Sickle Cell Disease Summit: From clinical and ... - Wiley Online Library

Position Paper Sickle cell disease summit: From clinical and research disparity to action Kathryn Hassell,1* Betty Pace,2 Winfred Wang,3 Roshni Kulkarni,4 Naomi Luban,5 Cage S. Johnson,6 James Eckman,7 Peter Lane,8 and William G. Woods8 The American Society of Pediatric Hematology/Oncology Sickle Cell Summit brought together a broad range of constituencies to identify a unified approach to healthcare and research disparities for sickle cell disease. Recommendations included the following: (1) speak with a unified voice representing all constituencies; (2) optimize access to care from knowledgeable health care providers and create a medical home for all individuals with the disease; (3) utilize population-based surveillance to measure outcomes; (4) develop overall approaches to basic, translational, clinical, and health services research; (5) enhance the community role in advocacy, education, service, and fundraising. Taskforces were identified to effect implemenC 2008 Wiley-Liss, Inc. tation. Am. J. Hematol. 84:39–45, 2009. V

federal institutions and community-based organizations. Furthermore, despite substantial accomplishments over several decades, translational and clinical research efforts have been hampered by limited funding and a lack of a fully effective, organized research structure. Comprehensive care models and coordinated research efforts for conditions such as hemophilia and childhood cancer were identified to inform approaches for SCD. This material is summarized on the ASPHO website (www.ASPHO.org). The Summit group was charged with developing actions by which the following vision might be achieved:

Introduction The Sickle Cell Summit meeting, held in Washington, DC, on June 28–29, 2007, was inspired by the recognition of healthcare disparity—both in the clinical care of persons with sickle cell disease (SCD) and in the research efforts directed toward understanding and treating this condition. The Summit represented the first major effort by the American Society of Pediatric Hematology Oncology (ASPHO) in advocacy and was convened to develop a unified front to improve research and the care of individuals with sickle cell disease. Healthcare disparity related to SCD was clearly described in 1970 by Dr. Roland Scott in an editorial entitled, ‘‘Sickle Cell Anemia: High Prevalence and Low Priority’’ [1]. Despite the National Sickle Cell Anemia Control Act of 1972, and, more recently, the Sickle Cell Treatment Act of 2003, disparity remains an issue in several areas. Smith et al. in a recent article entitled, ‘‘Sickle Cell Disease: A Question of Equity and Quality’’ [2], elucidated these disparities. Despite the major advances in the treatment of SCD that have occurred in the last 30 years, there is also a lack of equity in the quality of clinical care provided to patients with SCD as evidenced by a failure to apply knowledge obtained from research to much of the at-risk sickle cell patient population. Racial and ethnic disparities in health and healthcare are described in the Institute of Medicine report, Unequal Treatment: Confronting Racial and Ethnic Disparities in Healthcare [3], and the Secretary of Health and Human Services National Healthcare Disparities Report [4]. Thus, despite the substantial advances in the treatment of SCD which have occurred in the last 30 years, there is a lack of equity in the quality of clinical care provided to patients with SCD as evidenced by a failure to apply knowledge obtained from research to much of the at-risk population. The Sickle Cell Summit was conceived as a step in confronting the healthcare disparities associated with SCD.

‘‘An adequately funded, coordinated, comprehensive, and integrated national model for care of persons with SCD, involving all stake holders, will lead to improved outcomes for all Americans with the disease; lay the foundation for conducting health services, outcomes, and clinical/translational/basic research; and ultimately improve outcomes of future persons globally with SCD.’’ Through the Summit process, small working groups developed and ultimately all participants refined consensus with regard to goals and opportunities. Five major areas of opportunity, with specific actions for implementation over the next 1–3 years, were identified. The findings and conclusions in this report are those of the authors and do not necessarily represent the views of the Centers for Disease Control and Prevention. 1 Division of Hematology, Department of Medicine, University of Colorado Denver Health Sciences Center, Denver, CO; 2Department of Molecular and Cell Biology, University of Texas at Dallas, Dallas, TX; 3Department of Hematology, St. Jude Children’s Research Hospital, Memphis, TN; 4Department of Pediatrics and Human Development, Michigan State University, East Lansing, MI; 5Department of Laboratory Medicine and Pathology, Children’s National Medical Center, Washington, DC; 6Department of Medicine, Keck School of Medicine, University of Southern California, Los Angeles, CA; 7 Department of Hematology/Oncology, Emory School of Medicine, Atlanta, GA; 8Aflac Cancer Center and Blood Disorder Service, Children’s Healthcare of Atlanta, Emory University, Atlanta, GA.

