2020 Highlights the Disparities and Discrepancy of Sickle Cell Care

“Medicine is a mirror for the racial injustice in our society; it is a field riddled with racial disparities in everything from research funding to patient care to life expectancy.”

These words appear in an article published last month in The New Journal of Medicine. Co-authors Alexandra Power-Hays, MD, and Patrick McGann, MD, underscore this intrinsic—and perhaps under-appreciated—association between medicine and societal woes.

The authors argue, in far from oblique terms, that these medical-social relationships and issues of healthcare disparity—"from research funding to patient care to life expectancy”—are poignantly felt in the realm of sickle cell care.

It is perhaps safe to acknowledge that the events of this past year—whether that may be stringent lockdowns or the racial reckoning across the US—have augmented conversations and controversies surrounding healthcare access and related social inequalities.

Since sickle cell disease primarily affects people of color, most prominently African Americans, a group already at increased risk of socio-economic hardships, these conversations are especially relevant for this long-underserved patient community.

And yet, there is another layer to this issue of healthcare access—a problem that stems from new and innovative therapeutic options on the docket or horizon.

Medications like voxelotor (Oxbryta) and emerging technologies in gene therapy have no doubt offered hope in mitigating disease symptoms and ultimately curing it. But, with the promise of innovation comes the concern over access.

In an interview with HCPLive®, Admiral Brett Giroir, MD, United States Assistant Secretary for Health, expressed this unfortunate reality for much of the sickle cell community.

“All the science in the world is not going to help if you don’t have a care-delivery system that can meet the needs of patients,” Giroir said.

In order for these advancements to truly mean something, in order to ensure the sickle cell community at large can receive the most optimal outcomes, these underlying disparities in care-delivery, research funding, and resourcing cannot be overlooked.

The story of sickle cell disease, especially of this past year, dovetails with this increasing awakening to the gaps in healthcare access and the importance of equity.

As such, appreciating the therapeutic promises within this domain without acknowledging the inequities that plague sickle cell care would be a disingenuous reading of the narrative.

The Gap in Research and Healthcare

A cross-sectional study published this year by researchers at Stony Brook University Hospital and Johns Hopkins University School of Medicine noted that the US Food and Drug Administration (FDA) has approved only 4 medications for sickle cell disease—compared with 15 for cystic fibrosis, a condition that is likewise inherited, progressive, and life-threatening.

Even more, between 2008-2018, mean federal funding per person for sickle cell disease was $812 versus $2804 for cystic fibrosis.

The study concluded that government funding of sickle cell disease research roughly equaled research for cystic fibrosis, despite the former’s greater demand.

However, after the investigators factored in disease-specific private foundation funding, which significantly skewed towards cystic fibrosis, this disparity became even more pronounced.

When it comes to research, inadequate or disproportionate funding isn’t the only barrier that investigators face.

Similarly essential and worrisome are the challenges in patient recruitment. Certain barriers—including distrust of research, pain, difficult social situations, to name a few—may preclude individuals with sickle cell disease from participation.

Various formal analyses have sought to assess the effectiveness of diverse strategies—from recruiting through clinics and affiliated sites to participating in community events—in order to ensure study population size needs are met.

On the clinical side, healthcare providers have expressed dismay, recorded through investigator-led focus groups and questionnaires, over the lack of resources and support.

Many of these clinics, which are typically located in urban centers with diverse populations, may allocate funds in such a way that underserves the sickle cell population.

Further, issues stemming from lack of education on both the side of the patient and provider/staff as well as basic unmet patient needs (food, transportation, etc.) are underlying factors that can decrease the quality of care.

While challenges and disparities exist in various forms and degrees across the whole of sickle cell communities, issues with access to healthcare have tended to skew towards the adult population.

“I think there has been good access to care in pediatrics, and where there have been limitations has been on the adult side,” said Alan Anderson, MD, a pediatric hematologist-oncologist based in South Carolina and director of the Comprehensive Sickle Cell Disease Program at Prisma Health-Upstate.

