Category: News

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.

Rare Voices Australia (RVA) has engaged Monash University clinical registry experts, Professor Susannah Ahern and Dr Rasa Ruseckaite, to undertake an audit of Australian rare disease registries, and databases available to Australian participants.

The National Strategic Action Plan for Rare Diseases (the Action Plan) identifies the key role that registries play in its Research and Data Pillar. The Action Plan was informed by an extensive multi-stakeholder consultation process. Click here to learn more about the rationale behind this work.

RVA has invited RVA Partners, known rare disease registry managers and RVA’s Round Table of Companies to take part in a short survey as part of this work. However, this survey is open to everyone who manages or has managed a rare disease registry that collects Australian data. If this applies to you, we would be grateful for your input.

To complete this survey, you will need the following information:

• The type of registry – clinician led/patient led/other
• The data your registry collects and from what jurisdictions or sites
• Who is responsible for maintaining and populating the registry
• How it is funded
• If your registry has ethics approval
• Registry governance
• Outcomes, barriers and enablers of the registry

With this information handy, it should take approximately 10 to 15 minutes to complete all the survey questions. If you wish to take part, please click on the link below. 

https://monash.az1.qualtrics.com/jfe/form/SV_d5SBlmp3Dp1MatU

If you have trouble accessing this link, please copy and paste it into your usual web browser.

Thank you for taking part. The results will be available in the first half of 2022.

As part of the Rare as One Project, The Chan Zuckerberg Initiative (CZI) is seeking information about registry interoperability. Responses are encouraged from patient communities, researchers, clinicians, health technology developers and a diverse range of experts to share knowledge and submit information that can help address similar challenges from different perspectives. If this is relevant to you, click here for more information.

Submissions close 30 November 2021. 

Rare Voices Australia will be making a submission, however, we note that registry interoperability is an important issue that would benefit from the breadth of knowledge of the rare disease sector.

The Standing Committee on Health, Aged Care and Sport (the Standing Committee) intend to table the Parliamentary Inquiry report into approval processes for new drugs and novel medical technologies in Australia this Thursday 25 November between 10am – 11am AEDT in Parliament. The Chair and Deputy Chair will speak to the report for five minutes each in the chamber after its tabling. Other Standing Committee members will also have an opportunity to speak to the report in the Federation Chamber in the following sitting weeks.

The Committee’s report will be available on the Standing Committee’s website approximately 15 minutes after the report has been tabled in the chamber.

Rare Voices Australia (RVA) thanks 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 are central to this Parliamentary Inquiry. RVA looks forward to reviewing the Standing Committee’s report on Thursday.

Rare Voices Australia (RVA) is pleased to share a survey from one of our research partners seeking to understand consumer experiences with life insurance and genetic testing.

Led by experts at Monash University, this research aligns with the following actions in the National Strategic Action Plan for Rare Diseases:

Action 2.2.2: Develop policy that supports the implementation of diagnostic tools and tests.

Action 2.3.1: Ensure individuals and families known to have an increased chance of being carriers of genetic variants for rare diseases have equitable access to peri‑conception genetic testing and counselling, which can provide them with information about becoming pregnant and pregnancy.

Australian Genetics and Life Insurance Moratorium: Monitoring the Effectiveness and Response (A-GLIMMER)

Many people are concerned about the use of genetic information by life insurers. Researchers from Monash University are keen to understand the views and experiences of consumers. This includes people who have had genetic testing, as well as those who are eligible but choose not to have genetic testing. The findings of this research will contribute to a report to the Australian Government regarding the current regulation of life insurers, and your participation will assist with gathering critical data on the views and experience of consumers.

Please consider completing this important survey, whatever the experience you have had with life insurance and genetic testing. You can remain anonymous if you wish.

Feel free to forward this survey link to your family members who may have had or considered having a genetic test.

If you have any issues with accessing the links, copy and paste this link into your web
browser: https://redcap.link/aglimmer.consumer

At the beginning of the survey, you will find more information about the study and an explanatory statement.