The Summit Development and Goals A diverse group of 65 individuals representing major federal funding and health care policy agencies, foundations, professional societies, community organizations, patients, and expert clinicians and researchers with an interest in SCD (see Appendices A and B) were convened by ASPHO in June 2007. During the Summit, a number of issues were presented and discussed, including limitations in access and utilization for persons with SCD, despite the efforts of

Conflict of Interest: Nothing to report. *Correspondence to: Kathryn Hassell, Professor of Medicine, University of Colorado Denver Anschutz Medical Campus, 13121 East 17th Avenue, C222, P.O. Box 6511, Aurora, CO 80045. E-mail: [email protected] Received for publication 2 October 2008; Accepted 6 October 2008 Am. J. Hematol. 84:39–45, 2009. Published online 18 October 2008 in Wiley InterScience (www.interscience. wiley.com). DOI: 10.1002/ajh.21315

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American Journal of Hematology

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http://www3.interscience.wiley.com/cgi-bin/jhome/35105

position paper Summit Results: Opportunities and Actions Speak with one voice Overcoming the barriers, which inhibit the development of a coordinated, comprehensive system of health care and slow the progress of research in SCD, will take a concerted effort on the part of all stakeholders. Individuals and families living with the disease, their health care providers, community-based and advocacy organizations, state and federal agencies, and a variety of national organizations all must contribute to the identification of common goals and basic strategies to achieve them. The ASPHO Summit has initiated communication among a wide variety of constituents in order to: a. Establish a national task force with broad representation from all stakeholders b. Encourage open dialog, coordination, and collaboration between individuals with SCD, community-based organizations, the Sickle Cell Disease Association of America (SCDAA), federal agencies, national healthcare and professional organizations, and the investigator/provider community c. Develop and utilize a clear case statement describing common goals and strategic approaches to achieve those goals d. Substantially improve fundraising i. Identify an influential national spokesperson ii. Develop a national strategy for markedly increasing private philanthropic efforts in SCD iii. Lobby Congress to earmark specific funds for sickle cell activities, to include: 1. Funding for Centers for Disease Control (CDC) to conduct SCD surveillance 2. Funding for an expanded network of clinical care centers through the Health Resources and Services Administration (HRSA), which would also serve as clinical trials centers through National Heart, Lung, and Blood Institute (NHLBI) of the National Institutes of Health (NIH) 3. Agency for Healthcare Research and Quality (AHRQ) mandate for outcomes and quality assurance measures

Access to care from knowledgeable health care providers in a patient-centered medical home To optimize outcomes for all individuals in the United States living with SCD through the development of an adequately funded, coordinated, comprehensive, and integrated national model of care will require significant effort. Initial energy should focus in the following areas: a. Optimize access to and utilization of health care system In order to benefit from a coordinated, comprehensive healthcare system, individuals with SCD will need to seek care when appropriate and be assured of access to the health care system. i. Acknowledge potential lack of trust between affected individuals and the health care system ii. Increase individual and community education and expectations for appropriate health care from knowledgeable providers, optimizing the role of community-based organizations in: 1. Increasing public awareness of SCD and sickle cell trait 2. Individual and community education about SCD management

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3. Serving as liaison between the community and providers, and as advocate for individuals within the health care system iii. Optimize insurance coverage for individuals within currently available programs 1. Lobby states to adopt the Medicaid provision outlined in the American Jobs Creation Act of 2004 2. Ensure that state programs and insurance plans adequately cover appropriate SCD management iv. Lobby at the state and federal level for universal coverage of all individuals with SCD b. Build a system which provides a patient-centered medical home for all individuals with SCD i. Needs Assessment: Identification of the current setting of health care, utilization patterns, available resources, and individual preferences 1. Encourage state and federal agencies to utilize available administrative data sets 2. Engage community-based organizations to locate individuals with SCD and identify local medical resources ii. Care System Infrastructure: A medical home for all individuals with SCD should provide preventive care, assure ambulatory and inpatient care for acute illnesses, 24 hours a day, 7 days a week, identify the need for subspecialty consultation and referrals, knowing from whom and where these can be obtained, and provide continuity of care over an extended period of time. This medical home may reside within a primary or specialty care setting, depending on individual preferences and local resources. Primary care medical homes should be linked with specialty centers of excellence, distributed throughout the U.S. based on demographic and geographical considerations, where advanced expertise in SCD management is readily available and easily accessed. 1. Models from other disease states: Although coordinated comprehensive models of care have been successfully implemented for other conditions, the population affected by SCD may face unique challenges in areas of resources, funding, and advocacy. a. Assess the similarities and differences between SCD and other disease states for which coordinated, comprehensive care models exist (e.g. hemophilia, cystic fibrosis, childhood malignancies) as regards medical management, available resources, and compensation for healthcare providers. b. Identify strategies to expand or create necessary elements in order to implement an effective model which accounts for unique aspects of SCD 2. Models from prior and ongoing work in SCD: a. Compile examples of models of care and outcomes from programs providing comprehensive SCD management b. Adapt successful elements of the SCD Treatment Demonstration Program as they become available 3. Emphasis on pain management: Acute and chronic pain represents the most challenging aspect of SCD. a. Encourage development of multidisciplinary teams for pain management


