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National Academies of Sciences, Engineering, and Medicine; Health and Medicine Division; Board on Population Health and Public Health Practice; Committee on Addressing Sickle Cell Disease: A Strategic Plan and Blueprint for Action; Martinez RM, Osei-Anto HA, McCormick M, editors. Addressing Sickle Cell Disease: A Strategic Plan and Blueprint for Action. Washington (DC): National Academies Press (US); 2020 Sep 10.
Addressing Sickle Cell Disease: A Strategic Plan and Blueprint for Action.
Show detailsThere are approximately 100,000 people living with sickle cell disease (SCD) in the United States and millions more globally. Sickle cell trait (SCT) is even more prevalent and occurs in 1–3 million Americans and 8–10 percent of African Americans in the United States. Current estimates indicate that about 300,000 people are born with SCD each year worldwide and that more than 100 million people across the globe live with SCT. The sickle gene is found in every ethnic group, not just among those of African descent.
Since its discovery in 1910 by James Herrick, SCD has received relatively little attention and few resources from the scientific, clinical, and public health communities compared with other genetic disorders, such as cystic fibrosis (CF). Until December 2018 there was only one drug approved by the U.S. Food and Drug Administration (FDA) for the condition. A contributing factor to this lack of awareness and resources is that the affected population, which is primarily composed of racial and ethnic minorities, contends with persistent discrimination in the health care system and racism in society at large. As described by Keith Wailoo, a medical historian who has extensively studied the history of SCD, “Sickle cell disease is a microcosm of how issues of race, ethnicity, and identity come into conflict with issues of health care.” Thus, individuals with SCD have suffered from a lag in the development of treatments and cures as well as an often strained relationship with health care providers and limited resources for advocacy efforts.
To accelerate progress for those living with SCD, the Office of Minority Health at the Office of the Assistant Secretary for Health (OASH) at the U.S. Department of Health and Human Services (HHS) asked the Health and Medicine Division of the National Academies of Sciences, Engineering, and Medicine (the National Academies) to develop a strategic plan and blueprint to address SCD in the United States. (The full charge to the committee [Statement of Task] is provided in Box S-1.) This report is the answer to that request.
BOX S-1
Committee’s Statement of Task.
SCD and SCT status are currently identified at birth through universal newborn screening (NBS) in all 50 states, the District of Columbia, and U.S. territories. NBS has been highly successful at ensuring early access to much needed care, such as prophylactic penicillin for young children to avoid sepsis, which has saved countless children’s lives. Despite the effectiveness of NBS, there are wide variations in states’ short- and long-term follow-up practices regarding screening results (see Chapter 3). Most states also track individuals only if they remain within the same state, thus missing those who move out of the state. Additionally, while NBS identifies newborns who have SCT, there are currently no standardized practices for short- and long-term follow-up for carriers. This has important implications because of emerging evidence that SCT status might be a risk factor for certain clinical complications and because it is important for reproductive decision making. NBS also misses a large proportion of the SCD and SCT population who was born outside of the United States or before universal NBS was implemented in the country.
The genetic mutation responsible for SCD causes an individual’s red blood cells to distort into a C or sickle shape, reducing their ability to transport oxygen throughout the body. These sickled red blood cells break down rapidly, become very sticky, and develop a propensity to clump together, which causes them to become stuck and cause damage within blood vessels. The result is reduced blood flow to distal organs, which leads to physical symptoms of incapacitating pain, tissue and organ damage, and early death.
Pain is the hallmark of SCD, and individuals with SCD experience both acute and chronic pain. SCD pain is complex because it can be influenced by the disease pathophysiology as well as by psychological and social factors. The disease can also affect every organ in the body, as discussed in Chapter 4. While death rarely occurs among children with SCD in the United States, with 98 percent surviving to 18 years of age, SCD has a persistently high mortality rate in adults, and end-organ damage is the major driver of this mortality. Fatigue and emotional distress, such as anxiety and depression, become more prevalent with age and, along with chronic pain, pose a high burden and cause significant disability, which is under-recognized. In addition, childhood mortality for SCD remains very high in resource-poor countries.
