Rare Voices Australia: November 2018 e-news
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Vince's Story 

Vince's story highlights the role of diagnosis in rare disease and the reality that there is more work to be done to ensure Australian rare disease patients have equitable access to treatment and services. Read more.

A Word from our Chief Executive Officer

Hi everyone,

A lot can happen in a month! The Australian Government has committed to a National Rare Disease Framework!! Right now, I feel like I have the best job in the country! (To be honest, I feel like a bit of a rockstar!)

While opening our recent National Rare Disease Summit, Minister Hunt announced the Government’s commitment to a National Rare Disease Framework, the development of which is to be led by RVA. It is a truly amazing and momentous policy decision. The very first time I met Minister Hunt, he told me that he was a believer in systemic change. This is arguably the biggest systemic change in Australia for rare disease since… well, ever. Since taking over the Health portfolio, Minister Hunt has certainly been a champion for rare disease in the areas of access to rare disease treatments including the Life Saving Drugs Program Reform, the Medical Research Future Fund’s rare cancers and rare diseases calls, investment in genomic medicine, the endorsement of National Newborn Bloodspot Screening Guidelines and more. While at RVA, we say that such effective rare disease policy transforms patients’ lives, Minister Hunt’s commitment now to a National Rare Disease Framework is his real legacy - a commitment to prioritising Fair for Rare now and in the future. 

RVA has been leading the call for a National Rare Disease Framework since the organisation’s inception in 2012. I would like to personally thank the whole RVA team, the Board and staff, the Scientific & Medical Advisory  Committee, our Round Table of Companies and especially and most importantly, our RVA Partners – rare disease patient groups that not only provide support to their patient communities on the ground but have also guided and contributed to our Fair for Rare campaign and the broader rare disease advocacy efforts.

Last year, as part of the Fair for Rare campaign, RVA developed our Call for a National Rare Disease Framework: 6 Strategic Priorities. The priorities were very much built on previous consultation and work done by RVA through our national roadshow and of course, the Communique to progress a national rare disease plan, which was endorsed at the first RVA National Rare Disease Summit held three years ago. This document was very much a collaborative effort and included key input and feedback from multiple stakeholders around the 6 Strategic Priorities: diagnosis, access to treatments, coordinated care, access to services, data collection and research.

So what’s next? RVA will build on our work to date and travel around the country holding a range of consultations, some open and some targeted, to put together a quality Framework report with effective recommendations. As always, RVA will collaborate with all stakeholders, especially our RVA Partner organisations. Initial planning around an advisory committee and a consultation schedule has already begun. More information will be shared soon.

The Summit also provided a wonderful opportunity to start this Framework consultation and collaboration. The themes of the Summit included the 6 Strategic Priorities, but also incorporated a focus on mental health/wellness, the youth perspective and supporting the rare disease workforce. All of these elements will need to be included in a quality rare disease Framework. Thank you to all involved in the Summit and all who attended. It was an amazing event. I was so pleased how everyone engaged with each other and actively participated. I was actually blown away by the level of enthusiasm, support and collaboration in the room.

Now that we are all inspired by the Summit, let us all remain solution-focused. Let’s look for both opportunities to collaborate and common ground. Let’s not get too bogged down by issues and challenges. With the Government’s support of a National Rare Disease Framework, we have the best opportunity ever to make a lasting difference to the rare disease community, now and in the future. We can all now be part of something bigger and contribute to the detailed development of our country’s National Rare Disease Framework. RVA wants to engage and collaborate. Look at what we have achieved together – and now, the real work begins!

Until next month,

Nicole Millis
Chief Executive Officer
Rare Voices Australia
Government supports National Rare Disease Framework

RVA welcomes the Government’s support of a National Rare Disease Framework. The Hon. Greg Hunt MP, Minister for Health, made the announcement as part of the official opening of RVA’s biennial 2018 National Rare Disease Summit, which was held on 16 and 17 November. The Morrison Government will commission RVA to work with all stakeholders to deliver the National Rare Disease Framework with $154,000 in grant funding. The Minister acknowledged the work of Rare Voices Australia which has “made such a difference… a driving force for change.” We thank everyone who sent a letter to their local MP asking our leaders to prioritise rare disease as part of our Fair for Rare campaign. Look at what we’ve achieved together! You can read RVA’s full response here.

2018 National Rare Disease Summit

A big thank you to everyone who made our National Rare Disease Summit a success! We take this opportunity to once again thank our event sponsors as without your support, the event wouldn't be possible. RVA also thanks the Hon. Catherine King MP, Shadow Minister for Health & Medicare, for joining us in person at the Summit and announcing Labor's support of a National Rare Disease Framework, which they have formally committed to in their draft Health Policy. The feedback we've received from all stakeholders has been overwhelmingly positive - from comments about the power of all stakeholders in the room working together towards a common purpose to reflections on the fostering of true connection over the course of the two days. We are currently organising for the presentations/slides/videos from the Summit to be made available to those who attended (where we have permission from presenters to share them). We're also in the process of putting together a selection of content that will be made available to RVA partners in 2019. Moving forward, RVA will be organising a National Rare Disease Summit every two years.