position paper b. Provide templates and evidence of improved outcomes for successful outpatient/day-hospital settings for pain management c. Collaborate with emergency department health care professionals to optimize management of SCD pain d. Exploit emerging scientific information on the biology of pain to encourage investigation into possible unique features of SCD for development of new approaches to prevention and management 4. Encourage adaptation of quality improvement and quality assurance initiatives in all health care settings directed at SCD management iii. Ensure an adequate number of health care professionals who have appropriate training to provide knowledgeable care for individuals with SCD 1. Ensure awareness and basic SCD management education for current providers in all health care settings, including primary and specialty care. 2. Encourage participation of providers from diverse cultural and practice settings, including those representative of the affected population 3. Lobby individual states to adopt the Medicaid provision of the American Jobs Creation Act of 2004 and to take other measures to ensure adequate reimbursement for appropriate SCD management 4. Begin awareness of SCD and associated career opportunities at an early level in education and continue throughout all levels of training (high school, preprofessional/ college, professional schools, postgraduate training) 5. Emphasize SCD in the medical training pipeline (e.g. NIH K career development awards, T32 training grants), with a special emphasis on adult sickle cell issues c. Develop, disseminate, and facilitate implementation of best practices recommendations for all individuals with SCD A coordinated, comprehensive healthcare system will require standardized basic SCD management strategies; this system will in turn enhance the effective implementation of proven therapeutic approaches to SCD management. Best practices recommendations developed through the consensus process provide needed guidance in areas where management remains to be clarified by well-designed clinical research. This process should include individuals and families, as well as input from the multiple disciplines and specialties, impacted by SCD. i. Consider adoption of the American Academy of Pediatrics recommendations for the management of SCD [5] in all pediatric care settings ii. Identify, coordinate, and expand ongoing consensus efforts to develop and disseminate adult SCD recommendations to all adult care settings iii. Incorporate best practices into quality improvement/quality assurance initiatives iv. Review criteria for current Joint Commission for the Accreditation of Healthcare Organizations (JACHO) disease-specific certification of hospital-based sickle cell programs [6]


d. Disseminate and facilitate adoption of standardized transfusion practicesRed blood cell transfusion is a resource-intensive, multidisciplinary therapy for SCD which requires close collaboration among transfusion specialists, hematologists, health care professionals, transfusion recipients, the donor community, and blood collection and distribution facilities. Successful development, dissemination, and implementation of a standardized approach will not only directly improve the management of SCD but will also inform efforts to institute a broad array of best practices. i. Convene an expert panel to establish standards for RBC minor antigen matching of blood products and the appropriate use of simple and exchange transfusion ii. Work with state Medicaid programs and other insurers to ensure adequate reimbursement for transfusion support iii. Increase public awareness and engage community-based organizations to increase both blood and hematopoietic stem cell donation among donor bases that will provide the best possible products for transfusion and transplantation

