As part of its efforts to support a global approach to the development of new antimicrobial medicines, EMA has published the final revised guideline on the evaluation of human medicines for the treatment of bacterial infections.
Antimicrobial resistance (AMR), which is the ability of microorganisms to resist antimicrobial treatments, especially antibiotics, has a direct impact on the health of people and animals and carries a heavy economic burden worldwide. In the European Union (EU) alone, it is responsible for an estimated 33,000 deaths per year. It is also estimated that AMR costs the EU €1.5 billion per year in healthcare costs and productivity losses.
EMA plays an important role in the fight against AMR by guiding and supporting the development of new medicines and treatment approaches, especially for patients with infections caused by multidrug-resistant bacteria, who currently have very few therapeutic options.
As AMR is a global threat, regulators in the EU, the United States and Japan have agreed to align as much as possible their respective data requirements so that medicine developers can design clinical trial that meet the evidence needs of multiple regulatory agencies. The revised document reflects the outcome of these discussions, and also includes:
- clarifications on recommended clinical development programmes for antimicrobials intended to address an unmet need;
- guidance on clinical trials to support treatment of uncomplicated urinary tract infections and uncomplicated gonorrhoea;
- updated guidance on displaying microbiological and clinical efficacy data in the summary of product characteristics.
The revised guideline is published together with an addendum aiming to steer clinical development programmes required to support the authorisation of medicines for treatment of bacterial infections in children.
For the treatment of some infections, efficacy results can be extrapolated in certain children age groups by looking at efficacy data from adults. The addendum mentions that companies developing new antibiotics need to develop an extrapolation concept and provide details about it in an extrapolation plan.
For some infections that occur only or mostly in children below a certain age, extrapolating efficacy data from adults is not possible. The addendum includes guidance on trials that may be conducted in these exceptional cases.
The revised guideline was under public consultation for six months in 2019. The final document with all the updates implemented was adopted by EMA’s human medicines committee (CHMP) at its May 2022 meeting.