SICKLE CELL DISEASE TREATMENT DEMONSTRATION REGIONAL COLLABORATIVES PROGRAM 2021 CONGRESSIONAL REPORT
Executive Summary
Ensuring that the approximately 100,000 children and adults living with sickle cell disease (SCD) and their families are offered and obtain current lifesaving treatments — and that they have equitable access to quality care and successful new treatments and cures for this condition — stands as a national priority.
Sickle Cell Disease Treatment Demonstration Regional Collaboratives Program In 2017, through a Health Resources and Services Administration (HRSA), Department of Health and Human Services Funding Opportunity Announcement, five Regional Coordinating Centers (RCCs) were chosen to establish a Sickle Cell Disease Treatment Demonstration Regional Collaboratives Program (SCDTDRCP). This program was originally authorized by the American Jobs Creation Act of 2004, Title VII, § 712(c), P.L. 108-357, Title VII, § 712(c)(42 U.S.C. 300b) (2004) and was reauthorized by the Sickle Cell Disease and Other Heritable Blood Disorders Research, Surveillance, Prevention, and Treatment Act of 2018, 42 U.S.C. § 300b-5 (2018). The Program was administered and funded by the HRSA Maternal and Child Health Bureau (MCHB), Division for Children with Special Health Care Needs. Built from the progress of the prior SCD programs reported to Congress in 2014 and 2017, the purpose of the funding was to support RCCs in establishing regional networks and enable them to provide leadership and support for regional and state activities that would develop and establish system-wide mechanisms to improve the prevention and treatment of SCD and collect select data in these three healthcare domains: 1. Increase the number of providers treating individuals with SCD using the National Heart, Lung and Blood Institute (NHLBI) Evidence-Based Management of Sickle Cell Disease Expert Panel Report (National Heart Lung and Blood Institute, 2014)
NOTE: Throughout this report, the Sickle Cell Disease Treatment Demonstration Regional Collaboratives Program is referred to as “the Program” or “the SCDTDRCP.” 2. Use telementoring, telemedicine, and other provider strategies to increase the number of providers administering evidence-based SCD care 3. Develop and implement strategies to improve access to quality care with emphasis on individual and family engagement/partnership, adolescent transitions to adult life, and care in a medical home The goals were to improve health outcomes in individuals with SCD, reduce morbidity and mortality caused by SCD, reduce the number of individuals with SCD receiving care only in emergency departments, and improve the quality of coordinated and comprehensive services to individuals with SCD and their families.
Overview of Sickle Cell Disease Approximately 100,000 Americans live with SCD, which refers to a group of inherited red blood cell disorders whereby red blood cells become hard and sticky, die early, and tend to impede blood flow leading to serious health problems. While this disease affects many races and ethnicities, in the U.S. it disproportionately affects Black, African-American, and Hispanic-American populations (Centers for Disease Control and Prevention, 2020). One of every 400-500 Black and about one out of every 16,300 Hispanic-American babies are born with SCD each year (Benson & Therrell Jr, 2010; Centers for Disease Control and Prevention, 2020; Feuchtbaum et al., 2012; Kato et al., 2018) making it one of the most common serious genetic disorders in the U.S. (Neumayr et al., 2019; Rees et al., 2010). Although historically SCD was considered a pediatric condition because children with SCD did not survive into adulthood, with medical advances and improvements in care most people living with SCD now survive into adulthood. In the mid-1970s, people with SCD lived to a median age of 14. By 2017, the median age had increased to 43 years. This represents a little more than half the national average lifespan of 81 years for women and 77 for men. Despite these gains, people living with SCD face a lifelong battle with pain, infection, and other chronic, serious health problems that can affect every organ in the body.
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Core Program Partners
Three Healthcare Domains
1. Five Regional Coordinating Centers (RCC) The RCCs established a regional infrastructure and network comprised of clinical locations and community-based organizations (CBOs) covering the U.S. states and territories. Each region had at least one designated regional lead. To improve the health of people living with SCD, this network conducted activities to increase access to and quality of care, used a shared measurement strategy, and submitted data to the National Coordinating Center (NCC).