Care delivery for SCD is inadequate. This stems in part from the fact that for most of the 20th century SCD was considered primarily a childhood disease because most affected individuals did not survive into adulthood. High childhood mortality rates for SCD led to an increased emphasis on improving the infrastructure for pediatric care in the United States and conducting research to prevent early death from infections.
The emphasis on early interventions for SCD led to seminal studies that provided the evidence base for clinical guidelines for the prevention and management of pediatric complications, such as the implementation of guidelines for prophylaxis against pneumococcal sepsis and stroke. Unfortunately, over the years there has not been a parallel development of guidelines and infrastructure for care delivery to adults living with SCD. Persistent gaps in the understanding of the natural history of SCD, predictors and biomarkers of morbidity and mortality, and the pattern of emergence of organ damage and other sources of disability persist, thus limiting optimal care delivery, particularly for adults (see Chapter 4).
Individuals who are transitioning from pediatric to adult SCD care are at a particularly high risk for morbidity and mortality because the robust care delivery systems available to pediatric patients are not replaced by matching or adequate resources for the adult patients (see Chapters 5 and 6). The lack of dedicated facilities and personnel caring for adult patients is compounded by the rising complexity of the disease, the emergence of comorbidities, and the vulnerability of individuals through the challenging period of adolescence and young adulthood. Young adults with SCD report being unprepared to engage optimally with and navigate the adult health care system independently after years of support in the pediatric care system. Areas of particular unmet need center on the domains of independence, self-care, vocation, and insurance coverage. When these factors are coupled with the aforementioned stigma, racism, and discrimination within the health care setting and society more broadly, individuals living with SCD often find themselves facing a solitary battle. This battle includes having to advocate for themselves in the health care and public sectors, at work, in schools, and sometimes even in their families.
Finally, even when interventions are well established, the delivery of appropriate and comprehensive care is uneven (see Chapter 6). Some of this may reflect payment mechanisms that do not support coordinated care. The majority of those with SCD are publicly insured, and covered services may vary across states. Furthermore, health care providers may not be well versed in accessing appropriate and available enabling services,2 or such enabling services may not exist. There is a lack of awareness among providers, especially those who do not regularly encounter patients with SCD, of the available clinical practice guidelines for evidence-based SCD care, which leads to inconsistent or substandard care. Members of a health care team may also be unaware of the implicit biases that influence their interactions with and medical decision making for those individuals living with SCD (see Chapters 2 and 6).
Improving the organization of care delivery can help ensure that individuals have access to appropriate services and can also enhance the quality of the care that is delivered, in turn improving the overall quality of life (QOL) for individuals living with SCD while increasing their access to new therapies. Two therapeutic products have been recently approved by FDA (voxelotor and crizanlizumab), and researchers are actively pursuing improved curative options by broadening access to stem cell transplant and developing gene therapy protocols (see Chapter 7). In addition to increasing access to new interventions, service delivery should be restructured by creating multidisciplinary care teams that can support delivery of whole-person care necessary to improve physical and social functioning and QOL for individuals with SCD.
The restructuring of care delivery would also provide a platform for patient advocacy groups and community-based organizations (CBOs) that have played an integral role in driving policy and much needed programming for the SCD population. These groups provide education about SCD and SCT, genetic support, psychosocial support, camps, care coordination, case referral, and transition assistance, among other services. However, it should be noted that patient advocacy groups and CBOs are under-resourced and are thereby limited in their ability to serve their constituents. Furthermore, there are no guidelines or unified infrastructure for the operation of these organizations and advocacy groups. Despite their key role for the SCD population, they also have traditionally not been recognized as part of the SCD care delivery system (see Chapter 8).
