May 3, 2023 — It’s been quite the day for clinical development!
I’m sharing two summary results below and a few prompt questions RE: benefits in special patient sub-populations and benefits of long-term follow up. Would love to hear your thoughts and reactions!
🧠 Eli Lilly and Company reported results that its Alzheimer’s investigational biologic donanemab showed positive results in a pivotal Phase III study. A patient sub-population saw a slow-down of cognitive decline by 35%, with the overall trial population seeing an average cognitive decline of 22%. Both are impactful results — if you’re an Alzheimer’s patient or their caregiver, you would see noticeable positive changes — but the sub-population seeing the better results (the “super-responders”) is quite intriguing. Looks like Alzheimer’s research will increasingly focus on targeted segmentation (progression stage, molecular profile, etc.), similar to what we’ve seen happen in oncology.
💡Prompt questions: It’s almost impossible (and, frankly, impractical) to collect every data element that might distinguish super-responders from non-responders in a trial. 1) How should the industry think about incorporating prospective and retrospective real-world data (e.g., medical histories) to help generate hypotheses for better understanding super-responder populations? 2) What *new* data elements (e.g., imaging, genetic sequencing, protein expression/localization) would you collect if you were to do a follow-up study on a super-responder population?
🦠GSK was granted FDA approval for its RSV vaccine today for a patient population of 60 years and older. This is an exciting development for public health, and I look forward to the Centers for Disease Control and Preventionanalysis and recommendations coming this summer. RSV infections pose significant risks for both elderly adults and children, so I look forward to how this and other RSV vaccine candidate approvals expand the patient population where these interventions are approved.
💡Prompt questions: 1) How do we get vaccine administration to more individuals where the intervention is approved? A certain level of vaccination will be needed to determine both long term vaccine efficacy and population level effects. 2) In a universe where more individuals are given access to RSV vaccines, I’m curious about the safety and efficacy of these vaccines in pregnant people. If a newborn can get significant protection at birth, that could be incredibly impactful for the neonatal / children population. How do we test this safely, effectively, and quickly, given pregnancy is often an exclusion criteria in trials?