For any queries regarding this research, please contact Jane Tiller, the research coordinator, on [email protected]

Limited data about rare diseases related to incidence and prevalence, natural history, epidemiology, impact, and treatment outcomes or a lack thereof, is driving a global emphasis on the value of rare disease registries.

Registries have the capacity to:

• Facilitate public reporting and knowledge building
• Reveal differences in care practices and process and the impact of both on patients
• Identify best practice and target areas for future improvement
• Reveal information about incidence, prevalence, epidemiology, impact, treatment outcomes, the natural history of diseases and much more

However, most rare disease registries collect different types of data and are independent of one another. Very few are meeting their full potential due to limited patient involvement, low levels of data sharing and interoperability, and a lack of consistent standards.

The need for a national approach to rare disease registries was emphasised during multi-stakeholder discussions at the registries workshop at Rare Voices Australia’s (RVA) 2018 National Rare Disease Summit. Since this time, RVA’s Scientific and Medical Advisory Committee (SMAC) has championed the need to progress this work. The National Strategic Action Plan for Rare Diseases (the Action Plan) identifies the key role that registries play in its Research and Data Pillar. The Action Plan was informed by an extensive multi-stakeholder consultation process. Recently, RVA was pleased to finally fund and oversee critical rare disease registries work in collaboration with an expert team at Monash University who have skills and knowledge in the development and operation of clinical quality registries.

Why is a national approach to rare disease registries so important?

The challenges of rare disease registries are multifaceted and complex. In line with the Action Plan, a national approach to rare disease registries is vital because it is not practical or economically feasible to create clinical quality registries for every rare disease. Global leaders in rare disease registries–EURORDIS, NORD and CORD–have jointly declared 10 key principles for rare disease registries. One of these principles is to ensure consistent use of a minimum set of common data elements. This aligns with Action 3.1.4 of the Action Plan, ‘Develop a national approach to person-centred rare disease registries to support national standards, best practice and minimum data sets’.

 Implementation 3.1.4.1. in the Action Plan outlines the first step to achieve this:

Develop a summary report of all existing Australian and relevant international rare disease registries, collecting information on:

• governance standards;

• management practices;

• data sets, including patient numbers, estimated incidence, prevalence and coverage; and

• classification systems used (for interoperability with other registries and health information

systems).

This information will support national coordination of rare disease registries and the establishment of minimum data sets, to provide a better understanding of who is currently being counted and aid identification of best practice.

Project Summary

To progress this work, RVA, together with Monash University registry experts, Professor Susannah Ahern and Dr Rasa Ruseckaite, are conducting an audit of Australian and relevant international rare disease registries. This rare disease registry audit project consists of the following parts:

Part 1: Scoping review of the academic and grey literature to identify all existing Australian rare disease registries. The aim of this component is to propose minimum data categories for rare disease registries in Australia.

The review is in progress.

Part 2: Survey and interviews with managers of Australian rare disease registries to collect metadata, including the number of patients in each registry, estimated incidence/prevalence, estimated coverage, and data elements stored. The aim of this component is to compare and contrast the registries, and to understand barriers, enablers, views of clinicians and consumers about rare disease registries in Australia. A sample of rare disease registries data custodians and registry managers will be drawn for qualitative interviews to identify their best practice models. 

The survey tool is currently under development. RVA will be inviting RVA Partner organisations, SMAC and the RVA Round Table of Companies to take part in this survey soon.

Part 3: Final report integrating all findings.

RVA will report more on this important work as it progresses. We are also pleased to welcome Professor Susannah Ahern to briefly speak about the project as part of the 2021 Virtual National Rare Disease Summit on 12 November.

This audit is a critical first step and will potentially help leverage other important work in the sector on this issue. We anticipate that this work will be completed in the first half of 2022. If you would like to learn more about this project, please contact RVA and stay tuned for updates.

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.

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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.

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.

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: [email protected]

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 [email protected] 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

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:

• Funding for new medicines
• Medicines Supply Security Guarantee  
• HTA review  
• Enhanced Consumer Engagement Framework

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.