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ARCS Summit Round Up - Stream 'B' - Real World Evidence & its impact on clinical, regulatory and market access

ARCS Summit Round Up - Stream 'B'

Real World Evidence & its impact on clinical, regulatory and market access​

B1.Introduction - What’s happening in the Real World?

“What’s happening in the Real World?” is the topic of the 2nd session at this Monday morning's ARCS Summit. There’s discussion on Real World Evidence & its impact on clinical, regulatory and market access.


Real World Data

are data relating to patient health status and/or the delivery of healthcare routinely collected from a variety of sources.

Real World Evidence

is the clinical evidence regarding the usage and potential benefits or risks of a medical product derived from analysis of RWD.

There’s an increased demand for RWE/D

due to advances in personalised and precision medicines; growth in rare and orphan pipelines and accelerated approval pathways.

RWE is an invaluable complement

to Randomised Trials (RCTs ie the gold standard) and the combination of both would become the 'Platinum Standard' ie 'the integrated evidence solution'.

RWE will assist in bringing innovative new medicines

to patients as it will highlight the true value of the treatment.

Engagement with all stakeholders

will facilitate the understanding of the value of RWE and drive transformational change.

Brittany Schoeninger
Jan Lewis
George Papadopoulos

B2. Regulatory applications of RWE

Real World Evidence to enhance regulatory submissions is being discussed during Session B2 of Tuesday’s ARCS Summit.


The 21st Century Cures Act (USA)

formalises and systematically expands the use of RWE to support changes to labelling about drug product effectiveness.

The European Medicines Agency

is “recently showing an enormous interest in RWE”

China’s National Medical Products Administration

has firm guidance with regards RWE.

A growing number of regulatory decisions

are informed by RWE in a range of therapeutic areas eg anti-psychotics, oncology, and rare diseases.


is providing important insights in further understanding COVID. Protocols are available at Clinicaltrials.gov

In TGA pre-market prescription medicines evaluations

RWE is used on a case by case basis; and generally as an adjunct to support other data. For example, for rare diseases; in severe/life threatening disease (where a placebo group is not possible); and for post market safety data analysis.


is used in pharmacovigilance signal detection monitoring and signal confirmation.

Claire Behm
Nancy A. Dreyer
Tam Nguyen
Monique Stone
Brittany Schoeninger

B3. RWE and market access

Innovative product launches require innovative evidence solutions. Real World Evidence (RWE) vs “gold standard” head-to-head randomised controlled trials is the topic of Session B3 at Wednesday morning’s ARCS Summit.

“The PBAC has always used the best evidence available (RWE and RCTs)”

“The PBAC is trying to manage the risk implications of using poor quality data”

When using RWE

decide the purpose of presenting the data. If robust RCT data is available consider any additional value that RWE would present.

Cell and gene therapies

hold great promise for patient care. However there are also significant uncertainties in patient populations, outcomes and comparative benefits.


have received conditional approvals. HTA agencies expressed concerns over uncertainties in clinical data; therefore early engagement with HTA agencies to plan for the generation of RWE is critical.


has a major role in ensuring that the value proposition of PBAC investment is realised in practice.

Greg O'Toole
Katrina Lapham
Andrew Wilson
George Papadopoulos

B4. Spotlight on execution: RWE challenges & future solutions

Innovations in Real World Evidence, is the topic of this Thursday morning’s ARCS Summit Session “B4. Spotlight on execution: RWE challenges & future solutions”.


The EVENT project (Evaluation of anticoagulation for venous thromboembolism in the community)

created a RWE data linkage project between the hospital and the community to provide better patient outcomes. This approach can be expanded into other areas to prevent avoidable hospital re-admissions.

Other areas that would benefit from a specialty-community data linkage approach

include cardiovascular, diabetes and respiratory with the aim of improving the quality of life of discharged patients and to reduce the burden on the health system.

Innovations for RWE data collection include:
  • Front door e-consent (upfront permission to use data at a later stage)

  • Remote monitoring using connected sensors (offers a more holistic view of a person's lived experience)

Patient-generated data

(Patient portals, personal health records and electronic medical records) can be leveraged to generate RWE.

Liliana Laranjo
Michael Winlo
Mike Da Gama
Brittany Schoeninger

B5. RWE Roundtable: The future of RWE in Australia

Over the last 5 days we have been hearing about ground-breaking Real World Data & Evidence innovations. Session B5 continues the discussion.


RWD is being used for planning

understanding market trends; how medicine is being used; understanding patient demographics and local environments; understanding current treatment algorithms; and for validating control arms.

More products are coming to market

with more early phase data (especially for rare diseases) and RWD provides additional insights around optimal treatment.


complements other pharmacovigilance reporting systems eg spontaneously reported adverse events.

RWE is becoming more complicated

because we are gathering more data, and the challenge is how the data can be optimally leveraged.

Emerging sources of RWD

include electronic patient reported outcomes and digital wearables.

Predictive and prescriptive analytics

using RWD and machine learning improves patient outcomes.

Integration and coordination

of healthcare systems will facilitate the capturing of RWD.

Margaret Wilson
Mike Da Gama
Jan Lewis
Monique Stone
Andrew Wilson
George Papadopoulos
Brittany Schoeninger