Rose-Marie Pennisi – Pharmaceutical Manager

With the costs of new product development now estimated to be around $1.4 billion USD, Real World Evidence (RWE) is more important than ever with companies looking for ways to do more with less. RWE) is emerging as invaluable in the clinical evidence landscape and transforming the way we collect clinical data. The evolution of the healthcare landscape is driving the need for change and we need to think differently about evidence approaches.

At the recent ARCS Virtual Summit 2020, a daily feature of the program was exploring different perspectives on RWE. The presentations focused on why RWE is now more important than ever, the Australian landscape and various stakeholder perspectives, the long-lasting impact that COVID-19 has had on RWE and its impact on clinical care, registration and market access applications.

Some of the major trends that are impacting this landscape are today’s significant costs of development, the growth in rare and orphan drugs coming to market (in 2018, these were 58% of FDA approved medicines), advances in personalised and precision medicine where target populations are very small numbers (in 2018, these were 46% of FDA approved medicines) and accelerated approval pathways for innovative medicines.

These trends are resulting in fewer products coming to market with traditional data packages, fewer blockbusters coming to market with common approaches and huge target populations, smaller and more dispersed patient numbers for rare diseases and precision medicines and products coming to market with lighter clinical evidence. These present great clinical challenges and RWE is part of the solution in developing these innovative and smaller population medicines and evolving healthcare.

While randomised controlled trials (RCTs) remain the gold standard, real world evidence (RWE) comes in many forms and has the flexibility to address a broad range of research questions across the life cycle. It’s not about lowering the bar with RWE. It is about having an ethically and practically driven approach that is scientifically robust. By combining RWE and RCTs, we can get the best of both worlds and create higher standards using both. Collection of RWE creates a holistic evidence package that can address limitations and uncertainties and overcome challenges of RCTs by filling data gaps and unmet needs.

With all it offers, RWE does present some challenges which can limit its utility to varying degrees. Key barriers can include the quality of the data collected, lack of standardised processes and methodologies, access to data being challenging due to separate and fragmented sources, governance, privacy issues and lack of experience.

Relative to major developed markets, Australia is earlier in the RWE journey. While the Australian ecosystem contains a breadth and depth of high-quality data, it is somewhat fragmented due to being available in separate sources. These sources include MBS and PBS data in federal systems, hospital data in state systems, claims databases, registries, mobile and digital devices, and commercially owned data sets (e.g., retail pharmacy data), private providers with electronic medical records (EMR) registry-based datasets, academic and research sectors.

So what is Real World Data?

RWD can be generated in early development, pre-launch and in-market phases, and can come from cost-effective sources such as survey questionnaires for healthcare professionals and patients, chart reviews, database studies using retrospective analysis and EMR, prospective, eCRFs and PROs from non-interventional study (NIS) and pragmatic RCTs and use of single arm trial designs as pivotal evidence.

RWE has an immense potential to transform the healthcare landscape and engaging stakeholders in government, regulators, payers, HCPs, policy makers, HTA, patients, patient organisations, industry, academics, researchers, data custodians and HRECs will be key to this integration into regulatory and market access applications to support regulatory decision making, accelerate innovation and enable access to innovative medicines and ultimately drive improvements in clinical care.

There is a realisation that these opportunities will require significant work and collaboration across the healthcare sector, between industry, government and other stakeholders.

One of the speakers used the analogy of Netflix. Netflix is not a one size fits all approach and has revolutionised and personalised TV entertainment. So too will innovative, specialised and personalised medicines require customised, cost-effective, out of the box approaches to development.

Innovative Medicines require Innovative Approaches