THOUGHT LEADERSHIP

Due to the worldwide pandemic, the CDISC European Interchange, originally scheduled to take place in person in Berlin, Germany, was presented completely online. Susan Boquist, Associate Director, Statistical Programming, took advantage of the accessibility and logged in at 3:00 am her time both mornings. Though it may have become an April Fool’s Day to remember, with only a few weeks’ notice, the organizers were able to make the format transformation flawlessly. The conference schedule was consolidated into one track. A networking app was employed to allow for attendees to easily communicate with each other, ask questions of the presenters, and view and vote on the poster presentations. Training and workshops were rescheduled for another time. It was so successful that everyone hopes the organizers will consider adding a virtual option for future conferences. 

I recently served as the moderator for MassBio’s forum on “Closing the Real-World Evidence Gap: Pragmatic Clinical Trials & Observational Studies.” This lively and informative discussion was led by a panel of industry leaders, which included Robert Califf, MD, MACC, Head of Medical Strategy and Policy, Verily Life Sciences and Google Health; Jane Liang White, ScD, Senior Director, Statistical Group Lead for Oncology/Hematology Franchise at Pfizer; and Rebecca Miksad, MD, MPH, Senior Medical Director of Flatiron Health. 

Hwang et al.[i] completed a study looking at 640 phase 3 trials investigating novel therapies and found that 54% of these projects failed in clinical development; 57% of the failures were due to inadequate drug efficacy.  This means that 43% of the projects that failed were due to reasons other than the efficacy of the drug.  It is likely that many of these failures were due to operational deficiencies.  It is important to consider that a Sponsor’s investment in a trial is not only the investment made for the phase in which the project fails, but all investments from discovery leading up to the failure.