EuropaBio Position & Policy Recommendations to support EU ATMP Innovation

Europe has been a pioneer in the field of Advanced Therapy Medicinal Products (ATMPs) in terms of their development, authorisation, and regulation, thereby supporting patient access to these life-changing therapies. Between Jan. 2014 - Jun. 2019, 323 investigational clinical trials were initiated in Europe. However, this is less than half of what was observed in North America and Asia, with the number of new clinical trials increasing by <2% in Europe versus 36% and 28% in North America and Asia, respectively.1 To ensure the EU remains a leader in ATMP innovation, this paper outlines and elaborates EuropaBio’s position and policy recommendations to the European Commission (EC) across three key focus areas.

Clinical Trial Requirements
There is significant clinical trial complexity in the EU owing to Member States own interpretation of Clinical Trial Authorisation (CTA) legislation. This complexity creates confusion for ATMP developers and can lead to significant delays of planned trials, decreasing the attractiveness of conducting such trials in the EU. The upcoming Clinical Trial Regulation (CTR) (EU) No 536/2014 aims to promote the implementation of a harmonised CTA dossier and review timelines adopted by all Member States. However, EuropaBio is concerned that ATMP products will fall out of the CTR process owing to unrealistic review and response timelines as well as a submission portal that is not fit for purpose for ATMPs. As such, it is recommended that the EC and Clinical Trials Facilitation and Coordination Group (CTFG) build upon the learnings from the Voluntary Harmonised Procedure (VHP) for clinical trials and conduct ATMP-specific pilots to identify and resolve potential issues prior to wide-spread rollout of the CTR. Furthermore, enhanced centralised scientific advice procedures that allow for rapid advice on par with procedures in other jurisdictions, specifically the US, are requested to support timely EU involvement in global development programmes.

Genetically Modified Organism (GMO) Requirements
ATMPs that consist of or contain GMO are required to undergo additional approval procedures by GMO competent authorities in each Member State prior to CTA. This regulatory framework was not designed for pharmaceuticals and is not standardised across Member States resulting in developers undergoing multiple, inefficient and redundant procedures which are costly and can lead to significant delays to development programs. A single, networked approach conducted in parallel to CTA assessments is desired with the upcoming CTR providing an opportunity for the EC and CTFG to work with GMO authorities to achieve this. EuropaBio would like to collaboratively explore with the EC whether GMO requirements are appropriate for medicines given the current state of knowledge as well as how duplication between medicines and environmental agencies can be avoided to ensure optimised processes are in place to handle the increasing number of these products under development.

Optimised Evidence Requirements Including Real World Evidence (RWE)
It is often not possible to conduct large, randomised controlled clinical trials for ATMPs leading to uncertainty regarding their authorisation and reimbursement. However, RWE can be leveraged to complement ATMP clinical trial data and provide higher levels of confidence at the time of pre- and post-Marketing Authorisation (MA) decisions. There are no mature and relevant guidelines worldwide that clearly define the scope of application of RWE to drug development and there is therefore an opportunity for the EU to become a leading voice in defining its optimal use. EuropaBio recommends that a permanent working structure and information exchange platform is developed between the European Medicines Agency (EMA), national Heath Technology Assessment (HTA) bodies and payers with the intention to collect and utilise ‘’universal evidence,’’ i.e. data that supports the different needs of all stakeholders. This group would be tasked with initiating reviews and demonstration projects that ultimately allow for guidelines to be developed for the systematic integration of RWE to enhance drug development, authorisation and reimbursement processes.

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