The C3TI demonstration program is an excellent opportunity for pharmaceutical and biotechnology companies to closely interact with FDA and have meaningful input into the agency’s policy-making on clinical trial innovation. As such, we strongly encourage our clients to consider applying for this program.

Below we summarize the agency’s plans for the new clinical trial innovation center, and how this program demonstrates FDA’s goals of furthering the development of drugs to treat rare diseases, and enhancing the diversity of clinical trial participant populations.

Overview

In response to public comments regarding incorporating successful innovative clinical trial approaches, CDER announced this week plans to create C3TI: a central hub designed to enhance clinical trial innovation for drug development and regulatory decision-making. FDA said the C3TI will serve three main purposes:

1. Share lessons across CDER’s existing clinical trial innovation programs

2. Communicate and collaborate with external parties regarding innovative clinical trials

3. Create and manage a C3TI demonstration program for sponsors of innovative clinical trials in the pre-IND or IND phase

C3TI aims to achieve these purposes through various goals including: updating stakeholders with current clinical trial innovations; improving the efficiency, effectiveness, and diversity of trials; enhancing quality of trial data; and accelerating the development of safe and effective new drugs. In addition to coordinating with external stakeholders regarding information sharing, C3TI will also support knowledge sharing within the agency through discussion forums and a centralized knowledge repository, which will curate knowledge about completed CDER clinical trial innovation activities.

C3TI’s demonstration program is designed to enhance communication between CDER and innovative clinical trial sponsors. The program is currently open to sponsors conducting trials in the following 3 project areas:

1) Point of care or pragmatic trials (Streamlined Trials Embedded in Clinical Practice (STEP): This program is applicable to sponsors of trials incorporating pragmatic design elements such as broad eligibility criteria, decentralized procedures, and limited safety data collection. The program is aimed at addressing issues relating to statistical analyses, incorporation of real world data, and endpoint selection.

2) Trials implementing Bayesian analyses: This program is applicable to sponsors of phase 3 efficacy, safety, or non-inferiority standalone trial who are willing to supplement their primary statistical analysis methods with Bayesian analyses in order to increase understanding of Bayesian approaches.

3) Trials using selective safety data collection: This program is applicable to sponsors conducting trials on products with well-established safety profiles late-stage pre-approval or post-approval trials, including but not limited to, trials for approved products, seeking new indication, or trials designed to provide additional evidence of efficacy. The program is aimed at streamlining and reducing safety data collection in those trials.

Selected sponsors will have the opportunity for enhanced communication and interaction with CDER. These trials and subsequent interactions will serve as case examples that can be shared both internally and externally to foster innovation across various therapeutic areas.

C3TI demonstrates FDA priorities

In a press release announcing the C3TI, CDER Director Patrizia Cavazzoni, M.D., touted the agency’s “long-standing efforts to embed innovation in clinical trial design and conduct” in order to bring “new therapies to areas of unmet medical need.” Viewed alongside recent FDA actions, we see this effort as part of the agency’s work to promote drug development for rare diseases. For example, in October of last year, FDA announced the opportunity for a limited number of sponsors to participate in a new pilot program called Support for clinical Trials Advancing Rare disease Therapeutics (START), which offers additional meetings with FDA to sponsors of novel drugs that aim to treat rare diseases, as we summarized online here.

The creation of C3TI also demonstrates the agency’s goal of assisting sponsors where novel data-analytics tools and approaches to bioequivalence (BE) may be used, and when these approaches cannot be effectively addressed under existing regulatory frameworks. For example, FDA recently launched a pilot program to offer meeting opportunities to generic drug applicants who intend to use model-integrated evidence (MIE) approaches to establish BE in their abbreviated new drug applications (ANDAs); we provided an overview of that program online here.

In addition, CDER said the C3TI aims to “help increase the participation of diverse populations in clinical trials,” thereby enhancing the quality of trial data and providing more reliable information for underrepresented populations. Enhancing clinical trial diversity has been a major focus of FDA over the past few years, with the agency recently holding a clinical trial diversity workshop that revealed the agency’s expectations for sponsors to enroll historically underrepresented populations, as we discussed online here. And in February, FDA published a revision to its draft guidance “Collection of Race and Ethnicity Data in Clinical Trials,” demonstrating that the agency is laying the groundwork for additional guidance in this space, as we explain online here.

Next steps

C3TI’s web page is online here, and its demonstration program will accept up to nine proposals on a rolling basis within a 12-month period. Sponsors selected to participate in the program will have the opportunity for enhanced communication and interaction with FDA staff. Because the goal of the program is to share innovative information that has been gathered more broadly, sponsors will be required to share certain details of their development programs publicly. However, FDA has assured that necessary confidentiality will be maintained on proprietary information.

If you may be interested in applying to the C3TI program, or if you have questions on planning clinical trials with innovative designs, feel free to contact the Hogan Lovells attorney with whom you generally work or one of the authors of this alert.