Dialog Box

Children's Tumour Foundation of Australia

Addressing the revolving door to nowhere

Addressing the revolving door to nowhere


Neurofibromatosis (NF) is a complicated condition, so it makes sense that it requires a complicated solution…or does it? When it comes to NF, collaboration over complication is needed. 

 As Australia’s only patient advocacy and support organisation for those impacted by NF, we believe that the path to conquering NF will take a wide range of collaborative efforts from researchers, clinics and hospitals, government bodies, pharmaceutical industry, the private sector and other NFP organisations, as well as patients and their families.  

Problem shared is a problem halved.

 On Monday 20 February, our CEO Leanne Dib and Head of Support Services, Ruth Lindsay met with members of the local and international Alexion Rare (AstraZeneca) team in Sydney, including Senior Vice President Soraya Bekkali; International Medical Director, Dr Cezary Statuch and local General Manager, Medical Director and Patient Advocacy Advisor. 

 The intent of the meeting was to build awareness of the support and services the Children’s Tumour Foundation (CTF) provides to the community in Australia and hear directly from patients and caregivers about their journey.  

 NF Ambassador Janu Dhayanathan (NSW) and NF Mum, Jennifer Pearsall (QLD) gave moving accounts of their experience, with key insights shared about the inequities that patients and families experience with rare diseases in our community. They reported challenges in access to experienced specialists, gaps in clinician knowledge and experience with NF.  

Jennifer’s youngest son, Grayson (6) was diagnosed with NF1 at 18 months, and she has been fighting for answers and better treatments ever since but is increasingly frustrated by the lack of knowledge amongst healthcare professionals.

Like many other parents, she has become an expert in NF and has had to navigate her own path through a very complex set of medical issues.  

 During this meeting, she was able to express the frustration and constant stress families experience with what she fittingly describes as “a revolving door to nowhere”. She went onto explain a recent example where she waited seven months for an appointment with a neurologist to discuss access to a drug trial for Grayson, only to leave the appointment more frustrated and heart broken. 

 “The specialist had no knowledge about the trial, despite this being the reason for the original referral. The only good thing to come from this experience was that I was able to use it to highlight the issue within our existing health system to the team at Alexion. Without affordable access to treatments like Selumetinib through the PBS, accessing these life-changing treatments would be completely out of reach for most families, including my own.” 

 Alexion are currently working to secure PBAC support to approve Selumetinib (Koselogo®) for children aged between two to 18 years who have symptomatic, inoperable plexiform neurofibromas (PN).  You can read about the most recent update here.  

 Unfortunately, they did not receive a positive recommendation from the PBAC at the November meeting, which is why meetings with the Children’s Tumour Foundation and community members like Janu and Jen are so important to improve the chances of a positive outcome in the future.   

“It is really encouraging that pharmaceutical companies are striving to make treatment more accessible to the community. Not only that, but they are also trying to affect change in other areas that impact people living with NF”, said Janu. 

Our support teamwork with hundreds of families and adults with NF each year, and through this work we are able to identify many of the missing links in patient care and hearing these stories helps drive our mission of supporting every person who is impacted by NF. 

However, it is important that there are opportunities for community to share their stories and experiences directly too, to help advocate, raise greater awareness and highlight the issues that are important to them. 

"Alone we can do so little; together we can do so much.”  

Helen Keller 


27 February 2023
Category: Blog
Tags: Alexion, AstraZeneca, diagnosis, Neurofibromatosis, NF,