Unravelling the CAR T-cell therapy reimbursement riddle

Cost, accessibility, and real-world side effects

The key concerns about the therapies are cost and accessibility, especially for the Medicare population. Cost estimates have put the cost of CAR T-cell therapies as high as $1.5 million per patient and that could make them inaccessible for many.

“There remain unanswered questions about value and cost in older adults,” said Walid F Gellad, MD, codirector for the Center for Pharmaceutical Policy and Prescribing at the University of Pittsburgh. “There are many life-saving treatments in the medical system that cost much less than this therapy. Presumably, its cost will go down as the indications expand and the experience with creating the CAR T cells improves. At least, one would hope.”

The creation of off-the-shelf, third-party products would help improve accessibility for CAR T-cell therapies and lower cost, said Helen Heslop, MD, director of the Center for Cell and Gene Therapy at Baylor College of Medicine, Houston. “In the longer term, there’re obviously a lot of people looking at how [the treatments] can be made more accessible. These are the first-generation CAR T [products], and I think there’ll be lots of refinements both to make them more effective and safer and also to use a third-party product to bring the cost of goods down.”

Other lingering unknowns about CAR T-cell therapies include how many patients in real-world clinical practice will have serious side effects, compared with those in trials, and the long-term recurrence rates after therapy use, Dr Gellad noted. He recently proposed in an article that government payers reimburse only the cost of manufacturing and some predetermined mark-up for such therapies until confirmatory trials demonstrate clinical benefit (N Engl J Med. 2017;376[21]:2001-4).

The current CAR T-cell therapies are only the beginning, said Dr Richard T Maziarz, MD, a bone marrow transplantation and blood cancer specialist at the Oregon Health and Science University Knight Cancer Institute in Portland. “Genetically engineered cell products are going to explode over the course of the next decade. This is not the end of the line, this is the starting point.”

Disclosures. Dr Maziarz has received consulting fees from Novartis, Juno Therapeutics, and Kite Pharma. Dr Heslop has received consulting fees from Novartis, has conducted research for Cell Medica and holds intellectual property rights/patents from Cell Medica, and has ownership interest in ViraCyte and Marker Therapeutics. Dr Gellad reports grants from Express Scripts.