Joint Standing Committee on the National Disability Insurance Scheme Parliamentary Inquiry: Independent Assessments Final Report Tabled

On 19 October 2021, the Parliamentary Joint Standing Committee on the National Disability Insurance Scheme (NDIS) tabled its final report into the Parliamentary Inquiry into independent assessments. Click here to access the report.

Rare Voices Australia (RVA) welcomes the report’s six recommendations which align with the Submission lodged by RVA into the Parliamentary Inquiry. The announcement from Minister for the NDIS, Senator the Hon Linda Reynolds CSC, that independent assessments would not proceed in their proposed form, is a win for Australians living with a rare disease, as well as their families and carers.

The report’s six recommendations address:

  • Approaches to co-design and consultation
  • Broader matters of financial sustainability
  • Bulk-billed appointments with medical and allied health professionals

RVA thanks the 25 representatives from our RVA Partner organisations (rare disease support groups) who provided input during the development of RVA’s Submission. As the national peak body for Australians living with a rare disease, RVA will continue monitoring and engaging with the Government and the National Disability Insurance Agency (NDIA) to ensure the voices of people living with a rare disease are included in any future consultation around co-design and any proposed revised forms of assessment.

Launch of the first Parliamentary Friends of People with Rare and Undiagnosed Diseases in Western Australia

Rare Voices Australia (RVA) was proud to attend the official launch of the first Parliamentary Friends of People with Rare and Undiagnosed Diseases in Western Australia (WA) on 19 October.

The Parliamentary Friendship Group sees the Hon Matthew Swinbourn MLC, Hon Donna Faragher MLC and Hon Stephen Pratt MLC come together to increase awareness about rare and undiagnosed diseases in WA.

We thank the WA Premier, the Hon Mark McGowan, and Leader of the Opposition, the Hon Mia Davies MLA, for attending the event and for their support of Western Australians living with a rare disease, as well as their families and carers. RVA also thanks Mr Swinbourn for championing the formation of this Parliamentary Friendship Group and for his ongoing support of people living with a rare disease. Additionally, we thank RVA’s Deputy Chair, Kane Blackman, Clin/Prof Gareth Baynam, a member of our Scientific and Medical Advisory Committee, and Andrew Bannister, RVA supporter and Rare Disease Day champion, for the key roles they have played in the formation of the Parliamentary Friendship Group. You can listen to the speech that Kane delivered at the launch event here.

RVA looks forward to working with the Parliamentary Friendship Group on the issues that impact those living with a rare disease, their families and carers, including WA’s response to the priorities, actions and implementation steps outlined in the National Strategic Action Plan for Rare Diseases.

Pharmaceutical Benefits Advisory Committee (PBAC) Outcomes and Rare Diseases – September 2021 Meeting

A key Action of the National Strategic Action Plan for Rare Diseases (the Action Plan) is:

Action 2.4.3: Ensure people living with a rare disease have equitable access to medicines with demonstrated clinical benefit for a rare disease, including those that are already funded for another condition.

Many medicines that clinicians believe would be beneficial for a number of rare diseases, are currently only funded (listed) on the Pharmaceutical Benefits Scheme (PBS) for more common conditions. Due to the inherent very small patient numbers of rare diseases and lack of data, there is often no commercial incentive for companies to seek PBS listings for additional conditions (indications). This has resulted in people living with a rare disease having uncertain, inequitable or simply no access to such treatments recommended by their clinicians. Rare Voices Australia (RVA) has been advocating strongly for some time on this systemic rare disease issue to industry, the Therapeutic Goods Administration (TGA), the Pharmaceutical Benefits Advisory Committee (PBAC), the Office of Health Technology Assessment (OHTA – Department of Health) and the Federal Minister for Health’s office.

RVA welcomes the PBAC recommendation to change all listings for brands of rituximab to Unrestricted Benefits listings. Rituximab is a medicine often prescribed as part of the treatment for a number of rare diseases. This listing change will ensure that people living with a rare disease have equitable access to rituximab when it is prescribed by their clinician.

