Bluebird bio has launched its innovative gene therapy Zynteglo in Germany, its first commercial market.
The one-off gene therapy was approved by the European Medicines Agency (EMA) last June, for patients 12 years and older with transfusion-dependent beta thalassaemia (TDT) who have no matching donor for a stem cell transplant.
TDT is a hereditary disease caused by mutation in the β-globin gene that results in significantly reduced or absent adult haemoglobin. Those who suffer with this condition depend on blood transfusions to maintain haemoglobin levels throughout their lifetime.
Zynteglo (autologous CD34+ cells encoding β A-T87Q-globin gene) promises to permanently free beta thalassaemia patients from the frequent blood transfusions by restoring working copies of the β-globin gene into a patient’s own haematopoietic (blood) stem cells.
To prepare for the launch, bluebird has established a collaboration with the University Hospital of Heidelberg (pictured), as the first qualified treatment centre in Germany.
Institutions offering the therapy must have expertise in stem cell transplants as well as treating patients with TDT, due to the extremely technical and specialised nature of administering the gene therapy.
As it had previously planned, bluebird has entered into an outcomes-based payment agreement with multiple statutory health insurance providers in Germany. The total treatment cost of Zynteglo will be split into five instalments, with payers only having to pay if the therapy works at the time of infusion.
This means that an initial payment is made at the time of infusion, with four additional annual payments made, only if no transfusions for TDT are required by the patient thereafter.
This system varies only slightly from another model, namely the payment scheme offered for Novartis’ one-shot treatment for spinal muscular atrophy Zolgensma.
Zolgensma is priced at $2.12m, with its cost payable via a series of five instalments of $425,000 – however, this is not contingent on the outcomes of the treatment, which promises to immediately cure the degenerative disease.
With the rapid development of novel gene therapies also comes the need for new payment models that can accommodate the high cost of accessing these treatments.
Annuity models, such as what Novartis is offering for Zolgensma, and outcomes-based models, such as that being offered by bluebird for Zynteglo, could prove to be the only viable solutions for gene therapy developers in the coming years.
A report from The Alliance for Regenerative Medicine (ARM) published in July last year called for healthcare reimbursement systems across Europe’s leading nation to quickly adopt new payment models, including conditional reimbursement, pay-for-performance and annuity-based payments.
Now that bluebird has launched Zynteglo is Germany, the UK will be firmly in its sights. It may offer NICE a confidential discount, a move that many specialised treatment developers have taken, but its hard to tell if the cost-effectiveness watchdog will recommend it for use on the NHS even with a discount.
NICE’s system of appraisal has been criticised for being ‘inflexible’ when it comes to highly specialised treatments for rare diseases, like Zynteglo. NICE has a Highly Specialsed Technology (HST) appraisal process, but many rare diseases do not qualify for this pathway, and must therefore be appraised via its standard Single Technology Appraisal (STA) process.
This is the case for Zynteglo, which is set to undergo the NICE STA process – Biogen’s spinal muscular atrophy (SMA) treatment Spinraza (nusinersen) also followed this pathway, and was met with a rejection last year.
NICE will be looking at its HST system at an upcoming methods review, due to begin in the summer of this year. It will focus on what conditions and diseases are included in the HST programme, and at aiming to approach drugs reviewed under this system with increased flexibility.