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This page is an AI-assisted Turkish summary of the study presented at the 26th World Congress of Psychiatry. The study belongs to the presenting authors; please refer to the original source when citing it.

EV0332

Bridging the gap: the role of the FDA expanded access (compassionate use) program in treating serious neuropsychiatric conditions

BRIDGING THE GAP: THE ROLE OF FDA EXPANDED ACCESS (COMPASSIONATE USE) IN TREATING SERIOUS PSYCHIATRIC CONDITIONS.

The FDA expanded access pathway provides treatment access for patients with serious neuropsychiatric conditions who have exhausted approved options and generates real-world data, but it entails ethical constraints concerning safety, equity, and participation in clinical trials.

Objective: This review examines the role of the FDA expanded access (EA) pathway in treating serious neuropsychiatric conditions, given the inadequacy of approved treatments for treatment-resistant psychiatric and neurological disorders and the exclusion of the most vulnerable patients by clinical trial eligibility criteria. Methods and framework: The regulatory framework (21 CFR Part 312, Subpart I) is described under three categories: single-patient IND (individual EA), intermediate-size population EA, and treatment IND (widespread EA). It is noted that the FDA generally responds to emergency requests within 24 to 48 hours and that institutional review board oversight and documented informed consent are required. As a clinical example, a 74-year-old woman with amyloid PET-confirmed mild to moderate Alzheimer's disease (MMSE 23) in 2022 is presented: in the LIFT-AD trial (NCT04488419), she was initially randomized to the double-blind placebo phase, followed by the open-label subcutaneous fosgonimeton (ATH-1017) 40 mg/day arm; her MMSE score declined to 15 during the trial and to 3 at trial completion. To maintain continuity of treatment, she transitioned to expanded access, with an application submitted on October 24, 2024, and authorization granted on November 14, 2024, within three weeks. The patient continued receiving ATH-1017 40 mg/day with monitoring of vital signs, safety laboratory tests, ECG, MMSE, and C-SSRS; the last treatment was reported as February 28, 2026. High-impact areas identified include EA data on ketamine and esketamine accelerating the 2019 approval process; cannabidiol (Epidiolex) reaching more than 300 pediatric patients through EA before the submission of its approval application, with real-world data directly supporting FDA labeling; psilocybin in patients with cancer and MDMA-assisted therapy for combat-related posttraumatic stress disorder; gene therapies for FTD-GRN and Niemann-Pick disease type C; and novel agents for schizophrenia unresponsive to clozapine. From an ethical perspective, EA is described as supporting the right to access potentially lifesaving treatments and aligning with the principle of nonabandonment, while risks are highlighted, including unknown long-term safety profiles, placebo effects and reporting bias, inequities favoring educated and affluent patients, patients turning away from clinical trials, and manufacturers denying access for commercial reasons. Conclusions and recommendations: Recommendations include simplifying EA applications, mandating manufacturer transparency, expanding payer coverage, translating outcomes into real-world evidence through centralized EA registries, and harmonizing FDA regulations with the EMA compassionate use, Health Canada, and TGA frameworks.

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