What's the value of Real World Evidence in the context of the Life Sciences Industrial Strategy?

Reported by CSaP Communications Coordinator Kate McNeil & Alex Kell, EPSRC-funded Policy Intern (September - December 2019)

CSaP, the Office for Life Sciences and the Department of Health and Social Care brought together policy makers, researchers, and experts from industry and practice in the field to discuss the opportunities and challenges of real world evidence in the context of the Life Sciences Industrial Strategy.

Chaired by Helen Munn, Acting CEO of MQ, the discussion on the potential and limits of real-world evidence and its place in the UK’s approach to life sciences was timely, as the United Kingdom faces competition from other life science hubs.

Some argue that the cost and complexity of running clinical research has increased over the last 25 years due to the focus on highly targeted and personalised medicine. These high costs and long lead times have led to the promotion of real world evidence to assess the efficacy and safety of new treatments. At present, the way real world evidence is accepted varies across nations. The UK has access to world leading real world data and has the potential to drive innovation and research in the life sciences sector. The potential use of real-world evidence as an alternative or as complementary to randomised clinical trials (RCTs) was felt to be promising by some, particularly in research areas with long time scales, such as gene therapy, and in cases where rare diseases or precise medicine make RCTs impossible due to the small population size involved. Moreover, it was noted that real world data and evidence could offer more diverse populations than clinical trials, which often have difficulty recruiting participants diverse enough to generate representative samples.

Barriers identified during the workshop included public opinion and consent. According to one study funded by the Wellcome Trust on commercial usage of patient data, the general public does not presently understand how patient data which informs real world evidence is being stored or captured. Meanwhile, other research has shown that the public does have preferences concerning how this data is used, and that current assumptions concerning who is likely to consent to data sharing do not align with reality. While attitudes towards data sharing have shifted in the aftermath of revelations about Cambridge Analytica, the prevailing attitude of patients in the UK has been that the use of data for research is acceptable, but that information should not be used for insurance or marketing purposes.

Debate concerning data management also needs to consider the return on datasets to the NHS, which have more value when highly curated, and reciprocity for the clinicians who contribute to them. This includes paying closer attention to primary care, which faces fragmentation of systems and a high quantity of data handled by GPs.

The use of real world evidence is not without its limitations. Breakthroughs in standardising evidence in such areas will only occur once more treatments which do not fit the current model are introduced and require scale-up, while the data underlying this evidence remains less rigorously consistent than that found in formal trials. It was also pointed out that clinical trials must remain an important part of our approach to generating evidence in the life sciences, as they have the potential to spot problems otherwise unseen, as was the case in a 2014 trial of Niacin, which had been prescribed widely for 50 years, to find that Niacin caused infection, bleeding and diabetes.

A report of the workshop can be downloaded here.