The PBAC noted the “numerous enquiries/requests in previous years” to extend the listing of rituximab. RVA is aware that RVA Partner, Myasthenia Alliance Australia (MAA), was behind many of these enquiries/requests. RVA would like to acknowledge and congratulate MAA President, Susan White, who has been such a tireless and effective advocate on this issue on behalf of her community and the broader rare disease sector. It has been a privilege for RVA to advocate alongside Susan on this issue for the last two years. Susan effectively partnered with RVA and was able to successfully engage all key stakeholders with her authentic credibility, her polite persistence and her measured yet firm manner. Her advocacy style helped encourage other key stakeholders’ willingness to continue to problem solve regarding this critical rare disease issue.

Systemic change and access to repurposed medicines for rare diseases

Whilst this PBAC recommendation does not resolve the systemic rare disease challenge of access to repurposed medicines for all medicines, it is an important and encouraging milestone. This PBAC recommendation highlights potential drivers and settings to enable such solutions – in the case of rituximab, the removal of the reference (original) brand; and current government policy encouraging the adoption of biosimilars. RVA looks forward to continuing its advocacy with key stakeholders and decision-makers to identify more drivers and policy settings to facilitate more solutions to improve access to repurposed medicines for people living with a rare disease.

Timely and equitable access to emerging technologies

Another key action identified in the Action Plan is:

Action 2.4.1: Develop policy that supports people living with a rare disease to have timely and equitable access to new and emerging health technologies.

RVA welcomes the positive PBAC recommendation of a gene therapy for spinal muscular atrophy (SMA). This recommendation is an encouraging step forward in implementing the Action Plan’s focus on timely and equitable access to new and emerging health technologies such as gene therapy. While it is important to note that pricing negotiations are yet to occur and that pricing for innovative treatments is often challenging, we congratulate RVA Partner, SMA Australia, and their community for their advocacy efforts in achieving this positive recommendation.

RVA will continue its advocacy with key stakeholders and decision-makers to identify and facilitate more timely and equitable access to emerging technologies in the future.

New Parliamentary Senate Inquiry into Current Scheme Implementation and Forecasting for the National Disability Insurance Scheme

The Joint Standing Committee on the National Disability Insurance Scheme (NDIS) (the Committee) is conducting a new inquiry into the current Scheme implementation and forecasting for the NDIS. The Committee will focus on how the NDIS is implemented and funded, and what supports are or should be available for people with disability in addition to the NDIS.

The Committee has set two closing dates for submissions:

29 October 2021: any submissions received before this deadline will be considered if the Committee releases an interim report in late 2021.

28 February 2022: any submissions received before this deadline will be considered in the Committee’s final report.

Note: submissions received by 29 October may also be considered in the final report.

Click here to read the Terms of Reference.

Rare Voices Australia (RVA) has contacted the Committee Secretary who reassured RVA that stakeholders are welcome to lodge a submission ahead of the deadline that is most convenient for them. Interim reports generally contain few, if any recommendations and are often quite short. Stakeholders are welcome to lodge a short submission this year before organising a more detailed supplementary submission next year.

RVA’s advocacy

RVA will lodge a submission to meet both deadlines and we encourage those living with a rare disease who are NDIS participants, or whose community members are NDIS participants, to lodge a submission. This will ensure that the voices of people living with a rare disease, as well as their families and carers, are heard.

RVA’s 29 October 2021 submission will align with the National Strategic Action Plan for Rare Diseases (the Action Plan), which was informed by an extensive multi-stakeholder consultation process, as well as the Disability & Rare Disease: Towards Person Centred Care for Australians with Rare Diseases (Disability & Rare Disease) report. In developing the report, consultations with all stakeholders in the rare disease sector were held and a nationwide survey of people living with a rare disease and their carers was conducted. The Disability & Rare Disease report also informed the Action Plan.

