‘Come Early, Come Often,’ EMA Chief Tells Industry

Too many drug developers fail to come early enough to the European Medicines Agency to discuss potential regulatory challenges, according to Emer Cooke, chief executive of the agency. “Come early, come often” to solve problems that may subsequently delay regulatory approval, she urged developers at the Alliance Of Regenerative Medicines’ Meeting on The Med, on 15-17 April.

“We have too many developers who didn’t come to talk to us and had to redo things. That would have actually helped, [the problems] were quite easy to solve, but we weren’t able to have that conversation,” commented Cooke.

Early communication is especially important for supporting innovation when it comes to complex development plans, she added. “The door is open. Please come and talk to us, formally, or informally. We want to learn,”

Cooke added that if developers stayed away, the agency would be unlikely to “know what the questions are,” let alone “have all the answers.”

She said the agency offered several mechanisms that innovators could use to instigate early discussions. These involve:

• Pipeline meetings, which allow developers to discuss their pipelines with regulators to help the EMA ensure it has the right expertise available when the product is filed for marketing authorization.
• The Innovation Task Force,  which offers a platform for regulators and developers to discuss scientific and regulatory issues relating to innovative medicines. Developers “can come with your ideas, with your regulatory challenges, and we’ll have an informal discussion about what that means and what you might need to look out for,” explained Cooke. She added that this informal mechanism might not provide all the answers but will help the agency to understand what the questions are. “Our goal here is to prepare us for what’s coming, and also to make sure that the development pathways are robust so that they will deliver products for patients.”
• The Quality Innovation Group, which is an expert group that the EMA created to support the translation of innovative approaches to the design, manufacture and quality control of medicines. The mechanism helps regulators keep abreast of manufacturing developments so that it can understand how to regulate them and what needs to be updated from a regulatory point of view.
• The Office for Small and Medium Sized Enterprises, which advises and offers support to small and medium-sized companies developing medicines.
• Academia Briefings Meetings, which the EMA runs for academics to allow discussion and exchange on regulatory science.
• The PRIME scheme, which Cooke said “provides early, proactive support to medicines developers, to optimize the generation of robust data and enable accelerated assessments.” She added that almost half of the PRIME-designated products are advanced therapy medicinal products (ATMPs) and that most of the ATMPs approved in the EU since 2018 had PRIME support during development.
• A pilot program for academic and non-profit sponsors that are developing advanced therapies and which was launched in 2022. The pilot enables sponsor to access regulatory support for up to five products that address an unmet clinical need. The pilot’s second call for candidates in December 2024 attracted 11 candidates, Cooke said, adding that these types of sponsors find “it extremely hard to navigate the regulatory complexities, we feel it’s our responsibility to help.”

HTA Regulation

Cooke also pointed out that the EU Health Technology Assessment (HTA) Regulation, which came into force earlier this year, was “a great opportunity for collaboration” between HTA bodies and regulators. She commented that the EU-level joint clinical assessments, which evaluate the relative effectiveness of a new product, would help ensure that clinical trials serve both the needs of regulators and HTA organizations and increase “mutual understanding and dialogue” between the two types of organizations.

Cooke explained that regulators and HTA bodies operated in “very different cultures,” which means they find it hard to “get to know each other” and talk to each other effectively.