Shining light on treatment benefit: How Roche is leveraging Real World Data to improve patient outcomes

Niko Andre, Head of Global Medical Affairs

Real World Data (RWD) are becoming increasingly important in assessing the benefits and risks of new medicines, helping to inform healthcare reimbursement decisions worldwide. Several initiatives are ongoing to enhance our RWD knowledge and capabilities at Roche. We asked Niko Andre, Head of Global Medical Affairs, for his perspective on how RWD can be used to improve pharmaceutical development and medical practice.

What are Real World Data (RWD) exactly?

RWD are data collected from patients treated with widely available medicines in a regular clinical setting. As data collection and storage capabilities are now so advanced, RWD are not limited to patient registries. They can be generated in a number of ways; through analyses of the patient databases from insurance companies or healthcare providers, for example.

Randomised Clinical Trials (RCTs) have been used for a long time to assess the safety and efficacy of medicines. What issues are RWD expected to address?

RCTs will remain an essential part of proving that a potential treatment provides added benefit relative to the standard of care – in particular for demonstrating the risk-benefit ratio of new compounds. Yet there are limitations on these data as they are generated in highly selected patient groups, so not entirely representative of the general population; the elderly or fragile may be excluded, for example. RCTs also occur within a limited time frame and don’t allow insight into long-term clinical effects or the effects of multiple treatments. These data gaps may be addressed with RWD; providing a holistic view on the real patient population and long-term impact of treatment.

RWD seem to have become a “hot topic” in Pharma in a relatively short period of time – why is that?

New medicines undergo intense evaluation prior to being granted regulatory approval. In the past, the primary focus has been on the risk-benefit ratio of a new medicine to ensure its safety and efficacy. Today, regulators and payers increasingly assess whether a new drug is adding significant benefit over an established standard of care, and whether the price of the drug correlates with a positive health impact for the patient. Individual countries may also require locally generated or collected data to help inform their reimbursement decisions. Answers to these questions in a practical, real-life setting have only recently been feasibly addressed by RWD.

What benefits are RWD expected to bring to the industry, and ultimately to patients?

If used appropriately, RWD may be an enormously powerful source of information, providing greater understanding of medicines, diseases and their progression, and the impact of change in treatment standards over time. RWD may also detect new safety signals and explore the real, long-term impact of medicines on patient health.

What are the major challenges that face companies wanting to generate and analyse RWD and incorporate them into their operation?

Compared to RCTs – where data are generated in very well defined conditions – RWD data sources (e.g. registries, treatment databases, online patient platforms, etc) vary widely in both quality and reliability. So RWD evaluation comes with a certain level of risk.

What is Roche’s vision for RWD and what is it doing to make this vision a reality?

At Roche, we aspire to be a leader in utilising RWD. To ensure that we are using RWD in the most appropriate and accurate way, we have established a ‘Real World Data Science’ (RWD-S) group within Product Development. The group consists of data-science experts who provide guidance to the organization and cross-functional teams on how to best use the potential of RWD, enable better decision making and ultimately improve patient care. RWD is still a relatively young field, but we believe it will become a hugely important step towards achieving optimal patient benefit.