Refreshed National Medicines Policy (NMP) Delivered to the Australian Government

News

The Hon Mark Butler MP, Minister for Health and Aged Care, has announced the updated National Medicines Policy (NMP). The refreshed NMP is available on the Department of Health and Aged Care’s website.

RVA is currently reviewing the refreshed NMP in detail and what it may mean for Australians living with a rare disease.

As the national peak body for Australians living with a rare disease, RVA has been actively involved in the NMP Review.

RVA has contributed to the NMP Review to date in several ways, including:

First Ever Global Consensus Clinical Care Guidelines for Sanfilippo Syndrome

News

The first-ever global consensus clinical care guidelines (guidelines) for Sanfilippo Syndrome have been published. RVA Partner Sanfilippo Children’s Foundation (Australia) has collaborated with Cure Sanfilippo Foundation (United States of America), people living with Sanfilippo Syndrome and over 100 clinicians globally to develop and publish the guidelines.

Establishing consensus among medical professionals with expertise in the care of individuals with Sanfilippo on recommended care guidelines is a key step to elevating the care and support for children around the world who are living with Sanfilippo Syndrome.

How were the guidelines developed?

The guidelines were established through a multistage process to arrive at consensus recommendations, with input from more than 100 clinicians around the world with a range of specialist expertise, who have experience in the care of individuals with Sanfilippo Syndrome. Lived experience was integrated through participation of the collaborating Sanfilippo advocacy organisations.

What’s in the guidelines?

Accessible to anyone providing medical care, rehabilitative care, or support services for individuals with Sanfilippo Syndrome globally, the guidelines provide evidence-based, expert-led recommendations and guidance on Sanfilippo Syndrome-specific care management and monitoring of disease-related changes. Additionally, the guidelines are a practical resource for families to become well-informed advocates and can be shared with local care teams, who may not have previous experience with this rare disease. 

Topics addressed in the guidelines include: 

  • Symptoms that should raise suspicion for the diagnosis of Sanfilippo Syndrome
  • Methods of establishing the diagnosis
  • Evaluating, monitoring and managing neurological, gastrointestinal, airway, musculoskeletal and the many other complications that may develop
  • Special focus on the evaluation of unexplained pain and distress
  • Rehabilitative therapies
  • Support services 

Download a copy of the guidelines here.

Visit the Sanfilippo Children’s Foundation website for more information about the guidelines.

What are the next steps?

Cure Sanfilippo Foundation and Sanfilippo Children’s Foundation, in collaboration with international Sanfilippo Syndrome organisations, are developing a streamlined version of the guidelines for easy reference and access in multiple languages.

Rare Voices Australia Statement: Budget October 2022-23

News

On Tuesday, 25 October 2022 the Hon Dr Jim Chalmers MP (Treasurer of Australia) announced the Albanese Government’s October 2022-23 Federal Budget. In a media release, the Hon Mark Butler MP, Minister for Health and Aged Care, stated, “This is a Budget for the health of every Australian.”

As the national peak body for Australians living with a rare disease, Rare Voices Australia (RVA) attended the Department of Health and Aged Care’s Portfolio Briefing Webinar Presentation and Q&A with Minister Butler; the Hon Anika Wells MP (Minister for Aged Care, Minister for Sport); the Hon Ged Kearney MP (Assistant Minister for Health and Aged Care); the Hon Emma McBride MP (Assistant Minister for Mental Health and Suicide Prevention, Assistant Minister for Rural and Regional Health); and Senator the Hon Malarndirri McCarthy (Assistant Minister for Indigenous Australians, Assistant Minister for Indigenous Health). The session was facilitated by Prof Brendan Murphy AC, Secretary of the Department of Health and Aged Care.

Click here to read RVA’s Full Statement where we have highlighted some areas of the Budget we believe are most relevant to Australians living with a rare disease and the rare disease sector.

Parliamentary Event: Parliamentary Friends of People with Rare and Undiagnosed Diseases in Western Australia

News

Rare Voices Australia’s (RVA) Chief Executive Officer, Nicole Millis, was pleased to attend a Parliamentary Event in Western Australia (WA) on 19 October hosted by the Parliamentary Friends of People with Rare and Undiagnosed Diseases in WA (the Parliamentary Friends Group). Clin/Prof Gareth Baynam, who is a member of RVA’s Scientific and Medical Advisory Committee (SMAC), spoke at the event. RVA’s Deputy Chair, Kane Blackman, Dr Kristen Nowak (SMAC) and RVA Ambassador, Andrew Bannister, also attended.

The Parliamentary Event marked the one-year anniversary of the official launch of WA’s Parliamentary Friends Group. The event gave clinical and support organisations within the rare disease community the opportunity to showcase who they are and what they do to Members of Parliament to increase awareness of rare diseases. Thank you to the Members of Parliament who attended, including the Premier, the Hon Mark McGowan BA LLB MLA.

RVA also thanks Co-Convenors of the Parliamentary Friends Group, the Hon Matthew Swinbourn MLC, the Hon Donna Faragher MLC and the Hon Stephen Pratt MLC, for their ongoing support of the rare disease community.

