My final newsletter as Chief executive for Rare Disorders NZ demonstrates the amazing work our small team have been completing over recent times. I am so proud of all the mahi and amazing outcomes over the last five years, and look forward to the next steps as an independent consultant continuing to work for equity for those with rare disorders.
We are in a time where there can be a mindshift of understanding of current systemic barriers for those with rare disorders, which offers opportunity for practical modifications to be applied to our transformed system, allowing for a holistic, inclusive, and most importantly functional pathway. The time is now to enable such a strategy and action plan for this to happen, and I am keen to offer my support to such progress.
I wish to offer my heartfelt thanks to all those who have supported me over these last five years, and especially the people who gave their voice, time and energy to our advocacy campaign.
I wish to name a few, including Sue Haldane, who launched the petition for a ‘National Framework for Rare Disorders’ and spoke directly to Hon Andrew Little with myself and James McGoram, about the challenges she had to live with, to help him to appreciate and understand the complexity and unmet need. Kelly McQuillan, for her tireless work to have Ministers notice and listen to the challenges for the rare community, and to Jaime Christmas who stepped forward recently to speak directly with GPs and specialists to share the difficult journey she and her recently departed husband Aubrey, who lived with a rare disorder, had endured. Her poignant statement expressed regret for lost opportunities where pathways for more appropriate coordinated care could have made such a massive difference, and would have given Aubrey more precious time together with his loving family. Jaime explained that most of the health professionals provided exemplary care within a system that did not see the rare patient – clarifying that it was the system and not those who work within it which needed improvement.
And to all those who have provided time, resources and their faces for videos and news stories, those who have visited their MP, attended MOH or other meetings with us or represented Rare Disorders NZ at key events: thank you.
Unity is power and that is what creates change!
It is these voices and this community that deserve recognition, respect and action.
Government commits to develop a strategy for rare disorders
‘A strategy for rare disorders needs to be developed’ – Minister of Health Andrew Little.
It was a watershed moment for the rare disorder community in Aotearoa to receive the news on 1 June that the Government had accepted the Pharmac Review’s recommendation to develop a strategy for rare disorders. The Minister acknowledged that improvements were also needed in the way Pharmac made decisions, and a critical part of this is “…talking to people at the receiving end of their decisions, namely health consumers and particularly people in the rare disorders’ community.”
We welcome this acknowledgement and look forward to engaging with the Ministry to ensure the voices of those with lived experiences are heard. We would like to thank everyone who has fought for recognition for the rare community – it is thanks to our collective mahi that we got to this point, and we now welcome this new phase where we will be working to ensure the strategy is directed by the rare voice and fit for purpose.
The Pharmac Review was finally released on 1 June, and it was pleasing to see the review panel’s thorough examination of the situation for rare disorders in New Zealand and the major detrimental impact a lack of coordinated approach for rare disorders has on rare patients. The panel had taken on board the submissions from Rare Disorders NZ and others, and fully endorsed Rare Disorders NZ’s call for a national rare disorder framework. Disappointedly, despite being applauded by politicians for the highest amount ever provided to Pharmac with a budget of $191million over two years, this is insufficient to meet the needs to clear those medicines already approved for funding (options for investment list) and the reality is that many will lose out and face uncertain futures due to this decision. Another factor is that there has been no prioritisation for rare disorder medicines to be funded and with a current mandate for Pharmac to get the best outcomes for the most people creates a negative bias which creates discrimination for the rare community. Our hope is that the reports around equity will include recommendations to rectify this situation as New Zealand is falling behind. Rare Disorders NZ has written to the Minister to request prioritisation for access to modern medicines for rare disorders alongside cancer.
You can read the government response to the independent review here.
Governor General becomes patron of Rare Disorders NZ
We are honoured to have received confirmation that Her Excellency, The Right Honourable Dame Cindy Kiro, GNZM, QSO, Governor-General of New Zealand, has accepted our invitation to become patron of Rare Disorders NZ. We look forward to involving Her Excellency in our work to support all New Zealanders who live with a rare condition, and the people who care for them.
