Disease burden heavy for SCD patients in US, despite promise of gene therapy
New report using real-world data cites care hurdles, need for multiple meds
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In the U.S., having sickle cell disease (SCD) continues to place a heavy clinical burden on patients, who often require multiple medications, frequent blood transfusions, and the use of opioids to control pain. Most individuals with SCD report high barriers to healthcare, with poor access to treatments and little effective pain management as major issues.
Meanwhile, though emerging treatments such as gene therapy are generating interest among doctors, these options are not yet widely used by patients, primarily due to barriers that include insurance denials.
These are among the findings of a new report, called the 2026 “Patient Chart Dynamix: Sickle Cell Disease (US),” from Spherix Global Insights, which reviewed 133 real-world patient charts collected from 86 doctors in the U.S. who specialize in blood diseases and cancer.
“As the therapeutic landscape evolves, the 2026 findings point to a pivotal transition period in sickle cell disease care — marked by rapid scientific progress, expanding therapeutic innovation, and rising physician optimism, yet constrained by operational and access barriers that must be resolved to translate breakthrough therapies into routine clinical practice,” Spherix stated in a company press release.
The company concluded that sickle cell care in the U.S. has reached what it called “an inflection point.”
SCD occurs due to genetic mutations that cause the body to produce a faulty version of hemoglobin, the protein in red blood cells that carries oxygen. This faulty protein makes red blood cells take on a sickle shape, making them fragile and prone to form clumps. Those clumps, or aggregates, block small blood vessels, limiting oxygen delivery to tissues.
For many patients, the condition is marked by sudden episodes of severe pain called vaso-occlusive crises (VOCs).
Managing SCD remains complicated, with patients on 5 or more drugs
The latest Spherix report highlights that managing SCD remains very complicated. Many U.S. patients require several different medications at the same time, known as polypharmacy. On average, each patient analyzed in the report was taking nearly six medications, which may reflect a moderate to severe disease.
Also, opioids continue to be the mainstay treatment to control both acute episodes of severe pain, such as VOCs, and chronic pain, data showed. While these medications can be effective, their long-term use typically triggers side effects that can be severe.
More than half of the evaluated patients received regular blood transfusions to help increase their numbers of healthy red blood cells and reduce complications caused by sickle cells. Even with medications and transfusions, however, many individuals continued to experience ongoing pain and vaso-occlusive crises, the report noted..
Many patients also experience complications caused by SCD-related damage to organs such as the kidneys, the heart, or the lungs, as well as other health problems, including anxiety and depression.
“Hospitalizations and transfusion requirements continue to affect a meaningful subset of patients, reinforcing the need for more durable and accessible treatment strategies,” Spherix stated in the release.
Surveyed doctors reported that, among their patients, SCD is now only moderately controlled. Many are interested in alternatives to opioids, which can lead to addiction among their other serious side effects when used for long periods of time.
As new therapeutic options emerge, doctors see those specifically designed for SCD as key to improving long-term care.
Few US patients using gene/gene-editing therapies, per report
Gene and gene-editing therapies, which can deliver genes to cells or modify genes within cells, are among the most promising options because they target the cause of the disease.
Two such therapies are approved in the U.S. for adults and children, ages 12 and older, with SCD and a history of VOCs. One is the gene-editing therapy Casgevy (exagamglogene autotemcel); the other is the gene therapy Lyfgenia (lovotibeglogene autotemcel). Both are given as a one-time infusion into the bloodstream.
Most surveyed doctors said they are familiar with gene/gene-editing therapy and believe many of their patients could qualify for it. However, only a small number of patients have started the process of receiving such therapies.
“Uncertainty around treatment center availability, payer navigation, referral logistics, and long-term efficacy data continues to slow real-world adoption,” the release stated.
The promise of non-opioid therapies to better manage pain — and potentially transformative gene therapies — gives patients and providers real hope. … [Clinicians in the U.S.] need stronger referral pathways and clearer infrastructure so that innovation translates into real-world impact.
Overall, the report suggests that care for sickle cell disease is entering a period of major change. While doctors are increasingly hopeful about future therapeutic options, healthcare systems still need to address practical challenges to make them widely available to patients, according to the report.
“We are at an inflection point in sickle cell care. The promise of non-opioid therapies to better manage pain — and potentially transformative gene therapies — gives patients and providers real hope,” said Nina Anderson, the director and founder of TOVA Community Health in Wilmington, Delaware, and a strong advocate for gene therapy.
Anderson, a specialty primary care provider, is “eager to refer appropriate patients for gene therapy, but even with major hospital systems in the area, it is not always clear where to send them or how to navigate access.”
According to Anderson, clinicians in the U.S. “need stronger referral pathways and clearer infrastructure so that innovation translates into real-world impact.”
