Type 1 diabetes drug unable to secure PBS listing

The Pharmaceutical Benefits Advisory Committee (PBAC) has recommended against listing a breakthrough drug on the subsidised medicines scheme which can delay the progression of type 1 diabetes (T1D).
 
But experts are hopeful a future submission can make stronger case for affordable access to the medication.
 
Teplizumab (sold as Tzield) was put forward for PBAC consideration ‘for the treatment of Stage 2 T1D in patients aged eight years or older in order to delay the onset of Stage 3 T1D’.
 
The therapy works by targeting the immune attack that causes type 1 diabetes, delaying progression to clinical diagnosis by an average of two years. Patients are given one teplizumab infusion each day for 14 consecutive days.
 
The bid to have teplizumab listed on the PBS follows its approval by the Therapeutic Goods Administration (TGA) in May – a move described by Diabetes Australia as ‘a game-changing shift in treatment and management’.

However, the PBAC recommended against listing teplizumab on the PBS at its July meeting, with Tzield’s manufacturer, Sanofi, expressing disappointment.
 
‘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,’ the PBAC outcomes report stated.
 
But it felt ‘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.’
 
RACGP Specific Interests Diabetes Chair Dr Gary Deed said while the PBAC position is understandable, it does offer a window for future submissions that may identify younger patients and may provide a better economic argument for the initial approval.
 
‘Evidence is suggesting and noted in the PBAC response that a delay in the onset of type 1 diabetes from use of this or emerging agents may have significant benefit in allowing for staged support for people identified or affected by providing careful multidisciplinary support networks and monitoring for the onset of hyperglycaemia before diabetic ketoacidosis emerges,’ he told newsGP.
 
‘GPs are vital in helping identify people at risk or also developing support networks for these people in Stage 2 type 1 diabetes – identified as having 2 diabetes autoantibodies and no hyperglycaemia at this stage.’
 
Dr Deed added he would support a future bid by the company for PBS inclusion as this is ‘the first drug class that is offering a window to the possibility of changing the landscape of early type 1 diabetes and preventing hard or crash landing – [the] unknown and unpredicted onset of severe hyperglycaemic crises or diabetic ketoacidosis at the point of diagnosis rather than [relying on] the multidisciplinary team support [networks and monitoring approach].’
 
A spokesperson for Sanofi told newsGP the company is currently reviewing all feedback from the PBAC and will make a decision on resubmission timing in the near future.
 
While teplizumab is currently not available on private script in Australia, healthcare professionals can consider connecting with the Australasian Type 1 Diabetes Immunotherapy Collaborative and/or reach out to Sanofi Medical directly to discuss any potential access pathways, the spokesperson said.
 
Dr Deed said he provided consultancy to Sanofi as sponsor of this submission in his capacity as a coinvestigator on type 1 diabetes screening in Australia and national and international work on educational initiatives regarding staging of type 1 diabetes.
 
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