Expressions of Interest Sought: Rare Awareness, Education, Support and Training Stakeholder Reference Group

  • Do you have lived experience of rare disease?
  • Are you actively connected with a rare disease community?
  • Would you like to contribute to an innovative rare disease awareness, education, support and training program designed to improve outcomes for all Australians living with a rare disease?

We are looking for people to join the Rare Awareness, Education, Support and Training (RArEST) Stakeholder Reference Group. The RArEST project is being led by the University of New South Wales (UNSW) in collaboration with Rare Voices Australia (RVA) and the University of Western Australia and Macquarie University.

Role description

The RArEST project is a federal government funded initiative that aims to increase awareness, education, support and training for rare diseases among health professionals and within the rare disease community. The RArEST project will progress implementation of the National Strategic Action Plan for Rare Diseases (the Action Plan). Read more about the RArEST project here.

We are inviting people to lodge an expression of interest (EOI) to join the Stakeholder Reference Group, which will help us to design what the project will deliver (e.g. guiding what rare disease awareness, education, training and support resources are developed and how they are delivered).

By bringing together this group, we hope to increase the relevance and impact of the RArEST project. Members will be supported to take part in the group and will be offered reimbursement for their time and expertise. It will be a prerequisite to complete introductory training.

We wish to hear from people who:

  • Have lived experience — personal experience of living with a rare disease or caring for someone with a rare disease.
  • Are able to influence and communicate across a broad network of individuals or organisations in the rare disease sector.
  • Can demonstrate their commitment to ensuring their input into consultative processes is representative of their community/ies and reflects community engagement.
  • Preference will be given to those who represent one of the priority populations identified in the Action Plan, which include people living with a rare disease, Aboriginal and Torres Strait Islander people, people experiencing socioeconomic hardship, people from culturally and linguistically diverse (CALD) backgrounds, people living in regional, rural or remote communities and people who are suspected of having an undiagnosed rare disease.

Those who feel they can contribute effectively are encouraged to submit an EOI to participate in the Stakeholder Reference Group, which will see them consulting and collaborating with the RArEST team. This team will include RVA representatives, rare disease clinicians and researchers and other members of the rare disease community. Selection will be based on building a diverse Stakeholder Reference Group with broadest possible representation from the rare disease community. Experience as a consumer representative is not necessary as training and support will be provided.

Participants will be asked to:

  • Share their own experiences and the experiences of the broader community they represent.
  • Provide feedback on the relevance, understanding and value of proposed projects.
  • Participate in discussions.
  • Take a person-centered approach to healthcare.
  • Act as a conduit between the RArEST project and consumers and/or community organisations to provide feedback from the Stakeholder Reference Group and seek input and advice to pass back to the group.
  • Prepare for meetings and read materials ahead of meetings.

Members of the Stakeholder Reference Group who are consumers living with a rare disease will receive an honorarium payment of $45.26 per hour for their time preparing for and attending meetings and undertaking pre-agreed training.

Translation and support services may be available. Requests are welcomed.

We are looking for people who have the capacity to dedicate approximately 40-60 hours to this project over the next 2.5 years. 

How to lodge an EOI

Please complete the online EOI form below by COB Tuesday 25 January. Questions regarding the Stakeholder Reference Group can be directed to education@rarevoices.org.au.

RArEST Stakeholder Reference Group EOI Form

Name(Required)
Please check any of the priority populations you identify with:(Required)

We are person-centred

We want the outcomes that are best for each person as an individual. We will be driven by the needs of each person and by what works best for them. We won’t impose our assumptions on people.

We will actively build and maintain partnerships

We are the voice of the sector, magnifying the voices of all stakeholders that share our goals. We recognise that achieving the transformation we want to see in Australia will require us working in partnership with stakeholders.

We aim for a unified voice

We recognise that without a unified voice, the rare disease sector cannot be successful in achieving the support for people living with a rare disease that we need. As a result, we will prioritise consultation and conversation within the sector to ensure that our voice to policymakers and the broader community is as unified as possible.

We work for equity of access and participation

We believe that equity of access and participation is vital for all people living with a rare disease in Australia. In our initiatives, we will ensure that equity is a key factor in our planning and design. And in our advocacy, the principles of equity will be foremost in our messaging.

We will equip and empower

We aim to equip and empower organisations and people to help them advocate and work for themselves.

We are solutions and results oriented

We want to see results for people living with a rare disease in Australia. We are not going to be bound to theoretical models. We will adopt what works and will test our initiatives against the results they deliver.

We are credible

We recognise that our effectiveness is only as good as our credibility and trustworthiness. We will ensure that all of our actions and words build our credibility, rather than erode it. We recognise that trust takes a long time to build but can be quickly lost.

We will adopt a systemic focus

We are uniquely positioned to engage in systemic advocacy. Effective reform to systems and processes must be prioritised to reduce individuals and groups repeatedly facing common challenges. This is vital to achieving sustainable change at scale.

Systemic National Disability Insurance Scheme Advocacy: National Disability Insurance Agency Roundtable

Rare Voices Australia (RVA) has continued to actively advocate for systemic improvements to the National Disability Insurance Scheme (NDIS), to ensure the program can better respond to the unique needs and challenges of people living with a disability caused by rare disease. Through this sustained and ongoing advocacy, RVA was invited to participate in an interactive virtual roundtable meeting with the National Disability Insurance Agency (NDIA) and RVA Partner organisations on 1 December. The NDIA limited the number of places available at the workshop to ensure it remained interactive.

The following RVA Partners joined RVA at the roundtable:

  • Angelman Support Association Australia
  • Battens Disease Support and Research Association
  • Fragile X Association Australia
  • Huntington’s NSW and ACT
  • Mito Foundation
  • Muscular Dystrophy Foundation Australian
  • SCN2A

The two-hour session provided a platform for the NDIA to hear directly from the rare disease community, so the NDIA can be better informed about the functional impacts and support requirements of people with rare diseases who seek to access NDIS services. The roundtable covered three broad areas of the NDIS for discussion, which had been identified as key pain points for the rare disease community:

  • Access Pathway
  • Planning
  • Assistive Technology

These focus areas align with Implementation step 2.1.2.2 of the National Strategic Action Plan for Rare Diseases, which prioritises the need to fast track access to the NDIS and ensure participants are able to secure the appropriate support services to meet the evolving needs of people with rare diseases and their families.

For RVA and attending RVA Partners, the session enabled a robust discussion with the administrators of the NDIS about the unique challenges in eligibility, access and planning experienced by rare disease applicants and participants. While RVA Partners represented a broad range of rare diseases, insights into their communities’ lived experiences with the NDIS clearly demonstrated the extensive policy issues that currently exist within the system, that impact all people with rare diseases. To select participants who could represent a broad range of rare diseases, RVA sought Expressions of Interest (EOI) from RVA Partner organisation leaders. RVA then considered:

  • The EOI lodged
  • Broad representation of the rare disease community
  • Diverse range of experiences with the NDIS
  • Demonstrated alignment with RVA’s advocacy approach
  • Willingness and ability to represent the broader rare disease community

Although this roundtable session was the first-of-its-kind to be held between the rare disease sector and the NDIA, RVA was able to raise the need for creating a fit-for-purpose mechanism to facilitate ongoing dialogue between the two groups; and we expect similar sessions may be held during 2022. RVA thanks the attending RVA Partner organisations for their participation at this session and their commitment to improving the NDIS experience for people living with rare diseases. We look forward to keeping all stakeholders updated on the outcomes of this NDIA roundtable and communicating with RVA Partner organisations regarding potential future opportunities to contribute to this systemic advocacy.