“We have seen that these strides to improve access to care, number of patients on modifying therapies, using preventative health guidelines that are put out by National Heart, Lung, and Blood Institute (NHLBI), ASH, and others, have really been pioneered on the pediatric side,” he noted.

As for adult care, Anderson cited that many medical providers have historically received inadequate education or training in sickle cell disease.

Additionally, there are few dedicated medical homes for these patients that can best serve their specific needs. In fact, many adults with sickle cell disease end up in medical homes that are predominantly for cancer treatment.

Even when patients are seen for their disease, they can end up in a model of care where the primary focus of treatment tends to be on symptom and pain management, as opposed to education and disease modification.

“What we’ve seen on the adult is that many patients end up in the emergency department or hospitals to receive their care,” Anderson said.

An Exacerbation of the Issues

These disparities long existed before the pandemic, but, as with most other healthcare-related issues, the lockdowns and ensuing restrictions have only exacerbated what has long been broiling.

From the summertime protests after the killing of George Floyd to growing and recurring conversations around race, this concern over healthcare disparity in sickle cell care has further become the focal point for the specialty. It can no longer be ignored.

“I think if we look at sickle cell disease as really being a health diagnosis that is a microcosm of the race-relations issues and socioeconomic issues associated with race that we’ve seen in our country historically, then you would expect this disease process would be very hard hit by COVID-19,” Anderson said.

The pandemic has meant restricting resources in an already resource-limited space. The shift to telehealth has placed a greater burden on those with limited access to technologies. And the at-risk nature of the population has revealed that the only way to receive COVID-19 testing is through an emergency department setting.

This is the reality that many struggling patients within the sickle cell population face.

If anything—and as noted by Anderson—this year has only pushed and tested that which is already fragile for these patients and the healthcare systems they turn to.

Mending the Gap: An Admiral’s Charge

The “cure” for these social and systemic ailments must be a product of the synergy among individual, community, and top-down efforts.

When Admiral Giroir, MD, took office as Assistant US Secretary for Health in 2018, he made sickle cell disease a top priority.

Although a pediatrician by trade, Giroir made it his mission to ensure all sickle cell patients are able to receive the standard of care. By current estimates, they are received by only 10-15% of the population.

“Being the Assistant Secretary for our health, as the senior public health person in the government, I wanted to do this not only for sickle cell, for people living with that, but for all people in the country who have diseases that are below the radar,” he said.

Under his tenure, he oversaw programs to increase awareness for the disease, education, and understanding of care, which included first national report of sickle cell for all those covered by Medicaid and CHIP this year.

This year, the NIH has invested $120 million dollars into the Cure Sickle Cell Disease Initiative.

Furthermore, government health agencies have teamed up with the World Health Organization (WHO), World Bank, and other organizations like the American Society of Hematology (ASH), as well as pharmaceutical companies, to develop a program that promotes screening in newborns, an essential aspect of sickle cell care.

And finally, under Giroir, the federal health agencies sponsored and tasked the National Academies of Science Engineering and Medicine task with developing a sickle cell disease treatment roadmap, a document which was published this September, during Sickle Cell Disease Awareness Month.

The roadmap lays out a comprehensive plan for treating patients in this population—and other similar disease populations with few cases—especially those who are members of underserved communities subject to discrimination.

“I’m very proud of that, and we can’t implement that tomorrow, but we have a team working to implement that right now,” Giroir said.

While efforts at the top are moving forward to help mitigate social, systemic, and racial barriers to sickle cell care across the country, community-based programs and strategies are just as—if not more—essential to combating these multi-layered shortcomings.

With the support of local and federal governments, healthcare systems and communities have the potential to develop a comprehensive care network that can help mend the socioeconomic, psychosocial, and racial gaps—a sentiment shared by Giroir, Anderson, Power-Hays, and McGann.

If patients of underserved communities are empowered to overcome basic physical and emotional needs; if healthcare systems are provided the support to meet these needs and reduce systemic/racial barriers; and if all parties are appropriately educated on the novel therapeutic options and the disease itself, then the domain of sickle cell care for all ages would assuredly take a grand leap forward.

But, in light of the history of sickle cell care and the tragedies of this year, such a task seems almost insurmountable—and all the more crucial.