Sickle Cell Disease Access-to-Care Summit 2018 Electronic version available at GBT.com. Sponsored by Global Blood Therapeutics, Inc Sickle Cell Disease Access-to-Care Summit 2018 1 M-UNB-US-00050 V1.0 Table of Contents 2 Abbreviations 3 Introduction 6 Enhancing Care Within the Institution: Establishing a Sickle Cell Disease Clinic and/or Day Hospital for Comprehensive Care Management 11 Utilizing Existing Care Infrastructure: Leveraging Available Capacity by Partnering with Non-sickle cell disease Stakeholders 16 Transitioning Adolescent Care: Structuring Transition Between Pediatric and Adult Care Settings 21 Expanding Care Outside the Institution: Building a Community Outreach Model to Extend the Reach of Care 27 References Abbreviations CBO=community-based organization DH=day hospital ECHO=Extension for Community Healthcare Outcomes ED=emergency department HCPs=healthcare providers NBS=newborn screening PCP=primary care provider SMART=Specific, Measurable, Achievable, Relevant, and Time-bound US=United States 2 Sickle Cell Disease Access-to-Care Summit 2018 M-UNB-US-00050 V1.0 this context of institutional and systemic racism,1 “ Of all the forms of inequality, people living with the disease face a system of injustice in health care is the parallel, fragmented care2 that rests on an unwieldy most shocking and inhumane.” infrastructure and a healthcare environment riddled - Dr. Martin Luther King, Jr. with injustice. A Steering Committee of eight stakeholders from Introduction academia and business (Figure 1) formulated the The Sickle Cell Disease Access-to-Care Summit, program and set the objectives which were to: held on September 12, 2018 brought together Identify and discuss existing access-to-care committed stakeholders from across the United models used successfully to improve healthcare for States to explore ways of improving the lives of both adults and children with sickle cell disease people living with the disease who labor under a Create draft roadmaps of successful access-to- myriad of burdens, blockades and beliefs that have care models by involving stakeholders from across weighed upon African Americans for four centuries. the healthcare spectrum Moreover, as a chronic illness sickle cell disease Develop implementation roadmaps suitable for brings additional hardships and challenges that are dissemination through existing channels to local unbounded by race, religion or national origin. In and regional access-to-care initiatives Figure 1. Steering Committee Members Biree Andemariam, MD Payal Desai, MD Associate Professor, Department of Medicine Director, Sickle Cell Program Director, New England Sickle Cell Institute Director, Sickle Cell Research and Connecticut Bleeding Disorders Center Assistant Professor of Internal Medicine University of Connecticut Division of Hematology Farmington, CT The Ohio State University Columbus, OH Andrew Campbell, MD Director, Comprehensive Sickle Cell Program John J. Strouse, MD, PhD Children’s National Medical Center Director of the Adult Sickle Cell Program Associate Professor of Pediatrics Associate Professor of Medicine and Pediatrics George Washington University School of Duke University School of Medicine Medicine and Health Sciences Durham, NC Division of Hematology Washington, DC Julie Kanter, MD Kim Smith-Whitley, MD Director, Adult Sickle Cell Program Director, Comprehensive Sickle Cell Center at University of Alabama at Birmingham Children’s Hospital of Philadelphia Birmingham, Alabama Clinical Director, Division of Hematology Associate Professor of Pediatrics Perelman School of Medicine at the University of Pennsylvania James R. Eckman, MD Philadelphia, PA Professor Emeritus, Hematology & Oncology Emory University School of Medicine Atlanta, Georgia Kenneth Bridges, MD Vice President, Medical Affairs and Principle Medical Director Global Blood Therapeutics South San Francisco, CA Founder and Former Director, Joint Center for Sickle Cell and Thalassemic Disorders Brigham & Women’s and Massachusetts General Hospitals Boston, MA Sickle Cell Disease Access-to-Care Summit 2018 3 M-UNB-US-00050 V1.0 Access-to-Care challenges were addressed under programs and institutions built on a funding infra- four overarching headings: structure that includes private, local, state, and Expanding care within the institution: establishing federal sources. The focus of healthcare often is a sickle cell disease clinic and/or day hospital for short-term with problems addressed most often on comprehensive management a year-to-year basis, an approach best suited to Utilizing existing care infrastructure: leveraging acute issues of limited duration. Acquiring adequate available institutional capacity by partnering with healthcare can be challenging for people who are