News – Apr 20, 2018

Why have RWE trials become "the thing to do"?

Real world data has been around for a very long time, but its significance has not been well understood, and therefore, the potential for improving health care has not been fully utilized.

In an increasing number of cases of drugs/treatments, a different risk/benefit balance was shown in the real world than the one found in highly controlled clinical trials. Even to the extent that drugs were withdrawn from the market or advice on preferred treatments changed. This led to the insight that for many new treatments the evidence at the moment of market approval is insufficient to fully guide decisions by physicians and policy makers to select the best treatment for patients in routine clinical practice. Not only have insights changed, technological advances make real world data also better accessible.

Over the past decades, the use of computers and other electronic tools to gather and store huge amounts of health-related data has been increasing. This data holds the potential to allow a better design and conduct of research in the real-life health care setting to answer questions were previously thought not feasible.

Real World Evidence is also about evidence that matters to patients. Social media is also emerging as a platform for patients to share information about their experiences with particular treatments. Advances in social media are helping to capture more about the patient journey and more specifically, what patients need or want when it comes to what a drug does for them. Social media are increasingly used in observational studies but may also be used in pragmatic trials to collect outcome data.

The real-world data gets us to the next, critical step: real-world evidence. Real world evidence can tell us:

  • What works for real-life patients, with real-life comorbidities, preferences and behaviours, in routine clinical practice?
  • What happens over a patient lifetime, not just in one budget year? Right now, many payers are reluctant to increase access to drugs that have high up-front costs but long-term savings.

In conclusion, pragmatic trials and observational studies can provide an invaluable perspective into real-world value of treatments. While traditional highly-controlled clinical trials are necessary, observational studies and pragmatic trials have an important role to play in the evaluation of health care interventions, to ensure that they are fit for purpose in real world settings.

In part 3 of the series, we will go into more detail about Observational studies.