FDA enhances Adoption of Innovative Clinical Trial Designs

FDA's Center for Drug Evaluation and Research (CDER) announced that the center is soliciting public comments to understand the state of innovation in clinical trial design and conduct. The CDER is gathering information from internal and external stakeholders on the barriers and facilitators to incorporating innovative clinical trial approaches in drug development programs.

How to advance innovation in clinical trial design and conduct?

Changes in the drug development landscape require new clinical trial designs and innovative strategies for trial execution, an expanding range of drug development tools, and wider application in regulatory drug development of real-world data (RWD).

These approaches include (amongst others) the following:

• Programs in complex innovative trial designs (CID), model-informed drug development (MIDD), real-world evidence (RWE), rare disease endpoint advancement (RDEA), patient-focused drug development (PFDD), and digital health technologies (DHTs)
• Guidance on implementing decentralized clinical trial (DCT) designs and the use of DHTs
• Developing efforts to enhance use of simpler trials that could more easily be integrated into clinical practice (often called “point-of-care trials”)
• Artificial Intelligence and Machine Learning (AI/ML) in drug development 
• Qualification Programs for Drug Development Tools
• Efforts to improve enrollment of participants from underrepresented populations
• International harmonization efforts related to innovative clinical trial design and conduct

Topics that could be addressed include, but are not limited to, those listed below.

• Key challenges or barriers that may hinder the implementation and adoption of innovative approaches in clinical trial design, conduct, and execution, like, for example:
  • Regulatory and Compliance Considerations (e.g., GCP compliance, investigator oversight and responsibilities, data quality) 
  • Patient-Focused Trial Design and Recruitment Innovations (e.g., concerns for patient safety, patient recruitment and engagement, informed consent) 
  • Clinical Trial Infrastructure and Organizational Considerations (e.g., technical tools for data collection, management, and analysis, information regarding innovative approaches and how to implement them, capabilities of trial organizations)
  • Overarching Barriers (e.g., collaboration and communication between stakeholders (e.g., sponsors, CROs, sites, networks), familiarity with new methodologies and technologies)
• Examples of instances where integrating new innovations into existing programs or systems became particularly challenging.
• Therapeutic areas or types of trials that face unique barriers or challenges to implementing innovative approaches.
• Challenges when trying to apply innovative approaches in new areas (e.g., in a new therapeutic area or different trial types).
• Considerations that become more important as innovation is scaled and approaches wide-spread implementation. 
• Effective ways to enhance and coordinate communications with CDER (e.g., review divisions, compliance/inspectorate) or across other FDA stakeholders as new clinical trial innovations are implemented.

Responses to this public docket will be accepted until 19 April 2024. More information is available on the FDA website under CDER soliciting comments regarding clinical trial innovation.