Board Positions Available: Rare Voices Australia

Rare Voices Australia (RVA) is seeking non-executive directors to join the RVA Board. Successful applicants will bring experience, skills and capabilities that contribute to the governance of RVA and enhance its advocacy for the best outcomes for Australians living with a rare disease.

RVA remains committed to board diversity and inclusion. Specific experience in fundraising or political/government engagement would be viewed favourably but is not essential. Applicants should complete and send an Expression of Interest form and their CV to Nicole Millis, RVA’s Chief Executive Officer: nicole.millis@rarevoices.org.au. Board meetings are held via teleconference every two months and involve up to two hours of pre-reading. A duty statement for RVA Directors can be provided on request.

Our high calibre RVA Board oversees the organisation’s strategic purpose and direction and comprises dedicated and passionate volunteers, including those with lived experience. The board consists of those with a professional skill set relevant to the organisation including legal, financial, medical and scientific expertise. You can find more information about RVA’s governance framework and reporting here.

RVA is the national peak body for Australians living with a rare disease. Our person-centred focus sees us working with all key stakeholders including people living with a rare disease, governments, key peak bodies, researchers, clinicians and industry. You can read more about RVA’s purpose, including our Vision and Mission here.

The Australian Government commissioned RVA to lead the collaborative development of the National Strategic Action Plan for Rare Diseases (the Action Plan) in 2018. The Action Plan was launched in 2020 with bipartisan support after an extensive multi-stakeholder consultation process. RVA is now leading the Action Plan’s collaborative implementation.

Please direct any queries you may have about these board positions to RVA’s CEO.

Update: United Nations General Assembly Resolution on Persons Living with a Rare Disease and their Families

Throughout 2021, the global rare disease community has been advocating for the United Nations General Assembly (UNGA) to adopt the Resolution on Addressing the Challenges of Persons Living with a Rare Disease and their Families (Resolution on PLWRD). This campaign has been led at a global level by the NGO Committee for Rare Diseases, Rare Diseases International (RDI) and EURORDIS – Rare Diseases Europe. Learn more and read about the significance of a UNGRA Resolution on PLWRD here.

On 10 December, RDI confirmed that on 16 December 2021, the UNGA will adopt the Resolution on PLWRD. This is a wonderful milestone for the global rare disease community!

Rare Voices Australia’s advocacy

In response to Rare Voices Australia’s (RVA) advocacy regarding the Resolution on PLWRD, we have received a letter sent on behalf of Australia’s Minister for Foreign Affairs, Senator Marise Payne, confirming that Australia plans to endorse this Resolution at the UNGA on 16 December. Click here to read the letter.

As communicated in recent RVA eNewsletters, in collaboration with RDI, RVA had reached out to Australia’s Permanent Mission to the United Nations and Senator Payne to request their support of the Resolution on PLWRD. We had also previously reached out to the Minister for Health’s office to flag the Resolution on PLWRD and request Australia’s support.

RVA thanks the Australian Government for its ongoing commitment to rare diseases in Australia and for its support of this important global initiative.

Introducing RVA’s New Online Education Portal

Since the launch of the National Strategic Action Plan for Rare Diseases (the Action Plan) in February 2020, Rare Voices Australia’s (RVA) Mentorship and Education Program has focused on Action Plan implementation and specifically, Action 2.1.4:

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

To complement the workshops, webinars and customised mentoring support provided to RVA Partner organisations, RVA is launching a new Online Education Portal. Available 24/7, the site will contain a number of open and partner only resources that will be added to over time. We are launching with two courses:

  • Amplifying Advocacy Using the National Strategic Action Plan for Rare Diseases
  • How to Illuminate Landmarks to Celebrate Awareness Days

These courses are both partner only access and are available to RVA Partners and the RVA Round Table of Companies. You can learn more about the benefits of becoming an RVA Partner here. Find out more about the RVA Round Table of Companies here. Open access resources will be added in 2022.

Accessing RVA’s Online Education Portal

If you are an RVA Partner or an RVA Round Table of Companies representative, after you register, you will gain access to both available courses after RVA approves your application (you’ll receive a confirmation email). We encourage all team members within your organisation to register and complete the trainings. Once you have successfully completed a course, you will receive a certificate. Register now.

You can direct any queries about RVA’s Education Program to Louise Healy, our Education and Advocacy Manager: education@rarevoices.org.au

If you experience any technical difficulties when trying to access the Online Education Portal, please reach out to Sarah Cannata, RVA’s Communications Manager: communications@rarevoices.org.au

The Hon Greg Hunt MP Announces Retirement From Politics

The Hon Greg Hunt MP has announced that he will retire from politics at the next election after more than 20 years in Federal Parliament. Watch Minister Hunt’s speech to Parliament.

Rare Voices Australia (RVA) thanks Minister Hunt for his ongoing support of Australians living with a rare disease during his time as Minister for Health and Aged Care. Minister Hunt’s leadership led to the Australian Government investing in the collaborative development of the National Strategic Action Plan for Rare Diseases, which was launched in 2020, with bipartisan support. In his speech to Parliament today, Minister Hunt touched on his dedication to people living with a rare disease. We acknowledge the many rare disease initiatives that Minister Hunt has championed, including:

  • Research investment into genomics as well as rare diseases and rare cancers via the Medical Research Future Fund (MRFF)
  • Mackenzie’s Mission
  • Reform of the Life Saving Drugs Program (LSDP)
  • Overseeing the addition of many rare disease medicines to the Pharmaceutical Benefits Scheme (PBS)
  • Leadership on newborn bloodspot screening (NBS) reform, work which is ongoing
  • His systemic approach, which has seen rare disease expertise appointed to the National Health and Medical Research Council (NHMRC) for the first time and rare disease consumer expertise appointed to the LSDP Expert Panel
  • His referral to the Standing Committee on Health, Aged Care and Sport of the Parliamentary Inquiry into approval processes for new drugs and novel medical technologies in Australia
  • His support for the ongoing review of health technology policy in line with advancements in health technology, which has resulted in achievements such as the Mitochondrial Donation Law Reform (Maeve’s Law) Bill 2021 passing through the House of Representatives

We thank Minister Hunt for his efforts throughout his tenure and for his willingness to engage with RVA and work towards the best possible health and wellbeing outcomes for the estimated two million Australians living with a rare disease.

