Help Secure Access to NEW Treatments

Unusually there are two treatments for neuroblastoma going through the regulatory approval process. This is exciting to see, but we need your help to help ensure they get approved. 

DFMO

We need your help to support the approval and funding of the therapy called DFMO. 

We recently helped secure interim Federal Government funding for access to DFMO in Australia.  Neuroblastoma Australia and families pushed for access as this therapy was shown to reduce the rate of relapse by 50% in a clinical trial. This represents a huge difference for children with high risk neuroblastoma.  

DFMO has now become available to families via an Early Access Scheme whilst it goes through regulatory approval – and this is why we now need your support to ensure future access to this therapy is approved and funded.  

The Pharmaceutical Benefits Advisory Committee (PBAC) is considering the application and there is now a window for public consultation (until 29 Jan 2025).  If approved, access to DFMO will be secure and free for all children with high risk neuroblastoma as part of standard frontline treatment.  

The following survey is designed for anyone with relevant experience. You do not need to have a child in treatment, or a child who is potentially eligible for DFMO, to participate.

Please complete the survey but before completing, please visit our website page for useful information here  

Further information about the review can also be found here

Survey Link

Survey deadline is January 29, 2025 

Dintuximab Beta

The second treatment that requires approval and funding its the immunotherapy treatment, Dinutuximab Beta (brand name QARZIBA). 

Dinutuximab Beta is currently used as part of standard treatment for children with high-risk neuroblastoma. However, it is NOT approved for children with relapsed or refractory (drug resistant) neuroblastoma.  

The Medical Services Advisory Committee (MSAC) is currently reviewing the application for Dinutuximab Beta to be used to treat relapsed or refractory neuroblastoma, in combination with chemotherapy. 

A key part of the MSAC’s consideration process is feedback from the community and there is a survey where you can provide this. Anyone with relevant experience can complete it. You do not need to have a child in treatment, or a child who is potentially eligible for Dinutuximab Beta, to participate. 

We urge families impacted by neuroblastoma and our supporters to complete the survey and share their experience of neuroblastoma and why having this additional treatment option is extremely important 

Before doing so, we have created some helpful notes here. 

And there’s also some information on the MSAC website here.

Survey Link 

Survey deadline is February 14, 2025