Neurodegenerative disorders present some of the biggest challenges in planning and conducting clinical drug trials. Identifying strategies to proactively address or mitigate these challenges can help to ensure a successful trial.
Since the first drugs for neurodegenerative diseases (NDDs) were approved in the 1990s, an increasing number of disease- modifying therapies have been approved for multiple sclerosis (MS) and several symptomatic therapies have been approved for Alzheimer’s disease (AD). However, despite extensive research, none of these available treatments demonstrate the ability to halt neurodegeneration. Hence, developing drugs that can stop clinical progression remains the biggest challenge in NDD.
Recent advances in our understanding of the genetic, molecular, and cellular basis of neurodegeneration make this an exciting time for NDD research. As our understanding of NDD has increased, so has our understanding of how to conduct clinical trials to study them, including design, assessments, and biomarker use. For example, we know that patient profiling and adjudication of endpoints or adverse events of special interest can have a significant impact on drug development.
In this white paper, we explore the complexity of neurodegeneration and the operational challenges inherent in NDD studies. We also introduce strategies for mitigating these challenges to ensure a successful trial.
