CDER provides scientific and regulatory advice to guide clinical trial development and implementation as a cornerstone in drug development, helping patients and consumers access the therapies they need. As part of an ongoing effort to innovate and enhance clinical trials, CDER is establishing the CDER Center for Clinical Trial Innovation (C3TI). C3TI will foster CDER’s innovation efforts and act as a central point for coordinating, sharing knowledge, and communicating with both internal and external parties.

In this CDER Conversation, Kevin Bugin, PhD, lead for C3TI and deputy director of operations in the Office of New Drugs (OND), explains the purpose of C3TI, the importance of clinical trial innovation, and ways in which C3TI can advance public health.

What is C3TI?

Kevin Bugin, PhD, Photograph
Kevin Bugin, PhD

C3TI will be a central hub within CDER to support the implementation of innovative approaches to clinical trial design and conduct. C3TI’s mission is to promote existing and future CDER clinical trial innovation through enhanced communication and collaboration. This will ultimately improve the efficiency of drug development, bringing safe and effective drugs to patients as optimally as possible. C3TI will foster innovation across industry and therapeutic areas, and across new and ongoing initiatives within CDER. The goals of C3TI are to:

  1. Facilitate the sharing of lessons learned across CDER’s existing and new clinical trial innovation programs.
  2. Communicate and collaborate with external parties about innovative clinical trials.
  3. Manage a C3TI Demonstration Program that will expand opportunities for sponsors of drug development programs to work with CDER experts in enhanced ways to illustrate the value and impact of innovative approaches to the design and conduct of clinical trials.

Why did CDER create C3TI?

For years, CDER has supported new approaches in the design and conduct of clinical trials. However, drug development continues to evolve and now, more than ever, trials need to leverage the most fit-for-purpose designs and tools to efficiently and effectively generate evidence to support the regulatory decision-making for safe and effective new medicines. For this reason, CDER opened a public docket in late 2023 and held a two-day public workshop in March 2024, in collaboration with the Duke-Margolis Institute for Health Policy, to better understand opportunities that could improve the development and adoption of innovative approaches in clinical trials.

CDER heard that sponsors want our support integrating innovation into their trials for new drug development. CDER staff noted they would benefit from greater exposure to or experience with the use of innovations in their regular review work. There is a desire across the drug development “ecosystem” to integrate innovation, but there is a need for more direction on how to better bridge the gap between policy and implementation.

Based on this insight, C3TI will aspire to create more opportunities for staff to participate in development programs planning to implement novel trial approaches to gain experience with a given innovation, via a C3TI Demonstration Program. C3TI will also explore how to create new opportunities for CDER staff to receive training and exposure to new clinical trial methods, technologies, and tools. Lastly, C3TI intends to better capture and disseminate lessons learned from implementing a given clinical trial innovation through improved communication and coordination with colleagues (e.g., dedicated rounds to discuss clinical trial innovation) and with external parties (e.g., public meetings or webinars).

How can C3TI leverage opportunities in clinical trial innovation?

With advances in healthcare and technology, there are many ways to innovate clinical trials. C3TI can help us take advantage of these opportunities by fostering collaboration and knowledge sharing. For instance, while guidance documents have been critical to guide clinical trial innovation, real-world examples help us better understand how to implement these innovations. The experience from these examples, either identified by C3TI and its partners or generated through the Demonstration Program, can be shared broadly.

C3TI is also involved in planning public workshops, which can offer a unique forum to share and discuss case studies or examples. Our public workshop in March 2024 with Duke-Margolis identified several driving forces for clinical trial innovation, such as increasing the participation of diverse populations in clinical trials, increasing the number of clinical trials for rare diseases with participants with unique needs, and incorporating decentralized trial elements and digital health technologies. Furthermore, it was clear from this workshop that collectively we all benefit from discussing our real-world experience with implementing clinical trial innovation and should seek out future opportunities for similar collaborative discussions.

How does C3TI impact public health?

Clinical trials are a key step in the drug development process and for generating the evidence needed to support approving safe and effective drugs. By improving the efficiency and effectiveness of clinical trials, we hope to accelerate this process.

C3TI is very interested in increasing diversity and inclusion in clinical trials. This can potentially enhance the quality of clinical trial data and provide more reliable information on populations that may have been previously underrepresented in clinical trials such as racial and ethnic minority and indigenous populations, individuals with underlying genetic conditions, or those affected by environmental factors.

Rare diseases are another challenging area that C3TI will address. Many rare diseases have little or no approved therapies, which means there is rarely a clearly defined clinical roadmap for future drug development. Another potential obstacle is finding enough individuals to participate in rare disease clinical trials. C3TI will help in this area by supporting the broader understanding and adoption of the most fit-for-purpose clinical trial designs, ultimately accelerating the development and approval of new rare disease drugs.

What plans does C3TI have for the future?

As we look to the future, the vision of C3TI is to accelerate the pace of clinical trial innovation through collaboration and the promotion and adoption of useful innovations. In the near future, we’ll focus on our C3TI Demonstration Program, consisting of three initial project areas. The program is for selected sponsors of clinical trials in these specific project areas to serve as case examples. If selected, sponsors will have the opportunity for enhanced engagement with additional CDER staff within the context of their traditional interactions with the review teams. The goal of this program and selecting these case examples is to generate new lessons learned to share more broadly with the clinical trial community.

Using our experienced staff, working groups and public workshops, we will continue to provide insight and guidance on clinical trial design and execution for drug development. As C3TI is fully implemented, we hope to increase engagement with sponsors. We look forward to C3TI’s growth over the years to come and its positive impact on drug development.