Fair for rare: Collette's editorial
Happy Spring everyone.
I was fortunate recently to attend the Australian Pompe Association meeting in Noosa where the focus was on advocating for the addition of Pompe Disease to newborn screening across Australia. The need for this is heartbreakingly illustrated by the story of baby Nate. NZORD supports the NZ Pompe Network in their efforts to get newborn screening for Pompe implemented in New Zealand.
Timely diagnosis, access to medicines, carer support, research and access to support services are all part of the wider picture of issues facing the rare disorder community. At NZORD we increasingly see our role as being to draw people together with the aim of bringing systemic change. We are commencing work on developing an overarching Rare Disease Policy document over the next 6-12 months. This will be a one-stop information and reference tool for the evidence-based policies the rare disorder community needs to have implemented in New Zealand. We will be actively seeking your views and input through this process.
In the meantime, it's business as usual as we work alongside PHARMAC to observe their process during the latest call for proposals for rare disease medicines which yielded 13 applications (see below). The new rare disease PTAC subcommittee meets in November and we will be keenly asking for updates on the outcome of those meetings.
We are looking towards 2019 and planning is underway for Rare Disease Day in February. Keep a close eye on our website and emails for some exciting announcements coming about events closer to the time!
Dr Collette Bromhead