Zbigniew Pawlowski: Patients with amyloidosis need access to the drug. Life is at stake

For the treatment of transthyretin amyloidosis, we have registered causal treatment. What is the availability of this therapy for patients in Poland?

In cardiac amyloidosis, we have registered drug therapy. In Poland, there is a preponderance of patients with just this type of amyloidosis. The second variety is the mixed form, in which gene therapies are already registered. As for cardiac amyloidosis, a group of patients had access to a charity therapy sponsored by a drug manufacturer. She was started three years ago, when the patients were a few dozen. Since then, the group of patients has grown to nearly a hundred. The reimbursement application was rejected twice by the Ministry of Health, for various reasons. The reasons were not only economic, but also formal. Now we have a third approach to the reimbursement of this drug. It seems to me that the experience so far will make the hope for a positive decision firmly set. The situation now is that the charity program was closed at the end of the year. So no new patients are being accepted, and this is probably the most tragic moment, because there are people diagnosed, unable to enter the program.