Image credit: Ammon Creative

Sector-Wide Activity Scan: National Strategic Action Plan for Rare Diseases Implementation

News

The Australian Government’s National Strategic Action Plan for Rare Diseases (the Action Plan) was launched in February 2020, over two years ago. Since that time, the rare disease sector has been collaboratively progressing implementation of the Action Plan. To gauge how far we have come, Rare Voices Australia (RVA) is leading an activity scan of the sectors’ collective efforts. The aim of this work is not only to map what is being done and what has been achieved, but also to highlight any gaps in the sector’s progress and any areas where we can encourage greater collaboration and prevent duplication.  

To encourage cross-sector participation in the Action Plan activity scan, RVA have chosen a simple tool—Google Jamboards—where each stakeholder group can share current, past or forthcoming projects, initiatives, tools, resources, networks and processes. These Jamboards are active currently and will remain open until 17 October 2022. If you have progressed implementation of the Action Plan but have not received an email with information and a link to a Jamboard, please contact Falak Helwani, RVA’s Research and Evaluation Manager at [email protected] or call 0448 505 184.  

The results of this mapping exercise will demonstrate how far the sector has come and what is left to do. The results will be shared at the 2022 National Rare Disease Summit (Summit) in November and inform Summit Workshops (for in-person Summit attendees only). During these small group workshops there will be an opportunity to discuss the most urgent gaps in Action Plan implementation and who could or should be responsible for furthering progress in those specific gap areas. Participants will be asked to share any activities that were not captured via the Jamboards and encouraged to identify any obvious duplication or risk for duplicated efforts. RVA hopes this exercise will facilitate increased collaboration among stakeholders with similar objectives (if any) and guide next steps to fill the gaps for full implementation of the Action Plan.

RVA is looking forward to the outcomes of this work and to showcasing the collective efforts of the rare disease sector. Thank you to those who have already contributed to this exercise. Your involvement is appreciated.

For more information or if you have any questions about this work, please contact Falak Helwani at [email protected] or call 0448 505 184.

Rare Voices Australia Parliamentary Event – 5 September 2022

News

Rare Voices Australia (RVA) thanks everyone who attended our Parliamentary Event in Canberra today hosted by the Chair of the Standing Committee on Health, Aged Care and Sport, Dr Mike Freelander MP. We thank Dr Freelander, Member for Macarthur and Co-Chair of the Parliamentary Friends of Australians Living with Rare Diseases, and the following Parliamentarians who spoke at the event:

  • Hon Ged Kearney MP, Assistant Minister for Health and Aged Care
  • Senator the Hon Anne Ruston, Shadow Minister for Health and Aged Care
  • Dr Monique Ryan, Member for Kooyong and Co-Chair, Parliamentary Friends of Australians Living with Rare Diseases

RVA has been working with Dr Freelander to re-establish the Parliamentary Friends of Australians Living with Rare Diseases, which both he and Dr Ryan referred to at today’s event.

Special thanks to our RVA Ambassadors and the RVA Partner organisation leaders who also joined us. The Parliamentary Event provided an opportunity for the rare disease sector to continue fostering strong relationships with all political parties and to welcome newly elected members of the 47th Federal Parliament, while introducing them to key rare disease issues.

In addition to celebrating RVA’s 10th anniversary, the event demonstrated the need for the entire rare disease sector to collaborate to ensure the ongoing successful implementation of the National Strategic Action Plan for Rare Diseases.

RVA Welcomes Equity of Access for Australians Living with Phenylketonuria (PKU)

News

RVA welcomes the announcement that the Pharmaceutical Benefits Scheme (PBS) listed treatment for Phenylketonuria (PKU) will now be accessible to all Australians living with PKU who are responsive to treatment. RVA provided a consumer comment regarding this treatment that, in line with the National Strategic Action Plan for Rare Diseases (the Action Plan), highlights the importance of equitable access to treatments for adults who have lifelong conditions.

Congratulations to RVA Partners, the Metabolic Dietary Disorders Association (MDDA) and PKU Association of NSW, and their communities for advocating for the best outcomes for Australians living with PKU.

RVA also acknowledges the disappointment that the other treatment for PKU considered for listing on the PBS by the Pharmaceutical Benefits Advisory Committee (PBAC) at the July 2022 meeting was rejected. RVA provided a consumer comment for this treatment that highlighted the importance of new and innovative therapies becoming available to Australians living with a rare condition as soon as possible, which aligns with the Action Plan.

RVA provides consumer comments to the PBAC regarding systemic issues for Australia’s rare disease community.

National Medicines Policy Review Resumes

News

Rare Voices Australia (RVA) welcomes the resumption of the National Medicines Policy (NMP) Review. Finalisation of the NMP Review was extended until after the Federal Election in May 2022. The Hon Mark Butler MP, Minister for Health and Aged Care, has reappointed Professor Michael Kidd AM as the sole reviewer to complete the Review and provide a final report to Government in late 2022.

