Gaining Market Authorisation in Europe: Responding to Complex EMA Questions When You’re on the Clock
by Chris Miller
An emerging specialty pharmaceutical company submitted a Marketing Authorisation Application (MAA) for a product that had been in extensive clinical use in the United States for several years.
At the critical Day 120 assessment in the review cycle, the European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) posed dozens of questions on the rationale for study designs, statistical methods, and interpretation of results for several pivotal studies in the development program.
Meeting the Agency’s deadline posed strategic and logistical challenges. Some of the studies had been conducted more than a decade earlier, so there was no one on the client team who had first-hand knowledge of the design or conduct of the studies in question. The EMA’s questions were not only numerous – they wanted a lot of detail. The team was faced with a tremendous amount of work to gather and synthesize the information by the EMA deadline.
The 3D team collaborated with the client to create an effective response strategy, develop clear answers to the EMA’s complex clinical questions, and refine key messages to ensure consistency across the submission.
The 3D team’s expertise in the therapeutic area, combined with their knowledge of the clinical development program, allowed them to dig deep into the primary clinical documents and develop effective answers that supported the clinical trial designs, statistical analyses, and clinical meaningfulness of the results.
3D helped the client successfully deliver responses to the Day 120 List of Questions on time. The response strategy and key messages remained consistent in all subsequent communications with the Agency throughout the rest of the MAA review process. Ultimately, the product received a positive CHMP opinion without the need for an Oral Explanation.
It’s essential to closely listen to the EMA’s concerns and make sure you’re clearly and directly addressing each issue – leaving nothing unanswered. The next step is ensuring message consistency so that all parts of the submission culminate in one “voice” to tell a compelling and persuasive scientific story for approval.