June 24, 2020


Genetically modified organisms need special consideration for clinical trial applications; additional GMO authorities’ approvals according to the regulation of the country have to be obtained.


Either the GMO requires specific containment measures to limit the contact with the environment (contained use) with focus on biosafety classification of the risk and its minimization by implementing appropriate control measures, or it is not contained use (deliberate release) with focus on the impact on human health and the environment to be provided in an ERA.


In Europe clinical trials with GMO medicines viewed as deliberate release have to follow Directive 2001/18/EC; the contained use GMO follow Directive 2009/41/EC. Although the approaches of the two directives are different, both require a risk assessment that will be evaluated by GMO boards. While in some countries a single application to the Competent Authority is sufficient and the CTA approval includes the GMO approval, other countries require interaction with different national and/or regional GMO boards, some with lengthy review timelines that can delay the start of the clinical trial by up to 12 months.


To overcome these hurdles some processes have been initiated, and agreement has been made among the member states to harmonize the application, as recently with IMPs of human cells genetically modified by means of retro/lentiviral vectors or in vivo gene therapy products that contain or consist of AAV.


Other initiatives and harmonization of GMO applications of IMP of other categories are urgently awaited.


A streamlined and harmonized process would be desirable in Europe to be competitive concerning biomedicinal innovation and development of gene therapies. Furthermore, the GMO application should be aligned with the new Clinical Trial regulation No 536/2014. The advantages from the new Clinical Trial Application will not become visible if separate GMO applications on country level still apply in Europe. Sponsors might avoid running clinical trials in Europe, and patient access to innovative treatments could be delayed or impeded.

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