Rare Voices Australia: May 2018 e-news
View this email in your browser


Arianna's  Story 

Meet Arianna, whose story highlights the importance of diagnosis in rare disease. Her story reiterates how many rare diseases equate to an uncertain path for patients, with access to treatments being vital over the course of a lifetime. Read more.

A Word from our Executive Officer

Hello all,

I cannot believe May is just about over. Although looking back at the busy month, I can see how it just flew by. It was of course, Budget month. For the second time, I represented RVA at the Health Portfolio Budget Briefing and I am very pleased to say it was a much-improved outcome for rare disease this year. The profile of rare disease has definitely lifted.  On Budget night, the Turnbull Government made the following rare disease announcements:  the listing of Spinraza® on the PBS for Spinal Muscular Atrophy; the signing of a landmark agreement with Medicines Australia to improve access to life saving medicines for rare disease through key reforms to the Life Saving Drugs Program; $248 million worth of support will be dedicated to clinical trial activity through highly successful rare cancers, rare diseases and unmet need clinical trials and registry program; and the genomics project, Mackenzie’s Mission, which will see $20 million provided for a pre-conception screening trial for rare and debilitating birth disorders including Spinal Muscular Atrophy, Fragile X and Cystic Fibrosis. RVA welcomes these announcements and thanks Health Minister Greg Hunt for his ongoing support of the rare disease community. Such policy transforms lives. RVA continues to strongly call for a National Rare Disease Framework to help guide continued effective investment into rare disease into the future.

Another important rare disease policy announcement in the last few days is the Newborn Bloodspot Screening National Policy Framework. Over the last 15 months, RVA has strongly called for the endorsement of the Framework, a landmark policy achievement which for the first time ensures nationally-agreed, consistent, transparent, best-practice policy to guide and strengthen newborn bloodspot screening in Australia now and in the future. This Framework has helped to future-proof newborn screening in Australia. In the absence of this policy, Australia has not added any new disease test to the NBS in the last 15 years despite major progress in diagnostics and treatment options during that time. For the best outcomes for babies and families, it is important that screening and diagnostic programs are up-to-date and accessible. The Newborn Bloodspot Screening National Policy Framework is a critical factor in this. RVA looks forward to the Framework’s implementation, the next critical step.

RVA continues to be a strong key participant in the consultation around the details of the Life Saving Drugs Program Reform. We are working hard to do all we can to ensure that the patient voice is central to these consultations. RVA prides itself on being patient-centred but also skilled and experienced in working with a range of stakeholders. This experience is holding us in good stead in these discussions.

It is important that RVA has a good understanding of the perspectives of other stakeholders. As such, I was pleased to attend the recent PharmaDispatch conference, ‘What future, the PBS?’ Rather than formal presentations, the day consisted of mediated panel discussions, expertly MC’d by PharmaDispatch’s Paul Cross. I felt this format helped highlight the different speakers’ key messages (in fact, it also sometimes highlighted the gaps or lack of consistent key messages!). I came away from the day proud of the work RVA has done over the last 18 months, better articulating our own consistent key messaging. Our RVA partners and the broader rare disease community have both helped shape these key messages, and also promote them in the broader community. This is a collaborative effort of many ‘rare voices’. The more our messages are unified, the louder we become. Thanks to you all! It is definitely making a difference.

Finally, RVA is incredibly honoured that our Fair For Rare campaign has been nominated for Best Patients’ Campaign at the Australian Patients Association's Annual Patient Awards Night 2018. We thank our RVA partners and everyone who bravely shared their story and supported the campaign - without you all, the Fair For Rare campaign wouldn’t be possible. The awards night will be held on the 31st May so we’ll keep our fingers crossed!

Until next month,

Nicole Millis
Executive Officer
Rare Voices Australia

RVA's National Rare Disease Summit
Our biennial Rare Disease Summit will take place on 16 and 17 November at Bayview Eden Melbourne. It will build on the themes and successful approaches of the Fair for Rare campaign and RVA’s Call for a National Rare Disease Framework: 6 Strategic Priorities. We will demonstrate how effective rare disease policy transforms people’s lives with a focus on collaboration to highlight opportunities for implementation and improvement. Our key focus is to ensure policy reform to create real and meaningful change for Australians living with rare disease, now and well into the future.

The Summit brings together all rare disease stakeholders - patients, clinicians, researchers, the Government and the pharmaceutical industry. Please note that this is an invitation only event - if you'd like to express your interest in attending, please do so by filling out this form.
Newborn Bloodspot Screening National Policy Framework
Rare Voices Australia (RVA) welcomes the announcement of the Newborn Bloodspot Screening National Policy Framework. Over the last 15 months, RVA has strongly called for the endorsement of the Framework by the Australian Health Ministers’ Advisory Council (AHMAC). RVA is very pleased that this has now eventuated and would particularly like to congratulate the Newborn Bloodspot Screening Working Group, chaired by Professor Craig White. This Framework was developed from their 18 months of intense work and significant stakeholder engagement. Read RVA's response in full here.

Time is ticking until the EOFY
June 30 is approaching fast! That makes it a great time for you to donate what you can and help us to fight for fair for rare. All donations to RVA are tax deductible and each of your contributions, regardless of how big or small, help you to reduce or save on your tax at the end of the financial year. Donate today!

2018 has been a positive year and we've shared a number of recent advocacy wins but there is always more work to be done. Help us to continue fighting for Fair For Rare!

Fair For Rare update
We're very excited to almost be ready to reveal our revitalised Fair For Rare campaign. The campaign wouldn't be possible without the rare disease patients who have so generously and willingly given up their time to share their personal stories. The purpose of the campaign is to generate groundswell support from people who don't have a rare disease because they're able to identity with those who have been brave enough to share their stories and experiences. A big thank you to everyone who has been involved so far and we encourage everyone to get onboard! Watch this space.

My Health Record

In mid-May, the Consumers Health Forum (CHF) issued a media release informing Australians of the date when they’ll be able to opt-out of having a My Health Record created for them. The first date that people can opt out is 16 July and ends on 15 October 2018. One month after that October date, every Australian who has chosen not to opt out, will have a My Health Record created for them. Learn more here.

Public Lecture: Genetic Discrimination in Australia
Members of Queensland Genomics Health Alliance and the Australian Centre for Health Law Research are inviting people to a public lecture with Professor Margaret Otlowski on Genetic Discrimination in Australia: A Timely Reappraisal. The public lecture will review the available evidence of genetic discrimination in Australian life insurance based on predictive genetic tests, and critically evaluates the regulatory response. Learn more about the event and register your attendance by Friday 8th June 2018 via this link.

Upcoming events

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

Submit to RVA June 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: Please 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 June 2018 edition of RVA News is 8th June 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.
Become a Partner
Make a Tax Deductible Donation

This email was sent to <<Email Address>>
why did I get this?    unsubscribe from this list    update subscription preferences
Rare Voices Australia · PO Box 138 · MENTONE, VIC 3194 · Australia