RVA welcomes the announcement that the Pharmaceutical Benefits Scheme (PBS) listed treatment for Phenylketonuria (PKU) will now be accessible to all Australians living with PKU who are responsive to treatment. RVA provided a consumer comment regarding this treatment that, in line with the National Strategic Action Plan for Rare Diseases (the Action Plan), highlights the importance of equitable access to treatments for adults who have lifelong conditions.
Congratulations to RVA Partners, the Metabolic Dietary Disorders Association (MDDA) and PKU Association of NSW, and their communities for advocating for the best outcomes for Australians living with PKU.
RVA also acknowledges the disappointment that the other treatment for PKU considered for listing on the PBS by the Pharmaceutical Benefits Advisory Committee (PBAC) at the July 2022 meeting was rejected. RVA provided a consumer comment for this treatment that highlighted the importance of new and innovative therapies becoming available to Australians living with a rare condition as soon as possible, which aligns with the Action Plan.
RVA provides consumer comments to the PBAC regarding systemic issues for Australia’s rare disease community.