Summary Stakeholder Consultation Report

The NMP Review’s Expert Advisory Committee (the Committee) has released a report that provides a summary of the consultation findings to date, including the Committee’s commentary on how stakeholder feedback informed the development of the draft 2022 NMP. RVA is currently reviewing the report and will consider any potential implications for Australians living with a rare disease. We welcome the Committee’s recommendation that the NMP aligns with other policies, strategies and agreements, including the National Strategic Action Plan for Rare Diseases (the Action Plan). RVA also welcomes the Committee’s acknowledgement of the importance of person-centredness (‘Person-centred’ is a foundation principle of the Action Plan).

Stakeholders invited to provide feedback on the revised National Medicines Policy draft

Stakeholders are invited to provide feedback via a range of channels, including a consultation survey, which is open from 17 August 2022 until 27 September 2022 at 11:59pm. RVA will complete the survey as the national peak body for Australians living with a rare disease. A public stakeholder forum will be scheduled in September 2022. Professor Kidd will also engage in several open targeted consultations with key stakeholder groups.

Learn more and download the revised NMP draft and supporting documents here.

How RVA has contributed to the National Medicines Policy Review to date

RVA has contributed to the NMP Review to date in several ways, including:

Webinar for Health Consumers and Consumer Advocates with the Hon Mark Butler MP: Consumers Health Forum of Australia 

News

The Consumers Health Forum of Australia (CHF) hosted a webinar on Friday 5 August with the Hon Mark Butler MP, Minister for Health and Aged Care. Rare Voices Australia’s (RVA) Chief Executive Officer, Nicole Millis, was pleased to be one of eight health consumer advocates who had the opportunity to ask the Minister a question during the webinar.

Nicole’s question drew on Action 2.1.4 of the National Strategic Action Plan for Rare Diseases:

Develop the capacity of rare disease organisations to represent and advocate for people living with a rare disease and their families.

You can watch the recording of the webinar here.

RVA has noted the significant issues with the subtitles/closed captions on the recording uploaded by CHF. To ensure that members of the rare disease community who are reliant on captions can access Minister Butler’s response to RVA’s question, we have created a transcript of the question and answer.

The below transcript begins at 30 minutes and 50 seconds.

Nicole Millis: Hi Minister. Thank you for today. I’m also on the land of the Wurundjeri people of the Kulin nation today. My question is around consumer-led health and disease organisations. They’re an important part of the health sector, particularly in the rare disease sector. Yet those organisations are often under-resourced, largely volunteer based, often reliant on volunteers who themselves are living with a rare disease, or their long-term organisational sustainability is uncertain. How will the government ensure that this person-centred part of the sector builds capacity and is strong and sustainable?

Minister Butler: Thanks Nicole. Great to see you again, [I] look forward to working with you in a new capacity. I’m in a new capacity, you’re still in the same, but really look forward to working with you. As I said, one of the things I’m doing as a new Minister is trying to get a sense of what support arrangements were in place from the former Government.

So, as you know, many others on the webinar might know, there’s a range of different arrangements that could entice this sometimes core funding, particularly with some of the peak organisations and then grant funding that can be provided for particular projects.

I know that that places a lot of pressure on organisations that are already not well-resourced to have to sort of continue to apply for grant after grant after grant. That can take you away from doing your core work if you’re constantly involved in putting in these applications.

I don’t yet have a good, good and complete sense of where the department is up to with its arrangements. You know, frankly I think some of those funding support arrangements were cut after we lost government last time. So, you know I know a number of organisations I’ve been dealing with had their funding removed. We’ve already been able to reinstate that, particularly in areas that we’re very concerned about, health areas we’re very concerned about right now. But I just encourage organisations to you know, we’re at the point as a new government, where we need feedback, you know about where the gaps are. And I can talk to my department, I can read briefs. That’s no substitute though for getting direct feedback from people on the ground who are doing this really important work. So that’s about all I can say to you about that Nicole nine weeks in. Still pretty early days but I’m finding gaps and trying to fill them where I think it’s really important.

Nicole Millis: That’s fine and we look forward to further discussion. Thanks.

RVA Welcomes Announcement That Pharmaceutical Benefits Scheme Listed Treatment for Spinal Muscular Atrophy (SMA) Will Be Extended to Adults

News

Rare Voices Australia (RVA) welcomes the Hon Mark Butler MP’s announcement that, from 1 August 2022, the Pharmaceutical Benefits Scheme (PBS) listed treatment for spinal muscular atrophy (SMA) will be extended to adults whose symptoms appeared before 19 years of age. Read more.

Until now, there have been no treatment options available on the PBS for adults living with SMA. Advocating for access to this treatment for adults living with SMA has required much persistence and a solutions-focused approach. RVA had previously provided consumer comments regarding this treatment that highlighted the inequity of restricting access to subsets of a rare disease community, and the importance of utilising real-world evidence in Health Technology Assessment (HTA) in the absence of traditional clinical trial data. 

Congratulations to RVA Partner Spinal Muscular Atrophy Australia and their community for their dedication to advocating for the best outcomes for the SMA community!