We don’t know whether rates have improved since then because there’s no national registry for sickle cell disease.
One way to increase these numbers would be to use quality measures. The Medicaid program has a set of 25 measurements they recommend to states to assess the health care provided to patients. Although reporting on the measures is currently voluntary, many states and organizations have adopted them.
Last year, the pediatric Measure Applications Partnership, an expert panel set up by the Centers for Medicare and Medicaid Services (C.M.S.), voted by 19-1 to add two sickle cell measures — one for preventive antibiotics and one for transcranial Doppler screening — to the core set of pediatric measures. These measures were also endorsed by the National Quality Forum, an independent group that assesses quality measures for reliability and validity.
Dr. Gary L. Freed, a professor of pediatrics and health policy at the University of Michigan, and a voting member of the partnership in 2018, said: “Right now, no one is held accountable for the current standard of care that is provided to children with sickle cell disease. C.M.S. had the opportunity to send a clear message to states, and, by extension, to Medicaid health plans and to providers, that the quality of care for these children matters.”
Last fall, though, the Centers for Medicare and Medicaid Services decided not to add these measures to the core set. The main argument for that position: Some states have a low incidence of sickle cell disease, most likely because they have fewer African-American residents.
But reporting by states on the measures in the core set is voluntary, and each state could determine for itself if the sickle cell disease measures were appropriate for its population.
Moreover, although sickle cell disease is rare, other rare diseases achieve much more attention.
Cystic fibrosis is another inherited disease that reduces life expectancy. In the United States, it’s about one-third as common as sickle cell disease. As with sickle cell disease, there are no definitive therapies, but a number of recommended actions and screenings can improve life immensely. In 2013, a study published in Blood compared spending on the two diseases by the National Institutes of Health and national foundations.