Europe – When Can RWE Translate Into Credible Evidence? EMA Officials Discuss

Real-world evidence (RWE) may provide an opportunity to learn more about a drug’s benefits and risks, but officials from the European Medicines Agency (EMA) said in an article published Tuesday in Clinical Pharmacology & Therapeutics that there will need to be adequate statistical methods to extract, analyze and interpret RWE before they can translate into credible evidence.

In order to ensure that any new analytical methods are acceptable for regulators, the EMA authors say they will require testing and validation in nearly the same way as a new medicine is evaluated: “prospectively, well-controlled and according to pre-agreed plan.”

They add: “The ultimate key to achieving credibility is to start with an open but ‘agnostic’ mind-set and submit novel methods to a fair, transparent and prospective validation exercise; this cannot be done only by dry runs with old products. It is understandable that drug developers are wary of jeopardising the development programs for their valued new assets. However, we emphasise that if developers want trial assessors to accept novel methods, they will have to expose some of their experimental drugs to methodology development exercises.”…