Population-based surveillance to measure outcomes The ability to accurately determine the benefits of a coordinated, comprehensive healthcare system and assess ongoing needs is dependent on the ability to identify the population affected by SCD and determine health outcomes, cost-effectiveness, quality of life, and satisfaction. The optimal approach to this task for the SCD population remains to be determined. The unquestionable utility of a representative database, surveillance system, or population registry must be weighed against the effort and resources needed to overcome potential challenges, including individual privacy concerns. Coordinated, nonduplicative efforts are needed to obtain useful information while avoiding the waste of scarce resources and the overburdening of participating individuals. a. Optimize use of the information collected through universal newborn screening i. Assess the accuracy and completeness of current efforts to collect national data ii. Expand newborn screening follow-up programs 1. Build a surveillance system based on the identification of affected newborns, which can be used to compare outcomes within coordinated systems of healthcare as compared with care outside such systems 2. Encourage federal support to individual states for this extended activity b. Encourage federal agencies (HRSA, CDC, AHRQ) to extract information regarding SCD from available administrative data sets and surveillance systems c. Encourage collaboration among federal agencies and organizations (HRSA, CDC, AHRQ, Centers for Medicare and Medicaid Services, National Quality Forum, JCAHO) to: i. coordinate and enhance surveillance activities ii. develop and monitor outcome measures. d. Coordinate ongoing efforts directed at an SCD registry to avoid duplication e. Clarify legal issues around Health Insurance Portability and Accountability Act (HIPAA) for each of these activities f. Develop and validate sickle-cell specific quality of life instruments

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position paper Basic, clinical, translational, and health services research Building on the significant progress made in the understanding of underlying pathophysiologic processes, translating findings through clinical research to improve life expectancy and quality of life for individual with SCD will require appropriate allocation of available resources and significant increases in research funding. Redistribution of resources and identification of new partners for research activities may alleviate some of the effects of the current funding constraints, but ultimately new resources from both the public and private sectors will be necessary to achieve research equity for SCD. a. Encourage NHLBI, as the historical ‘‘home’’ for SCD within the federal government, to: i. Assess and attempt to coordinate ongoing activities across institutes (e.g. National Institute of Diabetes and Digestive and Kidney Diseases, National Institute of Child Health and Human Development) ii. Publicize research opportunities from other institutes iii. Solicit cooperation from other Federal agencies to coordinate programs b. Review NHLBI’s new strategic plan to identify areas of emphasis for the Institute which pertain to SCD in all arenas c. Continue emphasis on basic science research i. Encourage NIH to improve scientific peer review process: 1. Utilize reviewers with appropriate level of knowledge about SCD 2. Assist with assignment to or creation of appropriate review groups, especially when applications cross disciplines or fields of expertise ii. Improve communication among SCD researchers to better disseminate awareness of grant opportunities across various institutes within NIH and from other sources d. Expand opportunity for translational/clinical research Conduct of translational and clinical research entails taking basic science advances into the clinical arena and directs the implementation of proven therapies into practice. The application of proven management strategies is enhanced when the research is conducted through a large, representative, communitybased network of clinical centers. These centers can coordinate and manage the disease and can provide a broadly representative, efficient, and cost-effective clinical research network. i. Consider a cooperative group network model similar to Children’s Oncology Group for sickle cell clinical research 1. Consider reallocation of current resources from duplicative programs to establish a network with subsequent increased funding to enhance support 2. Ensure that the network is broadly inclusive of qualified research centers demographically and geographically representative of the SCD population and reaches a substantial proportion of affected persons within the U.S. 3. Incorporate partnerships with communitybased organization ii. Explore the extent to which Clinical Translational Science Awards (CTSA) can be used to support sickle cell research

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iii. Encourage pharmaceutical company support in SCD research e. Enhance emphasis on health services research i. Identify, publicize, and utilize current grant opportunities which emphasize work in fields characterized by health disparities ii. Encourage a focus on SCD, especially as it related to quality of care, care delivery, and health policy f. Engage the community and community-based organizations in the development and completion of research