APEC Rare Disease Action Plan
The Asia-Pacific Economic Cooperation (APEC) has launched a Rare Disease Action Plan to provide APEC Economies with a framework for policy action to tackle the challenges of rare diseases.  RVA partner organisations as well as RVA’s Scientific & Medical Advisory Committee provided input into the Action Plan during the consultation phase. The Action Plan outlines 10 key areas for action in relation to rare disease including raising public awareness, the better use of technology to enhance the purposeful use of data in clinical decision-making and healthcare system design to ensure that patients are diagnosed and cared for in a timely manner. The Action Plan calls for APEC’s 21-member economies to improve the economic and social inclusion of people living with rare disease, with clear targets, by 2025. View the Rare Disease Network website via this link.
Galafold® (migalastat) added to the LSDP 
RVA welcomes the Hon. Greg Hunt MP's announcement earlier this month that Galafold® (migalastat) has been added to the Federal Government’s Life Saving Drugs Program (LSDP) for the treatment of Fabry disease. We congratulate RVA partner, Fabry Australia and their community, for the advocacy work that has gone into this positive outcome. Galafold® has the potential to greatly reduce the burden of treatment for many Fabry patients. Read Minister Hunt’s full announcement here.
Health Voices - November 2018 issue
Amy Mills, RVA's Social Media Officer, contributed a piece to the Consumer Health Forum's November Health Voices issue this month. Amy writes about valuing voices of young people living with rare disease. You can read her article here. As our recent National Rare Disease Summit demonstrated with its focus on engaging and listening to youth, RVA is committed to working with young people as we head into 2019 and beyond. You can also read the Call to Action from the Consumers Health Forum of Australia's Youth Health Forum that was held earlier this year.
Congratulations to an inspiring future rare disease advocate
RVA congratulates Gidon Goodman, one of the founding members of RVA partner Gaucher Association of Australia and New Zealand, on being one of four Australians aged between 15 to 30 who were shortlisted for the NSW entry for the Young Australian of the Year award. Gidon and his family continue to do incredible work in the rare disease space and we were thrilled to welcome Gidon to our 2018 National Rare Disease Summit as one of our panelists for our Future Rare Disease Advocates Q&A session. Gidon and his mother Karen, also participated in our Fair for Rare campaign earlier this year. Learn more about Gidon's story here.
IDFA 2019 National Conference
On 13 and 14 (Saturday and Sunday) April 2019, RVA partner, Immune Deficiencies Foundation Australia (IDFA), is hosting its National Conference in Brisbane. The Conference is open to patients with primary and secondary immune deficiencies, carers, IDFA members, healthcare professionals and stakeholders. See full details by clicking here to view the event flyer and register now for the Conference via the IDFA website.
Survey - paediatric EoE
Do you live in Australia and are the parent or carer of a child who has Eosinophilic Oesophagitis (EoE) who is between the ages of 1 month and 18 years? You are invited to participate in this research which involves completing an online survey (approximately 25 mins duration). Questions will explore quality of life and experiences in the healthcare system for paediatric EoE. Survey participants will be given an option to go into the draw to win one of five $50 gift vouchers. Find out more and take the survey here. The researchers involved in this project are from Griffith University. Ethics approval has been obtained from the Griffith University Human Research Ethics Committee (GU Ref No: 2018/120). Please contact Nicole Hannan for more information.
Second Asia Pacific Lysosomal Conference - Feb 2019
Lysosomal Diseases New Zealand has announced the second Asia Pacific Lysosomal Conference to be held in Auckland, New Zealand in February 2019. Ten years after the first meeting, the aim of the Conference is to bring together scientists, clinicians, carers and families in a unique conference format to discuss the latest advancements in treatments, therapies and management of these very complex conditions. The theme of the Conference is “Lysosomes and disease: Today and tomorrow”. View the event flyer here and click here to see the full program and to register.
Workshop - Understanding genomics: empowering patient advocates
Are you a health or patient advocate for those seeking support for a genetic condition? Have you been asked questions about genomics, but don’t feel you have the knowledge or capacity to answer? Presented by the Melbourne Genomics Health Alliance, this half-day workshop held on 4 December from 8:30am to 1:30pm AEDT, will arm you with the tools you need to support your community - including easy-to-understand information about genomics, new genomic sequencing tests and test results. A series of talks about genomics will be followed by small discussion groups featuring clinical geneticists, genetic counsellors, genome interpreters and data experts. This is an opportunity to ask your questions, including about ethical and legal issues, of those at the coalface of genomic testing in Victoria. Learn more and register here.
RVA office closure for the holidays
Please note that RVA's office will be closed for the festive season from Monday, 24 December - we'll be back in the office on Monday, 7 January 2019. We thank you for your patience and understanding throughout this period.
Become an RVA partner
RVA is busy planning for 2019. As a small not-for-profit organisation that does not receive any government funding, we rely on the support of our RVA partners to continue doing the work that we do. The more funds we have, the more we can do to fight for a fair go for those living with a rare disease. Individuals and organisations can sign-up to become an RVA partner here. In addition to advocacy, mentorship and education, RVA offers a number of other benefits to RVA partners including social media promotion, entry into our closed Facebook group which allows patient organisation leaders to share strategies and so on, rare disease policy support and more. For more information, please reach out to Sarah, our Stakeholder Engagement and Communications Officer via email to organise a phone call. Alternatively, become an RVA partner today.

Upcoming events

If your organisation has an event coming up and would like it advertised in our newsletter, please email with the details. 

Submit to RVA December edition of e-News

RVA encourages partner organisations and individuals to consider making a submission to a future RVA e-newsletter. If you have a rare disease meeting, event or announcement to share via RVA enews, please submit it to: Send a website link (preferable) or short paragraph with details such as: the title of event, date, cost, venue, details/contacts. The deadline for submissions for the December 2018 edition of RVA News is 8th December 2018.

Support Rare Voices Australia

Rare Voices Australia is a small but vibrant non-profit organisation that relies on the support of organisation partners and the community to be the unified voice of all Australians living with a rare disease. RVA's online partnership area enables individuals and organisations to join our cause. Click here for more details.
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