Edward Smith, MD, a neurologist, discusses why starting a new therapy can be anxiety-provoking in rare disease and how concerns about effectiveness, side effects, and future options shape patient decision-making.
Transcript
The biggest anxieties, understandably, are typically around, “What if it doesn’t help?” Or, “What if it doesn’t help like we’re hoping it would help?” Or, “What if it doesn’t help the way that we’re seeing it help certain people on social media?” And then, “What if it hurts? What if I have a bad outcome, a bad side effect?”
And depending on the recommended treatment, we can be talking about something relatively mild: An upset stomach, or maybe some transient GI issues. Or we could be talking about kidney failure and hospital admission, and even all the way to potentially dying from a particular, treatment recommendation.
The level of anxiety that comes with starting a new therapy … I think a lot of that is driven by the fear of not having an adequate response and rare disease.
There’s also the anxiety that, “If I start treatment A, am I going to be unable to do treatment B that might save me in a clinical trial right now but not be available yet?”
“My decision to start this one treatment: Will it make me ineligible potentially for better treatments down the road?”
And that’s a real thing in the rare disease space. Whether it’s, you know, access to another commercially available drug or one that in trials looks promising and may be available soon.