Enhanced role of the community Individuals, families, and communities affected by SCD are best able to advocate for themselves when they are aware of and educated about SCD. These empowered consumers should expect expert disease management from knowledgeable providers and the receipt of supportive services from appropriate governmental, private, and community sources; they are perhaps the most significant force for change. A support network consisting of academic centers, federally qualified healthcare centers (FQHC), local healthcare systems, SCD community-based organizations (CBOs), social agencies, and spiritual and religious groups, enables individuals with SCD to better cope with the disease. To sustain and grow this comprehensive support network will require rational integration of several inter-related activities including legislation, research, and fundraising among all stakeholder groups. The participation of individuals and families affected with SCD and local community leaders in the development and implementation of healthcare services, research priorities, and advocacy is essential. Additional funding from public and private resources to support SCD-CBOs, federally qualified healthcare centers (FQHCs), and community healthcare systems will be necessary to strengthen community-based programs and future collaborations with academic centers. a. Empower individuals, families, and communities living with sickle cell disease i. Develop public service campaigns and identify local/regional/national spokespersons to increase awareness of SCD ii. Ensure education and genetic counseling services to at-risk communities iii. Emphasize patient/family education in the medical home, promoting knowledgeable self-advocacy iv. Encourage utilization of medical identification cards or other methods of portable personal health information b. Maintain sustainable, culturally competent CBOs in communities at risk i. Incorporate CBOs into the provision of comprehensive care to individuals/families with SCD via the medical home, utilizing community-based programs for education, resource identification, and other support services ii. Offer all SCD-CBOs membership in SCDAA, which would 1. establish a set of minimum criteria defining features of successful CBOs 2. provide support to CBOs for program/services development 3. expand its constituency base for advocacy and lobbying activities


position paper c. Support clinical and basic research in sickle cell disease. i. Educate the sickle cell disease lay community and general public on the importance of research to improve clinical care. ii. Report to the community on the results of clinical trials and basic research findings and the consequent availability of new treatments. iii. Disseminate information about locally available clinical trials and assist in recruiting patients. iv. Advocate to legislators on behalf of funding for sickle cell disease research. v. Raise funds for direct support of sickle cell research on the local, state, and national levels Next Steps Progress in the area of SCD will require coordinated cooperative effort across a diversity of organizations, agencies, communities, and individuals. The Summit participants envision the formation of national task forces consisting of invested members in the following areas: National coordinating committee Access to care/medical home/health care services

delivery practices development, dissemination, and implementation Transfusion medicine and transplantation SCD surveillance system and data systems utilization/development Basic and clinical research Fundraising/lobbying

Best

Conclusion The ASPHO Sickle Cell Summit brought together 65 participants from a broad range of constituencies to discuss current challenges and opportunities, and develop a unified approach to research and the care of individuals and families affected by SCD. Moving forward to overcome disparities will require the sustained efforts of newly developed task forces and ultimately a long-term commitment to achieve the ambitious goals of the Summit meeting. References 1. Scott RB. Health care priority and sickle cell anemia. JAMA 1970;214:731–734. 2. Smith LA, Oyeku SO, Homer C, Zuckerman B. Sickle cell disease: A question of equity and quality. Pediatrics 2006;117:1763–1770. 3. Institute of Medicine. Unequal Treatment: Confronting Racial and Ethnic Disparities in Healthcare. Washington, DC: National Academy Press; 2003. 4. US Department of Health and Human Services and Agency for Healthcare Research and Quality. National Healthcare Disparities Report. Report 05– 0014. Rockville, MD: US Department of Health and Human Services and Agency for Healthcare Research and Quality; 2004. 5. Section on Hematology/Oncology Committee of Genetics: American Academy of Pediatrics. Health supervision for children with sickle cell disease. Pediatrics 2002:109:526–535. 6. Disease Specific Care Certification, Joint Commission for the Accreditation of Health Care Organizations. Available at: www.jointcommission.org/CertificationPrograms/Disease-SpecificCare/.

Appendix A: Planning Committee (*Presenters/Facilitators) William G. Woods, MD (Chair) Director, Aflac Cancer Center & Blood Disorders Service Emory School of Medicine/Children’s Healthcare Atlanta President, American Society of Pediatric Hematology/Oncology Atlanta, GA James R. Eckman, MD* Department of Hematology/ OncologyGrady Memorial Hospital Emory University, School of Medicine Atlanta, GA


Kathryn L. Hassell, MD* Director, Sickle Cell Treatment Research Center University of Colorado Health Sciences Center Denver, CO