Parliamentary Inquiry Position Statement: ‘The New Frontier – Delivering Better Health for All Australians’

On 25 November 2021, the Standing Committee on Health, Aged Care and Sport (the Standing Committee) tabled its report into the Parliamentary Inquiry into approval processes for new drugs and novel medical technologies in Australia (Parliamentary Inquiry) in Parliament. The New Frontier – Delivering better health for all Australians (the New Frontier report) contains 31 recommendations, including dedicated sections to rare diseases and the patient voice. It also aligns with a number of the priorities, actions and implementation steps outlined in the National Strategic Action Plan for Rare Diseases (the Action Plan).

Trent Zimmerman MP, Chair of the Standing Committee, and its Deputy Chair, Dr Mike Freelander MP, each spoke to the report for five minutes in Parliament after the report was tabled. Rare Voices Australia (RVA) thanks Mr Zimmerman and Dr Freelander for engaging with the rare disease sector throughout the process and for their ongoing commitment to Australians living with a rare disease. Additionally, we thank all members of the Standing Committee for their work on this report.

RVA’s Chief Executive Officer (CEO), Nicole Millis, was invited to a private briefing with Mr Zimmerman and Dr Freelander to discuss the report’s findings immediately after the report was tabled on 25 November. The CEOs of Medicines Australia and the Medical Technology Association of Australia (MTAA) were also invited to the briefing.

Thanks once again to our RVA Partner organisations, as well as other key stakeholders in the rare disease sector, who lodged a Submission and participated in the Public Hearings earlier this year. As a sector, we have ensured that Australians living with a rare disease have remained central to this Parliamentary Inquiry throughout the process.

RVA’s Submission

RVA’s Submission into the Parliamentary Inquiry highlighted the critical issues that continue to persist for people living with a rare disease. It also suggested how Australia could build on the existing strengths of our current system in the most coordinated and systemic way to ensure processes enable Australian rare disease patients to access the fullest benefits of new medicines and emerging technologies now and in the future. You can read RVA’s Submission on the Standing Committee’s website (see Submission #86).

Next Steps

The Standing Committee included a number of the recommendations that RVA called for in our Submission and as opening witness during the Public Hearings. The recommendations in the New Frontier report are positive steps forward for people living with a rare disease and the entire rare disease sector. However, as these recommendations are yet to be implemented, it is imperative that the rare disease sector continues to leverage the Action Plan to ensure that these recommendations translate to policy that transforms people’s lives. RVA looks forward to the Government’s response to this report and in the meantime, will continue to actively engage with key political leaders on implementation.

Related Reviews

In July 2022, the Department of Health is scheduled to expand its independent Health Technology Assessment Review. Meanwhile, the National Medicines Policy (NMP) Review recommenced in August 2021 and the consultation process continued throughout October and November. You can read RVA’s Submission here. RVA also had the opportunity to participate in a one-on-one interview with the NMP Review Committee. We are confident the Review Committee now has a strong understanding of considerations from the rare disease sector. The Review Committee noted where possible, they would look to align their recommendations with the Action Plan. 

As a sector, we must continue to ensure that the views of people living with a rare disease are heard as part of both reviews. As the national peak body for Australians living with a rare disease, RVA will be advocating and engaging in consultations regarding these Reviews at every given opportunity. We will also consult with our RVA Partner organisations and other stakeholders as required.

This Position Statement

This Position Statement is designed to be read alongside the New Frontier report. RVA’s early analysis focuses on the strengths of the recommendations. We acknowledge the breadth and complexity of the New Frontier report which is 360 pages long. RVA intends to undertake further analysis of the report to identify any potential gaps or concerns on behalf of Australians living with a rare disease. We will continue to advocate to Government and key-decision makers and communicate to stakeholders accordingly. RVA Partners and other stakeholders are encouraged to reach out to RVA to discuss the report and its potential implications for Australians living with a rare disease. You can also download a PDF copy of this Position Statement.

Recommendations: The New Frontier – Delivering better health for all Australians

Recommendation 1: Establish a Centre for Precision Medicine and Rare Diseases

In RVA’s Submission, one of our key recommendations was the establishment of a Rare Disease and Precision Health Office in Government, acknowledging the importance and future promise of precision health in driving person-centred healthcare.

In its recommendation, the Standing Committee specifically states, “The objective of the Centre should be to ensure that the capacity of the Department of Health is enhanced to provide Australians with timely access to new drugs and novel medical technologies, including for rare diseases…”

In line with RVA’s Submission, the recommendation also states that, “The Centre should provide advice to governments on the establishment of a dedicated regulatory Health Technology Assessment pathway for cell and gene technologies, in consultation with state and territory governments, industry, patients and other relevant stakeholders. The Centre should regularly provide advice to government on the effectiveness of those pathways and areas for further reform.” Additionally, the recommendation adds, “The Centre should provide advice to the Department of Health and the Australian Medical Research Advisory Board on research priorities.”

In time, RVA envisions that the focus of a Centre for Precision Medicine and Rare Diseases could also include:

  • Data collection and epidemiology
  • Workforce and systems capacity
  • Investment into rare disease centres of excellence

High level Government policy coordination in the form of a Centre for Precision Medicine and Rare Diseases would go a long way towards progressing the Action Plan’s Vision: “The best possible health and wellbeing outcomes for Australians living with a rare disease.”

Recommendation 2: Establish a National Genomics Testing Program

RVA’s Submission noted that for decades, the rare disease community has heard about and waited eagerly for the future promise of gene and cell therapies, gene editing, genomics, precision and personalised medicine. RVA also acknowledged that gene therapy is time critical and that currently, there is no process in Australia for translating and utilising valuable real-world data as it emerges, yet this remains a potentially invaluable strategy to facilitate timely regulatory approval and to enable equitable therapeutic access. This is a long-standing equity issue for rare disease and will increasingly become an issue with the global trend towards personalised and precision health. It is a significant barrier preventing Australians accessing new medicines and emerging technologies.

Action 2.4.2 of the Action Plan is:

Ensure funding and reimbursement pathways are fit-for-purpose and sustainable for current and new health technologies for rare diseases.

RVA welcomes Recommendation 2 of the New Frontier report, which calls for “… the Health Technology Assessment (HTA) process for cell and gene therapies be simplified to establish a clear and certain pathway for such therapies.” In line with “equity of access” and “person-centred,” which are both foundation principles of the Action Plan, we also support the recommendation’s stipulation to “… establish a jointly funded national genomics testing program to provide equitable access to genomic testing nationwide. As part of the program, governments should ensure the provision of genomics counselling for all patients.” Importantly, Recommendation 2 also calls for the Australian Government to “prioritise and simplify the regulation of cell and gene therapy pathways for clinical trials in Australia,” which will expedite access to treatment for Australians living with a rare disease where a proven therapy is available.

Recommendation 3: Establish an Office of Clinical Evaluation

Recommendation 3 speaks to an “Office of Clinical Evaluation within the Department of Health to assess the best and most effective care for patients in the context of new and emerging health technologies.” This is yet another promising recommendation for Australians living with a rare disease.

