Regulatory agencies face operational challenges due to government funding uncertainties, affecting drug submissions and approvals. The FDA is suspending acceptance of new drug submissions amid a looming shutdown, while complete response letters have hindered critical rare disease treatments like Menkes disease. Several drugs, including novel BTK inhibitors for chronic hives and orphan-designated gene therapies, have received approvals or designations, reflecting ongoing innovation despite policy headwinds. Drug pricing policies negotiated with industry players like Pfizer also shape market dynamics and patient access.