Synthetic data and a common data register – the future of real-world evidence is fast approaching

Utilisation of real-world evidence (RWE) progresses at speed and development steps are fast.

Utilising real-world data (RWD), such as data accumulated in the healthcare registries, in different phases of the life cycle of a medicine advances quickly. Medaffcon’s Managing Director Jarmo Hahl describes the development as a path, which is expanded and strengthened constantly.

– First, RWE was not used at all, whilst now companies and authorities utilise it in many ways and some years from now, it is likely that the use of RWE becomes automatised and the demonstration of treatment effectiveness with RWE becomes obligatory.

Fimea’s Head of Assessment, Vesa Kiviniemi, also considers that the significance of RWE can increase in the future. The most essential issue to remember is, however, that the nature of RWE data is always complementary to clinical trials.

A national data source to assess medicine

Medaffcon’s Jarmo Hahl deems it important to have one national data source to assess medicinal products. It could be realised, for example, by gathering hospital medicine first into one data source that would eventually be combined with a Social Insurance Institution (KELA) register into a unified data source.

Synthetic data would alleviate data protection concerns

One possibility to bypass data protection concerns related to RWD is the use of synthetic data. It means that a synthetic version is created from a genuine data, such as patient information. It enables producing the same statistical quantities as genuine data without the patients of the original data being recognisable even in theory.

– Synthetic data is already used in academic research. At the moment, authorities assessing medicine are discussing if conclusions could be made in their assessments based on synthetic data.

One of the advanced manners to utilise RWD is the use of virtual control arms. Their use will increase in the future, states Medaffcon’s Data Analysis Lead Iiro Toppila.

A virtual control arm means describing the control group with the help of an RWE study in a situation where a clinical trial would be an unethical alternative. For example, in cancer treatments, the control group of a clinical trial is usually not realisable, because the patients cannot be left without treatment.

The use of virtual control arms is typical with especially promising therapies, where market authorisation is received on the basis of a phase 2 study without a control therapy or evidence of patients’ overall survival.

Medaffcon has conducted studies that have used virtual control groups to produce additional information for decision-makers, for example, in cancer treatment and rare diseases.

The approach of authorities towards single-arm clinical trials is changing. For example, according to Fimea’s Head of Assessment Vesa Kiviniemi, the European Medicines Agency seems to have changed their policy with regard to single-arm case series.  Approximately 10 years ago, it was very rare that a medicinal product received market authorisation with a single-arm case series, but now it has become more common.