RVA’s Submission acknowledged that in addition to a lack of investment, there is often no policy or legislative support for increased development and use of novel rare disease therapies in Australia. There can also be challenges and barriers to appropriately integrate these therapies into clinical care.

We also welcome the ongoing thread throughout the New Frontier report that encourages international liaison and collaboration where beneficial and appropriate. “State, national and international partnerships” is one of the critical enablers of the Action Plan.

Recommendation 4: Improving the Life Saving Drugs Program

The Standing Committee recommended “… that the assessment process for the Life Saving Drugs Program (LSDP) be streamlined and delays in access to treatments be reduced by ensuring that a sponsor only need lodge one application for one Health Technology Assessment pathway.”

The Standing Committee has put forward two different avenues to facilitate this recommendation. As when reforms to the LSDP were announced in 2018, RVA has always highlighted the importance of a streamlined and transparent LSDP process. RVA remains committed to working with the Australian Government regarding any potential policy reform of the LSDP.

Recommendation 6: Improving Education and Engagement for the TGA and the HTA processes

Health Technology Assessment (HTA) approval processes directly impact Australian patients’ access to new drugs and emerging technologies. In RVA’s Submission, in line with the Action Plan, we emphasized the importance of embedding the consumer voice throughout systems that impact rare diseases. It is particularly important in rare disease for consumers to participate in HTA processes. With limited data and uncertainties, consumers are able to provide much needed narrative and context to the data presented in HTA. It is critical that HTA processes formally embed, capture and promote the voice of people living with a rare disease and their families and carers.

People living with a rare disease can only contribute meaningfully to HTA processes if they understand the differences between the approval processes. Pillar 1 of the Action Plan is dedicated to Awareness and Education. Priority 1.2 is:

Ensure Australians living with a rare disease have access to information and education that enables them to be active participants in their rare disease journey.

RVA welcomes the Standing Committee’s recommendation “… that the Department of Health increase its efforts to educate and engage with patients, clinicians, industry and the public and develop education campaigns on all aspects of the regulation and reimbursement system.” RVA has been encouraging clearer and more timely communication between the Department of Health and all stakeholders in the rare disease sector for some time and we believe the proposals outlined in Recommendation 6 would go some way to addressing the issues that stakeholders have raised with RVA in the past. However, it is not the whole answer. Additionally, it is important to ensure that approval processes are fit for purpose.

Recommendation 8: Submission Fee Waivers

Due to small patient numbers, it is not always commercially viable for companies to seek reimbursement for a rare disease indication. In RVA’s Submission, we acknowledged this and the need for Government to respond to current disincentives. RVA also reaffirmed our support for reforms in recent years relating to expediated pathways, parallel processes, orphan drug designations and fee waivers, which are important incentives for pharmaceutical companies.

RVA welcomes Recommendation 8, recognising that legislation and regulation can greatly inhibit the development and use of novel therapies for rare diseases.

Recommendation 9: Funding for Submissions without a Sponsor

RVA’s Submission highlighted that Australia’s current Health Technology Assessment (HTA) system is over-reliant on pharmaceutical company sponsor-led applications. If companies are not commercially interested in submitting an application, there is currently no viable way to assess a health technology, regardless of levels of unmet need. This means that many people with a rare disease have challenges accessing a repurposed medicine that is prescribed by their doctor but only funded for use with a more common condition.

While RVA supports the sentiment behind Recommendation 9, as funding is only one part of the challenge in this instance, we maintain that there needs to be a way that clinicians and rare disease organisations can work with the HTA Consumer Evidence and Engagement Unit to submit an application for public reimbursement of a technology eligible for assessment by the Office of Health Technology Assessment (OHTA) via an appropriate, supported and sustainable pathway.

Action 2.4.3.3 of the Action Plan is:

The TGA [Therapeutic Goods Administration] and OHTA must work together to develop clear processes and pathways for sponsors considering submitting applications for the repurposing of medicines already approved for use in treatment of other conditions.

Recommendation 10: The PBAC and Managed Access Programs

RVA acknowledges the potential of Managed Access Programs (MAPs) and the current low uptake rates. Due to the small patient numbers and systemic data challenges in rare disease, MAPs have the potential to provide people living with a rare disease with earlier access to treatment while also collecting data and building knowledge in the long-term. A key priority of the Action Plan is to enable all Australians to have equitable access to the best available health technology.  

Recommendation 11: Review Repurposing of Drugs

RVA’s Submission highlighted the importance of repurposing of medicines already approved for use in treatment of other conditions. These medicines play an important role and present an opportunity to address unmet need in the treatment of rare diseases.

RVA welcomes the Standing Committee’s recommendation that “… the Department of Health conduct a comprehensive consultation process with industry to establish a more flexible way forward for the repurposing of drugs in Australia. This should include:

  • Establishing a new pathway that incentivises the repurposing of drugs for all diseases…”

Existing medicines funded for more common conditions can often be clinically beneficial as treatments for rare diseases. Clinicians often prescribe off-label use of medicines in the treatment of rare diseases. Currently, Australian patients are unable to reap the full benefits of repurposing of medicines as reimbursement of health technologies for rare diseases is challenging, even for an approved medicine for a more common condition. For rare diseases, there are a lack of transparent and equitable pathways for repurposing existing treatments that are already being reimbursed for more common conditions. This is a common and systemic issue for rare diseases. Companies commonly state that it is not feasible for them to submit an application for new indications due to extremely small patient numbers, lack of conventional clinical trials etc. This means that many Australians who are living with a rare disease have to rely on the uncertainty of off-label use or self-fund (often equating to thousands of dollars in costs) their access to a medicine that is recommended by their clinician. This is both unsustainable and inequitable.

Recommendation 12: TGA Reform

RVA supports Recommendation 12’s proposed changes to the Orphan Drugs Program. The recommendation to, “Provide automatic access to the Priority Review Pathway for all medicines granted an orphan drug designation,” will expedite access to treatment for many Australians living with a rare disease. This aligns with Action 2.4.1 of the Action Plan:

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

Likewise, we are encouraged by the focus on international liaison and collaboration where relevant, which is evident in the following recommendation: “Where the prevalence of a disease is unknown in Australia, accept evidence of prevalence in other comparable countries or, in diseases of extremely low prevalence, worldwide for the purposes of the eligibility criteria.”

Recommendation 15: Membership of the PBAC and MSAC

RVA’s Submission highlighted the lack of rare disease expertise and understanding that currently informs approval processes for rare disease therapies. Action 2.4.2.3 of the Action Plan is:

Ensure rare disease expertise exists, or can be accessed, on all reimbursement pathways and HTA advisory bodies.

Additionally, we said we were not clear on the Medical Services Advisory Committee’s (MSAC) level of understanding of rare disease and precision medicine.

RVA supports the Standing Committee’s recommendation “that the Australian Government ensure the membership of the Pharmaceutical Benefits Advisory Committee and Medical Services Advisory Committee provides the appropriate expertise for all applications.” Additionally, given the challenges typically at play in rare disease, we welcome the inclusion of “…enhanced cross-membership between the two committees and the appointment of temporary members to consider individual applications” as part of Recommendation 15.

