Sickle Cell Disease Association Holistic Patient Support and Education

Sickle Cell Disease Association of America, Inc.

We aim to provide holistic healing, support, and a cure for those with sickle cell disease (SCD) and improve overall health outcomes. We do this through education, research, advocacy, clinical trials, and mental health support for those with SCD and their caregivers. In the United States, it is estimated that over 100,000 people have sickle cell disease. About 2,000 babies are born with the disease each year in America. The blockage of blood flow caused by sickled cells leads to many complications, including chronic pain, infection, lung tissue damage, blindness, kidney disease, and stroke. While SCD can affect anyone, Blacks and Latinos are most commonly affected. Due to institutionalized racism, the lasting impact of the Tuskegee experiences, and the perception among many Emergency Department healthcare providers that this is a community of drug seekers, the sickle cell community is rightly suspicious of medical providers and researchers. With this as a backdrop, SCDAA has become the trusted source of SCD information due largely to the fact that our leadership and employees come from the communities that we serve, and many have lived experience with SCD or are SCD warriors themselves. 

SCDAA empowers sickle cell warriors with information: tools for self-care, tools for shared decision-making on current medications, tools for finding clinical trials, and tools for shared decision-making on gene therapy. Having a rare disease can be painful, confusing, isolating, expensive and exhausting. SCDAA works to optimize holistic care to address all of these challenges for people with rare diseases and their caretakers—including physical, mental, social, and other types of support.

Our solution is a patient-centered, holistic approach to care that focuses on education, data collection and analysis, and access to clinical trial and gene therapy information. We  SCD warriors, their caregivers, doctors, and the community to ensure the highest rate of patient health and success. We collect data to fill in missing information about those we seek to serve and close gaps in access to quality care. We use innovative practices to connect our SCD warriors with clinical trials and the latest gene therapy to ensure patients are well-informed and have access to the best care possible. 

With SCD specialists in short supply, a legacy of medical mistrust, and a history of poor access to care, it’s no wonder that approximately two-thirds of adults do not visit an SCD center for comprehensive care. African Americans and Latinos are disproportionately affected by SCD. Eight percent of African Americans are carriers, many of whom do not know they have it. Latinos have the second most common incidence in the United States; however, SCD affects all ethnicities. Because many of these warriors are isolated in rural locations and challenged by immigrant subcultures, physical disabilities, poverty, mental health issues, and reliable transportation, they have to overcome many barriers to care.

Our solution will address their needs by (1) supporting public awareness among public health professionals, (2) developing survey tools to fill data gaps, and (3) developing educational activities–all of which will help patients with this rare disease receive better care (4) increasing holistic care and support (5) improving access to treatment, including the new curative treatments. We carry out these activities at the national level, as well as through our 56 local SCDAA member organizations located in 30 states across the country, which collectively serve 500,000 individuals living with or affected by SCD.  The regulatory response to the opioid abuse epidemic stigmatizes people with legitimate needs for prescription opioids for the intense vaso-occlusive SCD pain. 

SCDAA has been the national voice for SCD for over 50 years and serves over 500,000 adults, children, and caregivers annually. We are far from being a pilot program. We connect patients to clinical trials, educate patients and doctors, and advise and advocate for those living with SCD and their caregivers. We are in the scaling phase of our organization–we aim to serve more individuals each year with SCD and expand access to treatment, including the new, potentially curative gene therapy treatments.  As the SCD landscape continues to change, we must remain timely and nimble in order to remain the trusted voice in a community that has often been rightly wary of medical providers and clinical research.

Other accomplishments include:

• Community health worker training: Our training program covers the core competencies of community health workers and includes an additional concentration on sickle cell disease. Virtual lectures, online in-class assignments, and homework provide a wide array of learning opportunities. This structured course is offered throughout the year.
• Leadership training: We train sickle cell organizational leaders on topics such as (1) Member Capacity Building, (2) Nonprofit Governance, (3)Advocacy, (4) Funding Sustainability, and (4) Leadership Development
• In partnership with Forma Therapeutics, we host a comprehensive clinical trial finder. We host annual SCD advocacy days in Washington, D.C. 

