Section 7 | National Academies for Sciences, Engineering, and Medicine (NASEM): A Blueprint for Action

Section 7: National Academies for Sciences, Engineering, and Medicine (NASEM): A Blueprint for Action

The Office of Minority Health at the Office of the Assistant Secretary for Health at the U.S. Department of Health and Human Services (HHS) requested that the National Academies for Sciences, Engineering, and Medicine (NASEM) convene a committee to develop a strategic plan and blueprint to address SCD in the United States. The Committee on Addressing Sickle Cell Disease: A Strategic Plan and Blueprint for Action was established in response to this request. Several members of the Program RCC teams and Oversight Steering Committee were involved with this project, able to provide both their clinical expertise and first-hand experience from the Program during the creation of the NASEM report. As part of the application process for the Program funding, each of the regions was charged with creating regional Action Plans that outlined current available resources and infrastructure. These action plans were important to supporting state SCD programming growth as well as instrumental resources in the creation of this blueprint. Although this Blueprint was not part of this project funding, the NCC strongly recommends reviewing the NASEM report for the research done in these areas and the important perspectives shared. Without

concerted attention and change, forward progress in the care of patients living with SCD will be inherently limited.

The NASEM ad hoc committee was charged to examine: • The epidemiology, health outcomes, genetic implications, and societal factors associated with SCD and sickle cell trait (SCT), including serious complications of SCD, such as stroke, kidney and heart problems, acute chest syndrome, and debilitating pain crises; • Current guidelines and best practices for the care of patients with SCD; • The economic burden associated with SCD; and • Current federal, state, and local programs related to SCD and SCT, including screening, monitoring and surveillance, treatment and care programs, research.

The committee offered deep insight into critical areas, including screening, registries, and surveillance topics, along with recommendations on delivering high-quality SCD care with a prepared workforce. In addition, the authors did an extensive look at societal and structural contributors to poor disease outcomes, including racism, implicit bias, and socio-economic barriers. These latter areas are significant in the context of SCD care and require great attention, which was outside of the scope of this program. The strategic plan consists of eight overarching strategies, or “pillars,” that support the vision, and seven foundational principles that undergird the strategic plan.

Strategic Vision: Long, healthy, productive lives for those living with sickle cell disease (SCD) and those with sickle cell trait (SCT).

• Establish a national system to collect and link data to characterize the burden of the disease, treatment outcomes, and the needs of those with

SCD across the life span • Establish organized systems of care that ensure both clinical and nonclinical supportive services to all persons living with SCD • Strengthen the evidence base for interventions and disease management and implement widespread efforts to monitor the quality of SCD care • Increase the number of qualified health professionals providing SCD care • Improve SCD awareness and strengthen advocacy efforts through targeted education and strategic partnerships among the U.S.

Department of Health and Human Services, health care providers, advocacy groups, community-based organizations, professional associations, and other key stakeholders (e.g., media and state health departments) • Address barriers to accessing current and pipeline therapies for SCD • Implement efforts to advance understanding of the full impact of SCT on individuals and society • Establish and fund a research agenda to inform effective programs and policies across the life span • Foundational Principles: Safe ∙ Effective ∙ Patient-centered ∙ Timely ∙

Efficient ∙ Equitable ∙ Ethical

The NCC highly recommends that Congress review the NASEM report and recommendations along with this report, as both provide valuable insight into the comprehensive nature of the work that is being conducted to improve care provided to people living with SCD and their families and outstanding work still to be done. Findings of the NASEM blueprint and this report often reflect and support each other, as the work done for this condition is carried out by a limited but intensely dedicated group of medical teams, researchers, and stakeholder advocates. The challenges and recommendations conveyed in the blueprint and this report are similar in addressing comprehensive, evidence-based care to help people living with SCD live their best lives.