THOUGHT LEADERSHIP

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?

Drug Safety & Pharmacovigilance professionals have long awaited consistency in the manner in which the US Food and Drug Administration (FDA) accepts Individual Case Safety Report (ICSR) submissions. For years, the industry has navigated different submission procedures for FDA compared with other regions, as well as differences between Investigational New Drug (IND) reporting and post-marketing pharmacovigilance reporting. The FDA has been working towards harmonization of ICSR submissions and the final guidance is here! FDA will require that both premarket and post-market safety reports be submitted electronically in the ICH E2B(R3) format, the format that is currently the standard in the EU. Let us explore the current FDA practices, the need for updated processes, timelines for implementation, and what pharmacovigilance professionals need to do to prepare for the change.

PROMETRIKA team members make continuous learning a priority. As a statistician, it is important to keep up-to-date with the FDA’s current thinking on analytic approaches applied in clinical trials. In May 2021, the FDA published a draft guidance for industry, “Adjusting for Covariates in Randomized Clinical Trials for Drugs and Biological Products,” which provides recommendations for use of covariates in the analysis of randomized, parallel group clinical trials that are applicable to superiority trials and noninferiority trials.  I share some highlights and insights on this topic below.

All team members at PROMETRIKA take responsibility for remaining well versed in the changes in regulations and trends that impact their work. As a biostatistician at PROMETRIKA, I recently attended the American Statistical Association annual Biophamaceutical Section Regulatory-Industry Statistics conference where we discussed the addendum to the E9 (R1) Statistical Principles for Clinical Trials titled “Estimands and Sensitivity Analysis in Clinical Trials.”  The addendum introduces the Estimands Framework and strategies for selecting an estimand. PROMETRIKA invites you to explore this addendum and learn about the estimand strategies put forth in this update. PROMETRIKA can assist you with these and other clinical trial needs.