The landscape for patients living with myasthenia gravis (MG) is quickly changing. Just six years ago when I was diagnosed, there was only a single FDA therapy approved specifically for MG. Although there were other treatments which had been used to address the symptoms of MG, these were developed for other diseases or disorders. Though they were functional and helped MG patients, they weren’t specific for how myasthenia manifested the symptoms.
Now we have four companies who have FDA-approved therapies specifically for MG. They are Alexion, argenx, UCB and J&J. Plus, there are a number of other companies who are developing therapies for MG which could be transformative. Their approach is unique compared to the existing therapies available.
There have been significant advancements in science and research in the rare disease community over the past 10 years — and it continues to accelerate. You will be hearing more about chimeric antigen receptor (CAR) T-cell therapy and nanoparticle therapy in the myasthenia gravis world.
The potential in digital health tools
Another area of advancement is with symptom understanding in real time. It’s at the beginning, but as we see development in digital health tools, I can envision time in the not-too-distant future where AI and the predictive nature of the technology will be able to warn us of a potential catastrophic event. Even our phones and watches are providing some of that today. In the past year I have received two warnings on my iPhone letting me know I have a high probability of falling based on the ”Walking Steadiness” data being collected in real time via my iPhone, which I carry in my pocket.
At this point our medical professionals do not know what to do with that information. Many healthcare professionals are not aware of that feature. The real challenge is the limitation of nonclinical measurements. As such the data cannot be used for a clinical assessment, only anecdotally.
One of the companies I am engaged with is working on a proof of concept integrating this type of digital health technology into patient electronic health record systems, such as EPIC. This allows the HCP to see in a side-by-side view the patient’s clinical data along with their personal health data. This includes data from Oura rings, FitBits, sleep monitors and more. It allows for a more complete picture of a patient. If the patient’s quality of life and true overall health is the goal, this type of view of a patient’s life in real time is critical.
So many companies are involved in meaningful data collection and forward-thinking designs, as well as analysis in real time, that prevention of potential catastrophic events should be imminent. Or at least over the next year or two.
The challenge will continue be compliance and regulatory requirements in a clinical setting. Outside a clinical setting, the focus on patient quality of life will drive the acceptance, especially in the rare disease world.
It’s time to “change the playing field” by raising the bar for quality of life with digital health tools in real life complimenting the life-changing therapies which we are grateful for.
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