We will engage with our RVA Partner organisations in December 2021 to inform RVA’s more detailed submission for the 28 February 2022 deadline.

RVA Partner organisations that are looking to lodge a submission ahead of the 29 October 2021 deadline are encouraged to reach out to RVA for mentorship support: education@rarevoices.org.au

Why this consultation is important for people living with a rare disease

There is clear cross over between many rare diseases and disability. Aspects of the National Strategic Action Plan for Rare Diseases (the Action Plan) specifically address the NDIS and the arbitrary and unhelpful line that is often drawn between medical issues and disability. In particular, the Action Plan highlights the need for coordinated and integrated care (see Appendix 1).

Since 2012, RVA has consistently highlighted the impact of disability on the lives of people living with a rare disease. In 2019, RVA commissioned the McKell Institute to deliver the report, Disability & Rare Disease.

Making a submission

Submissions should include the name of the inquiry: Inquiry into the future of the NDIS.

The Committee is seeking written submissions in electronic form submitted online or sent by email to ndis.sen@aph.gov.au as an attached Adobe PDF or MS Word document. Alternatively, written submissions may be sent to:

Joint Standing Committee on the National Disability Insurance Scheme
PO Box 6100
Parliament House
Canberra ACT 2600

Australian Government Announces Five Year Strategic Agreements with the Medicines Industry

On 7 September 2021, the Australian Government announced two new Strategic Agreements with Medicines Australia and the Generic and Biosimilar Medicines Association (GBMA). The agreements will be in place from 1 July 2022 to 30 June 2027.

The agreements contain a comprehensive package of reforms that aim to ensure Australians gain access to transformative medical advancements developed around the world, while protecting the supply of medicines that Australians use daily. These agreements will further improve and safeguard access to new medicines in a range of ways including through:

  • Equitable and sustainable access to the most effective medicines, including highly specialised, expensive and at times lifechanging medicines, through the Pharmaceutical Benefits Scheme (PBS).
  • An Enhanced Consumer Engagement Process to facilitate enhanced consumer and patient engagement through the Pharmaceutical Benefits Advisory Committee (PBAC) assessment process.
  • Continuous improvement of health technology assessment (HTA) processes for listing new medicines on the PBS to ensure they keep pace with the rapid advancement of health technology, remain world class and keep pace with rapid advances in medicine enabling them to be marketed and funded in Australia as they emerge.
  • A new Medicines Supply Security Guarantee which will bolster medicine supply to Australian patients.

The Department of Health has developed the following fact sheets to expand on key elements of the agreements:

What these new agreements mean for Australians living with a rare disease

The new agreements detail commitments to supplying affordable medicines, earlier patient involvement in PBAC processes and the first independent review of Australia’s HTA system in almost 30 years. HTA encapsulates a range of processes and mechanisms that use scientific evidence to assess the quality, safety, efficacy and cost effectiveness of new medicines and technologies before they are made available in Australia (for example, medicines being funded under the PBS).

The HTA review will primarily be a technical review focused on HTA policy and methods. It will run parallel with the higher level review of the National Medicines Policy and be informed by submissions to the Parliamentary Inquiry (the Inquiry) into approval processes for new drugs and novel medical technologies in Australia that was conducted by the House of Representatives Standing Committee on Health, Aged Care and Sport. The HTA review will also be informed by any recommendations arising from the Inquiry. This is encouraging for the rare disease sector. We know one of the drivers for the Inquiry was the National Strategic Action Plan for Rare Diseases (the Action Plan). The rare disease sector strongly contributed to this Inquiry. It is pleasing to see the continued impact of this in these complementary reviews, and the continued emphasis on rare diseases. Rare diseases are specifically referenced in the Department of Health’s HTA review fact sheet. The detail within these new agreements generally align with priorities identified in the Action Plan. As the peak national body for Australians living with a rare disease, Rare Voices Australia (RVA) looks forward to utilizing our extensive HTA experience as we engage in the HTA review and contribute to the co-design of the Enhanced Consumers Engagement Process.   

