Rare Disorders NZ: November Update
19 Dec 2022
 In this issue Rare Disorders NZ and Costello Medical Roundtable Accessibility for New Zealanders Bill Win for Spinal Muscular Atrophy Community Neurological Alliance Letters New Zealand Selected for International Rare Disease Study Health Quality and Safety Commission Consumer Code Spotlight on the Board of Trustees Rare Disease Day 2023 Save the Dates How You Can Help Join us on Social Media Thank You to Sponsors CE Update As you will see from this newsletter, we are very busy at Rare Disorders NZ working to support those with rare disorders but also in our advocacy work. I thought I would share with you our current priorities; Support – we continue to support individuals and our collective of support groups, and running our enquiries line. This is a precious service, and we help with connections on a daily basis. Please do get in touch with any feedback on how we can support the rare disorders community. Advocacy – we continue to advocate for better health, medicines, education and disability access and outcomes for those with rare disorders. You will see below lots of our advocacy work. Our main current focus is advocating for being a co-design partner in developing the National Strategy for Rare Disorders. Funding – as you may know we are funded through government and fundraising. Our current focus here is to ensure the government through its agencies funds us sufficiently to ensure the voice of those with rare disorders is heard and included, and we can be a partner. We are also working to increase our fundraising to be able to do more in supporting and advocating for those with rare disorders. You can find ways to help us at the end of this newsletter. This is an exciting time for us and there are real opportunities to make a difference for those living with Rare Disorders in New Zealand. We appreciate you taking the time to read about our work and maybe being able to support us. Please help get our message out widely and share this newsletter with friends, family and colleagues. Michelle Arrowsmith CE, Rare Disorders NZ  Rare Disorders NZ hosts roundtable to discuss NZ’s poor track record on rare disorders  On 26th October, Rare Disorders NZ, in collaboration with Costello Medical UK, hosted a virtual one-day roundtable to provide a platform for rare disorder advocates, healthcare professionals and researchers from New Zealand and abroad to share insights from their own experiences with rare conditions, either personally or professionally, and to discuss opportunities for improving the current situation in New Zealand for individuals with a rare disorder and their whānau. There was consensus among attendees that inclusion of all stakeholders, including those with lived experiences, is key to ensuring better health outcomes, and that the costs of doing nothing are too high for families, the health system and the country as a whole. An infographic summarising key takeaways and recommendations from discussions will be developed by Costello Medical. The infographic will be shared widely with the aim of encouraging the prioritisation of the unmet needs of the rare disorder community on the policy agenda and driving policy change. Read our media release here. Accessibility for New Zealanders Bill Submission and Petition  Rare Disorders NZ made a submission to the Accessibility for New Zealanders Bill. This bill aims to address accessibility barriers experienced by disabled people, tāngata whaikaha and others, enabling them to live independently and participate fully in all areas of life. Rare Disorders NZ expressed concern that the current bill will not remove barriers or deliver real progress for disabled people and recommended changes. If you would like to make a submission, our website has a step-by-step guide on how to go about it. Submissions close on Monday 7th November. Access Matters Aotearoa has a petition calling for the Government to broaden the scope of the Accessibility for New Zealanders Bill to include: Standards A regulator A barrier notification system A dispute resolution process to remove access barriers. Rare Disorders NZ supports the petition by Access Matters Aotearoa. If you would like to lend your support, you can sign the petition here. A huge win for Spinal Muscular Atrophy community  It was a day of celebration on 28 September when Pharmac announced it has reached a provisional agreement with medicine supplier Biogen to fund Nusinersen (brand name Spinraza) for people with Spinal Muscular Atrophy (SMA). The SMA community has been fighting for years for this life-saving treatment to be funded and it is thanks to their hard mahi, and in particular Patient Voice Aotearoa advocate Fiona Tolich, that Spinraza will soon be available. It is now more important than ever to include SMA on the newborn screening programme so that babies born with SMA can be treated as soon as possible. SMA is the leading genetic cause of infant mortality, with babies dying at an average age of 13 months old without treatment. Pharmac plans for Nusinersen to be funded from 1 January 2023 in New Zealand. Read our media release here. Following the announcement Pharmac, issued a consultation for feedback on the proposal to fund Nusinersen. Rare Disorders NZ provided feedback, which you can read here. Neurological Alliance sends letters to government seeking more engagement  On 3 October, the Neurological Alliance, with Rare Disorders