EFPIA calls for harmonisation of GMO procedures in investigational drugs

The European Federation of Pharmaceutical Industries (EFPIA) will implement the new European Clinical Trials Regulation (EU 536/2014), obligating EU member states to enable a practical and efficient system to allow greater co-ordination of the genetically modified organism (GMO) assessment of clinical trials.

The initiation of a clinical trial with an investigational medicinal product that consists of or contains a GMO is currently a lengthy and complex process in Europe, according to EFPIA, due to the fragmentation of application procedures and data requirements implemented by each EU member state. Moreover, this fragmented landscape for GMO applications poses challenges when sponsors attempt to co-ordinate with clinical trial applications and their harmonisation under the European Clinical Trials Regulation to be implemented January 2022. These EFPIA-authored findings and considerations were published in Cell & Gene Therapy Insights.

According to EFPIA, the platform to submit Clinical Trial Applications, the Clinical Trial Information System (CTIS), lacks the functionality to receive national GMO Competent Authority documents, including the Environmental Risk Assessment, EU Common Application Form(s) and other possible national forms.

Since GMO submissions to national GMO competent authorities are not co-ordinated with clinical trial applications submitted via the CTIS, sponsors of clinical trials cannot leverage the advantage of the single dossier submission of the Clinical Trial Application and synchronous approval across those EU member states to initiate the clinical trial across the intended countries. Different EU member state GMO competent authorities implement different submission timelines, which has led to delays to the start to clinical trials of GMO medicine products.

Due to future challenges to obtain a parallel approval of a GMO submission and a clinical trial application under the clinical trial regulation, Europe could be perceived to be less attractive to sponsors. Other regions have less stringent GMO requirements. For example the US, since 2015, have adopted a “categorical exclusion” for investigational (GMO-) advanced therapeutics and vaccines.

“EFPIA reiterates its call for a permanent exemption from GMO requirements for advanced therapy medicinal products (ATMPs) containing or consisting of GMOs, as well as for vaccines, to accelerate European patients’ access to these potentially life-saving medicines,” commented Pär Tellner, Director of Regulatory Affairs at EFPIA. “However, in the meantime, a pragmatic and simplified approach for the GMO assessment should be considered by each EU Member State, ideally with solutions towards greater harmonisation to the clinical trial application.”