Meeting 1: Addressing Sickle Cell Disease: A Strategic Plan and Blueprint for Action
Due to inclement weather in Washington, D.C. area, which complicated travel plans for several of meeting speakers and participants, we have changed the status of the meeting from in-person to a webcast.
The Department of Health and Human Services (HHS), Office of Minority Health requests that the National Academies of Sciences, Engineering, and Medicine convene an ad hoc committee to develop a strategic plan and blueprint for addressing sickle cell disease (SCD) in the United States. In conducting its work, the committee will examine:
the epidemiology, health outcomes, 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;
to the extent possible, 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, and others.
The committee will provide guidance on priorities for programs, policies, and research and make recommendations as appropriate, regarding:
limitations and opportunities for developing national SCD patient registries and/or surveillance systems;
barriers in the healthcare sector associated with SCD, including access to care, workforce development, pain management, and transitions from pediatric to adult care;
needed innovations in research, particularly for curative treatments such as gene replacement/gene editing and increasing awareness and enrollment of SCD patients in clinical trials; and the expanded and optimal role of patient advocacy and community engagement groups.
Committee guidance should be formulated around strategic objectives (strategic plan) and action steps (blueprint).
Throughout all the deliberations, the committee will give consideration to ethical issues related to SCD.