Healthcare Disparities and Sickle Cell Disease
In the complicated U.S. healthcare system, navigating a serious illness such as cancer or heart disease can be remarkably challenging for patients and caregivers. When that illness is classified as a rare disease, those challenges can become even more acute. And when that rare disease occurs in a population that experiences health disparities, such as people with sickle cell disease (SCD) who are primarily Black and Latino, challenges can become almost insurmountable.
People with SCD often have less access to comprehensive care. A lack of treatment centers and specialists dedicated to the treatment of SCD is part of the problem, as is the fact that a majority of patients are insured through Medicaid, further limiting care options. In addition, SCD has not historically received the research funding and pharmaceutical investment directed to other orphan diseases. These disparities are thought to contribute to the difference in outcomes between people with SCD and people with other orphan diseases.
What weighs heavily on me is that we lack a standardized model for transitioning from pediatric to adult care in the U.S. There is a lack of information surrounding this transition, in part because children with SCD were unlikely to survive to adulthood before the introduction of the use of hydroxyurea for SCD treatment in 1984. However, existing data now show that the mortality rate for patients transitioning from pediatric to adult care more than doubles.i
There are far fewer comprehensive centers for adults with SCD than there are for children with the disease.ii These centers – which include hematologists, emergency room physicians, social workers, psychologists, education specialists and nutritionists, among others – have been available for children with SCD since the early 1970s. There are only 49 centers for adults with SCD in the U.S., which is less than half of what’s available to adults suffering from hemophilia or cystic fibrosis.ii
Periodic episodes of pain are a hallmark symptom of sickle cell disease, yet there are also issues surrounding how pain management is handled in children versus how it is handled in adults. In the adult population, there is a great deal of scrutiny and stigma associated with pain management; adults with the disease are often perceived as drug seeking. This is discouraging for patients, but it also impacts the care they receive. Once a patient is perceived as drug-seeking, they may not be seen for hours, often do not receive appropriate treatment and are subject to further complications as a result. One 17-year-old with SCD described her anxiety surrounding the transition to adult care being rooted in the experiences of her older sister who also has the illness:
“I’m honestly really, really nervous. I really hope I get a good doctor because my sister, who is always in and out of the hospital, told me that her doctors don’t believe a word that she says. There are these stereotypes of sickle cell patients as drug addicts… she’s never had an encounter with a good doctor as an adult.”
Despite these lamentable experiences, there is hope and enthusiasm surrounding new approaches to transitioning adults with SCD. The very same changes that resulted in improved care for children can also be enacted to benefit the adult population, including additional funding for comprehensive centers for adult care, as well as proactively combatting the influence of racism in our medical system. In fact, the state of California just recently secured $15 million from their legislature to fund sickle cell centers.
Fixing the transition issue in SCD requires the entire healthcare ecosystem – including those of us in biopharma – to innovate in our ways of working with the SCD community. Drawing on the ideas of sickle cell doctors, social workers, caregivers and patients, among others, calling for change, we need to create a new model for patient care, one that helps to overcome distrust and anxiety, focuses on engagement and outreach to the community and seeks to overcome the specific access, equity, healthcare literacy and quality of life issues this population faces.
The Science of Giving a Damn
At Forma Therapeutics, we are working with leaders in the SCD community to develop and share information on the challenges and opportunities to improve care for patients in transition. We’ve developed a patient outreach team that includes SCD advocates and experts, and we are dedicated to working alongside the communities we seek to serve. For us, keeping patients at the center of all that we do means approaching drug development differently, not just creating a drug that we hope will serve patients well but actually changing the care and treatment paradigm. We call this the science of giving a damn.