ACT Brief: Bayesian Implementation, Women in Statistics, and FDA Tightens Gene Therapy Standards

This is the Applied Clinical Trials Brief—your fast track to the latest insights shaping clinical operations and drug development.

• In the fourth installment of his video interview, David Morton, PhD, director of biostatistics at Certara, outlined the technical capabilities teams need to incorporate prior data into trial design. Teams must establish rigorous prior distributions, conduct sensitivity analyses, and ensure real-time data infrastructure for adaptive decision-making and early stopping triggers, with clinical input defining meaningful thresholds.
• Vicky Marriott, head of statistics at Phastar, examined gender representation in data science and statistics roles across drug development. While some organizations achieve gender parity in data teams, women remain underrepresented across STEM industries, requiring systemic changes in education, mentorship, career pathways, and workplace policies to address unconscious biases and support retention.
• The FDA rejected uniQure's request to use Phase I/II external control data for its Huntington's gene therapy, instead mandating a prospective, randomized, sham surgery-controlled trial. The agency cited the disease's heterogeneity and strong placebo effects, pointing to contradictory signals where sham-controlled data showed no benefit at 12 months despite external controls showing 75% disease progression slowing.

That's all for today's ACT Brief. Join us tomorrow for more updates shaping clinical operations and drug development. Thanks for listening.