stakeholders outside the sickle cell disease arena deft within the system and well-resourced. For Transitioning adolescent care: structuring transition those who are poor and marginalized the problem is between pediatric and adult care facilities daunting. For poor, marginalized people with chronic Expanding care outside the institution: building illness the mountain is almost insurmountable. outreach models to extend the range of care into the community and beyond Sickle cell disease is a complex, chronic illness affecting approximately 100,000 Americans (Figure Participating stakeholders included patient advocates, 2). The multifaceted disorder evolves over decades health care professionals (HCPs), hospital admin- in character and manifestations. Except for the istrators, as well as representatives of professional limited option of bone marrow transplantation, no medical societies and government healthcare agen- cure exists. The problems are further exacerbated cies. The Steering Committee created draft roadmaps by the fact that most physicians are unfamiliar and/ to kindle group discussions, the output of which or uncomfortable with sickle cell disease manage- is distilled into the guidance material below. The ment.3,4 Episodes of acute vaso-occlusive pain resulting implementation roadmaps are freely avail- are the hallmark of sickle cell disease and over able to interested stakeholders for use and adaptation the course of a year these drive most patients into to individual circumstances. an emergency department (ED) on one or more The US healthcare system is a patchwork of occasions.5,6 Unfortunately, the ED is an option of Figure 2. Sickle Cell Disease in the US SCD in births: Number of individuals with SCD out of Black or African 15 every 36 American births ~100,000 AND in Hispanic every American 1 16,300 births Sickle cell trait Life expectancy for those with sickle cell disease is million reduced by 1 carry sickle cell trait in Black or African 1 13 American births ~30 years 4 Sickle Cell Disease Access-to-Care Summit 2018 M-UNB-US-00050 V1.0 last resort for many who struggle with sickle cell Importantly, reductions in hospitalizations and length disease7 due to that fact that many ED providers of stay by DHs can substantially reduce the cost of harbor negative attitudes about people with the care for patients with sickle cell disease.15 By chan- disease8 who consequently often experience inade- neling patients into an alternate management setting, quate disrespectful and even humiliating treatment. DHs potentially can free ED facilities and personnel The sad sequela for patients suffering with excruci- to focus on problems they handle best, such as ating pain commonly are longer waits than people major trauma or acute myocardial infarction.19 9 with other pain issues and frustrating delays in Drs. Andemariam and Eckman summarize the 10 analgesic administration. When treatment finally implementation roadmap for establishing DHs and/ arrives, relief often is minimal and fleeting due to or sickle cell disease treatment clinics. Starting 11 suboptimal dosing despite evidence that applying such programs often involves tapping into other guidelines for higher opioid dosing improves pain departments at an institution whose resources can 12 outcomes and decreases hospitalizations. be shared. Comprehensive Cancer Centers are an Adding to the misery, the whirlwind created by example where synergy possibly exists, particularly the US opioid crisis has pulled people with sickle in the hematologic malignancy sections where staff cell disease into its violent vortex.13 Policy makers are familiar with issues involving the blood-forming have responded to the debacle by implementing elements. Drs. Desai and Strouse address this guidelines for opioid analgesic administration that approach to expanding access-to-care for people are restrictive and sometimes draconian. Patients with sickle cell disease. suffering with sickle cell disease commonly require Sickle cell disease is a chronic disorder that results opioid analgesics to manage the severe pain from a single amino acid substitution in the hemo- resulting from compromised blood flow through the globin molecule. Clinical manifestations begin in microcirculation. Some need very high doses of infancy and extend into adulthood. The life-saving these medications. However, opioid use by patients intervention of childhood prophylactic penicillin with sickle cell disease often is more moderate was the catalyst for sickle cell disease newborn than daily opioid doses for patients with other pain screening programs which currently exist in each 14 syndromes. Nevertheless, people with sickle cell state. Efficient screening technology combined with disease frequently are stereotyped