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.
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.
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.”
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.
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.