Role of regulators in establishing added benefit of novel therapies

Regulators have a role to play in ensuring that there is a solid evidence base to support the assessment of added therapeutic benefit of novel treatments compared with existing and potentially cheaper therapies, according to a website posting by the European Medicines Agency.

To help health technology assessment bodies, payers, clinicians and patients to separate merely new from truly better medicines, the regulators, firstly, should provide explicit reasoning on a medicine’s added benefit compared to other treatments at the time of approval. And secondly, they should insist on ‘evidence by design’. This means they must make companies aware of the need to plan the development programmes of medicines upfront, so that they are suitable to address the evidence needs of all relevant healthcare decisions-makers.

These are the main conclusions of an article by the EMA’s senior medical officer, Hans-Georg Eichler, Harald Enzmann, chairman of the EMA’s human medicines committee (CHMP) and head of European and international affairs at Germany’s Federal Institute for Drugs and Medical Devices (BfArM), and the EMA's executive director Guido Rasi, published today in Nature Reviews Drug Discovery.