The Report to Congress reflects work conducted to address three healthcare domains. RCCs collected data through an annual provider survey and quarterly medical record review of clinical quality improvement measures.
2. National Coordinating Center (NCC) In partnership with HRSA/MCHB, the NCC collaborated with the regional leads to finalize shared measures, align data collection activities, support communication, and receive data from the RCCs. From submitted data, the NCC created reports for HRSA/MCHB. The NCC also was responsible for writing the Report to Congress and a Model Protocol and for gathering resources for a Compendium of Tools and Resources. The NCC was the National Institute for Children’s Health Quality (NICHQ). 3. Oversight Steering Committee (OSC) The OSC was comprised of RCC leads and additional experts who brought specific knowledge, skills, and connections. For the duration of the Program, the OSC met twice a year to give input and assist in making recommendations.
Healthcare Domain 1: Increase Number of Providers Using NHLBI Guidelines Highlights and Select Data: • Showed robust frequency of hydroxyurea (HU) prescription •
Reflected an increased recognition and use of disease-modifying agents, in addition to HU
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Confirmed both the importance of immunizations and that the processes of immunization assessment and delivery must be improved
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Demonstrated higher rates of Transcranial Doppler (TCD) screening than some national findings but recognized that efforts to increase rates should continue
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Identified barriers to care, including systemic bias and racism, and planned activities to address them
Table 1. Areas of Data Measurement and Annual Percentage Over Project Period
Program Impact The Program has served well over 25,000 people with SCD – more than a quarter of the SCD population in the U.S. – through 51 clinical sites and 49 community-based organizations. The five RCCs identified over 1,200 Program SCD clinicians in their regions for whom Program funding could lead to improvements in care. These SCD clinicians were all able to prescribe important disease-modifying therapies to SCD patients. Through the Program, providers increased their knowledge to improve care of the SCD population: Over 3,700 attendees completed provider-to-provider telementoring, including specialized COVID-19 seminars.
DATA MEASUREMENT
AVERAGE PERCENTAGE OVER PROJECT PERIOD1
Pediatric Use of hydroxyurea
70.3%
Adult Use of hydroxyurea
57.7%
Pediatric Use of Other Disease-Modifying Therapies
14.1%
Adult Use of Other Disease-Modifying Therapies
24.6%
Pediatric Pneumococcal Immunization
82.0%
Adult Pneumococcal Immunization
63.5%
Pediatric Transcranial Doppler Screening
65.5%
Calculated as the average of each 6-month aggregate percentage from Quarter 1 2019 to Quarter 4 2020.
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Healthcare Domain 2: Use of Telementoring and Telemedicine Highlights and Select Data: • Demonstrated the ability to leverage the Program infrastructure to expand the knowledge base and comfort level of SCD providers across the U.S. •
Reflected an increase in engaging and training a broad range of providers, both primary care providers and hematology/oncology physicians, nurse practitioners, and physician assistants
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Showed the RCCs’ ability to use telementoring to quickly address information and provider education needs emerging from the COVID-19 pandemic
Table 2. Total Count of Provider Attendance in ECHO Sessions Over Project Period DATA MEASUREMENT
TOTAL OVER PROJECT PERIOD2
Total Count of Provider Attendance in ECHO1 Sessions
3,745
Extension for Community Healthcare Outcomes Calculated as the total of each 6-month aggregate count from Quarter 1 2019 to Quarter 4 2020. The number is a total count of attendance not the number of unique participants. 1.
2.
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Demonstrated improvements in developing and integrating innovative pediatric-to-adult care transition care programs to ensure seamless, comprehensive care at a time when patients are most vulnerable
Table 3. Transition Plan Average Percentage Over Project Period
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DATA MEASUREMENT
AVERAGE PERCENTAGE OVER PROJECT PERIOD1
Transition Plan for Pediatric-to-Adult Care
40.8%
Calculated as the total of each 6-month aggregate count from Quarter 1 2019 to Quarter 4 2020.
