Trial Design

Real-world data is increasingly used to optimize trial design, reduce recruitment burden, and support regulatory decisions, but adoption remains uneven due to challenges around data quality, integration, and internal alignment across functional areas.

Digital twin technology can reliably simulate standard-of-care treatment outcomes using historical clinical trial data, offering a practical pathway to virtual control arms that reduce recruitment burden and address ethical concerns around placebo designs.

Moving beyond vendor evaluation to incentive design in Phase II/III rare and genetic disease trials.

In part 1 of this three-part series, behavioral science reveals how slow patient recruitment and enrollment challenges often stem from design and startup decisions made long before recruitment begins, but can be identified and resolved early when they still matter.

In the first months of 2026, the FDA has moved on multiple fronts to modernize how drugs are developed, evaluated, and approved, signaling a broader regulatory shift toward flexibility, mechanism-based evidence, and human-centric science.

In this video interview, David Morton, PhD, director of biostatistics at Certara, explains how regulatory momentum is encouraging sponsors to move beyond traditional methods, leveraging Bayesian frameworks to improve efficiency, particularly in rare disease and small population studies.

In this video interview, David Morton, PhD, director of biostatistics at Certara, discusses how growing FDA support is accelerating the adoption of Bayesian approaches, enabling more flexible, data-driven trial designs through external data borrowing, adaptive decision-making, and simulation-based planning.

As rare disease trials face persistent feasibility challenges, Bayesian designs are gaining momentum by enabling more flexible, data-driven approaches that integrate prior knowledge, reduce sample size demands, and align with evolving FDA guidance to accelerate evidence generation.

Findings from the VA’s Diuretic Comparison Project show pragmatic trial designs can achieve strong provider participation when burden is minimized and patient care remains the primary focus.

Charlie Paterson, partner at PA Consulting, outlines how limited FDA guidance on innovative designs, decentralized models, and digital endpoints is forcing clinical operations teams to recalibrate expectations and minimize regulatory risk.

Charlie Paterson, partner at PA Consulting, discusses how fewer new guidance updates are pushing sponsors to rely on historical precedents and non-US standards when making trial design and operational decisions.

See how real-world evidence is enabling smaller, smarter, and more efficient trial designs through hybrid models, external comparators, and continuous patient monitoring—while preserving the role of traditional clinical trials.

Explore how large-scale, de-identified real-world datasets enable more representative trial design, improve site selection, and support patient identification beyond the limits of traditional clinical study populations.

Learn how real-time patient eligibility data is reshaping trial planning and site selection, allowing sponsors to design more inclusive studies based on current patient reality rather than past performance.

Pharmacovigilance has advanced in detecting population-level safety signals, but a critical gap remains in translating those insights into transparent, defensible patient-level decisions.

As highly effective anti-obesity therapies emerge and regulatory expectations evolve, sponsors are adopting innovative trial designs, addressing long-term weight maintenance, monitoring lean mass preservation, and preparing for expanded safety and demographic requirements that will define the next era of obesity R&D.

Explore how AI can optimize study design, speed patient recruitment, and streamline operational workflows to shorten development timelines and enhance trial efficiency.

Twelve former FDA leaders have publicly challenged the agency’s proposed overhaul of vaccine approvals, sparked by an internal memo linking child deaths to COVID-19 vaccination, arguing the changes threaten evidence-based standards, weaken immunobridging practices, and risk eroding public trust.

Examine the rise of personalized and biomarker-driven therapies, the logistical challenges of rare patient populations, and how historical controls and real-world evidence are supporting strategy-driven trial designs.

Novel trial designs are reshaping clinical development by improving efficiency, reducing redundancies, and accelerating timelines toward a new era of precision medicine.

Exploring how large-scale patient databases and AI analytics can accelerate site activation, strengthen recruitment, and improve trial design from the start.

Revisit top insights from SCOPE Summit 2025, where industry leaders explored how artificial intelligence is reshaping trial risk management and how pragmatic study designs are bridging the gap between clinical research and real-world care.

See how early patient inclusion, integrated regulatory planning, and seamless CRO partnerships can help biotechs accelerate progression from Phase I to Phase II.