New report charts future course for increased generation and adoption of Real-World Evidence (RWE)

11 November 2020: A new report, released today by Medicines Australia, draws attention to important opportunities for researchers, industry and the Australian healthcare system to fully realise the benefits of applying ‘Real World Evidence’ (RWE) to health technology assessment and healthcare decision-making.
The proposed course of action would bring Australia in line with comparable international jurisdictions allowing it to keep pace with the rapid advances in personalised medical treatment, with improved patient outcomes the ultimate goal.

According to the Report, ‘The evolving role of real-world evidence in Australia’, prepared by the Medicines Australia Oncology Industry Taskforce RWE Working Group, real world evidence has the potential to reduce uncertainty and enable more informed, evidence-based decision-making across the healthcare ecosystem. The recommendations of the report are integral to Medicines Australia’s formal submission to the current House of Representatives Standing Committee on Health, Aged Care and Sport ‘Enquiry into Approval Processes for New Drugs and Novel Medical Technologies’.

The Report, developed by a cross-industry collaboration between several of Australia’s leading developers of innovative oncology medicines, draws attention to several critical challenges associated with the planning, delivery and acceptance of RWE. It also considers steps to progress a more standardised systematic approach than currently in place.
‘Rather than being an inferior and less credible form of evidence, real world evidence, from a range of sources, has the potential to substantially increase to our understanding of the effectiveness, safety and, value of new medicines in ways that are simply not possible through other more traditional methods alone,’ stated Prof John Zalcberg, a medical oncologist from Victoria.
‘Whilst RWE cannot replace the scientific rigour associated with traditional approaches, such as the randomised controlled trial, it can prove vital as a source of valuable information when overcoming some of the practical, ethical and economic challenges the emergence of precision (or personalised) medicine have raised for us when attempting to bring new innovative and effective medicines to patients-.’ Prof Zalcberg added. ‘This can lead to lengthy delays as attempts are made by sponsors to bridge these gaps.’

The Report identifies two critical challenges to address, as a first step, in developing a long-term strategy for consistent adoption of RWE:

  • Methodological – the absence of agreed frameworks, methods and standards has impeded advances in the delivery of fit-for-purpose RWE
  • Procedural – opportunities may exist for pre-PBAC submission processes that assist in planning for non-RCT data collection and RWE generation that could support early access in areas of high unmet medical need and help align value and price

Against these challenges, the Report authors propose clear and immediate steps for improvement:

  • Generating an aligned view on the issues through formation of a task force comprising industry, government, healthcare professional, patient, academic and other relevant stakeholder representatives to discuss different views on the topic and to set a framework for the expansion and standardisation of existing guidelines for RWE
  • As one potential output from these discussions, task dedicated sub-groups to create a system to facilitate collection of accurate, structured data on a consistent basis, including applying lessons from similar programs e.g. the Early Access to Medicines Scheme in the UK.

‘Transformational change is always confronting,’ states Dr Amanda Ruth from Rare Cancers Australia, ‘but the status quo really has to be challenged if we are to overcome future delays in access to new innovative medicines that represent opportunities for patients in areas of high unmet need. These are complex matters, but they do require a level of public debate and engagement because the issues are too important to ignore.’

The report is being launched at the Clinical Oncology Society of Australia Annual Scientific Meeting 2020 on Wednesday 11 November 2020 at specific evening conference. The evening symposium – The Evolving Role of RWE – Potential Implications for Access to New Oncology Treatments features Prof John Zalcberg as the chairperson. He is the Head of Cancer Research and NHMRC (MRFF) Practitioner Fellow within the School of Public Health and Preventive Medicine at Monash University and Tony Charlton Chair of Oncology at Alfred Health.

A copy of the Report can be accessed at:

For further information, please contact Anne-Marie Sparrow at Cube on 0417 421 560.

About the ‘Evolving Role of Real-World Data in Australia’ Report 2020

For the purposes of preparing the Report, authors worked within a broad definition of RWD/E in line with the US Food and Drug Administration (FDA), i.e. “healthcare information derived from multiple sources outside of typical clinical research settings, including electronic medical records (EMRs), claims and billing data, product and disease registries, and data gathered by personal devices and health applications.”

The RWE Working Group commission research firm IQVIA to combine a quantitative analysis of information from public summary documents with a qualitative assessment of RWE guidelines and literature, to provide a holistic assessment of how approaches and attitudes to RWE differ in Australia from other major HTA markets of the UK, France, Germany and Canada. The analysis showed the UK and France more often referenced RWE in HTA submissions (55% and 47% respectively), whereas Germany, Canada and Australia show much more limited use: 10%, 6% and 5% respectively.