One challenge that has stifled progress in SCD is the lack of funding. To successfully garner attention and resources for those affected by SCD and SCT, there is a need for public education and awareness about the disease and the burden it places on individuals and the health care system. The contribution of racism, discrimination, mistrust of the health care system, socioeconomic disadvantage, and inadequate services across many sectors experienced by ethnic minorities in the United States cannot be overemphasized. However, the problems of access to high-quality health and social services are not exclusive to SCD, and the strategies developed to address them in other conditions can serve as a guide. CF and hemophilia, for instance, which are both rare and inheritable diseases, have well-organized and well-funded health care delivery systems despite there being smaller numbers of affected individuals (approximately 30,000 and 20,000, respectively, in the United States). Amyotrophic lateral sclerosis attracted widespread public attention and efforts fueled by social media, which resulted in dramatically increased funding. Other rare diseases have also benefited from an infusion of federal and private funding. These diseases set valuable precedents for addressing the needs of the SCD population.
SCOPE OF WORK
Charge to the Committee
The Office of Minority Health at OASH at HHS commissioned the National Academies’ Health and Medicine Division to develop a strategic plan and blueprint to address SCD in the United States. A committee was formed to direct the study titled Addressing Sickle Cell Disease: A Strategic Plan and Blueprint for Action. The charge to the committee (Statement of Task) is shown in Box S-1.
The Committee’s Approach
To accomplish its task, the committee focused the report to address the most salient issues surrounding SCD and SCT in the United States. Thus, most of the literature reviewed for the report originates from the United States. However, because the majority of the SCD population resides outside of the United States, seminal evidence and developments from other countries were included in the report where necessary. The bulk of the report focuses on the needs of the SCD population because this is the area of greatest need.
Conceptual Framework
Life-span approach In its assessment, the committee considered the needs and specific challenges at different stages in life. While most SCD complications are common to both children and adults, specific ones may be more prevalent at different ages. For example, dactylitis (painful swelling of the hands and feet), stroke, and enlarged spleen are all common in children, whereas retinopathy, pulmonary hypertension, heart failure, chronic leg ulcers, and cognitive burdens are more prevalent in the adult population.
The non-health-related needs of younger and older individuals with SCD may also vary. For instance, children who experience overt or silent strokes as a result of SCD develop cognitive impairment that may warrant additional educational support in order to increase the chances for academic success. With age, these cognitive challenges coupled with the additional burden of chronic organ damage may necessitate vocational rehabilitation support, specific workplace accommodations, or support to facilitate changes in vocation. Finally, the life-span approach is appropriate because existing resources for children and adults with SCD vary. High-quality care is currently better established for children, although there is room for improvement; quality indicators for adults with SCD are significantly underdeveloped and clinical guidelines for high-quality care lack a strong evidence base. As individuals with SCD survive into adulthood, information is needed about the appropriate service needs, and this need may require longitudinal data systems that will inform evolving care and service needs over time. The committee conceptualized the life-span approach in Figure S-1 in order to represent the need for targeted interventions at different life stages.
FIGURE S-1
A life-span approach to understanding and addressing the needs of the SCD population. NOTE: SCD = sickle cell disease.
It is also critical to include a focus on QOL factors and on understanding how QOL may change over a lifetime; this is a common approach in the study of other chronic conditions, such as cancer and cardiovascular diseases.
Person-centric SCD care SCD is a genetic lifelong condition; as the survival rate continues to improve for children, it must be managed as a chronic disease, which requires an ongoing person-centric, collaborative approach to care management. Building on previous National Academies work on epilepsy, another debilitating medical condition diagnosed in childhood, the SCD committee based its recommendations at least in part on the epilepsy model of care (see Figure S-2), which is built on Wagner’s Chronic Care Model. Wagner’s model emphasizes the foundational partnership between the care team and an activated and empowered patient as being crucial for care delivery. The model also underscores the importance of family members and community service providers in the care delivery system. This is especially relevant for the SCD population, which relies heavily on services provided by advocacy groups and CBOs.