Cage Johnson, MD* Director, Comprehensive Sickle Cell Center Keck School of Medicine University of Southern California Los Angeles, CA Stephanie Kart Government Relations and Practice Specialist American Society of Hematology Washington, DC Roshni Kulkarni, MD Director, Division of Blood Disorders National Center on Birth Defects and Developmental Disabilities Centers for Disease Control and Prevention Atlanta, GA Peter A. Lane, MD* Director, Pediatric SCD Program Aflac Cancer Center & Blood Disorders Service Emory School of Medicine/Children’s Healthcare of Atlanta Atlanta, GA Naomi L.C. Luban, MD* Director, Hemophilia Treatment Program and Transfusion-Hematology/Oncology Division Chair, Laboratory Medicine Children’s National Medical Center Washington, DC Marie Y. Mann, MD, MPH* Deputy Chief Genetic Services Branch Maternal & Child Health Bureau Health Resources & Services Administration Maternal & Child Health Bureau Health Resources & Services, Administration Rockville, MD Betty S. Pace, MD* Director, SCD Research Center Chief Medical Officer, SCD Association of America The University of Texas at Dallas, Department of Molecular and Cell Biology Richardson, TX Cynthia Porter Executive Director American Society of Pediatric Hematology/Oncology Glenview, IL Susan B. Shurin, MD Deputy Director National Heart Lung & Blood Institutes Department of Health & Human Services Bethesda, MD Winfred C. Wang, MD* Director, St. Jude Comprehensive Sickle Cell Center St. Jude Children’s Research Hospital Memphis, TN Gerald M. Woods, MD* Chief, Hematology/Oncology Director, Sickle Cell Program Children’s Mercy Hospital Kansas City, MO

Summit Participants (*Presenters) Abdu Alayash, PhD Chief, Laboratory of Biochemistry and Vascular Biology Center for Biologics Evaluation and Research Food and Drug Administration Rockville, MD

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position paper Hani Atrash, MD, MPH Associate Director for Program Development National Center on Birth Defects & Developmental Disabilities Centers for Disease Control & Prevention Atlanta, GA

Nancy S. Green, MD Associate Dean for Clinical Research Operations Columbia U. Medical Center Division of Pediatric Hematology New York, NY

Marcus Barnes Parent of Consumer Landover, MD

Scott Grosse, PhD Senior Health Economist National Center on Birth Defects & Developmental Disabilities Centers for Disease Control & Prevention Atlanta, GA

Lennette J. Benjamin, MD* Professor Clinical Director Comprehensive Sickle Cell Center Albert Einstein College of Medicine Department of Medicine Bronx, NY

Judith Hagopian, MSW Genetic Services Branch Division of Services for Children with Special Health Needs Maternal & Child Health Bureau Health Resources & Services Administration Rockville, MD

Richard Benjamin, MD, PhD Chief Medical Officer American Red Cross Blood Services Washington, DC

Johnson Haynes, MD Director USA Comprehensive Sickle Cell Center Mobile, AL

Sarah Beringer Staff, American Society of Hematology Washington, DC

Carlton Haywood, Jr., MA* Consumer Johns Hopkins Berman Institute of Bioethics Baltimore, MD

George Buchanan, MD* Director Southwestern Comprehensive Sickle Cell Center Dallas, TX Keyoni Carter Consumer Landover, MD Jeffrey Chell, MD Chief Executive Officer National Marrow Donor Program Minneapolis, MN Dennis Confer, MD Chief Medical Officer National Marrow Donor Program Minneapolis, MN Melissa Creary, MPH* Consumer Division of Hereditary Blood Disorders National Center on Birth Defects and Developmental Disabilities, Centers for Disease Control & Prevention Atlanta, GA Mark Del Monte, JD Assistant Director Department of Federal Affairs American Academy of Pediatrics Washington, DC George J. Dover, MD Johns Hopkins Hospital Baltimore, MD Matt Eckel Staff, American Society of Hematology Washington, DC Willarda V. Edwards, MD, MBA* President/Chief Operatoring Officer SCD Association of America Baltimore, MD

Cheryl A. Hillery, MD Program Director, SCD Children’s Hospital of Wisconsin Milwaukee, WI Christopher D. Hillyer, MD Immediate Past President American Association of Blood Banks Director, Transfusion Medicine Emory University Atlanta, GA Charles Homer, MD, MPH Chief Executive Officer National Initiative for Children’s Healthcare Quality Cambridge, MA Thomas Howard, MD Director Division of Pediatric Hematology/Oncology The Children’s Hospital Birmingham, AL Clinton H. Joiner, MD, PhD Director Sickle Cell Center Cincinnati Children’s Hospital Medical Center Cincinnati, OH J. Hoxi Jones* Consumer National Heart Lung and Blood Institute Advisory Council Houston, TX The Honorable John R. Lewis* Member, United States House of Representatives 5th Congressional District of Georgia Washington, DC Martha Liggett, Esq. Executive Director American Society of Hematology Washington, DC

Anita Estell, JD* Lobbyist Polsinelli Shalton Flanigan Suelthaus PC Washington, DC

