Navigating EU regulatory challenges with cell and gene therapies

Biopharma companies face multiple challenges when seeking to bring cell and gene therapies (CGTs) to market, not least of which are the differing country and regional regulatory requirements and even differences in product definitions. For companies seeking to bring their CGTs to market in the European Union, understanding these nuances is imperative for regulatory approval and market access.  

Here are five current and emerging challenges facing the developers of CGTs, known as advanced therapy medicinal products (ATMPs) in Europe:

•  Preparing for scientific advice 
•  Adapting to an overhaul of the regulatory system  
•   Understanding the changing definition of gene therapy
•  Taking advantage of platform technology
•  Preparing for the Joint Clinical Assessment

The following recommendations are to help you navigate these developments.

 Preparing for scientific advice 

CGT manufacturers in the United States are accustomed to the FDA’s approach, in which manufacturers may ask the agency questions about clinical trial designs and receive guidance on whether they can proceed with their plans. This is usually done in a meeting with scientific discussion.  

In the EU, scientific advice is primarily provided as written guidance from the European Medicines Agency (EMA), rather than through face-to-face discussion. Scientific coordinators may ask follow-up questions to clarify the product and concept during a “discussion meeting,” but these meetings are not intended to provide advice. This structure can be challenging for companies accustomed to receiving direct guidance during meetings with the FDA.

Recommendation

Be prepared to answer questions and provide information. Also, be aware that the advice is written and not provided in that discussion meeting. It is essential to understand the different types of meetings available to innovators, as well as the avenues and incentives available to micro, small, and medium-sized enterprises (SMEs) of biopharma companies, including developers of orphan drug products, which account for the majority of ATMPs.
Another option is to pursue scientific advice from a national authority before engaging with the EMA. The benefit of the national route is that these meetings are more interactive, and certain authorities are known for being more progressive and for having domain expertise. It is advisable to seek professional advice to select an optimal member state for your product, prepare the briefing book, and draft questions to help guide the clinical design strategy. While national authorities do not provide the final regulatory opinion, their input can inform and advance later discussions with EMA.  

Subsequently, it is advisable, though not mandatory, for sponsors seeking national advice from a national authority to also seek EMA scientific advice, as all ATMPs are centrally authorized. You can easily adapt a briefing book for national authority advice to later EMA advice since they follow a similar structure, so pursuing both pathways will not add significantly to the workload.

You can also take advantage of the new Joint Scientific Consultations (JSC)², which have an option for joint health technology assessment (HTA) and EMA scientifc².

 Adapting to an overhaul of the regulatory system 

The EU pharmaceutical legislation, agreed upon in December 2025, represents a significant overhaul of the regulatory system³. To streamline processes, the legislation proposes reducing the number of committees. The proposal would integrate the tasks of Committee for Advanced Therapies (CAT) and other specialized committees into the Committee for Medicinal Products for Human Use (CHMP). The next step would be endorsement by the EU Parliament and Council.  

The concern for sponsors is that the expertise housed in the CAT could become less accessible, potentially limiting engagement with regulators with deep ATMP-specific proficiency⁴...