FIGURE S-2
Model of person-centric care for SCD. NOTE: SCD = sickle cell disease. SOURCES: IOM, 2012; originally adapted from Wagner, 1998. Republished with permission of American College of Physicians - Journals from Chronic Disease Management: What Will It Take (more...)
The main focus for any model of care should be the individuals with SCD and their families, rather than the health care system. Bearing in mind the individual’s preferences, needs, and values, systemic efforts are required to facilitate access to comprehensive care and empower individuals to self-manage and remove barriers to treatment. Health care and social services are designed to benefit patients and improve their health outcomes, QOL, and ability to be productive. The engaged, supported, and empowered patients are then able to work collaboratively with the care delivery team and community resources to effectively manage their care.
There is also a need to establish acceptable minimum standards for the delivery of the complex care that individuals living with SCD require across the life span. Several centers of excellence in SCD have made efforts to establish learning collaboratives that define these care delivery standards, leveraging quality improvement measures to ensure that every patient has access to standardized care at all times, with ongoing data monitoring to track the processes’ effectiveness. For example, the SCD Emergency Department Learning Collaborative recently supported quality improvement interventions across three sites to improve time to first analgesia for acute SCD pain, and the Hemoglobinopathy Learning Collaborative has focused on strategies that result in more coordinated and appropriate care in order to achieve fewer complications, acute care visits, and hospitalizations; enhanced QOL; and more compassionate and respectful treatment from the health care system. Expanding the reach of such collaboratives will help spread nationally agreed-upon standards of care to other sites, with ongoing efforts to improve the consistency and ultimately the quality of care for individuals living with SCD. (See Chapter 6 for more information.)
RECOMMENDATIONS
Against the contextual backdrop described in the preceding sections, the committee developed a strategic plan and blueprint for SCD action and identified strategies and specific actions (or recommendations) for improving care and outcomes. The vision for the strategic plan is to ensure “long, healthy, productive lives for those living with SCD and those with SCT.” The committee found that the core message of the Institute of Medicine report Crossing the Quality Chasm: A New Health System for the 21st Century still holds true today for the SCD population, which has not benefited from medical science advances as much as the general population or even at the same rate as those living with other rare and heritable diseases, such as CF and hemophilia. There is insufficient up-to-date information about the SCD population to appropriately inform programming and policies that address the population’s specific needs. Finally, evidence-based interventions (preventative, acute, and post-acute services) that apply to the general population are not always available to individuals living with SCD.
The committee determined that, at minimum, the strategic plan and blueprint should ensure that the SCD population receives the same high-quality care that every American is entitled to. The committee based the foundational principles for action on the six aims for the health care system identified in the Crossing the Quality Chasm report, namely that health care be safe, effective, patient-centered, timely, efficient, and equitable. According to the authors of the report, “a health care system that achieves major gains in these six areas would be far better at meeting patient needs.” The committee felt that, due to the history of marginalization and racism experienced by the majority of the affected population, it was important to add a seventh principle: that health care be ethical.
The strategic plan (see Figure S-3) is made up of a strategic vision, eight overarching strategies or “pillars” that support the vision, and foundational principles, which undergird the strategic plan. The strategies take into account the multifaceted needs of the SCD and SCT population and the equally multidimensional interventions required to meet these needs. The strategies are equally important and need to be approached with the same amount of urgency.
FIGURE S-3
Strategic plan for improving SCD care and outcomes in the United States. NOTE: SCD = sickle cell disease; SCT = sickle cell trait.
The committee has also proposed a blueprint for implementing the strategic plan. The blueprint offers recommended actions for each of the strategies in the strategic plan. These action steps are the recommendations identified in the report’s various chapters after a thoughtful review of the available evidence. The action steps or recommendations are enumerated with the chapter that contains the supporting evidence and listed in order of implementation timeframe. The committee offers timeframes for accomplishing each of the recommendations. The timeframes take into account the complexity of the activity, the level of resources needed to accomplish the task, and the existence of current programs that can serve as vehicles for advancing action. Activities are also prioritized by actions that need to occur sequentially.
