Life science associations are calling for ATMPs to be exempted from the strict EU GMO rules, as their different legal interpretations in EU member states are delaying applications for multicentre clinical trials.
According to the Alliance for Regenerative Medicine (ARM), the European Federation of Pharmaceutical Industries and Associations (EFPIA), and the European Association of Bioindustries (EuropaBio), the existing patchwork of national regulations for the approval and environmental impact assessment of multicentre clinical trials of gene and cell therapies is hindering the development of life-saving therapies in Europe. While in Asia and the USA a strong increase in corresponding clinical studies can be observed, in Europe there is almost a standstill due to the complex and nationally different legislations, stress EuropaBio, EFPIA and ARM in the journal Human Gene Therapy.
In continuation of the current exemption for genetically engineered COVID-19 vaccines, they call for a Europe-wide deregulation within the framework of the EU pharmaceutical strategy by 2022. Harmonisation of national requirements and deregulation to the greatest possible extent of the requirements for therapeutic approaches working with genetically modified cells and virus vectors would accelerate the clinical development of the globally fast-growing field in Europe as well and help to eliminate competitive disadvantages for EU developers.
It is generally considered that genetically engineered advanced therapy medicinal products (ATMPs) pose negligible risk to the environment. Such gene and cell therapiess include human somatic cells modified ex vivo; recombinant virus-based vectors, including those containing genome editing nucleic acid sequences such as mRNA therapeutics; and bacterial DNA plasmids. Outside of controlled storage conditions, gene therapies cannot survive for any appreciable length of time.
According to a 2019 ARM report, the number of ATMP clinical trials in Europe stayed roughly flat over a four-year period (2014-2018) while increasing substantially in North America (+36%) and in Asia (+28%). In contrast, current figures from the GCT Catapult in Stevenage, UK, however demonstrate a 30% increase of such clinical trials in the UK in 2020.
"In 2020, we welcomed the derogation from GMO legislation for COVID-19 treatments or vaccines in clinical trials,” said Pär Tellner, Director of Regulatory Affairs at EFPIA. “Member companies are increasingly reporting how the derogation has removed the significant and time-consuming hurdles associated with GMO submissions, in addition to the clinical trial application. Swift action to a permanent exemption from GMO legislation allows the EU to prosper and most importantly for patients to continue to receive transformative, potentially life-saving therapies."