On the recordApril 4, 2022
Key among these challenges is reaching agreement with regulators about determining the appropriate efficacy endpoints to support approval of innovative medicines for rare diseases.
Source
congress.govKey among these challenges is reaching agreement with regulators about determining the appropriate efficacy endpoints to support approval of innovative medicines for rare diseases.
Emphasizes the need for regulatory agreement on efficacy endpoints for rare disease drug approvals.
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Thank you, Senator Casey, for organizing this hearing and for all you do to improve support for caregivers.
I am disappointed that Dr. de la Torre defied the subpoena this Committee authorized and has chosen not to appear today.