White Papers

Utilizing a Registry to Inform an External Control Arm: A case study in drug-induced liver injury

Endocrine and Metabolics, Rare Disease

  Clinical trials that rely on external data sources to provide a control arm pose an attractive alternative to an internally administered control group. In principle, they give study participants a higher likelihood of exposure to the developmental therapy, which is a particularly desirable proposition when the disease in question has high morbidity or mortality. This whitepaper explores the utility of registry data applied as an external control arm, providing a step-by-step description of the process in a real-life study.   View Article  

White Papers

A Tale of Two Agencies: FDA and EMA Regulatory Guidance for NASH and the Impact On Program Design

Cardiovascular

  Both the EMA and the FDA agree that identifying therapies to slow, halt, or reverse NASH/ NAFLD in patients represent a significant unmet need. Appropriate guidance has been structured to inform clinical programs across all key design elements, particularly for potentially pivotal trials. And although regulatory guidance may articulate similar strategic objectives, the detail required for program design and execution may deviate by phase of research. This whitepaper examines methods of reconciliation enabling one strategically focused clinical development program.   View Article  

White Papers

ALS Studies and Lessons Learned From COVID-19

Central Nervous System (CNS), Rare Disease

The disruptions caused by COVID-19 have complicated both the initiation and execution of clinical trials involving therapies targeting amyotrophic lateral sclerosis (ALS). Read our experts’ perspectives on how to flex during this global pandemic.

White Papers

Shifting Tides in Dementia Research

Central Nervous System (CNS), Clinical Research, Neuroscience

shifting tides

This white paper reflects on decades of applied clinical research to understand lessons learned about the unique and significant challenges present in dementia research.