In the U.S., there are approximately 100,000 people living with sickle cell disease (SCD), and of those, the vast majority are from minority populations. An estimated 1 in 365 Black or African American newborns and 1 in 16,300 Hispanic American newborns will have SCD. For the more than 100 years since it was first characterized in medical literature, SCD has tragically illustrated the pervasive effects of inequity and barriers to care across the health care system — from a lack of access to comprehensive, evidence-based care, to a lack of treatment options, and systematic bias, stigma, discrimination, and a lack of understanding within the health care system.
There is a glaring need for improvement of the quality of care that people with SCD receive — including closing the gaps in research and increasing access to care.
In 2020, the National Academies of Science, Engineering and Medicine (NASEM) issued a blueprint for how the U.S. could work to do just that — improve care for people living with SCD. Two years later, there is still a lot of work to do. After months of work, the SCD Partnership publicly launched in June as a multi-stakeholder coalition to advance federal policy and the implementation of the NASEM strategy.
The vision of the SCD Partnership is "a day when every individual with Sickle Cell Disease in the United States lives to his or her fullest potential because the individual has timely, sustained access to high-quality, equitable, coordinated care and treatment.”
This strategy outlines a number of objectives to improve identified gaps in research, care and treatment programs for people living with SCD. Driven by the community and the need to put these strategic blueprints in motion, the SCD Partnership came together. We’re honored to be a part of the group, alongside the SCD community, physicians, professional societies, payers and manufacturers. Together, the coalition is bringing its voices and perspectives together to highlight the policy needs of the community.
The members of the SCD Partnership include: American Academy of Pediatrics, American Society of Hematology, AmeriHealth Caritas, Beam Therapeutics, Black Women’s Health Imperative, CSL Behring, Editas Medicine, Global Blood Therapeutics, Graphite Bio, National Academies of Medicine, Novartis, Sick Cells, Sickle Cell Community Consortium, Sickle Cell Disease Association of America and Vertex Pharmaceuticals Incorporated.
The coalition established three main parameters of focus for the SCD Partnership to tackle first in terms of developing policy recommendations and proposals:
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Care delivery and access: This includes everything from access to care in the emergency room to finding a treating hematologist.
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Data and evidence: Without data, it’s difficult to understand where sickle cell warriors are in their journey and what their baselines of care and outcomes are. This will help to develop evidence-based policies that can truly meet their needs.
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Access to new therapies: It wasn’t until 1998 that the first medicine was approved in the U.S. for the first treatment of SCD, despite its discovery in 1910. Since then, there have been three medicines between 2017 and 2019 to treat SCD, but there are numerous barriers across the country for individuals attempting to access these, and potential future, care options, including genetic therapies.
These efforts represent another essential way all members of the coalition are working alongside our sickle cell partners to help improve the lives of warriors. By focusing on the facets above, the SCD Partnership aims to ensure warriors, physicians and families can have the choices they deserve when therapies are available. More broadly than that, we hope this legislative action can have an impact on the health disparities and bias in today’s health care system, a driving factor of mistrust for those in the SCD community.
Vertex is proud to work with our coalition partners to raise awareness among federal policy makers, legislators and stakeholders across the system in an attempt to drive a unified call to action. And this hard work would not be possible without the cross-functional efforts of this group — the voices and perspectives of many that can help provide a well-rounded description of the SCD experience. The involvement of sickle cell warriors themselves is critical, and I might say, the most important voices in the room. All too often, they have been left out of key policy discussions. It is crucial that we support, empower, and work alongside the community in keeping with their mantra of “nothing for us, without us.”
I’m especially excited to share the work of the SCD Partnership and these steps forward in the quality of care for SCD as we join the community this month to recognize Sickle Cell Awareness Month. This Sickle Cell Awareness Month, we want to not only think about the past and present of the disease, but look toward the future as well, in hopes that this Partnership will be the shift needed to fully shine the light on the burden of disease, and then work to lessen it.