Impact of COVID-19 On January 27, 2020, the U.S. Department of Health and Human Services declared a public health emergency as a result of confirmed cases of 2019 Novel Coronavirus. COVID-19 caused extreme disruption to health care systems throughout the U.S., with many of the providers at local and regional levels in the Program engaged in either front line care or planning and developing procedures and processes to respond to evolving needs. To provide essential care for people living with SCD who were at high risk for complications related to COVID-19, RCCs quickly implemented new workflows and treatment approaches.
Healthcare Domain 3: Implement Strategies to Improve Care Highlights and Select Data: • Reflected increased strategies to better meet the needs of patients and families, including coordinating care visits, securing dedicated spaces, co-locating providers, and utilizing multi-disciplinary teams •
Reflected the intentional efforts of RCCs and their CBOs to partner in new and innovative ways to facilitate authentic engagement with patients, families, and caregivers to ensure that they receive comprehensive physical and psychosocial care
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Showed expanded telemedicine offerings which increased access to care for some patients, which were especially important during the COVID-19 pandemic
Sickle Cell Disease Treatment Demonstration Regional Collaboratives Program
In sum, the 2017-2021 Sickle Cell Disease Treatment Demonstration Regional Collaboratives Program addressed clinical and psychosocial needs to improve the health and quality of life of people with sickle cell disease. The Regional Coordinating Centers conducted numerous regional activities in Program priority areas. These activities along with the Program recommendations will inform and enhance future efforts to provide quality care for people living with this complex condition. Details of the Program, including additional data and RCC activities are found in the Report to Congress and its Appendix A, and Recommendations. Additionally, this Report includes a Model Protocol and Compendium of Tools and Resources consisting of resources to improve care of people living with sickle cell disease.
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References American Jobs Creation Act of 2004, Pub. L. No. 108-357, 42 § 300b (2004). https://www. congress.gov/108/plaws/publ357/PLAW108publ357.pdf
Rees, D. C., Williams, T. N., & Gladwin, M. T. (2010). Sickle-cell disease. Lancet, 376(9757), 2018-2031. https://doi.org/10.1016/S01406736(10)61029-X
Benson, J. M., & Therrell Jr, B. L. (2010). History and current status of newborn screening for hemoglobinopathies. Seminars in Perinatology, 34(2), 134-144.
Sickle Cell Disease and Other Heritable Blood Disorders Research, Surveillance, Prevention, and Treatment Act of 2018, Pub. L. No. 115-327, 42 § 300b-5 (2018). https://www.congress.gov/bill/115thcongress/senate-bill/2465
Centers for Disease Control and Prevention. (2020). Data & statistics on sickle cell disease. Retrieved April 23 from https://www.cdc. gov/ncbddd/sicklecell/data.html Feuchtbaum, L., Carter, J., Dowray, S., Currier, R. J., & Lorey, F. (2012). Birth prevalence of disorders detectable through newborn screening by race/ethnicity. Genetics in Medicine, 14(11), 937-945. Kato, G. J., Piel, F. B., Reid, C. D., Gaston, M. H., Ohene-Frempong, K., Krishnamurti, L., . . . Costa, F. F. (2018). Sickle cell disease. Nature Reviews Disease Primers, 4(1), 1-22. National Heart Lung and Blood Institute. (2014). Evidence-based management of sickle cell disease: Expert panel report, 2014. https:// www.nhlbi.nih.gov/sites/default/files/ media/docs/sickle-cell-disease-report%20 020816_0.pdf Neumayr, L. D., Hoppe, C. C., & Brown, C. (2019). Sickle cell disease: current treatment and emerging therapies. American Journal of Managed Care, 25(18 Suppl), S335-S343.
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