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Optimizing Your Global Drug Development Program: Strategy for Regional Variations of the 505(b)(2) Pathway
At Camargo, we are often asked for strategic advice on drug development programs seeking approval in not only the United States (US), but also approval from other regulatory agencies, including the hybrid application in the European Union (EU).
It is useful at the beginning of a drug development journey to know what the goals are. If a company’s goal is to gain approval in the US and EU, it may be possible to create a plan to maximize potential efficiencies and reduce redundant efforts to support each regional approval. But to enable an effective strategy to fulfill the requirements of each regulatory authority, the plan and choices must be made early.
By evaluating the complete development program in the context of both US and EU requirements early in development, Sponsors can identify and develop strategies to create the most efficient development program while still addressing the unique requirements of each regional regulatory authority.
The United States (US) FDA’s 505(b)(2) submissions pathway is considered roughly equivalent to the hybrid application pathway in the European Union (EU). As such, several parallels exist between these pathways that can be used to support a more efficient global development program. Understanding the similarities and differences between the two can allow sponsors to optimize their approach to product development.
Approval of prospective medicinal products via either the US or the EU requires that all requirements of a full marketing application be fulfilled, potentially requiring full reports (or reference to full reports) of investigations of safety and effectiveness of a drug, where some of the information required for approval comes from studies that are not conducted by the applicant and for which the applicant has not obtained a right of reference. The supportive information for such applications can come from either published literature or findings of safety and effectiveness from the relevant regulatory authority for an approved product (“listed drug” or “LD” in the US or “reference medicinal product” or “RMP” in the EU). A 505(b)(2) application can reference EU RMP information, but an EU application must be based on EU-approved RMP.
While the two pathways appear similar, there are key differences that Sponsors should take into account if the goal is to market a product in both regions. The requirements for 505(b)(2) NDA – as well as issues and pitfalls of this submissions approach—have been discussed extensively by Camargo’s 505(b)(2) experts.
In this discussion, we highlight key considerations for when an approval is sought using both the 505(b)(2) procedure in the US and the hybrid procedure in the EU, adding to previous discussion here on the Camargo blog. Further details on the EU hybrid applications can be found on the European Medicines Agency’s (EMA) website here.
Similar regulations exist in both the US and EU that enable Sponsors to rely on information for which they do not have right of reference to support approval of a given product; however, requirements for approval may not align exactly for a product developed in both regions.
Whether the 505(b)(2) and the EU hybrid procedures can be utilized for parallel/simultaneous drug approvals in the US and the EU is not always clear from the outset of development. Each instance must be evaluated on a case-by-case basis as the requirements are product-specific in both regions.
Overall, both the US 505(b)(2) and the EU hybrid process offer the potential for a time- and cost-effective route to market. It is possible for Sponsors to achieve success in both markets with little to no delay. However, as noted here, the same application in the two regions may have different legal and regulatory bases, require vastly different approaches to regulatory interactions, and may be reviewed differently by regulatory authorities.
The regulatory scene in the EU is further complicated by the recent withdrawal of the United Kingdom (UK) from the EU (see our discussion on the implication of Brexit, here). Therefore, it is critical to engage experts with ample experience in developing a global drug development strategy for a swift and successful path to market. For more, contact us.
Manira Rayamajhi, PhD, Research Scientist, Camargo Pharmaceutical Services
Eric Kendig, PhD, Senior Scientific and Regulatory Manager, Camargo Pharmaceutical Services
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Camargo Pharmaceutical Services provides comprehensive drug development solutions, specializing in customized programs including the 505(b)(2) pathway.