14 December 2018.
The Metabolic Dietary Disorders Association is extremely pleased with the decision of the Pharmaceutical Benefits Advisory Committee (PBAC) to recommend the listing of Sapropterin (sapropterin dihydrochloride) on the Pharmaceutical Benefits Scheme (PBS) for children and adolescents under 18 years of age (with continued access into adulthood) living with phenylketonuria (PKU).
Following this recommendation, there are several more steps before a medicine can be listed on the PBS. The PBAC website provides information about these and indicates that the minimum time frame for the completion of these processes is 5 months. In the mean time we recommend that you review the suggested BH4 guidelines developed by ASIEM to be as prepared as possible when the responsiveness testing commences.
MDDA, with the support of our PKU community, has been actively campaigning for a number of years to secure access to this treatment for children and adolescents who are responsive to this therapy. This welcome result has been the outcome of extensive political, media and public advocacy by our PKU and broader metabolic dietary disorders communities.
Whilst the outcome is a positive one, we are disappointed that at this time it has not been recommended for adults and we intend to continue to campaign for widespread Sapropterin accessibility for all ages and will once again be seeking the support of the whole community.
We want to thank each and every one of you that helped to achieve this important outcome, whether you raised the issue with your Federal Member of Parliament, shared your story of living with PKU with media, or talked about the issue with your family and friends to raise awareness of PKU – this has been an enormous effort and you should feel proud of the part you have played.
We also want to thank everyone who submitted a consumer comment to the PBAC about how important it was for our PKU community to get access to this treatment on the PBS. We were astounded to be told by the PBAC that it received 965 consumer comments in support of listing Sapropterin on the PBS! This is not just extraordinary for a rare disease – but it is the first time the PBAC has ever received this volume of consumer comments for any treatment it has previously reviewed. This is a testament to your passion and commitment and we can’t thank you enough.
For those who wish to be involved in the next steps of advocating for adults, a short webinar will be held in early 2019.
Any questions can be emailed to [email protected].