Recommendation 15 also speaks to “…the nature of health challenges in Indigenous communities, membership should include representation from Aboriginal and Torres Strait Islander Peoples.” Aboriginal and Torres Strait Islander peoples are one of the priority populations identified in the Action Plan. Several factors increase the potential impact of rare diseases on Aboriginal and Torres Strait Islander people.

Recommendation 16: Increase International Collaboration

One of the critical enablers of the Action Plan is, “State, national and international partnerships.” As identified in the Action Plan, given the small populations and complexity involved in rare diseases, strong ongoing partnerships are invaluable. Global collaboration and the sharing of knowledge and expertise are often required to ensure the best outcomes for people living with a rare disease. As such, RVA welcomes the recommendation that “… the Department of Health investigate further opportunities for the formation of an international Health Technology Assessment consortium…”

Recommendation 21: Improve Newborn Screening Program

While the Standing Committee acknowledged that newborn screening was not in the terms of reference for this Parliamentary Inquiry, they dedicated Recommendation 21 to newborn screening in response to “…calls from rare disease patient groups for more funding for treatment pathways for actionable disorders across states and territories, where identified through newborn screening.”

Newborn bloodspot screening (NBS) is an important program that supports the earliest possible diagnosis of numerous rare diseases. Priority 2.2 of the Action Plan is:

Ensure diagnosis of a rare disease is timely and accurate.

RVA welcomes the Standing Committee’s recommendations regarding the NBS program, particularly the focus on “actionable disorders” rather than “treatable”; as well as recommending that “…federal, state and territory health authorities complete the standardisation of newborn screening across Australia.” RVA is pleased that the Standing Committee has recognised that, currently, important work and reform is already progressing at both a Commonwealth and state and territory level. However, RVA also welcomes the Standing Committee highlighting that there is more work to be done before the NBS process is fully completed.

RVA remains committed to continuing our work with the Department of Health as well as states and territories regarding NBS.

Recommendations 22 to 26: Improve the Clinical Trial System in Australia

In our Submission, one of RVA’s key recommendations was to develop policy and infrastructure that responds to the strong interrelation between approval processes, clinical trials and data collection. Action 3.2.4 of the Action Plan is:

Building on existing initiatives, continue to foster an environment conducive to clinical trials for rare diseases take place in Australia.

It is critical that Australia attracts and incentivises pharmaceutical companies to conduct rare disease clinical trials in Australia, enabling Australian rare disease patients to participate in international clinical trials. Currently, there is a lack of coordinated infrastructure to support a national approach for rare disease clinical trials with very small patient numbers. For many people living with a rare disease, participation in a clinical trial may be the only way to access treatment.

Recommendation 22, which “recommends that all levels of government prioritise and implement with urgency the harmonisation of Human Research Ethics Committee (HREC) and Site-Specific Assessment submissions into one Australian online platform and enable parallel review by HRECs and Research Governance Offices,” seeks to address the multiple and onerous ethics approvals and other bureaucratic processes currently involved in running a clinical trial in Australia.

In Australia, data for most rare diseases is not captured in either health information systems or registries and there is no coordinated strategy to collect, measure, build and translate data that does exist. For many rare diseases, there are many barriers to effective research and no active research programs. Recommendation 23 calls for “… the development of a national clinical trial register” and the inclusion “…of a sophisticated digital platform to collect and facilitate patient identification, patient recruitment, patient retention and completion rates for clinical trials.” This recommendation aligns with Priority 3.1 of the Action Plan:

“Enable coordinated and collaborative data collection to facilitate the monitoring and cumulative knowledge of rare diseases, informing care management, research and health system planning.”

Recommendation 24 states that, “National standards should include standardising clinical costs and fees that are competitive with international fees.” Again, this recommendation aligns with Action 3.2.4 of the Action Plan:

Building on existing initiatives, continue to foster an environment conducive to clinical trials for rare diseases take place in Australia.

Recommendation 25 proposes that “the Australian Government should develop a national standard approach, including nationally agreed systems and standard operating procedures to support and strengthen the capacity to conduct clinical tele-trials in rural, regional and remote areas.” People living in regional, rural and remote areas are one of the priority populations identified in the Action Plan. Where people live can have a significant impact on their ability to access services. The lack of rare disease expertise nationally is exacerbated by Australia’s vast size.

RVA’s Submission also called for the development of an Australian Rare Disease Clinical Trial and Registry Network; national infrastructure to support rare disease clinical trials to accelerate clinical trials in rare disease and to attract industry. RVA maintains that an Australian Rare Disease Clinical Trial and Registry Network is needed, however, we support Recommendation 26, which states, “…the Australian Government should continue to fund Clinical Trial Networks with a particular focus on developing seed funding for Indigenous Health Clinical Trial Networks.”

Recommendation 27: Research and Development

RVA welcomes the Standing Committee’s recommendations to:

  • “Develop additional reforms to data exclusivity timeframes to support research and development into new drugs and novel medical technologies in areas of unmet need.
  • “Consider future funding initiatives for novel drug discovery and support research and development partnerships in Australia. This would assist new drugs and novel medical technologies in early stage and pre-commercial development.”

Recommendation 28: The Patient Voice

The New Frontier report dedicates an entire section to the importance of the patient voice. “Person-centred” is a foundation principle of the Action Plan and Action 2.1.5 is:

Embed the voice of people living with a rare disease and their families and carers throughout the structures and systems that impact rare diseases.

In RVA’s Submission, in line with the Action Plan, we emphasized the importance of embedding the consumer voice throughout systems that impact rare diseases.

RVA welcomes all elements that form Recommendation 28. The recommendation reads, “The Department of Health integrate the patient voice upfront into the Health Technology Assessment system” and goes on to propose, “The Australian Government provide funding for organisations to support participation in the HTA process, including for very rare disease patient groups that have limited capacity for fundraising or access to alternative funding.” While we support these improvements, again, we highlight that funding is not the only barrier for consumers — they also need a pathway that is appropriate, supported and sustainable. RVA reiterates the important role of the HTA Consumer Evidence and Engagement Unit, which is well positioned to assist in the provision of education and support to people living with a rare disease and their families and carers, and/or rare disease organisations to support them to take a more active role in HTA processes. Equally as vital is building the capacity of rare disease organisations to facilitate their participation in the co-design and coordination of trials.

RVA strongly supports the following elements of Recommendation 28:

  • “The Department of Health provide patients and stakeholders with a concise sponsor’s submission summary to help facilitate their own involvement in the Health Technology Assessment process.
  • “The Department of Health should consider making patient evidence compulsory for certain applications, and should consider the role of patient evidence in the decisions of the Therapeutic Goods Administration.
  • “The Department of Health should notify relevant patient groups of the outcome of the assessment process by all HTA bodies.”

Recommendation 29: Improving the HTA process

There are many strengths in Australia’s current HTA processes, however, these strengths do not exist consistently across all approval processes. This is a real issue as every approval process assesses rare disease and precision health technologies.

