Update - On Thursday 2 April dinutuximab-beta (QARZIBA) was approved by the Australian Therapeutic Goods Administration (TGA). Thank you to all the families who took the time to make this happen.

Request to families affected by neuroblastoma

Can you take a few minutes to help get a drug for the treatment of high-risk neuroblastoma listed on the PBS? The drug in question is the anti-GD2 immunotherapy drug called dinutuximab-beta (qarziba) which is being used mainly in the consolidation phase of treatment of children with high risk neuroblastoma (patients 12 months and older).

Currently hospitals need to assess each individual child on a case by case basis as to whether there is a clear case to fund dinutuximab-beta (and potentially apply to the State Department of Health for funding). This means there is no standard or “fair” approach to funding as one patient may be declined in one region but not necessarily in another. The cost of the drug is also very high so this could also affect decisions (although we, like you no doubt, don't think it should). EUSA pharmaceuticals who own the distribution rights to this drug, are putting forward the case to the Pharmaceutical Benefits Advisory Committee (PBAC) in March that the drug be listed on the PBS which means the issue of funding will be removed as it will be subsidised by the Government. There is currently an opportunity for families affected/clinicians to comment on this proposal and we believe it would be useful for families to comment to support it being listed on the PBS.

If you are able to do so, please go to: https://www1.health.gov.au/internet/main/publishing.nsf/Content/PBAC_online_submission_form

The deadline is 12 February

More information regarding the medicine and the PBAC and funding process can be found here

We would like to recognise Dr Toby Trahair at Sydney Children’s Hospital for his tireless work in this area, working with EUSA (Europe) and getting the drug to this point. Based on current research this immunotherapy treatment is making a noticeable difference to survival rates for high risk neuroblastoma children – estimated survival improvement is between 10 to 15%. This is why we need it to be a definite and straightforward option for all children affected.

Thank you

Lucy Jones President Neuroblastoma Australia

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