PIPELINEs: Creating Comparable Clinical Knowledge Efficiently by Linking Trial Platforms.

Area of Science:

• Clinical trial methodology
• Translational science
• Health services research

Background:

• Traditional single-sponsor, single-drug clinical trials are often inefficient and may not reflect real-world populations.
• There is a need for more efficient and representative clinical trial designs to accelerate therapeutic development and refine treatment strategies.

Purpose of the Study:

• To explore the potential of adaptive, seamless, multisponsor, multitherapy clinical trial platforms.
• To evaluate how these platforms can generate superior evidence more efficiently than traditional designs.
• To identify the benefits and challenges associated with implementing large-scale clinical trial platforms.

Main Methods:

• Conceptual framework for adaptive, seamless, multisponsor, multitherapy clinical trial platforms.
• Analysis of potential benefits including efficiency, participation, representation, and systematic evidence development.
• Consideration of multiarm designs, shared comparator arms, and standardized endpoints.

Main Results:

• These platforms can diminish barriers to trial participation and increase the representation of real-world populations.
• Systematic evidence development throughout a therapeutic life cycle can continually refine treatment use.
• Comparable evidence can be generated through multiarm designs, shared comparators, and standardized endpoints, aiding various stakeholders.

Conclusions:

• Large-scale adaptive platforms offer a pathway to superior and more efficient evidence generation.
• These designs can significantly benefit sponsors, providers, patients, regulators, payers, and scientists.
• Overcoming statistical, operational, governance, and data exchange challenges is crucial for successful implementation.