RVA commends the work of the TGA and Pharmaceutical Benefits Advisory Committee (PBAC) around parallel processes. Similarly, RVA believes the PBAC is the gold standard in terms of transparent timelines and consumer engagement. However, its criteria regarding cost effectiveness is challenging in the rare disease context, as is the reliance on traditional clinical trial evidence.

RVA welcomes the Standing Committee’s recommendation that, “The independent Health Technology Assessment Review reassess relevant aspects of the Health Technology Assessment process to ensure there are future pathways for treatments and therapies that do not fit neatly into the current system such as rare cancers, antimicrobials, orphan drugs, and precision medicines.” Additionally, we commend the Standing Committee for highlighting “… that precision medicine approval pathways will require a different application assessment than current approaches designed for treatments for common conditions, with large data sets and comparative evaluations.”

RVA’s Submission also highlighted the lack of transparency across certain elements of the HTA process. We believe the following recommendations will assist in addressing these issues:

  • “The Department of Health publish data on application processing times and positive recommendation rates for the Pharmaceutical Benefits Advisory Committee and other Health Technology Assessment bodies. In addition:
    — The Department of Health should publish Health Technology Assessment processing times annually, benchmarked against other nations with advanced HTA processes.
  • “The Australian Government, in collaboration with relevant stakeholders, develop a suite of clear and measurable benchmarks to track the Commonwealth’s implementations of the recommendations made by the Committee and accepted by the Australian Government.
    — These agreed benchmarks along with measurable KPIs/metrics should be developed in such a way as to best facilitate the Department of Health, including its agencies and other relevant statutory bodies, in the tabling of an annual update to the Australian Parliament.”

Recommendation 30: Review of HTA

The Standing Committee has made a number of recommendations for the Australian Government’s independent Health Technology Assessment Review (which is scheduled to commerce in July 2022) to consider.

RVA looks forward to playing an integral role in this consultation in 2022 on behalf of Australians living with a rare disease and welcomes all of the Standing Committee’s recommendations. From a rare disease perspective, the following recommendations are of particular importance:

  • “Streamlining the interaction between hospitals and the Health Technology Assessment system
  • “Streamlining the interaction of the Therapeutic Goods Administration, the Pharmaceutical Benefits Advisory Committee, the Medical Services Advisory Committee and other Health Technology Assessment bodies
  • “Cooperation and harmonisation between Australian Health Technology Assessment bodies and equivalent bodies overseas
  • “Increasing the use of Real World Evidence in Health Technology Assessment
  • “Introducing a scoping process that includes patients and clinicians at an early stage to agree on the framework that the submission will be considered.
  • “Improving the independent review process for HTA decisions, including the potential for this to be made available to groups of patients and clinicians in addition to sponsors.”

Recommendation 31: MSAC

RVA’s Submission noted that in recent times, there has been a massive variation in the many areas the Medical Services Advisory Committee (MSAC) assesses. As already noted, the MSAC’s level of understanding of rare disease and precision medicine is unclear. We also highlighted the lack of transparency around timelines and formal consumer engagement and our concern that applications were typically unsuccessful. RVA called for the refinement of the approval process to ensure that the MSAC is able to respond in a timely manner to the new and novel therapies that provide much hope for the rare disease community.

RVA fully supports the Standing Committee’s recommendation that, “The Department of Health expand the independent Health Technology Assessment Review in July 2022 to include Medical Service Advisory Committee processes.” Likewise, RVA supports the following recommendations:

  • “The Medical Services Advisory Committee increase the involvement of clinicians in its assessments of technologies with which its members lack relevant expertise
  • “The Therapeutic Goods Administration introduce parallel processing of applications with the Medical Services Advisory Committee
  • “The Medical Services Advisory Committee consider developing international collaboration for complex assessment proposals
  • “The Medical Services Advisory Committee publish a full calendar timeline of meeting agenda and outcomes, including dates when minutes and Public Summary Documents will be made public
  • “The Department of Health establish a benchmarking system for MSAC assessments, including benchmarking against comparable overseas organisations.”

RVA is Hiring: Resources and Information Officer

Rare Voices Australia (RVA) is looking for a Resources and Information Officer 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 research and develop customised, evidence-based information about rare diseases for all stakeholders across the rare disease community for the Rare Awareness Rare Education (RARE) Portal. In line with the National Strategic Action Plan for Rare Diseases, the portal will be an accessible multi-purpose website that includes information and resources for rare diseases that are customised for the Australian context.

Download this PDF for the full position description. Applications close COB 7 January 2022.

Final Report Tabled: Parliamentary Inquiry Into Approval Processes for New Drugs and Novel Medical Technologies in Australia

The Standing Committee on Health, Aged Care and Sport (the Standing Committee) tabled its report into the Parliamentary Inquiry into approval processes for new drugs and novel medical technologies in Australia (Parliamentary Inquiry) in Parliament today (25 November 2021). The report, The New Frontier – Delivering better health for all Australians, contains 31 recommendations, including separate sections dedicated to rare diseases and the patient voice. It also aligns with a number of the priorities, actions and implementation steps outlined in the National Strategic Action Plan for Rare Diseases. Access the report by clicking here.

Trent Zimmerman MP, Chair of the Standing Committee, and its Deputy Chair, Dr Mike Freelander MP, each spoke to the report for five minutes in Parliament after the report was tabled. Rare Voices Australia (RVA) thanks Mr Zimmerman and Dr Freelander for engaging with the rare disease sector throughout the process and for their ongoing commitment to Australians living with a rare disease. Additionally, we thank all members of the Standing Committee for their work on this report.

RVA’s Chief Executive Officer (CEO), Nicole Millis, was invited to a private briefing with Mr Zimmerman and Dr Freelander to discuss the report’s findings immediately after the report was tabled. The CEOs of Medicines Australia and the Medical Technology Association of Australia (MTAA) were also invited to the briefing.

Thank you to our RVA Partner organisations, as well as other key stakeholders in the rare disease sector, who lodged a Submission and participated in the Public Hearings earlier this year. As a sector, we have ensured that Australians living with a rare disease have remained central to this Parliamentary Inquiry.

RVA is in the process of taking a detailed look at the report and will provide a full statement with more information about the implications of the recommendations for Australians living with a rare disease shortly. Initially, we have identified a number of recommendations from the report that appear consistent with what RVA called for in our Submission and as opening witness at the Public Hearings.

The following recommendations are positive steps forward for people living with a rare disease and the entire rare disease sector:

Recommendation 1: Establish a Centre for Precision Medicine and Rare Diseases

11.1 — “The Committee recommends the Australian Government establish a Centre for Precision Medicine and Rare Diseases within the Department of Health.”

Note: Recommendation 1 contains more information. See the report for full details.

Recommendation 2: Establish a National Genomics Testing Program

11.2 — “The Committee recommends that, consistent with Recommendation 1 and the establishment of a Centre for Precision Medicine and Rare Diseases, the Health Technology Assessment (HTA) process for cell and gene therapies be simplified to establish a clear and certain pathway for such therapies.”

