How can novel, non-randomised analytic methods be validated for regulators? The EMA discusses

Real-world evidence (RWE) such as electronic health records (eHRs) can be combined with patient-level data from completed randomised controlled trials (RCTs) and new data sources such as social platforms, to potentially understand more about drugs’ benefits and risks.

However, the European Medicines Agency (EMA) has warned that these novel analytic methods have many hurdles which will need to be cleared before the full potential of their data sources can be utilised and translated into credible evidence to enhance pharmaceutical research and care.

In an article published in Clinical Pharmacology & Therapeutics, regulators have explained that they will need adequate methodical validation of the data sources so that reliable scientific conclusions can be drawn. They have said that this testing and validation will need to be “prospectively well-controlled and according to a pre-agreed plan”.

One way of doing this could be to conduct several RCTs comparing two or more treatments that have been on the market for a period of time long enough to allow simultaneous real-world data (RWD) analysis. This would eliminate bias by matching the RCT and RWD analyses as closely as possible, while avoiding the possibility that RWD could be tuned to match the known results of a completed RCT.

Furthermore, collaborative platforms such as EMA’s methods qualification procedure or similar plans offered by the US Food and Drugs Administration (FDA) could also be used to achieve methodical validation. Through the EMA’s methods qualification procedure, the Committee for Medicinal Products for Human Use (CHMP) can issue an opinion on the acceptability of a novel analytic method for specific research questions.

The requirement for quality assurance and control procedures has been identified and a variety of initiatives are aiming to take RWE to a level of regulatory acceptability. To view the full article, click here.