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For Sickle Cell Disease Patients, Hope Has Arrived – but at What Cost?

By: Kelly E. Dempsey, Esq.

For generations of sickle cell disease (SCD) patients, the suffering has been unbearable – with no end in sight. SCD, an
inherited genetic red blood cell disorder that affects hemoglobin, the protein that carries oxygen throughout the body, torments nearly 100,000 Americans (20 million people worldwide), a disproportionate number of whom are African-American. Among other symptoms, SCD often triggers chronic bouts of excruciating pain that require regular hospitalization, organ failure, strokes, and shortened life expectancy. Meanwhile, the only known cure for the insidious disease has been a bone marrow transplant.

Until last month, that is. Developments in this area are exciting for patients, but may make some waves for employers and their self-funded health plans.

In the middle of the invariably hectic holiday season, some above-the-fold medical news dropped when the FDA announced approval of two gene-based treatments for SCD:
Casgevy, the first-ever therapy to incorporate the gene-editing technique CRISPR which enables scientists to make cuts in DNA, and Lyfgenia, grounded in an older gene therapy approach in which a virus is used to deliver a healthy copy of the gene that produces adult hemoglobin to compensate for the one producing the sickled form. Both Casgevy (produced by Vertex Pharmaceuticals and CRISPR Therapeutics) and Lyfgenia (generated by Bluebird Bio) have been cleared for patients 12 and older and are designed to serve as a one-time panacea.

Certainly, for those with sickle cell who have long felt they have been suffering in silence, the disease now being the focus of this dramatic new approach to therapy development is cause for celebration. In reality, the majority of sickle cell patients can’t find a matching bone marrow donor, so this scientific breakthrough could truly be life-changing for the masses dealing with the agonizing blood disorder. And the early signs are, in fact, promising: through rounds of clinical trials, Casgevy and Lyfgenia have proved to be so effective as one-time treatments that they have been widely considered to be “cures” by the scientific community.

But, like any cutting-edge therapy, these won’t be cheap. Consider that Casgevy will cost $2.2 million for the one-time treatment while Lyfgenia will cost $3.1 million. It also bears mentioning that these multimillion-dollar treatments will need to be administered in a monthslong process within large-scale medical systems that can perform stem cell transplants – systems that are inaccessible to millions of impoverished Americans living in states that have not broadened their Medicaid programs. The global picture is even bleaker. A recent New York Times story indicated that Vertex has been focusing on gaining approval in six wealthy nations – U.S., Italy, Britain, France, Germany, and Saudi Arabia – that represent merely 2 percent of the worldwide sickle cell population. Meanwhile, approximately 75 percent of the planet’s sickle cell patients reside in sub-Saharan Africa, where basic medical resources can often be scarce, to put it mildly. It should be noted that not all sickle cell patients have cases severe enough to warrant the new therapies; for reference, roughly only 20,000 in the US are believed to be sick enough to qualify for treatment.

But back to the daunting financial numbers. Why are the price tags so ridiculously expensive? Those pursuing Casgevy and Lyfgenia will need to have their cells harvested and transported to a lab for manufacturing; undergo multiple rounds of rigorous chemotherapy, which very well can pose its own risks; and be hospitalized for months on end. As Vertex’s chief scientific officer, Dr. David Altshuler, was quoted as saying in the previously mentioned New York Times article, “
A medicine that is so resource-intensive as this is may not be appropriate in many places where the amount of resources for health care is more limited.”

For employer-sponsored health plans, the multi-million-dollar question is whether they will have the resources to foot the bill. Will most plans simply be inclined to outright exclude this therapy? Will these new treatments be the most cost-effective method for providing services to those with sickle cell disease?  For those plans that do provide coverage, how will their respective stop-loss policies be impacted? What sort of coverage, if any, will be provided by Medicaid and/or Medicare? 

Gene therapy and cellular therapy have been hot topics in the last year, as well as the ever-increasing price tag on medical care and new treatments. It reminds me of the meme floating around social media about Kevin McCallister’s grocery list from Home Alone. In 1990 he purchased a half-gallon of milk, a half-gallon of orange juice, a TV dinner, bread, frozen mac and cheese, laundry detergent, cling wrap, toilet paper, a pack of army men, and dryer sheets all for $19.83. In 2022, that same grocery list cost $44.40 and in 2023 it’s now up to $72.28 … My intent was to make readers laugh, but if you need tissues, I understand as well (I’ll see if Matt has any Phia logo tissues on hand). 

Stay tuned.