1. Introduction

Australia’s regulatory system for bringing new medicines and devices to patients is regarded as being thorough and robust and is well respected internationally. Australians should be proud of our healthcare system. The Committee heard this from stakeholders, including the pharmaceutical industry, patient advocacy groups and clinicians throughout the inquiry. Many witnesses congratulated the staff working within the Department of Health, including the Therapeutic Goods Administration (TGA) for their professionalism and dedication working on the regulation and reimbursement systems.
Along with this praise came suggestions for improvements to make Australia’s healthcare system even better. A significant challenge for Australia’s regulatory system was to establish more flexible pathways to enable our system to keep pace with medical and technological advances, including precision medicine, that are available now.
Numerous stakeholders raised the issue of the length of time it takes for a new medicine to get approved and listed on the Pharmaceutical Benefits Scheme (PBS). The Australian system was compared with other international regulatory systems and the findings were variable depending upon which factors were included for comparison. It became clear to the Committee that international regulatory systems are all unique and complex.
Access to medicines and therapies for rare disease and precision medicine was discussed as a significant challenge that required solutions to enable more equity for patients. Some of the challenges for rare disease and precision medicine access raised issues relating to research and development, clinical trials and the status of using real world evidence.
The Committee launched this inquiry in August 2020 just months after the declaration that the world was living with the COVID-19 pandemic. At the time that the Committee was reviewing Australia’s regulatory system, the Therapeutic Goods Administration and the Department of Health were fast tracking approval processes for certain drugs to assist with the treatment of COVID-19 patients in Australia. Many submissions noted this unprecedented collaboration with international and Australian regulators, pharmaceutical companies and clinical researchers. It was suggested that lessons could be learned from the pandemic and that our regulatory systems should be streamlined and adapted to cope with the flood of new healthcare innovations coming in the near future.
The Committee was mindful of the increasing globalisation of the pharmaceutical and medical devices industries and the rapid pace of innovation and change within the healthcare sector and how this impacted heavily on Australia’s regulatory system. In addition, the Committee recognised that Australia’s ageing population and growing burden of chronic diseases reinforced the importance of continued and ongoing investment in the timely access to new medicines and devices.
Stakeholders urged the Australia Government to consider the recommendations from this report together with the National Medicines Policy Review that recommenced in August 2021. These two reviews present an opportunity to continue this collaborative approach to reform and work towards a more streamlined system to access medicines and devices in Australia. This report lists the recommendations in the final chapter.

About the inquiry

Objectives and scope

On 13 August 2020, the Minister for Health, the Hon Greg Hunt MP, referred the Inquiry into the approval processes for new drugs and novel medical technologies in Australia (the inquiry) to the Standing Committee on Health, Aged Care and Sport (the Committee). The inquiry included a particular focus on approval processes and novel medical technologies for the treatment of rare diseases and conditions where there is high and unmet clinical need.
As part of the inquiry, the Committee examined the range of new drugs and emerging novel medical technologies that are in development and progressing through the regulatory system in Australia and in other countries of the world.
Other focus areas included:
Examining the approval processes of new drugs and medical technologies including whether these processes could be made more efficient without compromising safety, quality and efficacy
Measures that could make Australia more attractive for clinical trials; and
Incentives to research and commercialise new drugs and novel medical technologies.
The Committee appreciated receiving informative submissions from individuals, family members, patient advocacy groups, and peak bodies from small and large disease/patient groups who spoke of changes that were needed to make Australia’s access to new drugs and medical devices more equitable and efficient. These submissions provided the Committee with insights into the importance of incorporating ‘the patient voice’ into the approval process.
The Committee thanks all stakeholders who were generous with their time and patience in bringing the Committee up to speed with Australia’s regulatory and reimbursement system. This inquiry was complex and technical and required the Committee to have a comprehensive understanding of the system before it could consider making any recommendations to adjust it.

Inquiry conduct

On 18 August 2020, the Committee issued a media release announcing the inquiry and calling for submissions. The Committee invited submissions from government agencies, industry groups and pharmaceutical companies, research centres and universities, patient advocacy groups and healthcare providers, and the general public.
The inquiry received 207 submissions and an additional 30 supplementary submissions and one exhibit, which are listed in Appendix A and B.
The Committee held public hearings over 13 days, as outlined below. A list of witnesses and organisations who attended these public hearings is listed in Appendix C.
Table 1.1:  Public hearings held
3 September 2020
Canberra ACT
5 February 2021
Canberra ACT
11 March 2021
Sydney NSW
12 March 2021
Sydney NSW
26 March 2021
Canberra ACT
22 April 2021
Melbourne VIC
23 April 2021
Melbourne VIC
7 May 2021
Sydney NSW
17 May 2021
Brisbane QLD
18 May 2021
Brisbane QLD
18 June 2021
Canberra ACT
24 June 2021
Canberra ACT
7 July 2021
Canberra ACT

Report structure

This report consists of eleven chapters. The final chapter is a list of recommendations:
Chapter 2 provides a general overview of the recent reviews conducted, and the agreements entered into, by the Australian Government in relation to Australia’s health programs and regulatory frameworks, which have had a bearing on the Committee’s deliberations and subsequent recommendations in this report.
Chapter 3 presents a high level overview of the regulatory and reimbursement frameworks, the general understanding of how these systems work, and where there are gaps in the system.
Chapter 4 describes the concept of the ‘patient voice’, how it is currently drawn on in decision-making by Australia’s Department of Health and in overseas models, and what further improvements to government engagement with the patient voice could look like.
Chapter 5 provides an overview of the Therapeutic Goods Administration including the general themes to have emerged throughout the inquiry, including the regulation of medicines and medical devices, and the financial and technical aspects of its regulation.
Chapter 6 outlines the Heath Technology Assessment (HTA) system. It discusses the processes of the Pharmaceutical Benefits Advisory Committee. Some of the main issues discussed include: the application process, length of time for review, fees, provisional access and international regulators.
Chapter 7 explores the Medical Services Advisory Committee, another advisory committee in the HTA system that focuses on medical devices and services. Again, issues of flexibility, length of time for review, resourcing and application processes are discussed, as with its approach to real world evidence. The chapter also looks at the Prostheses List Advisory Committee and the future of the Prostheses List.
Chapter 8 explores the important issue of rare disease, focussing on Government initiatives, potential HTA alternative pathways, the Life Saving Drugs Program, newborn screening and limitations on data, research and clinical trials.
Chapter 9 looks at clinical trials in Australia including our regulations and challenges, why we have a competitive advantage, and discusses what is needed for Australia to be ready for a surge in demand for novel medicines and devices in the clinical trial sector.
Chapter 10 discusses research and development in Australia and what the Australian Government is doing to fund initiatives, what research incentives are available, the need for further and greater horizon scanning, and the regulatory hurdles attached to the repurposing of drugs.

 |  Contents  |