Selumetinib pbs update
Alexion, AstraZeneca Rare Disease has been seeking to have Selumetinib (Koselugo®) funded by the Pharmaceutical Benefits Scheme (PBS) for the treatment of neurofibromatosis type 1 (NF1) paediatric patients aged two years and above, who have symptomatic, inoperable plexiform neurofibromas (PN).
Earlier in the year, the Pharmaceutical Benefits Advisory Committee (PBAC) invited patients, caregivers, family members and friends to share their views via their online portal, to better understand the impact of the condition and the challenges patients and families face.
Consumer comments are a way for the PBAC to understand issues that are important to patients and caregivers, and comments that contain specific information about why treatment is important based on lived experiences, needs and preferences can impact the PBAC’s advice.
Unfortunately, they did not receive a positive recommendation from the PBAC at the November meeting.
Although this is disappointing to us all, we can be encouraged by the PBAC's acknowledgement that Selumetinib addresses a high and urgent unmet clinical need for children with inoperable PNs and the lived experience of our community was critical in building that awareness.
Alexion has advised us that they will continue to work with the PBAC and the Department of Health and Aged Care to secure access to Selumetinib and will keep us informed of the next steps in that process.
Whilst this is difficult news for all involved, the pathway to funded access to new medicines for rare conditions such as NF1, with inoperable PNs is complex, and it is not unusual for the PBAC not to recommend a first submission.
It is important not to lose hope as we must continue to advocate for access to treatment options where there are no alternatives, and support clinical trials and efforts to extend access to the adult population as well.
PBS.GOV.AU (refer to page 35)
If you would like to learn more about how you can support future advocacy efforts, we would love to hear from you.
Contact us at support@ctf.org.au or by phone 02 9713 6111