Overview

A specialty pharmaceutical company partnered with G&L Healthcare Advisors to guide the US regulatory strategy for developing an EU partner’s drug-device combination product.

The goal was to achieve approval in the US via a 505(b)(2) New Drug Application (NDA) pathway. 

The client needed a comprehensive gap analysis to identify regulatory differences between the EU Marketing Authorisation Applications (MAA) content and the U.S. Food and Drug Administration (FDA) requirements, ensuring a smooth transition to the US market.

Challenges

G&L identified several key challenges in preparing the product for the US market:
Regulatory differences: The key challenge was identifying and addressing regulatory gaps between the European Medicines Agency (EMA) and FDA requirements, particularly for the drug-device combination product.
Device component and dosing concerns: Two significant gaps were identified, including a potential gap in device component/human factors studies, and a gap related to dose and dosing regimen due to differing drug concentration standards in the EU and US.
Contingency planning: The client required a strategy that not only addressed these gaps but also allowed for contingency planning based on FDA feedback, ensuring a smooth 505(b)(2) NDA submission.

G&L’s Approach and Solution

G&L conducted a comprehensive gap analysis and developed a strategic approach to address the regulatory challenges identified. This approach included:
Gap analysis: G&L conducted a thorough analysis of the EU MAA content, comparing it against US regulatory requirements and precedent set by similar US-approved drug-device combination products.
Identification of key gaps: G&l identified two key gaps, one related to the device component and the need for a human factors study to meet FDA expectations and the other related to dose and dosing regimen differences due to varying drug concentration standards between the EU and US.
Pre-IND briefing document: The identified gaps formed the basis for critical questions in the pre-Investigational New Drug (pre-IND) briefing document. G&L worked with the client to clearly present these gaps and potential solutions to the FDA.
Contingency strategies: G&L developed contingency strategies based on possible FDA feedback, ensuring that the client was prepared for different regulatory outcomes and could adjust their development plan accordingly.
NDA preparation: Following the gap analysis and pre-IND preparation, NDA preparation was initiated, setting the stage for a smooth and timely filing once the pre-IND phase was completed.

Impact and Outcomes

G&L's involvement delivered key outcomes for the client's regulatory process:
Seamless integration: G&L’s regulatory strategists integrated seamlessly into the client’s teams, working as an extension of their internal regulatory team.
Regulatory expertise: G&L’s detailed gap analysis and strategic guidance helped the client prepare effectively for their pre-IND meeting, addressing critical regulatory gaps and ensuring readiness for the 505(b)(2) NDA submission.
Expanded partnership: Based on the success of this engagement, the partnership expanded to include a similar scope of work for a second 505(b)(2) product, highlighting the trust and value placed on G&L’s expertise.

• Seamless integration: G&L’s regulatory strategists integrated seamlessly into the client’s teams, working as an extension of their internal regulatory team.
• Regulatory expertise: G&L’s detailed gap analysis and strategic guidance helped the client prepare effectively for their pre-IND meeting, addressing critical regulatory gaps and ensuring readiness for the 505(b)(2) NDA submission.
• Expanded partnership: Based on the success of this engagement, the partnership expanded to include a similar scope of work for a second 505(b)(2) product, highlighting the trust and value placed on G&L’s expertise.