The European Medicines Agency (EMA) approved the one-off gene therapy last June. Those patients who have TDT, transfusion-dependent beta-thalassemia and are 12 years of age or older and don’t have a donor with matching stem cells for a transplant.
TDT is a genetic disorder and is caused when there is a mutation in the β-globin gene. A person lacks a great number or even completely adult hemoglobin under this condition and to maintain their hemoglobin levels, one has to transfuse blood continuously throughout their life.
The β-globin gene working pipes can, however, be restored in the blood stem cells of a patient suffering from TDT through Zynteglo and thus relieve them from having frequent blood transfusions.
Bluebird has collaborated with the University Hospital of Heidelberg for the launch of the therapy and become Germany’s first qualified center for the treatment.
Only institutions that expert in the transplant of stem cells and experience with treating TDT patients are eligible to offer the treatment as the therapy has an extremely technical nature.
Bluebird, as per its previous plans, has made an agreement with various German health insurance companies for the payment of the therapy. The complete payment of Zynteglo is to be made in five installments in which the four out of five are to be paid only at the condition of success of therapy.
The very first payment has to be made at the infusion time while the rest of the four installments to be paid annually only if the patient does not need any transfusions for TDT.
There is only a slight difference between this system and that offered for the one-shot treatment of Novartis for the spinal muscular atrophy Zolgensma.
Zolgensma costs $2.12m and this cost can be paid in five installments, each of $425,000. This mode of payment is however not conditional on the results of the therapy which promises an immediate cure of the disease.
As the new gene therapies are being developed rapidly, we also need novel payment models to enable us to afford these extremely expensive treatments.