With the aim of delivering better health for all Australians, the report from the Parliamentary inquiry into the approval processes for new drugs and novel medical technologies in Australia has been delivered, with a particular focus on treatments of rare diseases and conditions where there is a high and unmet clinical need.

The national approach to reforming approval processes should produce outcomes that simplify and streamline processes to benefit all Australians ultimately.  ACTA is pleased to see good alignment across many areas of our response submission. 

The inquiry recommendations

The inquiry focused on approval processes for new drugs and novel medical technologies, primarily for treating rare diseases and conditions of a large and unmet clinical need. In alignment with the recommendations provided in ACTA’s submission, the Committee recommended that the Australian Government continue to fund Clinical Trial Networks (CTNs)  with a particular focus on developing seed funding for Indigenous Health CTN. Further, the Committee recommended the establishment of a Centre for Precision Medicine and Rare Diseases within the Department of Health, which aims to provide Australians with access to new drugs and novel medical technologies, in parallel with the Health Technology Assessment (HTA) process and Government research agenda, and improved patient input to decision-making.

ACTA welcomes recommendations for improving the HTA framework and a review of repurposing drugs.  The HTA framework, including increasing the use of real-world evidence, strongly aligns with ACTA’s advocacy work around a pre-investment scheme to fund trials of new and emerging interventions where effectiveness, comparative effectiveness or cost-effectiveness have not been established.  While the recommendation around incentivised pathways for repurposing specifies industry, this is highly relevant to investigator-initiated clinical trials.  Greater funding in this area would allow the sector to assess off-patent drugs that can be used to treat a broader spectrum of diseases.

Additionally, the Committee calls for the implementation of a nationally harmonised platform for Human Research Ethics Committee and Site-Specific Assessment submissions, which supports the efforts to achieve a national, interconnected, rapid and streamlined approvals platform through the National One-Stop-Shop.

There was also a heartening recommendation around equity of access in rural, remote and regional areas, with the Committee calling for a national standard approach to support and strengthen the capacity to conduct clinical teletrials.  This strongly supports ACTA’s current program to improve the efficiency and effectiveness of clinical trials and overcome known barriers to participation.

About the inquiry

The inquiry began in August 2020 and considered four key topics that would allow Australia to continue to be well-positioned to access new drugs and novel medical technologies in a timely manner and respond to emerging global trends.

ACTA submitted a response to the Parliamentary Inquiry in November 2020.  ACTA Director, Prof John Zalcberg OAM, also appeared virtually before the House of Representatives Standing Committee for Health, Aged Care and Sport (the Committee) during a Public Hearing in May 2021.

The inquiry Committee, chaired by Mr Trent Zimmerman MP, recently delivered a report with 31 recommendations, following the review of over 200 submissions and public hearings held over 13 days.  ACTA also congratulates the Committee on a rigorous and bipartisan approach to improve and streamline approval processes, improving trial access for patients and across all diseases.

ACTA's recommendations

ACTA’s key recommendations in response to the consultation were:

• Recommendation 1: Further increase funding to Clinical Trial Networks (CTNs) to ensure core infrastructure support so that CTNs can operate optimally to address not only the investigator-initiated questions but also develop mechanisms to liaise with industry.
• Recommendation 2: Enable pathways for the optimal utilisation of CTNs by promoting that new drugs and novel medical technologies are provided to CTNs.
• Recommendation 3: Increase the amount of funding available to investigator-led clinical trials, which will allow the sector to address conditions where there is an unmet need, in particular orphan, personalised drugs and off-patent that could be repurposed and used to treat new conditions.
• Recommendation 4: Establish a rigorous pathway for treatments, services, and technologies unproven in the real world to enter practice as quickly as possible through a conditional scheme.  This scheme would require participation in either a trial conducted by a Clinical Trial Networks (CTNs) or Clinical Quality Registries (CQRs) capable of generating important real-world data about the clinical effectiveness and value of the intervention in the real-world context. We believe that this would enable the Government to acquire more data, enhance patient access and enable doctors to gain invaluable experience with new drugs and medical technologies. Such an arrangement would enable Australian healthcare to be evidence-based as well as self-learning.
• Recommendation 5: Conduct a review of potential reforms to the Medicare Benefits Schedule (MBS) to facilitate the better generation of real-world evidence to improve outcomes and deliver value gains.  The review should consider ways to use savings generated through investigator-initiated trials (IITs) and CQRs as a means of funding these activities.
• Recommendation 6: Expand the current Parliamentary Inquiry to include all medical interventions, including tests, procedures, devices and new drugs.

What’s next?

The recommendations will now be considered by Federal Government.  One item that ACTA believes will need further consideration is parity in Network funding.  Currently,some CTNs receive core funding from the Federal Government, while others do not. Greater core funding for CTNs and CQRs is essential for ensuring better outcomes for Australians through high-quality clinical trials and evidence-based healthcare.

ACTA looks forward to continuing discussions with the Department of Health about the inquiry recommendations and opportunities for further involvement, on behalf of our Members.

View the Inquiry report: https://www.aph.gov.au/Parliamentary_Business/Committees/House/Health_Aged_Care_and_Sport/Newdrugs/Report

View ACTAs submission: https://clinicaltrialsalliance.org.au/latest-news/parliamentary-inquiry-into-approval-processes-for-new-drugs-and-novel-medical-technologies/