We need a roadmap, we need a system, we need something that is going to give us what many people already have, and what this country is going to need more of, as there are more people that are going to be living longer and living better with this new science.
—Adrienne S. (Open Session Panelist)
The strategic plan and blueprint for sickle cell disease (SCD) action identifies the strategic vision, strategies, and action steps for improving health care and health outcomes for individuals living with SCD. The fundamental vision of the framework is to advance and extend healthy, productive lives for individuals living with SCD and to advance understanding of sickle cell trait (SCT).
In developing the strategic plan, the committee found that most of the key messages from the Institute of Medicine (IOM) report Crossing the Quality Chasm: A New Health System for the 21st Century (IOM, 2001) still hold true today for the SCD population. Furthermore, SCD has not benefited in significant ways from medical advances compared with the general population or even other populations living with rare and heritable diseases, such as cystic fibrosis (CF) and hemophilia. Despite advances that have helped children living with SCD live longer, mortality and morbidity increase sharply in young adulthood (Treadwell et al., 2018) (see Chapter 1). Pregnant women with SCD are more likely to die or suffer adverse outcomes than the general population (ASH, 2015).
There is insufficient research and information about the needs of the SCD population, which makes it difficult to appropriately inform programming and policies to address their needs. However, there are evidence-based interventions (preventive, acute, and post-acute services) that every individual living with SCD should receive, although many do not. Clinical practice guidelines developed for SCD management are not applied consistently; even where there is strong evidence for certain services, not everyone with SCD receives them. People with SCD are dying at a much higher rate than the rest of the population, as discussed in Chapter 1 (IHME, 2020). One reason for these poor health outcomes is that the health care delivery system is not organized to address the health needs of individuals with SCD as they transition from pediatric to adult care. As the longevity of people living with SCD increases, so do the number and types of complications requiring comprehensive and coordinated care from a multidisciplinary team of experts and an array of health and non-health services. Only a limited number of health care professionals is willing and able to provide the necessary SCD care, and the health care delivery system is poorly organized to facilitate the needs of this population. As a result, people living with SCD often resort to episodic acute care to manage pain, the hallmark of the disease, as well as other acute complications. Lacking for this population is a system of care that can provide comprehensive coordinated care management aimed at preventing SCD complications and reducing the disease burden.
The sociopolitical and historical contexts underpinning the disease compound these problems. As discussed earlier, the roots of SCD in the United States can be traced back to the slave trade, where some African slaves transported involuntarily may have carried the gene. Because of persistent racism and discrimination, the disease was not addressed for years after it was discovered in the United States. This racism persists, and individuals with SCD and SCT have had to contend with discrimination in the health care system. Those living with SCD are also stigmatized in both health care and non–health care settings, in social circles, in places of employment, and schools. The far-reaching health and health-related social implications of SCD and SCT for those who live with these conditions and their families necessitate expansive action that takes into account the inherent complexities of the disease.
Taking these factors into consideration, the committee determined that, at a minimum, the strategic plan should ensure that the SCD population receives the same high-quality health care to which every American is entitled. The strategic plan (see Figure 9-1) is composed of a strategic vision, eight strategies in support of the vision, and foundational principles, which undergird the strategic plan.
The committee based the foundational principles on the six aims for the health care system identified in the IOM report Crossing the Quality
Chasm: safe, effective, patient-centered, timely, efficient, and equitable (IOM, 2001). According to the IOM report, “a health care system that achieves major gains in these six areas would be far better at meeting patient needs” (IOM, 2001, p. 6). The committee, however, believed that due to the history of marginalization and racism that has affected the majority of the population impacted by SCD, it was important to add a seventh principle: ethical. The seven foundational principles for action for SCD in the United States are described below.
- Safe: avoiding injuries to patients from the care that is intended to help them;
- Effective: providing services based on scientific knowledge to all who could benefit and refraining from providing services to those not likely to benefit;
- Patient-centered: providing care that is respectful of and responsive to individual patient preferences, needs, and values and ensuring that patient values guide all clinical decisions;
- Timely: reducing waits and sometimes harmful delays for both those who receive and those who give care;
- Efficient: avoiding waste, including waste of equipment, supplies, ideas, and energy;
- Equitable: providing care that does not vary in quality because of personal characteristics, such as gender, ethnicity, geographic location, and socioeconomic status; and
- Ethical: providing care that is free of provider prejudice or bias, avoiding unfair treatment because of SCD diagnosis and trait status, and addressing patient mistrust in the health care system.
The vision for the strategic plan is to assure “long, healthy, productive lives for those living with SCD and those with SCT.” The committee identified eight overarching strategies or “pillars” to support the attainment of this vision. These 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 all be approached with the same level of urgency.
The committee also proposes a blueprint for implementing the strategic plan. The blueprint offers action steps for each of the strategies in the strategic plan. The actions reflect the committee’s recommendations identified in the report’s preceding chapters after a thorough review of the available evidence. The action steps or recommendations are enumerated with the chapter that contains the supporting evidence. The committee recognizes that it will not be feasible to tackle all of the actions simultaneously and as such, 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 a vehicle for advancing action. Activities are also prioritized by actions that need to occur sequentially.
Each of the recommendations will require multistakeholder engagement, and the committee identifies some of the partners who need to be involved to achieve each action step. The committee, however, believes that in order to make meaningful and sustained progress on the strategic plan, it is critical that there be central oversight from the Office of the Assistant Secretary for Health (OASH) at the U.S. Department of Health and Human Services (HHS). OASH should appoint an oversight body with members from across HHS agencies to oversee the implementation of the strategic plan and action 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, is one option for such an interagency group (Azar and Giroir, 2019). The oversight group should be charged with advancing the strategic plan and blueprint by engaging and convening other stakeholders, developing interim milestones and monitoring progress, identifying funding sources, and overseeing other necessary activities.
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.
ASH (American Society of Hematology). 2015. Study pinpoints pregnancy complications in women with sickle cell disease. https://www.hematology.org/Newsroom/Press-Releases/2015/3852.aspx (accessed November 1, 2019).
Azar, A., and B. Giroir. 2019. Coming together to confront sickle cell disease. https://www.hhs.gov/blog/2019/06/19/coming-together-to-confront-sickle-cell-disease.html (accessed January 30, 2020).
IHME (Institute for Health Metrics and Evaluation). 2020. GBD results tool. http://ghdx.healthdata.org/gbd-results-tool (accessed April 21, 2020).
IOM (Institute of Medicine). 2001. Crossing the quality chasm: A new health system for the 21st century. Washington, DC: National Academy Press.
Treadwell, M., M. DeBaun, and J. Tirnauer. 2018. Transition from pediatric to adult care: Sickle cell disease. https://www.uptodate.com/contents/transition-from-pediatric-to-adult-care-sickle-cell-disease (accessed November 1, 2019).
1 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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