Bringing Consistency and Efficiency to Regulatory Decision-Making
u Guidelines provide the building blocks for the construction of a fit-for-purpose dossier
u Communicating the agency’s expectations for the dossier- pre-submission advice
u Communicating the agency’s assessment decision
Determining if a medicinal product should be authorized requires the assessment and integration of a varied set of data related to quality, safety and efficacy. Importantly, the basis for an efficient review is having a well-constructed data package for the submission. Guidelines such as ICH provide the building blocks for the construction of a fit-for-purpose dossier; having guidelines that communicate the agency’s expectations for the dossier is key; and offering pre-submission advice sets expectations regarding the content and limits surprises during the review. These three areas will be reviewed in detail. When conducting the review, using procedures that help standardize the review brings consistency to the process. The benefits of using tools such as assessment frameworks that permit a full review or a reliance-based review, and benefit-risk models will be discussed. How the decision to authorize or not, is actually made will be discussed- does the organization have systems and procedures in place to make decisions that at not influenced by biases; decision-making tools will be reviewed. Once the decision is made, how is it documented and communicated? The role of internal and external assessment reports will be discussed. The applicability of all of these concepts to new molecules, biologics, generics and vaccines will be illustrated.