NZ's support, sent two letters on the Alliance's position in relation to the Health Reforms, the new health system and engagement. The first letter was to Minister Little, expressing frustration about the lack of engagement with Health NZ, and seeking a meeting with him to start the kōrero to ensure the future outcomes in the new health system deliver for those impacted by a neurological condition, including their supporting whānau. The second was to the new CEOs of Te Aka Whai Ora (Riana Manuel) and Te Whatu Ora (Margie Apa) seeking engagement and collaboration on the work ahead in the new health system. You can find out more about the Neurological Alliance and read the letters here. New Zealand selected for international study on barriers to accessing treatments, with a focus on Cystinosis and Cysteamine This month we have been supporting a working group, coordinated by Rare Disease International in collaboration with International Rare Disease Research Consortium (IRDiRC) to analyse barriers to access essential medicines for rare diseases. The working group is running a case study on Cystinosis and Cysteamine, taking a country approach to analyse challenges accessing medicine for cystinosis in a few selected countries around the world. New Zealand was one of the countries selected for this study, and we have been engaging with the Cystinosis NZ support group for their input to this study, to help map the common barriers patients face in accessing treatments. Health Quality & Safety Commission new consumer code launched, opportunities to get involved At the end of August, as part of the Pae Ora legislation, the Health Quality & Safety Commission launched the ‘code of expectations for heath entities for engagement with consumers and whānau’. The code sets the expectations for how health entities must work with consumers, whānau and communities in the planning, design, delivery and evaluation of health services. You can find the code and a link to other relevant information about the code on the HQSCs website. Spotlight on the RDNZ Board of Trustees  In this issue we meet our newest board member, Awhina Hollis-English. Awhina lives in Hawkes Bay with her three children; one who has multiple medical complexities resulting from Cytomegalovirus (CMV). Awhina completed a PhD in Social Work at the University of Otago and worked as a lecturer in Social Work at Massey University. Her research focuses on Kaupapa Māori theory and practice. What interested you in becoming a member of RDNZ’s board? My whānau have a strong connection to RDNZ and have a number of rare disorders too. My Uncle John Forman was the founder of RDNZ, so our whānau are extremely proud of him and what RDNZ is today, and it was the first place I went when my daughter was diagnosed with congenital Cytomegalovirus (CMV). Through RDNZ I was able to connect with one other whānau in NZ, and later with a few others. As a board member I hope to give back to an organisation who supported us in the past. What are you personally interested in advancing for rare as a member of the board? I'm passionate about supporting whānau with rare disorders as I am a part of one myself. I have a background in social work and Māori advancement and feel that through whanaungatanga - building connections and manaaki, caring for each other, we are better able to advocate for ourselves and others. What is something from the last board meeting that stood out to you? RDNZ has achieved a great deal recently through the advocacy and hard work of many. The passion and care that staff, board members and whānau have put into RDNZ over the years has been wonderful to see and it is a privilege to be a part of RDNZ at this exciting time. Rare Disease day 2023 The team at Rare Disorders NZ are well underway with planning for Rare Disease Day 2023 (28 February). We are excited to share that the plan for the campaign includes extending Rare Disease Day out into a whole month, to allow us more time to get our messages out into the public and increase influence on decision makers. Save the dates  Some events taking place in Rare Disorders Month: Launch of a new Rare Disorders NZ Parent Guide by the Governor General 10-12 March Rare Beer Challenge, Wellington and Auckland 02 March PaintVine fundraiser, Wellington We look forward to updating you with more information soon! How you can help us help those with Rare Disorders across New Zealand If you would like to help our cause and support us to advocate for better healthcare, better access to vital medicines and ensure those with rare disorders receive the care they need to be able to live well and productively as New Zealanders there are several ways you can help; Donate regularly to Rare Disorders NZ the easy way through One Percent Collective. Or alternatively here is the link to RDNZ | Donate (raredisorders.org.nz) Join us on social media  Please help us to reach more New Zealanders so more people understand about Rare Disorders, where to get help for those they know with a Rare Disorders and our campaign for better healthcare. There are several ways to do this, so please join us on Thank you  We would like to acknowledge and thank the following sponsors for funding key projects for us over the past two months. Todd Foundation Thomas George Macarthy Trust Lottery Community Fund Kiwi Gaming Foundation Pub Charity Rata Foundation Trust House Foundation Four Winds Foundation The team at Rare Disorders NZ |
