TEPLIZUMAB

The PBAC did not recommend listing teplizumab (Tzield®) to delay the onset of Type 1 diabetes (T1D, Stage 3) in patients aged 8 years or older with Stage 2 T1D. 

Stage 2 type 1 diabetes is an early stage of T1D where the immune system attacks the insulin-producing cells in the pancreas and blood sugar levels start to become abnormal, however the person has not yet developed symptoms. Over time, people with Stage 2 T1D can develop Stage 3 T1D – where blood sugar levels are high enough to cause symptoms and require treatment with insulin. 

The PBAC welcomed input from consumers, health care professionals and organisations. The PBAC acknowledged the need for treatments that reduce the impact of T1D. 

The PBAC noted that the submission was based on one clinical trial that randomised 76 patients called the TN-10 trial, which included 44 patients treated with teplizumab and 32 patients treated with placebo. 

The PBAC considered that the submission’s proposed approach to identify patients with Stage 2 T1D through screening family members of those with diagnosed T1D and through studies of T1D screening would not provide equitable access to the medicine. This view was formed as the majority of individuals diagnosed with T1D do not have a family member with the condition. The PBAC considered a coordinated approach to diagnosing Stage 2 T1D would be important to ensure equitable early diagnosis and access to potential treatments such as teplizumab. 

The PBAC considered that teplizumab is effective in delaying the onset of Stage 3 T1D when compared with placebo. The PBAC also noted that teplizumab was associated with a higher rate of adverse events, including serious adverse events when compared with placebo. 

The PBAC considered that delaying the onset of Stage 3 T1D would be beneficial for all patients, but especially for children and adolescents, as it would provide additional time free from symptomatic T1D, and the demands of insulin treatment and blood glucose management. However, the PBAC considered that the limited evidence presented in the submission did not adequately support the submission’s claim regarding long-term benefits of teplizumab after being diagnosed with Stage 3 T1D such as increased life expectancy and reductions in cardiovascular diseases or other long-term complications of T1D. Further, the PBAC considered that the value of a delay in onset of Stage 3 T1D would likely differ by age, and that patients treated as children or adolescents are likely to benefit most. As a result, the PBAC considered that the results of the economic evaluation were highly uncertain and the submission had not demonstrated that teplizumab would be value for money at the price sought by the supplying company. 

Sponsor's comment:
Sanofi is disappointed with the PBAC’s decision not to recommend teplizumab (Tzield) to delay the onset of Stage 3 type 1 diabetes (T1D) in adult and paediatric patients aged 8 years and older with Stage 2 type 1 diabetes. As the first new therapy available for T1D in over 100 years, teplizumab (Tzield) offers a new approach for the treatment of this autoimmune condition. We thank patients, clinicians and organisations for their valuable contributions to the PBAC process and we are committed to working with all stakeholders to resubmit teplizumab again for funding consideration at the earliest opportunity.