Dialog Box

Children's Tumour Foundation of Australia

Selumetinib recommended for listing on PBS

The Pharmaceutical benefits advisory committee has recognised the significant unmet need in NF1 patients with inoperable Plexiform Neurofibromas

In very welcome news, the Pharmaceutical Benefits Advisory Committee (PBAC) has recommended Koselugo/selumetinib for listing on the Pharmaceutical Benefits Scheme (PBS). 

This update comes after years of advocacy and is the first step in bringing funded access to children under 18 years of age with neurofibromatosis type 1 (NF1) who have inoperable plexiform neurofibromas (PNs).

The Children's Tumour Foundation (CTF) was approached by Alexion, AstraZeneca Rare Disease, in November 2021 to be part of a Global Patient Advisory Board on Neurofibromatosis. These consultations lead to a collaborative workshop to provide a Patient Journey Map to illustrate the complexities of PNs and the usual health system experiences of people in Australia. 

Following these discussions, Alexion embarked on the process of having Selumetinib (Koselugo®) funded by the Pharmaceutical Benefits Scheme (PBS) for the treatment of symptomatic, inoperable plexiform neurofibromas (PN) in the paediatric population.

The Pharmaceutical Benefits Advisory Committee (PBAC) is the independent organisation which advises which medicines should be funded by the government.

Patients, caregivers, family members and friends shared their views via consumer comments; however a positive recommendation from the PBAC was not received at the initial meeting In November 2022. 

The CTF was then invited to attend a Facilitated Resolution Workshop in April 2023. Meredith, a CTF Support Services Coordinator, attended to represent the NF Community and to impress upon the PBAC the difference that Selumetinib would have for our children. 

To ensure the best possible chance at success, Alexion spent twelve months diligently incorporating all of the workshop’s recommendations and the CTF encouraged the community to provide comments to the PBAC. The Selumetinib application was resubmitted to the PBAC for consideration at the March 2024 meeting, and a recommendation was made public 26th April 2024. The outcomes from the March PBAC meeting can be found HERE .

Selumetinib is now the first treatment on the pathway to a PBS listing for these children where surgery is not an option. Alexion, AstraZeneca Rare Disease, the medicine's sponsor, will now work closely with the Department of Health towards a PBS listing which will see this medicine funded and available to any eligible child in need in Australia.


1. Department of Health and pharmaceutical company agree on details of listing (price and volume)

2. Cabinet approved for anything >$20m per year

3. Listing date finalised following approval by the health minister

This process can sometimes take several months – but every effort will be made to get this medicine on the PBS as soon as possible. This recommendation would not have been possible without the submissions of patients, caregivers, family members and friends sharing their lived experiences. 

The PBAC recognised the significant unmet need in NF1/PNs, and the challenges patients and families face when treatment options are limited.

We will keep you informed of the progress towards a listing and will update the community as more information becomes available. 

For any questions, please contact us at support@ctf.org.au or by phone 02 9713 6111


The PBAC is an independent expert body appointed by the Australian Government. It's primary role is to recommend new medicines for lising on the pharmaceutical Benefits Scheme based on clinical effectiveness, safety and cost-effectiveness compared with other treatments.

30 April 2024
Category: News