Note: Recommendation 2 contains more information. See the report for full details.

Recommendation 3: Establish an Office of Clinical Evaluation

11.3 — “The Committee recommends the Australian Government establish an Office of Clinical Evaluation within the Department of Health to assess the best and most effective care for patients in the context of new and emerging health technologies.”

Note: Recommendation 3 contains more information. See the report for full details.

Recommendation 4: Improving the Life Saving Drugs Program

11.4 — “The Committee recommends that the assessment process for the Life Saving Drugs Program (LSDP) be streamlined and delays in access to treatments be reduced by ensuring that a sponsor only need lodge one application for one Health Technology Assessment pathway.”

Note: Recommendation 4 contains more information. See the report for full details.

Recommendation 6: Improving Education and Engagement for the TGA and the HTA processes

11.6 — “The Committee recommends that the Department of Health increase its efforts to educate and engage with patients, clinicians, industry and the public and develop education campaigns on all aspects of the regulation and reimbursement system.”

Note: Recommendation 6 contains more information. See the report for full details.

Recommendation 8: Submission Fee Waivers

11.9 — “The Committee recommends that the Australian Government make the following changes to submission fees for the Therapeutic Goods Administration (TGA) and the Pharmaceutical Benefits Advisory Committee (PBAC) and where appropriate Medical Services Advisory Committee (MSAC) assessments in the following separate circumstances:

  • Replace the current orphan drug fee waivers with a HECS-style fee waiver, in which orphan drug application fees are payable on successful application, only once the drug has earned the sponsor a certain amount of revenue. The Department of Health should determine this threshold value in consultation with industry
  • To support smaller companies, HECS-style fee waivers for any sponsor company with revenue at or below $50 million per annum
  • HECS-style fee waivers for Australian start-up companies with a specified amount of revenue in the Australian market to promote innovation.

The Committee also recommends introducing a sliding scale for fees for resubmissions, with fees being lower for resubmissions.

Recommendation 9: Funding for Submissions without a Sponsor

11.10 — “The Committee recommends that the Australian Government establish a fund to support patients, clinicians and non-profit organisations to sponsor registration and reimbursement applications where there is no realistic prospect of a company serving as sponsor, and where the Department of Health is otherwise supportive of the application.

  • Such a fund should be targeted at treatments for conditions where low patient numbers in Australia serve as a market barrier and where there is a clinical demand and need. The fund should be available for applications to repurpose previously listed medicines and technologies.”

Note: Recommendation 9 contains more information. See the report for full details.

Recommendation 10: The PBAC and Managed Access Programs

11.11 — “The Committee recommends that the Australian Government amend the National Health Act 1953 (Cth) to give the Pharmaceutical Benefits Advisory Committee the power to authorise Managed Access Programs. The eligibility criteria for these Managed Accessed Programs should be aligned as far as possible with the eligibility criteria for the Therapeutic Goods Administration’s provisional registration.”

Recommendation 11: Review Repurposing of Drugs

11.12 — “The Committee recommends that the Department of Health conduct a comprehensive consultation process with industry to establish a more flexible way forward for the repurposing of drugs in Australia.”

Note: Recommendation 11 contains more information. See the report for full details.

Recommendation 12: TGA Reform

11.13 — “The Committee recommends that the Therapeutic Goods Administration make the following changes to its Orphan Drugs Program:

  • Provide automatic access to the Priority Review Pathway for all medicines granted an orphan drug designation
  • Treat paediatric patient populations as separate to adult patient populations for the purposes of the eligibility criteria
  • Better account for the extra costs incurred by a sponsor in expanding its medicine to paediatric indications, for the purposes of assessing commercial viability as part of the eligibility criteria
  • Where the prevalence of a disease is unknown in Australia, accept evidence of prevalence in other comparable countries or, in diseases of extremely low prevalence, worldwide for the purposes of the eligibility criteria.

Recommendation 13: Molecular Indications

11.14 — “The Committee recommends that the Department of Health reform its regulatory and reimbursement processes to enable therapeutic goods to be registered and reimbursed by molecular indication in addition to by disease indication. This should include legislative change if necessary.”

Recommendation 15: Membership of the PBAC and MSAC

11.16 — “The Committee recommends that the Australian Government ensure the membership of the Pharmaceutical Benefits Advisory Committee and Medical Services Advisory Committee provides the appropriate expertise for all applications. This should include the possibilities of enhanced cross membership between the two committees and the appointment of temporary members to consider individual applications.”

Note: Recommendation 15 contains more information. See the report for full details.

Recommendation 16: Increase International Collaboration

11.17 — “The Committee recommends that the Department of Health investigate further opportunities for the formation of an international Health Technology Assessment consortium…”

Note: Recommendation 16 contains more information. See the report for full details.

Recommendation 21: Improve Newborn Screening Program

11.22 — “The Committee recommends:

  • The federal, state and territory health authorities complete the standardisation of newborn screening across Australia
  • As part of that process, the Australian Government work with states and territories to expand the newborn screening program based on new understandings of genomic testing for conditions and international best practice
  • That the Australian Government in collaboration with states and territories, conduct reviews every two years to determine whether the screening program should be further expanded based on new Australian and international scientific and medical knowledge.

“While not in the terms of reference for this inquiry, the Committee recognises and supports the calls from rare disease patient groups for more funding for treatment pathways for actionable disorders across states and territories, where identified through newborn screening.”

Recommendation 22: Improve the Clinical Trial System in Australia

11.23 — “The Committee recommends that all levels of government prioritise and implement with urgency the harmonisation of Human Research Ethics Committee (HREC) and Site-Specific Assessment submissions into one Australian online platform and enable parallel review by HRECs and Research Governance Offices.”

Note: Recommendation 22 contains more information. See the report for full details.

Recommendation 23

11.24 — “The Committee recommends that all levels of government jointly provide funding for the development of a national clinical trial register. It should include:

  • Development of a sophisticated digital platform to collect and facilitate patient identification, patient recruitment, patient retention and completion rates for clinical trials.”

Note: Recommendation 23 contains more information. See the report for full details.

Recommendation 24

11.25 — “The Committee recommends the Australian Government develop policies that encourage modernising digital technologies and practices to position Australia as the premier destination for international clinical trials. This would include developing national standards for the use of e-consent, esignature, and electronic medical records to enable remote monitoring and participation in clinical trials across Australia.

  • National standards should include standardising clinical costs and fees that are competitive with international fees.”

Recommendation 25

11.26 — “The Committee recommends the Australian Government should develop a national standard approach, including nationally agreed systems and standard operating procedures to support and strengthen the capacity to conduct clinical tele-trials in rural, regional and remote areas.”

Note: Recommendation 25 contains more information. See the report for full details.

Recommendation 26

11.27 — “The Committee recommends the Australian Government should continue to fund Clinical Trial Networks with a particular focus on developing seed funding for Indigenous Health Clinical Trial Networks.”