Rare Disorders NZ took every opportunity at the largest annual gathering in New Zealand of General Practitioners at the General Practice Conference and Medical Exhibition to raise awareness of rare disorders, by presenting a workshop, running a stall and including our flyers in each delegate’s bag. It was the first time Rare Disorders NZ has presented at the conference, and CE Lisa Foster’s workshop was the second most sought-after workshop, which highlights the huge demand among health professionals for more knowledge about rare disorders.
GP Awareness video
To support Lisa’s presentation at the GP CME, we commissioned a video targeted to GPs, conveying directly from three rare patients their experience of living with a rare disorder and what they would like GPs to know. The video is now accessible to the general public through our YouTube channel.
By the end of June, every GP in NZ would have received an information pack from us about rare disorders. That’s over 5000 GPs in the country being made ‘rare aware’ and advised about how best to manage rare disorder cases that come through their practices.
Rare Beer challenge a huge success
On 3 June, Fortune Favours hosted the Rare Beer Challenge fundraiser for Rare Disorders NZ, and what a night it was! Fortune Favours recorded one of their busiest nights, and raised over $10,000 for Rare Disorders NZ! We are so grateful to Fortune Favours, the 18 participating craft breweries, Hills Hats for donating the trophy hat, the judges who donated their time and everyone who supported the event in one way or another. It was truly uplifting to experience so much support for the rare disorder community. We were also thrilled to be on TVNZ Breakfast on the morning of the challenge to raise awareness of rare disorders.
Following the success of the Rare Beer challenge, on 16 June Fortune Favours hosted another unique event to support Rare Disorders NZ’s work – a tattoo art auction with help from the team at Wellington Tattoo Convention. Tattoo artists from around New Zealand donated pieces of art, and with the help of auctioneer Nu Taramai, over $3,000 was raised for Rare Disorders NZ!
Global Genes Award Nomination
We are honoured to have been nominated for the Global Genes’ RARE Champions of Hope Award under the Advocacy (Foundations) category! These awards ‘celebrate, honor, and recognize champions of Hope in the rare disease community.’ There are seven categories and the winners will be announced in September.
Rare Disorders NZ is pleased to introduce our newest board member Awhina Hollis-English.
Ko Tāwhiti-nui-a-Paoa te maunga, ko Horouta te waka, ko Taharora te marae, ko Ngāti Porou te iwi.
Awhina lives in Hawkes Bay with her three children, one who has multiple medical complexities resulting from 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.
Education Highest Needs Review
Our CE is proud to represent the voice of rare disorders within the Highest Need Review Advisory Group so that the needs of children with rare disorders can be heard within this Kaupapa. The direct engagement from our webinar session has been incorporated into stakeholder views and all submission have been given recognition in this review.
This process so far has been extremely thorough with all submissions and stakeholder views included in a respectful and collaborative way. Hopefully, this can lead to an inclusive outcome that can lead to improved access to educational supports for our rare children.
Costello Medical Videos
Costello Medical – a UK-based medical communications and health economic services agency have kindly offered to produce four videos for us pro bono to support our advocacy work.
All they need from us is the raw video footage and they will put it all together for us – so this is where we need your help!
The themes for the videos will be as follows:
Equity – what does equity for rare mean to you?
Diagnosis – what difference would early diagnosis mean for your condition and your wellbeing?
Data – why is it important to collect data on rare disorders and be visible to decision makers?
Framework – what would it mean to you for the Government to implement a rare disorder framework that incorporated priority areas?
We would like to get individuals/families from the rare disorder community in NZ to take a video of themselves on their smartphone answering one of the above questions and sending us the video file. The video would only need to include a short 10-15 second soundbite.
Please firstname.lastname@example.org you would like to be involved and which theme(s) you would like to participate in. You are very welcome to be involved in all four themes if you would like!
Once we have confirmed who will be participating, we will send out instructions on how to take the video, to ensure they are all taken in a similar way and work together.
Thank you – we really appreciate your help on this exciting project!
Global conference on rare disease policy
This week the European Conference on Rare Diseases, recognised globally as the largest, patient-led rare disease policy event, is taking place from 27 June - 1 July 2022.
This event, which has been designated as an official event of the 2022 French Presidency of the Council of the European Union, provides a valuable platform for collaborative dialogue, learning and conversation, forming the groundwork to shape goal-driven rare disease policies and allow for important and innovative discussions on a national and an international level to take place.