Harnessing the Benefits of Externally Controlled Clinical Trials (ECTs) to Accelerate Development of Better Medicines

Parexel experts from regulatory, epidemiology and biostatistics discuss the use of external control arms (ECAs) as an alternative to randomized controlled trials (RCTs) to accelerate clinical development. Based on literature analysis, as well as available EMA and FDA guidelines, they provide a detailed assessment of ECAs pros and cons. 

The digital era has enhanced the potential for real-world evidence (RWE) in regulatory decision-making, as demonstrated during the COVID-19 pandemic. Artificial intelligence (AI) could play a crucial role in analyzing RWE data to identify trends and important factors to control, potentially expanding ECA applications to dosage optimization, new administration routes, and accelerated treatment development. However, ECA implementation based on RWD still remains limited due to challenges such as ensuring data robustness, matching patient populations, and controlling bias.

What is more, regulatory acceptance of ECAs also requires thorough justification and early discussions with authorities to address potential biases and confounding factors. Therefore, while ECAs have the potential to streamline drug development and bring treatments to patients faster, further advancements in regulatory approaches, methodologies, and technologies are necessary to realize this goal fully.  

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