In order to make meaningful and sustained progress on the strategic plan, OASH at HHS should appoint an oversight body with members from across HHS agencies to oversee the roll-out of the strategic plan and blueprint. The appointment of the oversight body should be immediate, and the current HHS Sickle Cell Disease Workgroup, which has representation from 11 HHS agencies, would be one option for such an interagency group.
Finally, to ensure continued progress, the oversight body should conduct regular assessments of the implementation of the strategic plan, with the first evaluation occurring no more than 5 years after the release of this report.
Chapter 9 includes a complete description of the components of the strategic plan and blueprint.
Strategy A: Establish a national system to collect and link data to characterize the burden of disease, outcomes, and the needs of those with SCD across the life span. This can be accomplished by building on current and previous data collection efforts by the Centers for Disease Control and Prevention, developing a clinical registry for SCD, and linking existing datasets on the SCD population as described below.
Recommendation 3-1: The Centers for Disease Control and Prevention should work with all states to develop state public health surveillance systems to support a national longitudinal registry of all persons with sickle cell disease.
Timeframe: 1–2 years
Recommendation 3-2: The Health Resources and Services Administration, the National Institutes of Health, and the Agency for Healthcare Research and Quality should develop a clinical data registry for sickle cell disease. The registry would allow for identifying best practices for care delivery and outcomes.
Timeframe: 1–2 years
Recommendation 3-3: The Office of the Assistant Secretary for Health should establish a working group to identify existing and disparate sources of data that can be immediately linked and mined. These data can be used to provide needed information on sickle cell disease health care services usage and costs in the short term.
Timeframe: 1–2 years
Strategy B: Establish organized systems of care that ensure both clinical and nonclinical supportive services to all persons living with SCD. Such systems would ensure access to high-quality, evidence-based, comprehensive primary and specialty (acute and chronic) care delivered by a multidisciplinary team; supplemental enabling services; and behavioral health and social services. The following action steps are recommended to achieve this strategy.
Recommendation 2-1: The Social Security Administration should review disability insurance qualifications to ensure that the qualification criteria reflect the burden of the disease borne by individuals with sickle cell disease.
Timeframe: 1–2 years
Recommendation 2-2: States should expand and enhance vocational rehabilitation programs for individuals with sickle cell disease who need additional training in order to actively participate in the workforce.
Timeframe: 2–3 years
Recommendation 5-1: The Office of the Assistant Secretary for Health, through the Office of Minority Health, should convene a panel of relevant stakeholders to delineate the elements of a comprehensive system of sickle cell disease (SCD) care, including community supports to improve health outcomes, quality of life, and health inequalities. Relevant stakeholders may include the National Minority Quality Forum, National Medical Association, American Society of Pediatric Hematology/Oncology, American Academy of Pediatrics, American Board of Pediatrics, American College of Physicians, American Society of Hematology, Sickle Cell Disease Association of America Inc., Sickle Cell Adult Provider Network, and other key clinical disciplines and stakeholders engaged in SCD care; health systems; and individuals living with SCD and their families.
Timeframe: 2–3 years
Recommendation 5-2: The Centers for Medicare & Medicaid Services should work with state Medicaid programs to develop and pilot reimbursement models for the delivery of coordinated sickle cell disease health care and support services.
Timeframe: 3–4 years
Recommendation 5-3: The U.S. Department of Education should collaborate with state departments of health and education and local school boards to develop educational materials to provide guidance for teachers, school nurses, school administrators, and primary care providers to support the medical and academic needs of students with sickle cell disease.
Timeframe: 1–2 years
Strategy C: Strengthen the evidence base for interventions and disease management and implement widespread efforts to monitor the quality of SCD care. Existing evidence to support the care and management of SCD needs to be updated to reflect the current evolution of the SCD population, where individuals are living into adulthood and contending with complications that arise later in life. Excess mortality can be attributed to not receiving appropriate care. There also needs to be widespread efforts to track and improve the quality of care that accredited comprehensive SCD centers (as described in Strategy B) provide. This strategy can be accomplished through the following recommended action steps.
