STAT+: FDA gets pointed input on ‘optimizing’ advisory committee panels

The day an FDA advisory committee met to weigh the merits of an experimental treatment for amyotrophic lateral sclerosis was one of the worst of Mitze Klingenberg’s life. 

Her son Matt had benefited, she said, from an ALS treatment under review for approval by the Food and Drug Administration. However, the expert panel voted against the therapy on that day, Sept. 27, 2023. Panelists dismissed his experience, and that of other patients, as anecdotal, saying it could stir up false hope, Klingenberg said Thursday during an impassioned speech at an FDA town hall meeting. The meeting, which several patient advocates attended, was meant to dissect existing advisory committee protocols — and come up with ways to “optimize” the agency’s use of the committees.

“This was personally devastating, as my son did have hope, as this treatment worked for him,” she said. “And to be basically ignored and unacknowledged during that time rates the ad comm as the second-most difficult day in our ALS journey — topped only by his initial diagnosis.”

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