Streamlined Trials Embedded in clinical Practice (STEP) Demonstration Project

C3TI aims to promote the adoption of pragmatic design elements to integrate randomized trials into clinical practice and improve coordination and collaboration between CDER and sponsors to effectively support these innovative trials. To this point, CDER will partner with sponsors on trials that include limited procedures outside of routine clinical care, decentralization of procedures that can be done outside of designated research sites, the use of real-world data to obtain outcomes, and, where appropriate, the integration into point-of-care practice. These types of trials are advantageous as they can be more resource-efficient, able to better attract broader study populations, can be completed more rapidly, and yet still robustly assess study objectives.

With the Streamlined Trials Embedded in clinical Practice (STEP) demonstration project, C3TI looks to partner with sponsors planning pragmatic/point-of-care trials to provide an opportunity to address and resolve issues (e.g., statistical analyses, incorporation of real-world data and evidence, trial endpoint selection, inspectional approaches) around trial design and conduct, the lessons learned from which will be made available broadly and used to inform updates to relevant CDER guidance.

Benefits of participating

By participating, sponsor(s) would receive additional CDER engagement support for trial design and implementation aspects, which includes leaders across several CDER offices (e.g., Office of Medical Policy, Office of New Drugs, Office of Translational Sciences). Engagement may include access to additional coordination support with CDER subject matter experts and an inspection process that is fit-for-purpose for the innovative design (i.e., focused on a quality by design approach).

Eligibility Criteria for STEP Demonstration Project Proposals

• The sponsor has an active pre-Investigational New Drug (IND) or IND for the product(s) included in the proposal.

• The trial incorporates pragmatic design elements that are reflective of routine clinical practice to improve trial efficiency and enhance patient centricity while maintaining patient safety and data integrity. Examples of pragmatic design elements include (but are not limited to) broad eligibility criteria, limited visits and procedures outside of what might occur in routine care, including incorporation of decentralized procedures, and limited safety data collection consistent with ICH E19.
• The trial fits one of the following criteria:
  • Trial later in pre-market development, when the safety profile is reasonably well-defined.
  • Post-approval trial (either that the sponsor initiated or in response to a post-marketing requirement), wherein the population, trial procedures, and endpoints can all be appropriately incorporated into a large simple trial. 

Of note, trials with narrow entry criteria, complex procedures, complex drug administration, or challenging endpoint collection will likely NOT be appropriate.

• The sponsor and FDA can reach agreement on the trial design information to be publicly disclosed.
  • Sponsors participating in demonstration projects will be expected to share select details of their clinical trials and the implementation of clinical trial innovations as they progress, starting as early as the finalization of study design. This sharing may include updates, lessons learned, and relevant insights gathered during the trial. It is understood that these shared details will reflect general principles and innovative aspects, while maintaining the necessary confidentiality of proprietary or sensitive information.

Instructions on how to submit a proposal can be found on the C3TI Demonstration Program Proposal Submission webpage.