Gaining regulators’ marketing approval is – together with launch – one of the two major value points in a drug’s life cycle. Marketing Authorization Applications (MAA), New Drug Applications (NDA) or Biologic License Applications (BLA) detail how the product is manufactured and controlled, and summarize years of testing.
Registration applications provide comprehensive information about the drug, enabling regulatory agencies to ensure that therapeutic benefits outweigh risks. Regulators assess quality, safety and efficacy, and evaluate the ability of the future marketing license holder to ensure and monitor a sustainable benefit/risk ratio. In addition to summarizing quality, nonclinical, and clinical data, marketing application submissions also present how the safety reporting system transitions from development to the post-approval phase.
A core registration file or an initial national application is adapted in country-specific submissions – depending on whether marketing applications are submitted in several markets in parallel, or sequentially. Regulators’ review is a thorough process, which can be expedited in priority review or accelerated assessments for eligible products and medical conditions.
Commercial launch is prepared in parallel to late-stage clinical development and registration. Based on registration label claims, the product’s value proposition is finalized, and the launch strategy is refined.
Key challenges encompass:
- Are clinical trial results adequate for both regulatory approval and market access?
- Where are the key challenges of the overall development, and how to present them?
- What are the national requirements and procedures for registration?
- Is the product eligible for Accelerated Assessment Procedures, Fast track and Priority Review?
- What should be included in the Risk Management Plan? Is a Risk Evaluation and Mitigation Strategy document (REMS) necessary?
- Is it more appropriate to file in several markets sequentially, or in parallel?
- What would be the optimal launch sequence?
- How to develop a value dossier?
- How about countries with lower permitted price ranges?
- How to prepare for negotiations with payers?
- Organize expert panels with medical/clinical, regulatory and health economics experts
- Enhance communication and smoothen liaison between Sponsor and regulators before and during review processes
- Design customized risk management mechanisms, to optimize commercialization
- Finalize BLAs, NDAs, MAAs (authoring or review)
- Detail roadmaps for submitting applications in various countries of interest, and managing review processes
- Manage communication with regulators on behalf of Sponsor throughout review procedures
- Identify EU Qualified Person for PharmacoVigilance for MAA
- Adapt post-approval safety surveillance to regional/national requirements, including safety data exchange agreements (SDEAs) set-up, – literature surveillance and medical information
- Monitor safety profile, organize global safety review boards
- Finalize value claims and data
- Develop launch strategy, set optimal global pricing strategy