[co-author: Alexander Wenzel]

The concept paper

The EMA has published a “Concept paper on preparation of a revised guideline on the evaluation of medicinal products indicated for treatment of bacterial infections.” In the Concept Paper, the EMA proposes to merge the “Guideline on the evaluation of medicinal products indicated for treatment of bacterial infections (CPMP/EWP/558/95 Rev 2)” with the “Addendum to the guideline on the evaluation of medicinal products indicated for treatment of bacterial infections (EMA/CHMP/351889/2013).”

Since the adoption of the two Guidelines, several new antibacterial agents have been approved in the EU. One of these is a pathogen specific indicated for use in patients with “limited therapeutic options”. During the interactions with the applicants for marketing authorisation, agreement was reached on aspects of clinical development programmes that are important for programme feasibility and conduct. The aspects agreed differ from or are not included in the current guidance.

It became evident that the current guidance needs to provide a detailed explanation on how to prove that antibacterial agents, are expected to address an unmet need. Medicinal products that address an unmet need benefit patients that have no other satisfactory treatment option. A medicinal product that offers a major therapeutic advantage over existing treatments, can also address an unmet need. The incentives provided to applicants for authorisation of medicinal products addressing an unmet need, are an accelerated assessment and a conditional marketing authorisation.

The concept paper’s proposals

The paper proposes to clarify which clinical programmes are more suited to identification of antibacterial agents expected to address an unmet need. The Paper also proposes to add an explanation that would clarify which indications can result from each clinical programme. Merging the two Guidelines while making the necessary revisions, would remove repetition and provide a single core document of reference.

The comments on the concept paper

The European Federation of Pharmaceutical Industries and Associations (EFPIA) has submitted comments on the Paper. EFPIA welcomed the EMA’s decision to merge and provide clarification concerning the two existing Guidelines. EFPIA also raised several issues, including the need for new antibacterial treatments. In order to facilitate the development of antibacterial treatments, EFPIA proposed to broaden the definition of an “unmet need”.

EFPIA believes that anti-bacterial medicinal products that address a longer-term need for society and public health should also be considered to constitute an unmet need. This would be to “anticipate the resistance mechanism of bacterial pathogens”. The current definition is limited to conditions for which there is no adequate treatment. Expanding the scope of the definition of unmet needs to include long-term society and public health needs, would support the timely development of new anti-bacterial medicinal products.

EFPIA believes that the “availability of multiple products with different mechanisms of action” will be helpful to ensure that treatments are available for emerging resistant pathogens.

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