04/30/2024

On April 15, 2024, the Center for Drug Evaluation and Research (CDER) of the US Food and Drug Administration (FDA) announced the establishment of the CDER Center for Clinical Trial Innovation (C3TI). Clinical trial design and conduct must be held to rigorous standards and policies in order to safeguard the well-being of trial participants and the public. These standards and policies are outlined within the ICH E8(R1) Guideline: General Considerations for Clinical Studies. ICH E8(R1) details the protection of participants, scientific approaches to design, planning, conduct, analysis, and reporting of studies, as well as provides guidance on designing quality into the studies (Clinical Pathways offers an interactive eLearning course on the ICH E8(R1) Guideline here for those interested in learning more).

Trials must be open to innovation in order to adapt to the ever-evolving drug development processes. Recently, CDER, requested feedback from stakeholders involved in clinical trials on recent innovation efforts in clinical trials. This consisted of conducting interviews, listening sessions, opening a public docket for comment, as well as a public workshop for external and internal parties in March 2024 (recordings from the workshop can be found here). As a result, CDER heard a consensus from the stakeholders that integrating innovation is necessary to improve the efficiency and effectiveness of clinical trials.

C3TI is intended to be a central hub within CDER that focuses on supporting the implementation of innovation within clinical trials by having sponsors of innovative trials work alongside CDER staff to help them overcome the obstacles that policies pose for implementation. Three major goals of C3TI are:

1. Promoting communication and collaboration between CDER and external parties. This will allow for a more diverse array of ideas and opinions to be implemented in CDER research that otherwise may not have been considered.

2. Facilitating the sharing of lessons learned. This means providing external and internal parties access to information on existing CDER clinical trials as well as new innovation programs.

3. Manage a C3TI Demonstration Program. C3TI has chosen three initial project areas to kickstart the program:

Bayesian Supplementary Analysis (BSA)
- Bayesian analysis/statistics is a form of data analysis that takes prior knowledge or evidence about a parameter and applies it to Bayes’ theorem which results in the probability of a specified event occurring. Examples of clinical research questions that BSA could be applied to are: “what is the probability of a patient’s resting heart rate increasing when prescribed medication X” or “what is the probability of cancer remission on treatment Y”. BSA will be used as a supplement to the primary analysis as the name suggests.
Selective Safety Data Collection (SSDC)
- SSDC is an approach to streamlining data collection in specific clinical trials by reducing unnecessary data collection throughout the trial. This also reduces the overall cost of the trial and allows for optimal resource allocation.
Streamlined Trials Embedded with clinical Practice (STEP)
- The STEP demonstration will be supporting trials that include pragmatic design elements such as broad eligibility criteria, limited visits and procedures outside of what might occur in routine care, incorporation of decentralized procedures, use of real-world data to obtain outcomes, and more (see link for full list). These elements are intended to be resource efficient, able to increase trial participation, completed faster, all while remaining robust when assessing study objectives.

As C3TI continues to grow more project areas will be supported including more difficult areas such as clinical trials for rare diseases that often struggle to find enough participants and have no approved therapies. This growth will in turn help to develop the C3TI knowledge repository (a curated bank of information on completed CDER clinical trial innovation activities) and lessons learned which will hopefully generate further innovation in future trials. All of these C3TI efforts will ultimately lead to safely accelerating clinical trial research and new drug development that benefits public health. More information on C3TI including how to participate can be found on their website here. Supplementary information can also be found at the following links:

- The Clinical Pathways Team

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