As the lead doctor of a team researching the quality of pediatric health care across the country, Gary Freed was shocked when the group reached the topic of sickle cell disease.

Until developed by the team at the Susan B. Meister Child Health Evaluation and Research Center there were no validated quality measures to test the quality of care for children with the painful and often fatal inherited condition. And while some sickle cell centers followed the best standard of care, many children were not receiving critical services proven to make a difference.

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"We found that most children in the US with sickle cell disease were not receiving fundamental preventative care despite overwhelming evidence of its effectiveness in improving outcomes. That was a wake-up call," says Freed, M.D., M.P.H., a pediatrician and researcher at the University of Michigan C.S. Mott Children's Hospital.

"Once we realized the gravity of the situation, we knew we needed to try and do something more publicly to help make a difference in the lives of these children."

But Freed says the Centers for Medicare & Medicaid Services (CMS) missed a key opportunity to improve care for children when it ignored a strong recommendation by an expert committee to add sickle cell disease to the Medicaid core set of pediatric quality measures. Freed shares his perspective in JAMA Pediatrics.

"CMS's decision not to add recommended measures failed children with sickle cell disease. It sends the message that these children are not a priority in our health system," he says.

"The nation falls terribly short in providing preventive care for children with sickle cell disease – and now we have missed a historic opportunity to try to fix it."

Freed is the principal investigator of one of the Centers of Excellence in the US Pediatric Quality Measures Program. In the first phase of the program, these Centers were charged with developing and testing quality measures on topics assigned by CMS. Quality measures were evaluated based on evidence of importance, scientific soundness, usability and feasibility. 

In June 2018, a national committee formed by CMS to make recommendations for inclusion of new measures into the pediatric core set of quality measures recommended that CMS include two sickle cell disease quality measures. These measures were based on the research done by the team at the University of Michigan.

Approximately 1 in 365 black or African-American babies is born with sickle cell disease.

The quality measures recommended to CMS involved antibiotic prophylaxis treatment to prevent life- threatening infections and transcranial Doppler screening to help prevent strokes. These measure recommendations were made based on the low percentage of children receiving these necessary preventative services.

Despite overwhelming recommendations (19-1) by the expert panel to include the two sickle cell disease quality measures, CMS opted to not include either of them.

This is especially significant as 90% of children with sickle cell disease are enrolled in Medicaid.

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"Sickle cell disease is woefully underfunded and not prioritized in the medical profession. The disease is not a focus of state Medicaid programs and many sickle cell treatment centers are hurting, especially in terms of funding, resources and personnel," Freed says.

"We need to utilize quality measures to protect one of the most vulnerable groups in our society - children with sickle cell disease," he says. 

Freed explains more about sickle cell disease and topics highlighted in his opinion piece.

What is sickle cell disease?

Freed: The disease is a group of disorders that affects hemoglobin, which is the molecule in red blood cells that delivers oxygen to cells throughout the body. People with sickle cell disease have atypical hemoglobin molecules called hemoglobin S, which distorts red blood cells into a sickle shape.

What are signs and symptoms of the disease?

Freed: Sickle cell disease signs and symptoms typically begin in infancy. Symptoms may include anemia, infections and episodes of pain. Painful episodes can occur when sickled red blood cells get stuck in small red blood cells. This can lead to organ damage in the lungs, kidneys, spleen and brain.

The JAMA opinion piece is very direct at pointing out flaws in the system. What made you decide to speak up?

Freed: These are children that need to be prioritized and not marginalized as they have often been for decades. These are vulnerable kids and families, and they deserve our support, effort and concern on their behalf. If one of these children has a stroke, it can be devastating. If even one stroke can be prevented, to not try to help do so is a shame on our society.

How does the lack of proper care for children with sickle cell disease affect society today?

Freed: More than 100,000 people in the United States are diagnosed with sickle cell disease. One in 365 African American individuals are born with the disease Our data demonstrate these children are not provided the fundamental preventive care needed despite the evidence of its effectiveness. We as a society need to do more for these children and the quality of care they receive.

What does the situation with CMS not adding the sickle cell quality measures to the core set show you?

Freed: The decision to not add the recommended measures to aid critically ill children further shows that sickle cell disease is not a priority for the Medicaid program. We would not stand for this level of preventive care for any other life-threatening disease.

What is the goal of sharing your experience and opinions?

Freed: The public needs to be made aware of how the healthcare system is failing these children. I hope the public will question CMS and request answers as to why they did not add the sickle cell disease quality measures to their core set. CMS needs to be held accountable for the decisions they make that affect the care of these children.