Health Regulation

The 'New Frontier - Delivering better health for all Australians' report by the House of Representatives Standing Committee on Health, Aged Care & Sport covers the Committee's Inquiry into approval processes for new drugs and novel medical technologies in Australia. It picks up a submission by myself and Dr Wendy Bonython (Bond University) and my testimony at a hearing of the Committee.

The Committee offers the following recommendations 

 Recommendation 1 

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

• 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, and that the HTA process and government research agenda aligns with this outcome. 

• The Centre should provide advice to the Department of Health and the Australian Medical Research Advisory Board on research priorities. 

• The Centre should provide education and training information including support for patients and a comprehensive horizon scanning unit for new medicines and novel medical technologies. 

• 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. 

Recommendation 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.

• This simplified process should be considered together with a new HTA pathway for cell and gene therapy. 

• Building on the Medical Research Fund Genomics Mission, the Australian Government and state and territory governments should 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. 

• The Australian Government should prioritise and simplify the regulation of cell and gene therapy pathways for clinical trials in Australia.

Recommendation 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.

• The Office should enable evaluation of both pharmacological and non-pharmacological interventions, combination products and products with different sponsors. It should also establish a “living evidence” function to ensure Health Technology Assessment is based on the most up-to-date global health practices. 

• The Office, in consultation with relevant stakeholders, should conduct a review of how the Department’s Health Technology Assessment system assesses combination products, particularly combinations with different sponsors, with a focus on: - Value attribution between the different products - Challenges to cooperation between sponsors due to competition law - Disincentives for a sponsor with an already listed product to participate in its combination listing 

• The Office should consider collaboration with the National Institute for Health and Care Excellence (NICE) in the United Kingdom to establish similar clinical evaluation processes in Australia that links in with Australian Health Technology Assessment processes. 

• The Office should cooperate and share information with the state and territory governments to ensure that patients receive treatment where it is safest and most efficacious for them and that there are no gaps in continuity of care. 

Recommendation 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. The Committee recommends either:

• Providing sponsors with an immediate pathway to the LSDP Expert Panel (instead of waiting for a PBAC determination), or 

• Providing a pathway by adjusting the Pharmaceutical Benefits Scheme section 100 program, with specific criteria, as with other section 100 programs.

The Committee believes it is critical that consideration be given to how the LSDP will integrate with an increasing number of precision medicine applications into the future. 

Recommendation 5   

The Committee recommends that the Australian Government develop a labour market and skills strategy to expand the number of health economists in Australia. This could include encouraging training within Australia as well as seeking expertise from overseas. 

Recommendation 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.  The Committee recommends that the Department of Health improve information available on the websites of the Therapeutic Goods Administration (TGA) and its Health Technology Assessment (HTA) bodies for all users including patients, clinicians, industry and the public. This would include:

• Using plain English language, infographics and videos to explain general processes and timelines 

• Explanations on the TGA and all HTA’s websites of how that entity fits into the overall regulation and reimbursement system, similar to the Medical Services Advisory Committee’s Australian Government HTA Processes factsheet. 

• The Department of Health expanding the Pharmaceutical Benefits Scheme Medicines Status website to include technologies funded through the Medicare Benefits Schedule or create an equivalent website for such technologies. 

Recommendation 7   

The Committee recommends that the Department of Health and the National Blood Authority, in consultation with state and territory governments, reform the Health Technology Assessment processes for blood products to provide better alignment with the Health Technology Assessment system, including:

• Publication of guidance documents for applicants 

• Establishment of timelines for applications, and publication of an assessment cycle calendar 

• Creation of a parallel Therapeutic Goods Administration and Health Technology Assessment process. 

Recommendation 8   

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   

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. 

• The fund should be annually capped with clear and transparent eligibility rules. 

Recommendation 10 

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   

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. This should include: 

• Establishing a new pathway that incentivises the repurposing of drugs for all diseases, not just rare disease. 

Recommendation 12   

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   

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 14   

The Committee recommends that the Australian Government reconsider the current cost recovery funding model for the Therapeutic Goods Administration, paying attention to future staffing and IT infrastructure needs in an environment where demand on its services and systems are expected to increase in future years. The Committee recommends funding specifically for: 

• IT systems upgrades, to modernise and match the IT capability of other overseas Tier 1 regulators. 

• An expansion of its staffing capacity in areas of new medical and technological advances including for horizon scanning. 

• The release of TGA Australian Public Assessment Reports at the same time as a prescription medicine is listed. 

• The implementation of the HECS-style fee waivers outlined in Recommendation 8. 

Recommendation 15   

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. 

• Recognising the nature of health challenges in Indigenous communities, membership should include representation from Aboriginal and Torres Strait Islander Peoples. 

