Interview with Zbigniew Pawlowski, president of the Association of Families of Patients with Amyloidosis.
Zbigniew Pawlowski: Access to TTR amyloidosis therapy is humanly imperative for patients
Published Nov. 28, 2023 11:51
In an audit conducted earlier this year, representatives of patients with transthyretin amyloidosis once again highlighted an urgent and unfortunately still unmet need. This is access to causal treatment, so that patients could be managed in accordance with international guidelines. What is the situation for these patients now?
Invariably... It's been four years since the drug was registered in Poland. For four years we have been fighting for any drug to be available. Normally in Poland it takes about 2 years to solve such problems. Twice the application for reimbursement of this drug has already been submitted. Twice it was rejected, and now it is submitted for the third time. I hope that this time, the process will be successful, that is, the introduction of this one drug to the list of reimbursed drugs. The problem is growing because excellently developing diagnostics means that the disease is being detected in more and more people. Just two years ago, there were about thirty of us. Now there are nearly 100 people with this diagnosis. At the same time, all forecasts indicate that annually, there will be about 50-70 new patients. Without this drug, the survival period of patients averages 2 to 4, at most 5 years. Each year of delay in reimbursement results in unnecessary deaths.
Here we touch on the key problem, which is time. That time is life.
Exactly. That time is just life. I will relate to myself. Five years ago, when I got the diagnosis and read about the disease, I took stock and was prepared that the survival time would be several years. I am currently in my fourth year of therapy at a charity program. This is not a curable disease. It is a disease that will progress, and the drugs are designed to stop or slow the progression of the disease. Patient results clearly show that the disease process really is very slowed down. In some situations, as in my case, the process is virtually stopped. Studies clearly indicate that this drug really works. Access to it I believe is humanely indispensable for us patients.