Recommendation 27: Research and Development

11.28 — “… The Australian Government should:

  • Develop additional reforms to data exclusivity timeframes to support research and development into new drugs and novel medical technologies in areas of unmet need. 
  • Consider future funding initiatives for novel drug discovery and support research and development partnerships in Australia. This would assist new drugs and novel medical technologies in early stage and pre-commercial development.

Note: Recommendation 27 contains more information. See the report for full details.

Recommendation 28: The Patient Voice

11.29 — “The Committee recommends that:

  • The Department of Health integrate the patient voice upfront into the Health Technology Assessment system. Earlier patient engagement with the Health Technology Assessment system would include:

– Representation from peak patient bodies that is refreshed every three – five years

– Representation of Aboriginal and Torres Strait Islander Peoples.

  • The Department of Health implement a notification system for all HTA bodies and the TGA to advise relevant patient groups of the receipt of an application.
  • The Department of Health provide patients and stakeholders with a concise sponsor’s submission summary to help facilitate their own involvement in the Health Technology Assessment process.
  • The Department of Health should consider making patient evidence compulsory for certain applications, and should consider the role of patient evidence in the decisions of the Therapeutic Goods Administration.
  • The Department of Health should notify relevant patient groups of the outcome of the assessment process by all HTA bodies.
  • The Department of Health be funded to implement these recommendations.
  • The Australian Government provide funding for organisations to support participation in the HTA process, including for very rare disease patient groups that have limited capacity for fundraising or access to alternative funding.”

Recommendation 29: Improving the HTA process

11.30 — “The Committee recommends that:

  • The Department of Health produce a pre-submission advice framework for submissions to the Therapeutic Goods Administration, Pharmaceutical Benefits Advisory Committee, Medical Services Advisory Committee and other Health Technology Assessment bodies, explaining the interaction between those bodies and their evidentiary and other requirements, to be provided to sponsors before they make their submissions.
  • The independent Health Technology Assessment Review reassess relevant aspects of the Health Technology Assessment process to ensure there are future pathways for treatments and therapies that do not fit neatly into the current system such as rare cancers, antimicrobials, orphan drugs, and precision medicines

– The Committee is of the clear view that precision medicine approval pathways will require a different application assessment than current approaches designed for treatments for common conditions, with large data sets and comparative evaluations.

  • The Department of Health publish data on application processing times and positive recommendation rates for the Pharmaceutical Benefits Advisory Committee and other Health Technology Assessment bodies.”

Note: Recommendation 29 contains more information. See the report for full details.

Recommendation 30: Review of HTA

11.31 — “The Committee recommends that the Australian Government’s independent Health Technology Assessment Review (which is scheduled to commerce in July 2022) consider and develop reforms in the following areas:

  • Reducing the frequency and need for applications to HTA bodies to be resubmitted
  • Streamlining the interaction between hospitals and the Health Technology Assessment system
  • Streamlining the interaction of the Therapeutic Goods Administration, the Pharmaceutical Benefits Advisory Committee, the Medical Services Advisory Committee and other Health Technology Assessment bodies
  • Improving the measurement of the performance of the Pharmaceutical Benefits Advisory Committee and the publication of data on that performance
  • Improving the mechanisms for communication between sponsors and the Pharmaceutical Benefits Advisory Committee during the submission process
  • Increasing the use of Managed Access Programs to facilitate earlier access to innovative medicines
  • Increasing the use of Real World Evidence in Health Technology Assessment
  • Introducing a scoping process that includes patients and clinicians at an early stage to agree on the framework that the submission will be considered. This process could draw on the approach taken by the United Kingdom’s National Institute for Health and Care Excellence
  • Improving the independent review process for HTA decisions, including the potential for this to be made available to groups of patients and clinicians in addition to sponsors”

Note: Recommendation 30 contains more information. See the report for full details.

Recommendation 31: MSAC

11.32 — “The Committee recommends that:

  • The Medical Services Advisory Committee increase the involvement of clinicians in its assessments of technologies with which its members lack relevant expertise.
  • The Therapeutic Goods Administration introduce parallel processing of applications with the Medical Services Advisory Committee.
  • The Medical Services Advisory Committee increase opportunities for sponsors of particularly complex applications to present to it at its meetings and expand the opportunities for pre-submission meetings
  • The Department of Health expand the independent Health Technology Assessment Review in July 2022 to include Medical Service Advisory Committee processes
  • The Medical Services Advisory Committee publish a full calendar timeline of meeting agenda and outcomes, including dates when minutes and Public Summary Documents will be made public
  • The Department of Health establish a benchmarking system for MSAC assessments, including benchmarking against comparable overseas organisations.

Note: Recommendation 31 contains more information. See the report for full details.

Recap: 2021 Virtual National Rare Disease Summit

Thank you to those who attended Rare Voices Australia’s (RVA) 2021 Virtual National Rare Disease Summit (the Virtual Summit) on 12 November. The Virtual Summit brought together key stakeholders in the rare disease sector including people living with a rare disease, governments, key peak bodies, researchers, clinicians and industry. The theme was, ‘A person-centred approach to implementing the National Strategic Action Plan for Rare Diseases’. Person-centred is one of the Action Plan’s foundation principles and shaped the Virtual Summit. People living with a rare disease opened the event and shared their voice throughout. Panel sessions addressed the following key areas in rare disease: 

  • Lived experience
  • Emerging therapies
  • Research 
  • Rare disease workforce

RVA thanks the Hon Greg Hunt MP (Minister for Health and Aged Care), for formally opening the Virtual Summit, as well as the Hon Mark Butler MP (Shadow Minister for Health and Ageing) who delivered an address. We also thank Dr Mike Freelander MP (Co-Chair, Parliamentary Friends of Australians Living with Rare Diseases; Deputy Chair, Standing Committee on Health, Aged Care and Sport) and Trent Zimmerman MP (Co-Chair, Parliamentary Friends of Australians Living with Rare Diseases; Chair, Standing Committee on Health, Aged Care and Sport) who joined the ‘Emerging Therapies and Their Potential to Revolutionalise Care for People Living with a Rare Disease’ panel discussion and answered questions from attendees.

The Virtual Summit also featured a diverse range of speakers to reflect various stakeholder expertise. A big thank you to our speakers for sharing your invaluable insights with the rare disease sector. We also thank our Master of Ceremonies, Julian Morrow. Finally, thanks to all attendees for your questions and contributions throughout the day.

Over 140 people nation-wide registered to attend the Virtual Summit with each session stimulating much discussion that will further inform implementation of the National Strategic Action Plan for Rare Diseases. 

Feedback from attendees:

What a powerhouse RVA has become in just 10 years. So professional, so well connected and with such wonderful achievements all growing daily…” – RVA Partner 

“It was one of the best online events I attended this year.” – Researcher

“The caliber of the speakers was very impressive and their contribution to the Summit was invaluable… Australia’s approach to rare diseases will go from strength to strength I am sure due to all the hard work by everyone involved in Friday’s Summit.” – Person with lived experience

“The quality of speakers and presenters was exceptional – but the focus and flow was excellent.” – Industry representative