EMA on the Development of Novel Therapies

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The European Medicines Agency weighs in on the role of regulators in determining added benefits of novel therapies.

The European Medicines Agency (EMA) announced on April 11, 2019 that agency officials have published an article analyzing the role of regulatory agencies in assessing the benefit of a novel treatment and how this is affected by the drug pricing debate.

According to EMA, regulators provide specific reasoning on the added benefit of a medicine compared with other treatments at the time of its approval. This reasoning can help health technology assessment bodies, payers, clinicians, and patients to understand what makes a new medicine a ‘better’ treatment and not just a ‘new’ treatment.

The article, which was published in Nature Reviews Drug Discovery, takes a look at different proposals regarding the role of regulators in the development of new therapies. One such proposal suggests that only new medicines that have demonstrated an added benefit should be authorized by regulators. Another proposal suggests that there should be a mandatory comparison of new therapies with other available treatments. The authors, however, say that limiting the flexibility of regulators may “not produce good results for patients and healthcare systems” and that evidence by design is the best approach. EMA stressed in a press release that mutual understanding between decision-makers on clinical trial designs is best to view “the entire spectrum of available methodologies.”


The article was written 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.

Source: EMA