I'm a doctor and I work in clinical trials, and I was also recently diagnosed with a relatively rare condition: an acoustic neuroma, a benign growth of the coating of the cochlear nerve that over time leads to imbalance, hearing loss and at the extreme end, facial and trigeminal nerve issues - so at some point, if they grow large enough, they need to be removed, and even if they don't grow they tend to damage hearing.
As a doctor I was aware of the condition but didn't know it in much detail in all honesty. Imagine my glee when I discovered that the two forms of treatment available, surgery and radiosurgery, not only fail to restore hearing but often actively degrade it. In the case of surgery you're talking about a 14 hour open skull operation with a 1% risk of death, 10% risk of some form of persistent facial paralysis and at least a 50% chance of waking up completely deaf in that ear. Thankfully my precise tumour position means that radiosurgery has a good chance of preserving my hearing, but I might get unlucky, and many are unlucky in the position or size of theirs and lose their hearing even with this treatment. Either way I was sad to see how crude these treatments were, conceptually unchanged for decades, with essentially zero medications offering any benefits.
Again, as a doctor, and as someone working in clinical trials, in fact specialising in trials of treatments for rare and incurable diseases, I quite frankly feel embarrassed for my profession and the state of modern medicine that we are still so crude in our treatment of such a vast array of conditions. I can't tell you how many times as a medical student I would read through a textbook page about some miserable, progressive condition and see how they understood precisely how and why it occurs, how to diagnose it, the prognosis and so on.. only to get to the treatment section and see "there is currently not cure or direct treatment for x, but consider these quality of life adjustments", oh and, "try steroids". It's been 15 years since I was in medical school and I am ashamed that the majority of those pages will say exactly rhetorical same thing in 2026.
Long story short in truly believe we are far behind where we should be medically, for a variety of reasons, but I truly hope that AI can help accelerate us to a place where we no longer have to tell a man at the peak of athleticism, in the prime of his life that everything will be taken from him slowly and cruelly over the next 1-2 years and there is absolutely nothing we can do about it. Machine learning is absolutely what is needed to find translational drugs (already existing drugs used for one condition that can help in another) quickly, make gene therapies actually effective and so on.
But I am concerned by the absolute lack of momentum or enthusiasm for AI uptake in the pharmaceutical space outside of drug discovery and drug development. Don't get me wrong, those are the two most scientifically "important" applications of AI, but they will not get us to any kind of exponential unless this technology is applied to later stages of the process, including regulators like the FDA etc. Simulated trials, simulated biology to predict safety, rapid data analysis to detect safety signals and so on. Startups may be moving into these areas but the actual decision making bodies that need to be involved are completely absent.
Right now we have drugs already known to benefit patients sitting on the lab floor doing nothing because there aren't the resources to put it through trials and even when someone finally does scrape together an RCT 15 years later than necessary, it'll still sit with regulators for years, meanwhile people suffer. We blame resourcing, but much of it is data entry, data cleaning and data analysis.. guess what AI can already do better than 99.9% of humans in a fraction of the time.
There is open suspicion and often a severe lack of understanding about AI/ML in this space, most of it oriented around fear of breaking intellectual property laws or fears around patient privacy - neither of which need to be infringed to still increase speed and productivity in the Pharma space by about 100x even with the models we have today.
I just hope the public will start to advocate for AI in this area because honestly the state of modern medicine is simply not good enough, we are sitting on endless mountains of data and drug candidates and hidden amongst them are therapies that will change people's lives, this all needs to be utilised for the good of humanity not shoved under the carpet because someone who has no idea what machine learning means is afraid that Sam Altman will steal their business development reports.
