THOUGHT LEADERSHIP

The 2025 New England Rare Disease Statistics (NERDS) workshop, held on October 9-10 in Boston, brought together leading biostatisticians, data scientists, clinical researchers, and regulatory professionals from academia and industry. This workshop served as an important forum for exchanging innovative ideas aimed at addressing the unique statistical and regulatory challenges inherent to rare disease drug research and development. At PROMETRIKA, we continue to advance collective expertise and strengthen our contributions to successful clinical development.

As the role of statisticians continues to change in a rapidly advancing digital landscape, adaptability and continued learning matter now more than ever. To this end, PROMETRIKA supports its team members in attending conferences where the most current methods and industry guidance are discussed. the annual American Statistical Association (ASA) Biopharmaceutical Section Regulatory-Industry Statistics Workshop in Rockville, MD,features representation from the pharmaceutical industry, academia, and the FDA. The most recent conference focused heavily on the evolving role of statisticians in the artificial intelligence (AI) and machine learning (ML) era. A consensus emerged regarding AI and ML, following innovative perspectives shared by several speakers. While both are viewed as powerful tools with great potential to more efficiently deliver life-saving therapies to patients, caution must be exercised in model implementation.

The ASA Biopharmaceutical Section Regulatory-Industry Statistics Workshop is held annually in Rockville, MD to provide biostatisticians with the opportunity to collaborate and expand the frontiers of statistical knowledge. The 2025 ASA conference centered around the future of statistics in the era of artificial intelligence (AI) and machine learning (ML). Two statisticians attended on behalf of PROMETRIKA to explore current AI/ML research efforts and use cases from statisticians across industry, academia, and regulatory agencies. Across a three-day series of short courses, speaker sessions, panels, roundtables, and poster sessions, a clear message emerged that AI/ML has a strong potential to improve statistical frameworks throughout the drug development life cycle.

Team members at PROMETRIKA are encouraged to stay abreast of scientific and regulatory advancements in biopharma by attending industry conferences. Earlier this year, I took this opportunity to attend the World Orphan Drug Congress 2025 in Boston. Some of the brightest minds in biotech, pharma, patient advocacy, and regulatory sciences met to discuss and brainstorm around one of healthcare’s most complex challenges: rare diseases. Urgency and innovation were highlighted through powerful keynote speeches, deeply personal fireside chats, and content rich panels focused on everything from patient identification to artificial intelligence in clinical care. I’d like to share some highlights from my experience and the sessions I joined.

The annual CDISC US Interchange conference is an opportunity for PROMETRIKA’s Statistical Programming team to learn about the newest ideas and trends for data dissemination in the industry. At this year’s gathering, between the keynote speaker’s sentiments, the FDA’s opening thoughts, and the breakout sessions – one idea carried through each talk; how do we increase communication and collaboration across all functions to make everyone’s life easier and reduce the work load placed on all involved – from Sponsors to CROs to the FDA to patients?