NHLBI Realigns Sickle Cell Disease Research Program

By Susan B. Shurin, MD

2008-07-01

Dr. Shurin is Deputy Director of the National Heart, Lung, and Blood Institute.

On May 16, 1972, Congress passed the National Sickle Cell Anemia Control Act (PL 92-294). This Act called for a national diagnosis, control, treatment, and research program to be implemented by the Department of Health, Education, and Welfare (now known as the Department of Health and Human Services [HHS]). Its implementation was assigned to the National Heart and Lung Institute, which established Centers to conduct research and provide education and services to people with sickle cell disease (SCD). At the time, many people born with SCD died in childhood and many more in young adulthood.

Biomedical research and health-care delivery has been transformed over the past 36 years. The costs of health-care delivery have increased exponentially. Health-care disparities remain, and in some areas have increased. Most people born with SCD now live well into middle age and older, with substantial burden of disease experienced by them and their families, who often have serious problems accessing quality health care. The findings of a recent NIH Consensus Development Conference on Hydroxyurea Treatment for Sickle Cell Disease 1 underscores the problems of ensuring that all who may benefit have access to research advances.

In this setting, NHLBI felt that it was appropriate to analyze its portfolio in SCD to assess the effectiveness of its investment in achieving its goals and identify areas in which adjustments should be made to funding mechanisms. We examined the history of investments and advances, solicited comments and suggestions from multiple constituencies, including ASH,2 and asked a subcommittee of our Advisory Council to provide an objective summary with recommendations. The report of the committee3 was adopted by the full Advisory Council at its February 13, 2008, meeting.4

In response to the findings and the wisdom of the community, we have implemented significant changes in our approach to SCD research.5 We have reconfigured the Centers to form a Basic and Translational Research Program in SCD, which will undergo further modifications as the full research agenda evolves. We expect to address an aggressive basic science agenda, designed to bring investigators in disciplines who have not previously been involved in SCD research into the field, to ensure that scientific approaches not yet brought to bear can enhance the research agenda, and to create the "Manhattan project" for SCD research recommended by the ASH workshop. We plan to create new mechanisms for translational clinical research, which will require that the investigator community rigorously establish priorities, and we will invite all capable investigators and patients who wish to participate in research to do so. We will enhance our support of training and career development, including broadening the specialties involved in such activities. We will collaborate with the Clinical and Translational Science Award (CTSA) Consortium to both conduct clinical research and support training of investigators. We will involve multiple constituencies in the development of practice guidelines and educational materials for professionals, advocacy groups, patients, and families.

We are now actively engaged in discussions across HHS to support the missions of our sister agencies, including surveillance and assessment of public health needs by Centers for Disease Control and Prevention and Health Resources and Services Administration's goal of ensuring access to care for the medically vulnerable and those affected by health disparities. The NIH missions are research and education. The Institute is not positioned to implement changes in medical practice, and is thus heavily dependent upon partnerships with payors and providers to address these issues. We will both repurpose existing funds and partner extensively to achieve our ultimate goals, which are to realize the promise of intervention in the prototypical molecular disease and to prolong and enhance the quality of life for patients with SCD. ASH members and leadership are an integral part of implementation; we all have a lot of work to do!

References

  1. National Institutes of Health. Panel Finds Hydroxyurea Treatment Is Underutilized for Sickle Cell Disease. Accessed May 21, 2008.
  2. American Society of Hematology. Toward a New Research Paradigm: Building A New Sickle Cell Disease Research Agenda. Accessed May 21, 2008.
  3. National Heart, Lung, and Blood Institute. Report of the National Heart, Lung, and Blood Advisory Council Subcommittee Review of the NHLBI Sickle Cell Disease Program.Accessed May 21, 2008.
  4. National Heart, Lung, and Blood Institute. Brief summary of the February 13, 2008, meeting of the National Heart, Lung, and Blood Advisory Council. Accessed May 21, 2008.
  5. National Heart, Lung, and Blood Institute. NHLBI Announcement: Institute to Realign its Sickle Cell Disease Research Program. Accessed May 21, 2008.

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