The European Commission’s proposed revision of the pharmaceuticals legislation remains focused on the conventional medicines of the past rather than the Advanced Therapy Medicinal Products (ATMPs) of the future. It fails to build upon the Commission’s statement in its 2020 Pharmaceutical Strategy for Europe that cell and gene therapies represented ‘milestones of major progress’ in healthcare.
The legislative revision is an opportunity for EU policymakers to help address the EU’s stagnation in ATMP clinical trials, company formation, and investment in comparison to the US and China. While the 26 April Commission proposal in some ways improves upon an earlier draft version, it still falls significantly short of building a sustainable future for the ATMP sector and providing access for EU patients.
The proposal fails to harmonize the implementation of the hospital exemption based on high regulatory standards across EU Member States, undermining the EMA’s centralized authorization process and potentially risking patient safety.
While it streamlines the GMO clearance process for ATMP clinical trials and makes it more specific for medicines, the proposal misses the opportunity to provide a derogation from such requirements.
Lastly, the Commission’s focus on product launch in all 27 EU member states fails to recognize the unique nature of ATMPs, many of which are delivered in specialized treatment centers for small rare disease patient populations.
ARM and its members look forward to engaging with all relevant EU decision-makers to ensure that the future of medicine is a major focus in this once-in-a-generation opportunity to revise the pharmaceuticals legislation. This legislation, along with the implementation of the EU HTA regulation, will in large part determine whether patients in the EU have access to the durable treatments and potential cures of today and tomorrow.