Guys – I’m wildly embarrassed for us in drug development. If your days are like mine, the letters A and I are sprinkled like fairy dust. But the sad reality is my clinical endpoints for Cervical Dystonia (CD), like so many rare disease patients, are trapped in the 80s. I kid you not – I fill out PAPER TWSTR self evaluations (I love the humor of the person who named this IN 1997!) and get video taped during my visits for a POINT IN TIME PHYSICIAN ASSESSMENT. In 2026!
If I were to draw a Venn diagram of all the things missed by these old timey endpoints and uncontrolled risk when developing a treatment for CD, it would be a circle. Here’s the breakdown of what causes wide variation in my twisties: mood, menstrual cycle, illness, sleep duration, muscle fatigue. It is NOT CAPTURED ANYWHERE!
I am dying for someone to shove me into one of those sensor suits used by Andy Serkis playing Gollum and get some real data. I would LIVE in that suit (stink be damned) and run through the alps like a feral teletubbbie!
Longer monitoring of movement with AI analysis is far more precise than current point in time measuring. With our focus in rare disease research, we must embrace the need for technology enabled endpoints, include it in our development timelines and validate it against the old guard assessments.
The technology exists. But we are often risk averse. A critical miss, however, is that if we are not brave with digital endpoints, we will never have the data to show us our blind spots. As a patient with huge unmet need, it terrifies me that we could kill game changing drugs because we miss sufficient precision in our measuring. We should be able to measure both what I can do and what I can’t, to ultimately impact QoL. It’s time to embrace the regulatory red tape and make validation happen. It’s a worthy mountain to climb.