NICE Draft Guidance Rejects Zynteglo for Transfusion-dependent Beta-thalassaemia

LONDON, United Kingdom – In an appraisal consultation document, the National Institute for Health and Care Excellence (NICE) has recommended against the use of Bluebird Bio’s Zynteglo (betibeglogene autotemcel) for the treatment of patients with transfusion-dependent beta-thalassaemia (TDT).

The one-time gene therapy was rejected on the grounds that clinical trial data is small and insufficient to justify reimbursement, according to NICE, which means that the organization has uncertainties about the cost effectiveness. Further, the cost estimate for Zynteglo is “considerably” higher than what NICE normally considers an acceptable use of NHS resources at £1,450,000 per patient.

Despite recognizing the innovative and life-changing gene therapy as a potentially curative treatment, NICE’s methods – which have often been criticized for being behind the times when it comes to evaluating non-traditional drugs such as cell and gene therapies – mean that it will not be available to patients who live with the significant burden TDT.

Following a consultation period, the appraisal committee will meet again to consider the evidence after the opportunity for comments closes on 4 March 2021.

“Living with thalassaemia is a difficult and challenging experience not just for patients but for their entire families,” commented Romaine Maharaj, Executive Director, UK Thalassaemia Society.

She continued to say that the organization is “extremely disappointed with NICE’S decision not to recommend betibeglogene autotemcel as a treatment option in the UK.”

“We also feel disheartened that our patient experts were misquoted and used out of context and feel that NICE needs to rectify this. Having an option and the access to a potentially curable treatment is vital and should be offered to patients.”

Nicola Redfern, UK General Manager at bluebird bio, also expressed the company’s shock and disappointment at the decision, saying that Bluebird “strongly believe that NICE has failed to act in the best interests of people with TDT and their families in England and Wales.”

She continued to say, “It is baffling that NICE disregarded the testimonies of patients, advocacy groups and clinicians and, despite recognizing beti-cel as a potential cure for patients with TDT, has chosen to deny access and has dismissed the obvious unmet need. This decision is deeply concerning and will not only come as a huge blow to the TDT community but also sets a dangerous precedent which could limit access to other gene therapies in the UK in the future.”

Zynteglo received a conditional marketing authorization valid throughout the EU on 29 May 2019.

The costly drug has already been launched in Germany under a value-based payment agreement with multiple statutory health insurances. The deal means that payers only pay if the therapy delivers on its promise. Bluebird’s proposed payment model is limited to five payments issued in equal installments: an initial payment at the time of infusion and four additional annual payments if no transfusions for TDT are required for the patient.