The 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. 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 EMA’s Senior Medical Officer, Hans-Georg Eichler, Harald Enzmann, Chair of EMA’s human medicines committee (CHMP) and Head of European and International Affairs at the Federal Institute for Drugs and Medical Devices (BfArM) and EMA's Executive Director Guido Rasi, published today in Nature Reviews Drug Discovery. In this article, the authors analyse the benefits and risks of different proposals regarding the role of regulatory agencies in establishing the added therapeutic benefit of novel treatments in light of the ongoing debate about medicines pricing. Health-care payers emphasise that ‘innovation’ is not always synonymous with ‘added therapeutic benefit’ and increasingly resist paying high prices for new medicines with no or only minor added benefit.

The most fundamental proposal is to only authorise new medicines that have demonstrated added therapeutic benefit. Other suggestions include the mandatory comparison of new therapies with the best available treatment at the time of authorisation. However, according to the authors, limiting flexibility in medicine development and authorisation may not produce good results for patients and health-care systems.

The article concludes that the best approach is ‘evidence by design’, which can be achieved by mutual understanding among all relevant decision-makers on appropriate clinical trial designs, with a view to using the entire spectrum of available methodologies.

The article entitled Added therapeutic benefit and drug licensing is available through open access in Nature Reviews Drug Discovery.