Michele A. Lloyd-Puryear, MD, PhD* Chief, Genetic Services Branch Division of Services for Children with Special Health Needs Maternal & Child Health Bureau Health Resources and Services Association Rockville, MD

Bruce Evatt, MD Director Emeritus Division of Hereditary Blood Disorders Centers for Disease Control Atlanta, GA

Bertram H. Lubin, MD President/Director, Medical Research Children’s Hospital Oakland Research Institute Oakland, CA

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position paper Yvonne T. Maddox, PhD Deputy Director National Institute of Child Health and Human Development National Institutes of Health Bethesda, MD Lawrence A. McAndrew, FACHE President & Chief Executive Officer National Association of Children’s Hospitals and Related Institutions Alexandria, VA Ernestine W. Murray, RN, MAS EPC Program Task Order Officer Agency for Healthcare Quality and Research Department of Health and Human Services Rockville, MD Kwaku Ohene-Frempong, MD Director Comprehensive Sickle Cell Center Children’s Hospital of Philadelphia Philadelphia, PA Maura C. O’Leary, MD Administrative Officer Children’s Oncology Group Bethesda, MD Eileen M. Ouellette, MD JD Immediate Past President American Academy of Pediatrics Newton Center, MA Christopher S. Parker, PhD, MPH Deputy Director Division of Blood Disorders National Center on Birth Defects and Developmental Disabilities Centers for Disease Control and Prevention Atlanta, GA Charles Peterson, MD Director Division of Blood Diseases and Resources National Heart Lung and Blood Institute Bethesda, MD Winston Price, MD Past President National Medical Association Baldwin, NY Gladys Ashe Robinson, MEd* Executive Director SCD Association of the Piedmont Pleasant Garden, NC Monique Rorie Parent of Consumer Landover, MD Sonya I. Ross, MS Vice President, Programs and Services SCD Association of America Baltimore, MD Lauren A. Smith, MD, MPH Medical Director Medical-Legal Partnership for Children Boston Medical Center Boston, MA Sidney L. Strickland, Jr Government Relations Committee National Board of Directors SCD Association of America Washington, DC Francisco S. Sy, MD, DrPH Chief, Office of Community-Based Participatory Research & Outreach


Program Director National Center on Minority Health & Health Disparities National Institutes of Health Bethesda, MD Marilyn Telen, MD* American Society of Hematology Duke University Medical Center Division of Hematology Durham, NC Joseph Telfair, DrPH, MSW, MPH Professor Public Health Research & Practice Department of Public Health Education School of Health and Human Performance University of North Carolina at Greensboro Greensboro, NC Sharon Terry, MA President and Chief Executive Officer Genetic Alliance Washington, DC Alexis A. Thompson, MD, MPH Director, Pediatric Hematology Services Children’s Memorial Hospital Chicago, IL Ruth Tiernan Director of Education American Society of Pediatric Hematology/Oncology Glenview, IL Edwin Trevathan, MD, MPH Director National Center on Birth Defects and Developmental Disabilities Centers for Disease Control and Prevention Atlanta, GA Robert L. Wright, PhD Chairman and Chief Executive Officer Dimensions International, Inc. Alexandria, VA Barry S. Zuckerman, MD Chair, Department of Pediatrics Boston University School of Medicine Boston Medical Center Boston, MA Appendix B: Represented (*Sponsoring) Organizations Agency for Healthcare Quality and Research *American Society of Pediatric Hematology/Oncology *American Academy of Pediatrics American Association of Blood Banks American Red Cross Blood Services *American Society of Hematology *Centers for Disease Control and Prevention National Center on Birth Defects & Developmental Disabilities Division of Hereditary Blood Disorders Children’s Oncology Group Dimensions International, Inc. Food and Drug Administration Genetic Alliance *Health Resources and Services Administration Genetic Services Branch, Division of Service for Children with Special Health Needs Institute of Medicine *March of Dimes Medical-Legal Partnership for Children Minority Health Office, U.S. Department of Health and Human Services National Association of Children’s Hospitals and Related Institutions National Center on Minority Health and Health Disparities National Initiative for Children’s Healthcare Quality National Institutes of Health National Heart, Lung and Blood Institute National Institute of Child Health and Human Development *National Marrow Donor Program Polsinelli Shalton Flanigan Suelthans, PC Porter Novelli *Sickle Cell Adult Provider Network *Sickle Cell Disease Association of America, Inc. *United Health Foundation

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