Our solution is innovative because we connect our SCD warriors with clinical trials through our clinical trial finder and our mobile clinical trial app. We provide timely, accurate information and data on gene therapy treatments to ensure patients are informed and can increase their access to the best care possible. We focus on holistic care–treating mind, body, and spirit. SCDAA continues to innovate with our SCD Masterclass series, Mindful Mondays series, mental health campaign, clinical trial information, and journey map to help patients with this rare and debilitating disease.  We offer disease and treatment information, access to clinical trial information, the most up-to-date research, and holistic support for SCD patients and their caregivers. We are educated in the latest treatment options–including gene therapy–to educate SCD warriors about their treatment options.

Our innovation in patient communication is successful because we have worked extremely hard over the last 50 years to gain the trust of those we serve. Unlike some other rare diseases, our community has a deep-seated mistrust of the medical community. Because of our care and our consistency, we have earned their trust, and that is not something we take lightly. This means that the delivery of the information is just as important as the information itself.

How we work with our warriors and their caregivers can be a model for other organizations, especially those trying to reach a hesitant population. As we have seen with COVID-19, trust in the medical community is still low. Our concentration on holistic support can be a model for other organizations, and this approach can change the landscape of care by increasing trust in the system. Increasing trust in treatment will improve overall health outcomes, particularly for marginalized communities. 

The Amgen Prize can help us overcome financial barriers to our success. With our current budget of about $2 million, our footprint covers 30 states. With additional funding, we could enhance the capacity of existing community-based member organizations, assist emerging CBOs in additional states, increase access to clinical trials, and offer additional programming.

We are the primary source of information and education on sickle cell disease and sickle cell warriors and their families throughout the  United States. With the advent of gene therapy as a potentially curative treatment,  our need to communicate and educate has grown faster than ever. SCD is a complex disease, and families often require a huge amount of emotional, social, and family support.

Primary care doctors and other health care providers look to SCDAA for accurate, timely information on the range of disease-modifying and curative treatments available. The public is aware of how costly gene therapy is and how difficult it is to find comprehensive sickle cell centers in their geographic areas. Not all living with sickle cell will be eligible or will choose, to undergo gene therapy treatment. It is important to ensure that access to disease-modifying treatments existing and in the pipeline, as well as new forms of gene therapy, remain accessible to all who seek it.  We must continue to provide holistic support to SCD warriors and their families. 

The SCDAA leadership team comprises individuals who reflect the community they serve. Most are of color, many have been connected to or impacted by sickle cell disease in one way or another, some are SCD Warriors themselves, others have cared for those who lost their lives to SCD complications, and all are deeply committed to improving the lives of those living with the SCD.  They have experienced and/or witnessed how race can determine the quality of healthcare a person receives. They have experienced and/or observed how social determinants play a critical role in, for example, how hospitals–large and small–are ill-equipped to treat SCD or are located in urban cities or rural areas inaccessible to the average person with SCD.

SCDAA Medical and Research Advisory Committee (MARAC) is comprised of doctors, nurses, and researchers who are world-renowned experts in SCD and have years of experience treating adult and pediatric patients or conducting translation research, all in the search for a universal cure.  They, too, are the trusted advisors the community looks to for guidance. This background is critical to allowing us to connect with those we serve. SCDAA can better advocate and educate on the community’s behalf because we hear and understand their concerns. For 52 years, SCDAA has built enduring and trusted relationships, collaborations, partnerships, and trust, which makes us the national voice for SCD

Impact goals for the next year:

• We will provide our solution to over 500,000 sickle cell warriors and their families as measured by social media and web metrics, as well as member reporting
• We will raise $25,000 in additional program funds
• We will host a national convention with SDC patients, caregivers, and the medical community, with an attendance of at least 500
• Create and distribute new informational materials, as COVID-19 has changed some of our vaccine recommendations.

