Patients. NABP Newsletter. 2013; June-July: 12830. Available at: http://www.nabp.net/system/redactor_assets/ documents/618/Final_June-July_2013_Newsletter.pdf. Accessed on 20/03/2014. 35) National Hemophilia Foundation: Strategic Summit Report. October 2012. National Hemophilia Foundation. Available at: http://www.hemophilia.org/NHFWeb/MainPgs/MainNHF. aspx?menuid=119&contentid=2120. Accessed on 20/03/2014.
Correspondence: Mark W. Skinner National Hemophilia Foundation 1155 23rd Street NW #3A Washington, DC 20037, USA e-mail: [email protected]
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18) Soucie JM, McAlister S, McClellan A, et al. The universal data collection surveillance system for rare bleeding disorders. Am J Prev Med 2010; 38 (4 Suppl): S475-81. 19) Soucie JM, Symons J, Evatt B, et al and the Hemophilia Surveillance System Project Investigators. Home-based factor infusion therapy and hospitalization for bleeding complications among males with hemophilia. Haemophilia 2001; 7: 198-206. 20) Guh S, Grosse SD, McAlister S, et al. Healthcare expenditures for males with haemophilia and employer-sponsored insurance in the United States, 2008. Haemophilia 2012; 18: 268-75. 21) Guh S, Grosse SD, McAlister S, et al. Health care expenditures for Medicaid-covered males with haemophilia in the United States, 2008. Haemophilia. 2012; 18: 276-83. 22) DiMichele DM. Immune tolerance in haemophilia: the long journey to the fork in the road. Brit J Haematol 2012; 159: 123-34. 23) Miller CH, Rice AS, Boylan B, et al, the Hemophilia Inhibitor Research Study Investigators. Comparison of clot-based, chromogenic, and fluorescence assays for measurement of factor VIII inhibitors in the U.S. Hemophilia Inhibitor Research Study (HIRS). J Thromb Haemost 2013: 11; 1300-9. 24) Healthy People 2020. Blood Disorders and Blood Safety Topic Objectives. Available at: http://www.healthypeople.gov/2020/ topicsobjectives2020/overview.aspx?topicid=4. Accessed on 20/01/2014. 25) HHS News Press Release: HHS announces the nation's new health promotion and disease prevention agenda. OASH Press Office. Released December 2, 2010. Available at: http:// www.healthypeople.gov/2020/about/DefaultPressRelease.pdf. Accessed on 20/03/2014. 26) Pub L. 111-148, 111th United States Congress. Washington, DC. March 23, 2010. 27) HHS Centers for Medicare and Medicaid Services. Letter to Issuers in the Federally-facilitated Marketplace. March 14, 2014. Available at: http://www.cms.gov/CCIIO/Resources/ Regulations-and-Guidance/Downloads/2015-final-issuerletter-3-14-2014.pdf. Accessed on 26/03/2014. 28) NHF MASAC Recommendations. National Hemophilia Foundation. Available at: http://www.hemophilia.org/NHFWeb/ MainPgs/MainNHF.aspx?menuid=157&contentid=347. Accessed on 20/03/2014.
Blood Transfus 2014; 12 Suppl 3: s542-8 DOI 10.2450/2014.0019-14s s548 All rights reserved - For personal use only No other uses without permission