Recommendation 16   

The Committee recommends that the Department of Health investigate further opportunities for the formation of an international Health Technology Assessment consortium similar to the Access Consortium to streamline the regulatory process for certain medicines and medical technologies. This investigation should include discussions with representatives of the Health Technology Assessment bodies of the United Kingdom, Canada and other countries with systems similar to Australia’s.

• The Committee recommends that the Therapeutic Goods Administration work with the United States Food and Drug Administration and other overseas regulators to establish an equivalent of Project Orbis for non-cancer rare diseases, or to expand Project Orbis to include such diseases. 

Recommendation 17   

The Committee recommends that the Australian Government establish a scheme that supports the domestic medical technology sector, similar to the Food and Drug Administration’s Breakthrough Devices Program in the United States. 

Recommendation 18   

Recognising the vital role that vaccines play in addressing many diseases, including its importance in providing protection against Covid-19, the Committee recommends that the Department of Health conduct a review of the National Immunisation Program. This review should focus on reforming existing approaches used to value vaccines to ensure early and rapid deployment of vaccines in Australia. 

Recommendation 19 

The Committee recommends that the Australian Government continue to address the following matters in its reforms to the Prostheses List:

• The lack of coverage for non-implantable devices under the current arrangements. 

• Improving coordination between the Medical Services Advisory Committee and the Prostheses List Advisory Committee to provide faster access for patients. 

Recommendation 20   

The Committee recommends that the Australian Government establish a last resort mechanism for directly securing ongoing supply of medicines that meet a high clinical need and lack suitable alternatives that are at risk of being delisted from the Pharmaceutical Benefits Scheme. 

Recommendation 21 

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 

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.

• The platform should be developed within the purview of the Australian Commission on Safety and Quality in Health Care. 

• This work should be a continuation from the work prepared as part of the National Clinical Trials Governance Framework. 

Recommendation 23 

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. 

• Linked data from existing national registers and consideration should be given to whether the register is best operated by a government agency or an existing Non-Government Organisation, or an academic body with appropriate experience. 

Recommendation 24 

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, e-signature, 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 

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. 

• This approach should be developed in consultation with industry and allied health workers. 

• This would include the need for education and training opportunities for General Practitioners and all allied health workers engaging in clinical trials using tele-trials and multi-centre trials. 

Recommendation 26 

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 

The Committee recommends the Australian Government reform data exclusivity provisions in Australia with a view to extending data exclusivity for orphan drugs and vaccines to a period of up to 10 years. 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. 

• In partnership with the states and territories, develop and implement a pilot scheme for value-based payments for new antimicrobial drugs. This pilot should apply the lessons learned from the Australian Government’s pilot scheme for payment for Hepatitis C drugs, as well as from overseas antimicrobial drug schemes. 

• Promote the recent research and development tax initiatives internationally as a way of encouraging industry to look to Australia for future investments in the healthcare sector. 

• Conduct a full review of the patent box scheme every two years after implementation to ensure it is operating effectively and driving increased expenditure and innovation within Australia. 

• Collaborate with the states and territories to review the funding of the research and development sector in health care to distribute funding in a methodical way that provides sufficient support throughout the research funding ‘pipelines’. 

• Noting the work underway through the Modern Manufacturing Program, the Committee supports the development of an updated roadmap to facilitate the manufacturing and commercialisation of novel drugs and technologies in Australia. 

Recommendation 28 

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 

The Committee recommends that:

• The Committee recommends that the Australian Government amend the National Health Act 1953 (Cth) to formalise the role and powers of the Pharmaceutical Benefits Advisory Committee Executive. The scope of the Executive’s role and powers should be determined by agreement between the Executive and the Department of Health. 

• 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. 

• It is imperative that appropriate clear pathways are considered for inclusion for paediatric medicines and technologies. 

• 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. 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 

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 

• Cooperation and harmonisation between Australian Health Technology Assessment bodies and equivalent bodies overseas 

• 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 

• Improving flexibility when choosing a comparator 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. 

Recommendation 31 

The Committee recommends that:

• The Department of Health should consider, in consultation with state and territory governments, industry, patients and clinicians, the introduction of fees for Medical Services Advisory Committee applications on a cost recovery basis, if this is necessary to increase the speed and effectiveness of assessments. If fees are introduced they should have similar features to those recommended by the Committee for Pharmaceutical Benefits Advisory Committee fees (including those arrangements outlined at Recommendation 8). 

• The Medical Services Advisory Committee increase the involvement of clinicians in its assessments of technologies with which its members lack relevant expertise. 

• The Department of Health introduce an equivalent to the Managed Access Programs for medical devices. The details of this scheme including eligibility criteria and duration should be formulated in consultation with patient groups, clinicians and industry. 

• 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 Medical Services Advisory Committee consider developing international collaboration for complex assessment proposals. 

• 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 Medical Services Advisory Committee publish additional guidance for sponsors of digital health technologies. 

• The Department of Health establish a benchmarking system for MSAC assessments, including benchmarking against comparable overseas organisations