Frequently Asked Questions about CDER Center for Clinical Trial Innovation (C3TI)

1. How will C3TI impact regulatory review?

   C3TI is likely to have an impact on regulatory review of new drugs in several ways, such as:

   • More efficient review: Innovative trial designs, like adaptive trials and master protocols, can require specialized expertise and C3TI can identify this expertise.
   • Optimized clinical development: In many cases, innovative designs and approaches to conducting trials can be more fit-for-purpose in generating the needed evidence of safety and effectiveness, and optimized for efficiency in recruitment, diversity or representativeness, and generalizability.
   • Improved patient access and inclusion: Novel designs and approaches, such as those that enable trials to leverage new drug development tools or decentralized trial practices, may reduce the burden for patients or sites to participate in trials and facilitate the inclusion of clinical trial sites in more diverse, community-based settings.
   • Enhanced communication: Early engagement and ongoing dialogue between sponsors and CDER can clarify expectations and resolve issues early in the review process, expediting overall drug development timelines.
2. How does C3TI impact how sponsors will work with CDER?

   C3TI can impact sponsors’ interactions with CDER in the following ways:

   • Facilitating innovative trial designs: C3TI promotes and disseminates information, such as guidance, precedents, and training on adopting novel clinical trial approaches. Adopting such approaches can require greater upfront communication and engagement with CDER experts, but ultimately these approaches can expedite the development.
   • Enhancing communication and collaboration: C3TI fosters a collaborative environment, where sponsors and CDER work together to ensure efficient and successful integration of innovative clinical trial approaches in drug development. This includes initiatives, such as:
     • Early engagement meetings: These meetings encourage sponsors to discuss proposed clinical trial designs with CDER before formally submitting them to their investigational new drug (IND) application.
     • Working groups and public workshops: These forums facilitate information sharing and discussion of best practices for innovative trial design and conduct.
3. Will C3TI expedite CDER decision-making for innovative trials?

   C3TI’s primary role is to support and guide innovation in clinical trials. Its efforts are expected to contribute to more efficient CDER review processes in the design of clinical trials with innovative approaches to provide the most fit-for-purpose data. This early efficiency is expected to improve overall development timelines. It is important to note that while C3TI can play a role in supporting innovative trials, the review and regulatory decision-making remain under the purview of the CDER review divisions.

4. Which types of innovation are within the scope of C3TI?

   The following are within the scope of C3TI:

   • C3TI focuses on a broad range of innovations related to clinical trials, including novel statistical methods for the design and analysis of clinical trials, selective safety data collection practices, patient-centric approaches, decentralized trial models, and integration of randomized trials into clinical practice. C3TI does not focus on innovation related to other areas of drug development, such as pharmaceutical quality and non-clinical research.
   • C3TI manages demonstration projects to explore scalable improvements in the adoption of select clinical trial innovations through collaborations with drug developers. These clinical trial projects, intended to support new drug approvals or changes to approved drug labeling, serve to enhance the existing body of knowledge and precedents supporting multiple clinical trial innovations.
   • As a starting point, C3TI is focusing on three demonstration project areas. More information can be found on the C3TI Demonstration Program webpage. Future demonstration project areas will be decided based on lessons learned and coordination with internal and external parties.
5. How does the C3TI Demonstration Program differ from the existing CDER and CBER co-led programs like Complex Innovative Trial Design (CID), Model-Informed Drug Development (MIDD), Innovative Science and Technology Approaches for New Drugs (ISTAND), and Rare Disease Endpoint Advancement (RDEA)?

   C3TI demonstration projects may have similar objectives to existing CDER and CBER co-led programs exploring innovative clinical trial approaches and drug development tools. The C3TI demonstration projects are intended to streamline the process required to collaborate among CDER experts, review teams, and drug development sponsors on the implementation of innovation in development programs, and expand the areas of innovation explored. C3TI demonstration projects will have an open application window permitting sponsors to propose planned trials as early as they are conceived. This enables review teams to identify demonstration projects for enhancement from CDER subject matter experts through C3TI’s coordination support to participate in any planned engagements (e.g., formal meetings). Additional areas of innovation may be considered in the future. More information can be found on the C3TI Demonstration Program webpage.

6. Will C3TI focus on specific therapeutic areas?

   C3TI aims to support innovation across therapeutic areas, recognizing unique challenges and opportunities in different fields of medicine.

7. Will C3TI provide funding for innovative clinical trial?

   C3TI does not provide funding for innovative clinical trials.