Clinical Trials are Inefficient, Slow and Scarce
Clinical trial designs are often inefficient, resulting in high costs, lengthy timelines, and suboptimal patient outcomes. Innovative trial designs and decision-support tools are required to streamline the clinical evaluation process and accelerate therapeutic development.
Foundational Capabilities (9)
Clinical trial designs such as challenge trials, adaptive trial design, and group testing can improve efficiency.
These can be complemented with analytics and decision-support systems to optimize design, patient enrollment, and outcome interpretation.
The ability to study the real-time dynamics of molecular pathways in living humans remains extremely limited in time-resolution (frequency and duration) and scalability (beyond lab testing). Continuous, minimally invasive technologies are needed to improve our understanding of human physiology, accurately diagnose patients, and assess the impact of clinical interventions. Molecular engineering, particularly protein engineering, and integrated circuit design can be combined to develop new, miniaturized devices.
Identify and validate robust biomarkers and surrogate endpoints to serve as effective proxies in clinical trials, enabling faster and more informative evaluations. This is especially important for aging (e.g., as proposed by the Norn Group).
Diagnostic tools for the top 10 hard to diagnose diseases could have a significant impact on patients and the healthcare ecosystem. Diagnostics as an industry is in a state of market failure. Many disorders remain challenging to diagnose, impacting patient outcomes and burdening the healthcare system.
Monitoring the epigenome can enable the identification of exposures to infectious disease and reveal exposure to threat agents.
Create high-fidelity computational models (digital twins) that accurately simulate human physiology, enabling synthetic clinical trials and faster hypothesis testing.
Recruitment is a bottleneck that could be addressed by socio-technical programs. Increased interoperability and unity of data access and patient recruitment across centers and disease states would help de-silo recruitment and improve efficiency.