As a physician, I support the FDA's role in protecting patients from adverse events, ensuring safety & efficacy.
As a physician living with #ALS, I've recognized there must be different regulatory flexibility for untreatable lethal diseases.
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As a physician living with #ALS, I've recognized there must be different regulatory flexibility for untreatable lethal diseases.
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https://twitter.com/NicholasFlorko/status/1401942843321634832
As a physician who was denied access to clinical trials for 8 yrs, I have been #DyingWaiting for some drug, any drug, with some efficacy to be approved.
I have been horrified by the FDA's regulatory rigidity & inhumanity toward the ALS community.
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I have been horrified by the FDA's regulatory rigidity & inhumanity toward the ALS community.
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After multiple meetings, a new #ALS Guidance Document in 2019, and a flawed subjective tool to evaluate efficacy as a trial endpoint, the FDA has refused to honor its promise to exercise regulatory flexibility in recognition of the "critical unmet need."
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There is no way to understand the horrific reality of ALS unless you experience it.
ALS suffering is ignored against a purported risk of an adverse event. What is more adverse than nearly dying every single day when you choke on your own saliva... ?
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ALS suffering is ignored against a purported risk of an adverse event. What is more adverse than nearly dying every single day when you choke on your own saliva... ?
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...when your computer freezes & you're trapped without eye gaze, when your trach dislodges so you begin to suffocate as you use the bed pan, when your caregiver puts your hand in scalding water without checking the temp & all you can do is hope that she notices your tears?
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If the FDA can approve Aducamunab, they must approve #NurOwn.
Regulatory flexibility must be applied to 100% fatal diseases like ALS with vast biological & clinical heterogeneity.
ALS has more heterogeneity than ALZ. The ALS population is miniscule, 30,000 vs 6,000,000.
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Regulatory flexibility must be applied to 100% fatal diseases like ALS with vast biological & clinical heterogeneity.
ALS has more heterogeneity than ALZ. The ALS population is miniscule, 30,000 vs 6,000,000.
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A smaller surviving trial population makes robust clinical trials difficult.
ALS has far more heterogeneity than ALZ making proof of efficacy even more onerous with trials of 100 patients & we don't live long enough to increase trial populations.
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ALS has far more heterogeneity than ALZ making proof of efficacy even more onerous with trials of 100 patients & we don't live long enough to increase trial populations.
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NurOwn had improvement in neuroinflammation, neurodegeneration & neuroprotection biomarkers. 100% in NurOwn arm & 0% of placebo arm.
I'm hopeful the FDA's humanity with Aducamunab will be the bridge to many more drugs in an ALZ cocktail for people fighting the disease.
/end
I'm hopeful the FDA's humanity with Aducamunab will be the bridge to many more drugs in an ALZ cocktail for people fighting the disease.
/end
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