Recap: Health Technology Assessment Consumer Consultative Committee’s ‘Making Connections’ Symposium

Rare Voices Australia (RVA) staff were pleased to attend the Health Technology Assessment Consumer Consultative Committee’s (HTA CCC) ‘Making Connections’ Symposium (the Symposium) on 23 September. The Symposium brought together consumers and consumer organisations with an interest in engaging with health technology processes to build connections, share experiences and receive updates about these processes. RVA’s Chief Executive Officer, Nicole Millis, also attended in the capacity of her role as the ministerially appointed consumer nominee on the Life Saving Drugs Program Expert Panel.

In line with the National Strategic Action Plan for Rare Diseases (the Action Plan), the Symposium emphasized the centrality of consumers/patients in Health Technology Assessment (HTA). This was highlighted in the opening address by Adriana Platona, the First Assistant Secretary of the Department of Health. The Symposium consisted of two plenary sessions. The first focused on genomic testing and cell and gene therapies; the second concentrated on the National Medicines Policy Review. Other sessions included a focus on Real World Evidence and an update on the work of the Consumer Evidence and Engagement Unit. Attendees also heard from the Pharmaceutical Benefits Advisory Committee (PBAC) and Medical Services Advisory Committee (MSAC) Chairs.

Additionally, breakout sessions included consumer HTA experiences. Congratulations to Naomi Ford from RVA Partner, XLH Australia, as well as Julie Cini from RVA Partner, Spinal Muscular Atrophy (SMA) Australia, who presented during these sessions and shared their learnings. Both RVA Partner organisations have previously utilized RVA’s mentorship support and guidance in their HTA experiences. Thank you to Naomi who clearly highlighted the importance of this informed support in contributing to the progress the XLH Australia community achieved. In RVA’s experience, rare disease group leaders with a working understanding of HTA policy are much better positioned to provide well-informed support to their community.

RVA congratulates the HTA CCC led by Jo Watson and Sally Wortley for this successful Symposium. We look forward to continuing our ongoing work in rare disease HTA through RVA’s successful mentorship program and our broader advocacy work with the Consumer Evidence and Engagement Unit and the Department of Health as we continue to actively contribute to key HTA policy reviews; the House of Representatives Standing Committee on Health, Aged Care and Sport’s Parliamentary Inquiry into approval processes for new drugs and novel medical technologies in Australia, the high level National Medicines Policy Review and the recently announced future independent HTA review – a technical review on policy and methods.

Rare Voices Australia is Hiring: Operations and Compliance Manager

RVA is looking for an Operations and Compliance Manager ​to join our passionate and dedicated team! RVA is Australia’s national non profit peak body, representing people who live with a rare disease. We are dedicated to working with all stakeholders to drive the best outcomes for Australians living with a rare disease.

The successful candidate will play a key role in identifying and facilitating the necessary operational compliance systems for the organisation. The Operations and Compliance Manager will engage with RVA’s Chief Executive Officer and staff to support operational compliance administration.

Download this PDF for the full position description. Applications close at 5pm (AEDT) on 4 October 2021.

Rare Voices Australia is Hiring: Project Officer – Mental Health and Wellbeing

RVA is looking for a Project Officer – Mental Health and Wellbeing ​to join our passionate and dedicated team! RVA is Australia’s national non profit peak body, representing people who live with a rare disease. We are dedicated to working with all stakeholders to drive the best outcomes for Australians living with a rare disease.

