Earlier this week, Tom Levenson linked to an excellent article by NPR on the promise of CRISPR technology for either long term symptom alleviation or cures for some people who have sickle cell anemia:
“I’m doing great,” Gray, now 36, said during a recent interview from her home in Forest, Miss. with NPR, which has had exclusive access to chronicle her experience for more than two years….doctors taking cells out of her bone marrow, and editing a gene in the cells in their lab, using the revolutionary gene-editing technique known as CRISPR….
The hope was the edited cells would produce a protein known as fetal hemoglobin, alleviating the symptoms of sickle cell…
And it appears to have worked, for Gray and other patients. Doctors have now treated at least 45 patients with sickle cell and a related condition known as beta thalassemia, and reported data indicating it’s working for at least 22 of them.
Sickle Cell Anemia is a genetic disease that is the result of two recessive genes expressing themselves so that red blood cells are bent instead of round. A single copy of the gene provides protection against malaria while both copies of the gene produce pain and lack of function.
Last week, the Duke Margolis Friday virtual lunch seminar had a speaker go over some of their new research on sickle cell. The TLDR from that talk is that sickle cell cures are a very nasty policy problem.
The first big thing is that the disease is very heavily racialized. African Americans are tremendously more likely to have the right combination of genes than people who identified as white.
Secondly, individuals with sickle cell are highly likely to be insured by Medicaid. Medicaid is a joint federal-state program. ACA Medicaid expansion has the federal government pay 90% of the costs and the states 10% while legacy Medicaid has the feds paying between 50% and 75% of the costs and states paying the remainder. The mostly former Confederacy states that have not (yet) expanded Medicaid under the ACA have a disproportionate share of individuals with sickle cell anemia.
Third CRISPR is expensive even in the context of US healthcare costs. A million dollar treatment cost is well within the realm of plausible and in some discussions that would be considered cheap. According to the NPR article, success is only happening in about half of the trial population. So there might be cases where a million dollars is spent for limited gains while other people can have massive improvements in quality and quantity of life for the same million dollars.
It is likely that a sickle cell cure will be cost effective under traditional health economics regimes. It is almost certain that a CRISPR based treatment will be a massive cash flow shock. Costs are incurred in the first period and the benefits are distributed to the individual over a twenty, thirty or forty year follow-on period. The Hep-C antivirals are a good analogy for a technological shock that is very cost-effective over a reasonable time horizon that is also a huge cost shock to the current payer period.
Several of my colleagues are thinking hard about how to structure contracts and payment arrangements that deal with massive expenditures with group level benefits but uncertainty and variance at the individual level. But no matter what, this is both very promising and one hell of a nasty policy problem.