An ad hoc committee of the National Academies of Sciences, Engineering, and Medicine will develop a strategic plan and blueprint for addressing thalassemia in the United States. In conducting its work, the committee will examine:

1. The epidemiology (including changing epidemiology with aging of the thalassemia population), health outcomes (including complications from the disease, complications from treatment (e.g., transfusion-related complications), mental health, and quality of life issues), and societal factors associated with thalassemia;
2. Current guidelines and best practices for the care of patients with thalassemia;
3. To the extent possible, the economic burden associated with thalassemia; and
4. Current federal, state, and local programs related to thalassemia, including screening and diagnosis, monitoring and surveillance, treatment and care programs, research, and others.

The committee will provide guidance on priorities for programs, policies, and research, including ethical issues, and make recommendations as appropriate, regarding:

• Challenges, limitations, and opportunities for developing national thalassemia patient registries (e.g., including antibody registries) and/or surveillance systems;
• Barriers in the healthcare sector associated with thalassemia, including inequities in access to care (including language/communication/cultural barriers between the patient/caregiver and healthcare providers), workforce development (including physician/healthcare provider education/training to ensure an adequate national workforce of thalassemia care providers to reduce disparities in care), and a formal transition program as patients transition from pediatric to adult healthcare providers;
• Needed innovations in research, particularly for curative treatments such as gene replacement/gene editing and increasing awareness and enrollment of thalassemia patients in clinical trials; and
• Patient/caregiver/community-based educational programs for informed decision-making (including primary prevention) and the expanded and optimal role of patient advocacy and community engagement groups to increase awareness of the problem and opportunities for obtaining necessary genetic counseling, prenatal testing, and appropriate medical care.

Committee guidance will be formulated around strategic objectives (strategic plan) and action steps (blueprint).