Recommendation 4-1: Private and public funders and health professional associations should fund and conduct research to close the gaps in the existing evidence base for sickle cell disease care to inform the development of clinical practice guidelines and indicators of high-quality care.
Timeframe: 3–5 years
Recommendation 5-4: The National Heart, Lung, and Blood Institute; Health Resources and Services Administration; Centers for Disease Control and Prevention; and U.S. Food and Drug Administration should collaborate with the American Society for Hematology, Pediatric Emergency Care Applied Research Network, Patient-Centered Outcomes Research Institute, and private funders of quality improvement initiatives to foster the development of quality improvement collaboratives.
Timeframe: 3–5 years
Recommendation 6-1: Federal agencies including the Agency for Healthcare Research and Quality; National Heart, Lung, and Blood Institute; Health Resources and Services Administration; Centers for Disease Control and Prevention; and U.S. Food and Drug Administration should work together with and fund researchers and professional associations to develop and track a series of indicators to assess the quality of sickle cell disease care including the patient experience, the prevention of disease complications, and health outcomes.
Timeframe: 1–2 years (to identify and develop list of quality indicators); 3–5 years (to implement monitoring program to track performance of those indicators)
Recommendation 6-2: The Centers for Medicare & Medicaid Services and private payers should require the reporting of expert consensus-driven sickle cell disease (SCD) quality measures and other metrics of high-quality health care for persons with SCD.
Timeframe: 3–5 years
Recommendation 6-3: The U.S. Department of Health and Human Services should fund efforts to identify and mitigate potentially modifiable disparities in mortality and health outcomes. Specific subgroups to consider include young adults in transition from pediatric to adult care, pregnant women, and older adults.
Timeframe: 1–2 years
Strategy D: Increase the number of qualified health professionals providing SCD care by enhancing existing health professional training and accreditation programs and incentivizing providers to provide compassionate and high-quality care. This strategy can be achieved through the following recommended action steps.
Recommendation 6-4: The National Institutes of Health should disseminate information on loan repayment opportunities to incentivize health care professionals interested in conducting research on sickle cell disease (SCD). The Health Resources and Services Administration should add populations with SCD as a designated population health professional shortage area under the National Health Service Corps program and create a loan repayment program for health care professionals working with SCD populations.
Timeframe: 1–2 years (disseminate information about existing programs); 3–5 years (develop criteria for loan repayment and similar programs for health professionals working specifically with the SCD population)
Recommendation 6-5: Health professional associations (American Society of Hematology, American College of Obstetricians and Gynecologists, American College of Emergency Physicians, American Academy of Family Physicians,3 American Academy of Pediatrics, National Medical Association, American College of Physicians) and organizations for other relevant health professionals such as advanced practice providers, nurses, and community health workers should convene an Academy of Sickle Cell Disease Medicine (SCD) to support SCD providers through education, credentialing, networking, and advocacy.
Timeframe: 2–3 years
Recommendation 6-6: Health professional associations and graduate and professional schools should develop early and effective mentoring programs to link early career health professionals with seasoned providers to generate interest in sickle cell disease care.
Timeframe: 3–5 years
Strategy E: Improve SCD awareness and strengthen advocacy efforts through targeted education and strategic partnerships among HHS, health care providers, advocacy groups and community-based organizations, professional associations, and other key stakeholders (e.g., media and state health departments). Strategic partnerships with advocacy groups and CBOs will enhance their capacity to provide supportive services and acknowledge their value as partners in promoting patient-centered policies and programs. The following recommended action steps will be necessary to achieve this strategy.
Recommendation 2-3: The U.S. Department of Health and Human Services should engage with media to improve awareness about the disease and address misconceptions about the disease and those affected.
