Rare Disorders NZ October 2025 update
24 Oct 2025
| Pānui October 2025  Message from the Chief Executive Kia ora koutou katoa, I’m writing this hard on the heels of the recent Valuing Life: NZ Medicines Access Summit – which means that improving access to rare disorder medicines is very much top of mind. It’s all the more poignant with James McGoram’s recent resignation as RDNZ’s Chairperson, and his imminent move to Australia as a medical refugee, solely because that’s where he’ll be able to access the lifesaving medicines he needs to combat his particular rare disorder. Despite several years of being on Pharmac’s list of medicines waiting for funding (aka the Options for Investment list), the medicine James needs is yet to be funded. In the absence of a significant lift in Pharmac’s funding and a separate assessment pathway for rare disorder medicines, low volume high-cost medicines continue to struggle for prioritisation. Although this will seem to many like an ongoing unresolvable issue, presentations and discussions at the Summit, my inclusion on Pharmac’s Consumer and Patient Working Group, new Pharmac leadership, and combined meetings of rare disorder medicines pharmaceutical companies, patient voice and Pharmac representatives offer faint glimmers of hope that maybe – just maybe – the walls are ever so slightly starting to crumble. We at Rare Disorders NZ support a collective of over 150 disorder specific support groups whose members have needs and issues which are distinct from access to medicines. These include early and accurate diagnosis, access to rare disorders expertise and evidenced based best practice care pathways, service coordination, visibility and equity within the health system, and opportunities to benefit from research and innovation. The importance of implementing the Rare Disorders Strategy (RDS) to address these issues and improve lives cannot be overstated. We hope that our forthcoming November meeting with the Health Minister and RDS implementing agencies, our recent meeting with Professor Stephen Robertson and the cross-party parliamentary group on rare and undiagnosed disorders, and ongoing discussions with Te Whatu Ora | Health New Zealand will result in kickstarting implementation of the RDS and progressing the vision for a coordinated network of rare and undiagnosed disorders expertise. Finally, I strongly encourage you to take the Voice of Rare Disorders Survey if you have not yet done so. It is imperative we have a strong response rate to strengthen our advocacy work to improve systems and services for rare. Thank you for taking the time to participate. Ngā mihi Chris Chief Executive  Survey closes 31 October The Voice of Rare Disorders Survey will be closing on 31 October, so if you – or someone you care for, lives with a rare disorder, please take the survey today. Your responses are valuable. We use the anonymised data from our surveys: - To inform health professionals on how best to support patients living with rare disorders. - When presenting to Ministers and officials about the impacts of living with rare disorders. - In our submissions on proposed policy changes that could impact the rare disorder community. - To advocate for improved systems and services for the rare disorder community. - When educating the public and raising awareness about rare disorders in Aotearoa. Access the survey here. Valuing Life summit RDNZ represented the rare disorder community at the two-day Valuing Life Summit organised by Patient Voice Aotearoa and Medicines New Zealand at Parliament last week on medicine access issues. Chris participated in a panel discussion on the perspectives of patient advocates, bringing attention to the unique issues people living with rare disorders face accessing medicines. It was noticeable how this year rare disorders were routinely mentioned among a range of stakeholders throughout the day, reflecting the growing recognition of the unique challenges around accessing rare medicines and the need for a separate assessment pathway. Cross-party group quarterly meeting The Cross-Party Parliamentary Group on Rare and Undiagnosed Disorders held a quarterly meeting in October, with a focus on how a leadership and coordination mechanism for rare could be established in the health system. Leading up to the meeting Rare Disorders NZ arranged opportunities for support group leads and members of their Clinical Advisory Panel to provide input on how they envision a leadership and coordination mechanism for rare disorders. The chair of the Clinical Advisory Panel, Prof Stephen Robertson, presented the consultation feedback to those present at the meeting. Input into the draft New Zealand Disability Strategy 2026 - 2030 Whaikaha is refreshing the New Zealand Disability Strategy and at Rare Disorders NZ’s request, Whaikaha hosted an interactive webinar for our support group leads so that they could directly ask questions and provide feedback. We made a submission to the consultation informed by this webinar. View the submission here.  New podcast series by Rare Disorders NZ Rare Disorders NZ is proud to introduce our new podcast series, Rare Aware, hosted by Alanna Peck. Through this series Alanna will be exploring what it means to live with a rare disorder in Aotearoa New Zealand and how individuals and families navigate the journey. She will also be speaking with experts about what work is being done to make New Zealand a better place for the rare disorder community. In the first episode, Alanna speaks with outgoing Chair of Rare Disorders NZ James McGoram, who is having to uproot his family and move to Australia to access the life-saving treatment he requires to manage his rare disorder, Fabry disease. You can access the first episode here or from any major podcast platform. Pharmac definition After public consultation and additional input from Rare Disorders NZ Pharmac has updated its definition of a rare disorder to align with the definition in the Rare Disorders Strategy. Previously, their definition of less than 1 in 50,000 people only captured those now defined as ultra-rare. The new definition will enable Pharmac to assess more rare disorder medicines earlier in the process as the rare disorder policy principles allow Pharmac to assess funding applications before an application has been made to Medsafe for regulatory approval. Impact Report for 24/25 now available Rare Disorders NZ’s Impact Report for the 24/25 financial year is now available on our website. Despite a tough year financially, the RDNZ team is proud to have continued to achieve for the rare disorder community. There were notable highlights throughout the year, including: - The establishment of the Cross-Party Parliamentary Group on Rare and Undiagnosed Disorders; - Hosting a roundtable at parliament on diagnosis and access to rare disorder medicines attended by the Minister of Health, Associate Minister of Health, the shadow Minister of Health, health agency executives, specialists, industry, parliamentarians and consumer representatives; - The establishment of the Rare Disorders Research Network and Clinical Advisory Panel, - Celebrating our 25th anniversary at Government House on International Rare Disease Day; - Another successful Rare Disorders Month raising awareness of rare disorders around the motu, concluding with the the Sky Tower lighting up for rare. Read the Impact Report here.  Media NZ Doctor: Calling all Kiwis with a rare health condition – your voice is needed TVNZ: Rare disease advocate forced to move to Australia for treatment NZ Herald: Family fights to keep baby with rare genetic disorder alive  Thank you! We would like to acknowledge and thank the following sponsors for supporting key projects over the past two months: - Pub Charity - TG Macarthy Trust Make a difference for people living with a rare disorder We’re a small organisation with a big heart. We rely on grants and donations to carry out our work. Please consider donating to help us continue to improve information for rare disorder patients, their whānau and professionals and to keep advocating for systemic changes to benefit the entire rare disorder community. Your support is greatly appreciated. Donate |