Over the next five years, we want to increase our impact by: 

• Increasing our reach by adding 5% more member organizations
• Increase our reach to serve at least 5% more sickle cell warriors and their families as measured by membership surveys
• Increase the number of sickle cell warriors who engage in clinical trials by 5%, as measured by our clinical trial finder. 

Our theory of change is based on some basic assumptions:

• Living with a rare disease is difficult, isolating, expensive, and painful
• Caring for a loved one with a chronic illness brings its own set of challenges
• Holistic approaches to health have the greatest impact on outcomes
• There is understandable mistrust of medical professionals in the African American and Latino communities
• Education is critical to the management of rare diseases
• Social determinants of health greatly affect African American and Latino populations, and those are the populations we serve

Theory of change:

If SCDDA provides holistic, person-centered care that addresses mental, physical and emotional health

Then, SCD patients and their caregivers will experience a higher quality of life

Which will lead to overall improved health outcomes and rare disease management

If SCDDA provides data-driven research and treatment information to medical professionals, patients, caregivers, and the public

Then, SCD patients will have better access to care and resources

Which will improve early diagnosis, increase access to treatment options, reduce stigma, and improve patients' overall quality of life.

If SCDAA continues to provide timely, accurate information delivered by professionals with lived experience with SCDAA

SCDAA will continue to grow as a trusted voice in the African American and Latino communities.

  This will lead to more SDC patients seeking clinical trials, trying new treatments, getting vaccines, and improving overall health outcomes. 

Full-time staff 7
Part-time staff 0
Contractors 4

52 years

Diversity, equity, and inclusion are at the heart of our work. We ensure that our staff reflects the diversity of the population that we serve. The majority of our staff and leadership team are Black. That includes the national and local levels of our work. Our community-based organizations throughout the U.S. are made up of diverse individuals working in the communities that they serve–rural and urban.

It is critical to SCDAA that our staff, research team, and doctors reflect the background of the minority communities we serve. Historically, and for good reason, there is distrust of medical professionals among the African American community because of the documented abuses the community has endured, from the U. S. Public Health  Service Study of Untreated Syphilis in African American Males (Tuskegee) experiments, Johns Hopkins Hospital’s use of Henrietta Lacks’ cancer cells without her consent, and up to the present day, minority communities have a well-founded distrust of the medical and research professions. 

SCDAA is a nonprofit organization, and we rely on grants and donations for support.  We also have member organizations in 30 states, and they are also nonprofits. We provide our services–education, programming, holistic support, and accurate information–to any sickle cell warrior and their caregivers free of charge. We do not provide direct medical services. We are able to do this because of outside funding. Sickle cell warriors and their families need our trustworthy voice in a world that is overcrowded with information and much misinformation about sickle cell and treatments. Having a chronic disease can be terribly isolating, and we provide a voice of hope

SCDAA has sustained its work for over 50 years through grants, donations, sponsorships, and corporate partners. We charge small fees to attend the annual conference and host fundraising events. SCDAA provides all of its services for free to patients and caregivers. We pursue state, federal, local, and foundation grants and partner with other organizations on their applications.

We receive funding from BlueBird Bio ($90,000), Agios Pharmaceuticals ($62,000), Genentech ($35,000), Kimberly Clark, Novartis, Pzifer, and more.

With the advent of gene therapy as a potentially curative treatment for SCD and the increased media attention that has been provided, we expect (and have already experienced) increased interest and awareness in our rare disease work. We will use this increased exposure and awareness to seek ways to improve access to the cure. As a trusted voice in the community, our mission is to advocate for people affected by sickle cell conditions and empower community-based organizations to maximize the quality of life and raise public consciousness, advancing the search for a universal cure.

Over the last 50 years, we have served as the national voice for those living with sickle cell disease and play a vital role in research, patient care, and education. Our dedication to increasing awareness and improving the quality of SCD life has helped us build trust in the community, and it is their support that will sustain us.