Timeframe: 1–2 years
Recommendation 8-1: The U.S. Department of Health and Human Services, in collaboration with health professional associations, health care providers, and other key stakeholders, should partner with community-based organizations and patient advocates to translate and disseminate emerging clinical research information to people living with sickle cell disease and their families in order to improve health literacy and empower them to engage in the care and treatment decision-making process.
Timeframe: 2–3 years
Recommendation 8-2: The U.S. Department of Health and Human Services, in collaboration with state health departments and health care providers, should partner with community-based organizations and community health workers to engage the sickle cell disease (SCD) population in designing educational and advocacy programs and policies and in disseminating information on health and community services to individuals living with SCD and their caregivers.
Timeframe: 1–2 years
Strategy F: Address barriers to accessing current and pipeline therapies for SCD, with the goal of ensuring widespread patient access to beneficial therapies. The following recommended action steps will be necessary to achieve this strategy.
Recommendation 7-1: The Centers for Medicare & Medicaid Services in collaboration with private payers should identify approaches to financing the up-front costs of curative therapies.
Timeframe: 2–3 years
Recommendation 7-2: The U.S. Department of Health and Human Services should encourage and reimburse the practice of shared decision making and the development of decision aids for novel, high-risk, potentially highly effective therapies for individuals living with sickle cell disease.
Timeframe: 1–2 years (to identify and synthesize criteria for the use of new medications); 3–5 years (to develop guidance for shared decision making and tools for implementation)
Recommendation 7-3: The National Institutes of Health, U.S. Food and Drug Administration, pharmaceutical industry, and research community should establish an organized, systematic approach to encourage participation in clinical trials by including affected individuals in the design of trials, working with community-based organizations to disseminate information and recruit participants, and conducting other targeted activities.
The Patient-Centered Outcomes Research Institute, American Society of Hematology, FDA, and National Institutes of Health all have existing activities to foster patient-centric clinical trials design. Lessons and best practices from these disparate efforts need to be standardized, scaled, and adopted for inclusion in every clinical trial involving individuals living with SCD.
Timeframe: 2–3 years
Strategy G: Implement efforts to advance understanding of the full impact of SCT on individuals and society. The committee recommends the following action steps but cautions that all activities pertaining to collecting and using data to raise awareness and improve interventions should be performed so as not to stigmatize those living with SCT in any way.
Recommendation 3-4: The Health Resources and Services Administration should work with states to standardize the communication of and use of newborn screening positive results in genetic counseling and should create a mechanism for communicating this information across the life span and ensuring access to needed support and services.
Timeframe: 2–3 years
Recommendation 4-2: The National Institutes of Health should fund research to elucidate the pathophysiology of sickle cell trait.
Timeframe: 2–3 years
Recommendation 4-3: The Office of the Assistant Secretary for Health should partner with community-based organizations, the media, and other relevant stakeholders to disseminate information to promote awareness and education about the potential risks associated with sickle cell trait.
Timeframe: 1–2 years
Strategy H: Establish and fund a research agenda to inform effective programs and policies across the life span. Federal and private funders should collaborate to provide funding to clinician scientists and scholars with expertise in SCD, race, and stigma to advance research on pressing topics. The oversight body established by OASH at HHS should collaborate with health professional associations, researchers, individuals living with SCD, and funders to develop a robust research agenda with priority topics that need to be studied.
Timeframe: 1–2 years (to develop research agenda); 3–5 years (to disseminate funding opportunities for researchers)4
Footnotes
- 1
Citations and references for all facts and figures mentioned in the Summary are included in the subsequent chapters of this report.
- 2
Enabling services are defined as patient services that are intended to improve access to health care and create better health outcomes. Some examples include health education, case management, and transportation.
- 3
This text was revised since the prepublication release of this report to correct American Association of Family Practitioners to American Academy of Family Physicians.
- 4
This text was revised since the prepublication of the report to include the timeline for implementation of this strategy. The prepublication version of the report listed the timeline as “Ongoing.”
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