CDER review staff encourages the sponsor to submit clearly worded questions so that the purpose of the meeting is clear. Let’s take a look at potential topics for questions at different phases of development: pre-IND, the EOP1 and EOP2.
The pre-IND meeting can be very valuable in planning a drug development program, especially if sponsors' questions are not fully answered by guidances and other information provided by FDA. Early interactions with FDA staff can help to prevent clinical hold issues from arising. A pre-IND meeting can also provide sponsors information that will assist them in preparing to submit complete investigational new drug applications. Efficient use of FDA resources can lead to more efficient drug development and reduce time to market. These questions and answers can be especially helpful to small businesses that may have limited experience interacting with the Agency, or are unfamiliar with pre-IND meetings.
At the end of phase 1 of development a number of topics are reviewed, including proposed phase 2 controlled trials (including safety and efficacy), optimal dose selection and pharmacokinetics (PK)/pharmacodynamics (PD).
The End of Phase 2 (EOP2) meeting should be held before phase 3 trials begin. The purpose of an end-of-phase 2 meeting is to determine the safety of proceeding to Phase 3, to evaluate the Phase 3 plan and protocols and the adequacy of current studies and plans to assess pediatric safety and effectiveness, and to identify any additional information necessary to support a marketing application for the uses under investigation. At the End of Phase 2 of development, potential topics for review of phase 1 / 2 safety data include safety signals; preliminary efficacy data in the intended population, early risk/benefit evaluation, and phase 3 studies.
EOP2 meetings may include a proposed protocol for a phase 3 pivotal study. The sponsor may wish to discuss the study design including dose selection and endpoint selection. For example, does the study design provide definitive support of efficacy and safety?
The purpose of a pre-NDA/BLA meeting is to discuss format and content of the anticipated application, including labeling and Risk Evaluation and Mitigation Strategies (REMS), if applicable, presentation of data, dataset structure, acceptability of data for submission, as well as the projected submission date of the application. The meeting should be held sufficiently in advance of the planned submission of the application to allow for meaningful response to FDA feedback and should generally occur not less than two months prior to the planned submission.
Potential topics for questions at the pre- NDA/BLA phase of development may include questions surrounding formatting of the submission, such as regulatory requirements, organization of the submission, and the electronic common technical document (eCTD).
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