The Office of the Assistant Secretary for Health at the U.S. Department of Health and Human Services (HHS) should appoint a team of experts from across HHS agencies to advance the strategic plan.
|Timeframe for Action
|Strategy A: Establish a national system to collect and link data to characterize burden of disease, outcomes, and the needs of those with SCD across the life span.
|Develop state public health surveillance systems to support a national longitudinal registry of all persons with sickle cell disease (SCD).
|The Centers for Disease Control and Prevention (CDC) and state public health departments
|Develop a clinical data registry for SCD in order to identify best practices for care delivery and outcomes.
|HHS (specifically, the Health Resources and Services Administration [HRSA], National Institutes of Health [NIH], and Agency for Healthcare Research and Quality [AHRQ])
|Establish a working group to identify existing and disparate sources of data that can be immediately linked and mined.
|Office of the Assistant Secretary for Health (OASH)
|Strategy B: Establish organized systems of care that ensure both clinical and nonclinical supportive services to all persons living with SCD.
|Review disability insurance qualifications to ensure that the qualification criteria reflects the burden of the disease borne by SCD patients.
|Social Security Administration
|Expand and enhance vocational rehabilitation programs for individuals with SCD who need additional training in order to actively participate in the workforce.
|Convene a panel of relevant stakeholders to delineate the elements of a comprehensive system of SCD care, including community supports to improve health outcomes, quality of life, and health inequalities.
|HHS (Office of Minority Health and OASH), stakeholders such as National Minority Quality Forum, American Society of Pediatric Hematology/Oncology, American Academy of Pediatrics (AAP), American Society of Hematology (ASH), 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 parents and individuals living with SCD
|Develop and pilot reimbursement models for the delivery of coordinated SCD health care and support services.
|Centers for Medicare & Medicaid Services (CMS), State Medicaid programs, and private payers
|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 SCD.
|U.S. Department of Education, state departments of education, and local school boards
|Strategy C: Strengthen the evidence base for interventions and disease management and implement widespread efforts to monitor the quality of SCD care.
|Fund and conduct research to close the gaps in the existing evidence base for SCD care to inform the development of clinical practice guidelines and indicators of high-quality care.
|Private and public funders and health professional associations
|Foster the development of quality improvement collaboratives.
|National Heart, Lung, and Blood Institute (NHLBI); HRSA; CDC; U.S. Food and Drug Administration (FDA); ASH; Pediatric Emergency Care Applied Research Network; Patient-Centered Outcomes Research Institute
|Timeframe for Action
|Fund researchers and professional associations to develop and track a series of indicators to assess quality of SCD care including patient experience, prevention of disease complications, and health outcomes.
|AHRQ, NHLBI, HRSA, CDC, FDA
|Require the reporting of expert consensus-driven SCD quality measures and other metrics of high-quality health care for persons with SCD.
|CMS and private payers
|Fund efforts to identify and mitigate potentially modifiable disparities in mortality and health outcomes for vulnerable groups (e.g., young adults in transition, pregnant women, older adults).
|Strategy D: Increase the number of qualified health professionals providing SCD care.
|Disseminate information about NIH and HRSA loan repayment programs and designate SCD as a 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.
|Convene an Academy of SCD Medicine to support SCD providers through education, credentialing, networking, and advocacy.
|Health professional associations (ASH, American College of Obstetricians and Gynecologists, American College of Emergency Physicians, American Academy of Family Physicians, AAP, National Medical Association, American College of Physicians, and organizations for other relevant health professionals such as advanced practice providers and nurses)
|Develop early and effective mentoring programs to link early career health professionals with seasoned providers to generate interest in SCD care.
|Health professional associations, graduate and professional schools
|Strategy E: Improve SCD awareness and strengthen advocacy efforts.
|Improve awareness about the disease and address misconceptions about the disease and those impacted.
|HHS, media, health care providers, patient advocacy groups and community-based organizations (CBOs), health professional associations, and state health departments
|Translate and disseminate emerging clinical research information to people living with SCD and their families in order to improve health literacy and empower them to engage in the care and treatment decision-making process.
|HHS, health professional associations, health care providers, CBOs, and patient advocates
|Engage the 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.
|HHS, state health departments, health care providers, and CBOs
|Timeframe for Action
|Strategy F: Address barriers to accessing current and pipeline therapies for SCD.
|Identify approaches to financing the upfront costs of curative therapies.
|CMS and private payers
|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 SCD.
|NIH, FDA, pharmaceutical industry, and research community
|Establish an organized, systematic approach to encourage participation in clinical trials by including affected individuals in the design of trials, working with CBOs to disseminate information and recruit participants, and other targeted activities.
|NIH, FDA, pharmaceutical industry, research community, and CBOs
|Strategy G: Implement efforts to advance understanding of the full impact of SCT on individuals and society.
|Standardize the communication and use of newborn screening positive results in genetic counseling and create a mechanism for communicating this information across the life span.
|HRSA and states
|Fund research to elucidate the pathophysiology of sickle cell trait (SCT).
|Disseminate information to promote awareness and education about the potential risks associated with SCT.
|OASH, CBOs, media, and other relevant stakeholders
|Strategy H: Establish and fund a research agenda to inform effective programs and policies across the life span.
|The oversight body appointed by OASH should appoint a research task force to develop the research agenda and identify sources of funding.
|OASH, federal and private funders, and researchers
|1–2 years to develop research agenda
|3–5 years to disseminate funding opportunities for researchers
a Define (i.e., identify and develop) list of indicators.
b Implement monitoring program to track performance on those indicators.
c Disseminate information about existing loan repayment programs (e.g., for health disparities work).
d Develop criteria for loan repayment programs for health professionals working specifically with SCD populations.
e Identify and synthesize the criteria for the use of recently approved medications.
f Develop guidance for shared decision making and tools for implementation.
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