Over the last 18 months many Australians have observed with awe and admiration the incredible work of medical scientists in finding vaccines and new treatments to a virus that has taken the lives or impacted the health of millions around the world.
Many of the innovations and medical understandings developed during the COVID-19 pandemic will have long-term benefits for health treatments for other conditions beyond COVID-19.
These innovations reflect the new frontier of medicine which is giving many hope for better treatments and technologies for conditions ranging from cancers to rare diseases. At its forefront is the development of personalised or precision medicine which is being delivered as our understanding of fields like genomics grows.
This report examines the opportunities to deliver better health care for Australians through our regulatory and health technology assessment process for both medicines and technologies.
At its heart are the needs of patients - Australians who are born with or who acquire conditions, many of which have so far eluded highly effective treatments. Everything in this report is about providing better options and hope for Australians with medical conditions.
Australia has long prided itself on having one of the world’s best health systems. By any measure we do. Our success in protecting Australians during a global pandemic is the latest evidence of both the strengths of our health care system and the quality and dedication of all those who work in health care.
However, no nation and no health system can rest on its laurels. With innovation happening at a fast pace, governments at both the state and federal level have a duty to ensure that Australians continue to have access quickly to medicines and medical technology and that our health systems facilitate that outcome rather than hinder it. Australians can also benefit by being at the forefront of innovation through clinical trials and a strong domestic research, development and manufacturing capacity.
Medical innovation has grown exponentially in recent years and pharmaceutical and Medtech companies are eager to bring new medicines and devices to market as efficiently as possible. The Committee also heard from clinical experts and patient groups and their families who urged us to support a more flexible system to provide for timely access to the latest medicines, devices and treatments.
One of the challenges facing the existing system is the trend towards delivering precision medicine to patients. Precision medicine is an emerging approach for disease treatment and prevention that takes into account individual variabilities in genes, environment and lifestyle for each person. This offers great hope for patients from a broad spectrum of conditions and diseases, including patients with rare diseases. However, these developments were not envisaged when the current regulatory and reimbursement system was designed and legislated.
The Committee recommends the creation of a Centre for Precision Medicine and Rare Disease within the Department of Health, to provide advice on research priorities, education and training for clinicians and patients, and the development of a comprehensive horizon scanning unit for new medicines and novel medical technologies. The Committee also recommends that a new pathway for cell and gene therapy be established to simplify the Health Technology Assessment (HTA) processes.
The Committee heard from patients and their families about the need for more patient involvement in the approvals decision-making process for new drugs and novel medical technologies. Patients have a crucial perspective on what treatments work best for them, including important lifestyle benefits, but this has traditionally not been given enough attention within the regulatory and reimbursement system. The Committee recommends reforms that will strengthen the central role of patients in the assessment system.
Many submitters to the inquiry suggested that there is little measurement and publication of how well the regulatory and reimbursement system is performing. The Committee believes this should be more transparent and recommends the Department of Health annually publish data on HTA processing times and benchmark these against other nations with advanced HTA processes.
The Committee heard from patients and clinicians who were frustrated that some medicines and technologies are available overseas and not in Australia, with companies seemingly deciding not to sell their products in Australia for commercial reasons. This is a particular issue that arises for orphan drugs and drugs for rare diseases. The Committee recommends changes to encourage companies to enter the Australian market with their products and technologies. This includes changes to the fee structure for applications to the TGA and HTA processes – particularly for orphan drugs and smaller companies, including Australian start-ups.
The Committee also recommends the creation of an annually capped fund with clear and transparent eligibility rules to provide funding for applications by patients, clinicians and non-profits, where there is no realistic prospect of a company serving as a sponsor.
The approval processes for new medicines and novel medical technologies are very complex, and this report discusses different ways to streamline them to provide better and faster patient access to treatments. While it is often difficult to achieve this without compromising on patient safety, efficacy or cost effectiveness, the Committee believes there are areas where major changes are necessary and possible. One example of this is the Life Saving Drugs Program (LSDP) for treatments for very rare diseases, which despite the urgent patient need, currently requires a lengthy two-step application process. The Committee recommends that this process be streamlined into a one step process to establish a new pathway to the LSDP Expert Panel or to establish an alternate pathway by adjusting the Pharmaceutical Benefits Scheme section 100 program.
Another cause of complexity in the approvals system for medicines and medical technologies is the interaction between the Commonwealth and the states and territories. The Committee found that there are several areas where the Australian Government can work better with the states and territories. An important example of this is newborn screening, which has the potential to ensure early intervention and more accurate diagnosis. The Committee recommends that the Australian Government lead efforts to complete the standardisation of this screening across the country, based on new understandings of genomic testing, and to review the newborn screening program every two years to keep pace with new medical developments.
Clinical trials are another area where Australia has considerable strong comparative advantages. Ensuring Australia remains a top-tier country for trials not only develops our own research capacity but, more importantly, can ensure early access to life changing drugs and technologies.
The Committee has recommended changes to streamline the system and ensure Australia is an even more attractive location for clinical trials. These include the immediate harmonisation of ethics and governance approvals into one online platform and the establishment of a national clinical trials register.
The research and development (R&D) of new medicines and medical technologies attracted considerable attention during this inquiry, and the Committee makes a number of recommendations to support stronger and more collaborative R&D. Patient groups advocated strongly for the repurposing of existing medicines to treat alternate disease or conditions. The Committee recognises this is an area that requires a more flexible vision for the future and recommends the establishment of a new pathway that incentivises the repurposing of drugs for all diseases.
This report is being delivered in an ever-changing environment. The Australian Government is reviewing the National Medicines Policy (NMP) and a further major review of HTA processes has been announced. It is our hope that many of the recommendations in this report can be implemented in the short-term and not await the outcome of these further reviews. We have also identified medium term issues that should be central to the HTA review.
It was clear to the Committee that there was a great deal of momentum behind the push to improve the regulatory and reimbursement system — not just a general desire for change, but a wealth of ideas for reform and a willingness to make the efforts and compromises necessary to implement them. The Committee hopes that this report captures those ideas, and paves the way for the improvements needed to provide Australians with the best possible health care now and into the future. Indeed, the Committee inquiry has already triggered change as government agencies have heard and considered the evidence we received.
I want to thank everyone who took the time to give evidence to this inquiry.
We were moved by the testimony of patients and their families and inspired by the work of our researchers and medical scientists. We were impressed by the professionalism of those working in the medicines and technology sectors and appreciative of the obvious dedication, co-operation and knowledge of those within the Department of Health who assisted our deliberations in public and private hearings and through their submissions.
I would also like of thank my fellow Committee members for their close engagement and their knowledgeable contributions that each member made to this inquiry. In particular, I wish to thank the Deputy Chair, Dr Mike Freelander MP, for his expertise, good judgement and good humour. In an area of such significance, the fact that we have emerged with a bipartisan and unanimously adopted report speaks to the commitment of all Committee members.
Finally, I want to thank our committee secretariat staff, particularly Kate Portus, Rebecca Gordon and Peter Richardson. This was the largest inquiry undertaken by the Committee during my five years as Chair and they have supported our work with exceptional dedication and quality – and occasionally some patience and forbearance!
The new frontier of medicine and technology is an exciting one for the health care we provide as a nation. Acting now to build on our obvious strengths in health will have enduring benefits for all Australians.