The Project Officer – Mental Health and Wellbeing will play a key role in delivering the RArEST (Rare Awareness, Education, Support and Training) Project. The RArEST Project will contribute to the collaborative implementation of the National Strategic Action Plan for Rare Diseases. The successful candidate will work alongside the Rare Voices Australia team to identify the unmet needs of Australians living with a rare disease. The Project Officer – Mental Health and Wellbeing will help to research, design and deliver customised and evidence-based mental health and wellbeing resources to a range of stakeholders in the rare disease community.

Download this PDF for the full position description. Applications close at 5pm (AEDT) on 4 October 2021.

Public Consultation Open: National Disability Insurance Scheme Legislation Reforms

The Australian Government is proposing changes to National Disability Insurance Scheme (NDIS) legislation with the goal to make processes easier and better for NDIS participants. The proposed changes are informed by the outcomes of the 2019 independent review of NDIS legislation, which was conducted by David Tune AO PSM. The Government is engaging in a public consultation to gather feedback about the legislation reforms.

The proposed Bill

The proposed Bill will make amendments to the NDIS Act and Rules, giving effect to 14 of the Tune recommendations, in whole or in part. A key recommendation is the introduction of a Participant Service Guarantee to set standards for how long NDIS processes (e.g. approving or amending an NDIS Plan) will take and how the National Disability Insurance Agency (NDIA) will work with those living with a disability. Read more via the Department of Social Services’ Engage website.

Public consultation process – open until midnight, 7 October 2021

The Government is accepting feedback and submissions regarding the legislation reforms until midnight, 7 October 2021. Click here to learn how you can register for one of the public consultation briefing sessions and here for how you can make a submission.

Rare Voices Australia (RVA) has written to the Minister for the NDIS and key Opposition spokespeople to express concern that the length of the consultation period is inadequate for such a significant consultation. We have requested that the consultation period be extended and will inform you of the outcome of this request.

RVA will lodge a submission and we encourage those living with a rare disease who are NDIS participants, or whose community members are NDIS participants, to lodge a submission. This will ensure that the voices of people living with a rare disease, as well as their families and carers, are heard.

RVA Partner organisations that are looking to lodge a submission into this public consultation are encouraged to reach out to RVA for mentorship support: communications@rarevoices.org.au

Why this consultation is important for people living with a rare disease

There is clear cross over between many rare diseases and disability. Aspects of the National Strategic Action Plan for Rare Diseases (the Action Plan) specifically address the NDIS and the arbitrary and unhelpful line that is often drawn between medical issues and disability. In particular, the Action Plan highlights the need for coordinated and integrated care (see Appendix 1).

Since 2012, RVA has consistently highlighted the impact of disability on the lives of people living with a rare disease. In 2019, RVA commissioned the McKell Institute to deliver a white paper, Disability & Rare Disease: Towards Person Centred Care for Australians with Rare Diseases, which informed the Action Plan.

Time Sensitive RVA Partner Opportunity: Influence Research and Interact with the Global Rare Disease Community

The EURORDIS Rare Barometer Project is developing a global survey on diagnosis. EURORDIS is working with Rare Voices Australia (RVA) to include Australian perspectives in this work. EURORDIS is keen to talk to Australian rare disease organisation leaders to help them develop the final questionnaire. 

The Project Team has decided to pursue a different approach to the methodology. Instead of conducting one-to-one interviews, they will invite patient advocates to a live online community where they can take part in individual activities and collective discussions with other participants.  

The online community will take place from 20 – 24 September. And include patient advocates from Europe as well as the identified target countries, which include Australia. Participants will take part in 15-20 minutes of activity each day for five days on an online platform where new questions will be posted, and new discussions will be assigned each day by a moderator.

Participants are asked to pre-register by filling in this pre-registration form no later than 16 September.

The online platform will be facilitated by an independent research institute (OpinionWay Healthcare). Participants can answer questions by posting a text, image or video. During this phase, there are only limited places available on the platform, and participants will be selected based on the study criteria by EURORDIS.

RVA staff have preregistered but were also keen to open up this opportunity to RVA Partner organisations with an interest in diagnosis and/or informing research design.