“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.”
Dr. Helen Heslop
“In the longer term, there’s obviously a lot of people looking at how [the treatments] can be made more accessible,” she said in an interview. “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, but also eventually with trying 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. A recent paper by Dr. Gellad in the New England Journal of Medicine proposes 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]:2001-04).