Gene therapy is a promising treatment for severe hemophilia B. In this inherited bleeding disorder, blood does not clot properly.
Clinical trials are underway to make sure gene therapy is safe and effective for adults. Information about the cost to make and deliver gene therapy has been missing.
“Gene therapy for hemophilia is at a critical point. It is proving successful in clinical trials, but without reimbursement by third-party payers and insurance companies, patients may not benefit,” said Nancy Bolous, MD, of St. Jude Global.
St. Jude has produced gene therapy for hemophilia B. Scientists recently used the hospital’s data to figure out the cost to make and distribute gene therapy. The cost included five years of follow-up care. The price was only $87,198 per person. That is far lower than current hemophilia B treatments. It is also much less than proposed prices for gene therapy if it is approved.
Researchers hope publishing the actual cost will lead to open discussions about prices of this possibly lifesaving medicine.
Blood published a report on this work.