Department of Medical Ethics, Philosophy and History of Medicine, Erasmus MC, Rotterdam, the Netherlands; GGz Breburg, Tilburg, the Netherlands.
Department of Biostatistics, Erasmus MC, Rotterdam, the Netherlands; Erasmus School of Health Policy and Management, Erasmus University Rotterdam, the Netherlands; Real-World Data Department, myTomorrows, Amsterdam, the Netherlands.
Psychiatry Res. 2023 Nov;329:115554. doi: 10.1016/j.psychres.2023.115554. Epub 2023 Oct 20.
Some psychiatric patients have exhausted all approved treatment options. Numerous investigational drugs are currently being developed and tested in clinical trials. However, not all patients can participate in clinical trials. Expanded access programs may provide an opportunity for patients who cannot participate in clinical trials to use investigational drugs as a therapeutic option outside of clinical trials. It is unknown to what extent expanded access occurs in psychiatry. We conducted a systematic literature search on PubMed, Embase, and PscyInfo, with additional information from ClinicalTrials.gov, the WHO International Clinical Trials Registry Platform and FDA/EMA approvals, in order to find all expanded access programs ever conducted, globally, in the field of psychiatry. This resulted in a total of fourteen expanded access programs ever conducted in psychiatry. Given the prevalence of psychiatric disorders, the activity in clinical research in psychiatry, the regulatory framework enabling expanded access, and the impact of psychiatric disorders on patients, their families, and society, we had expected a higher utilization of expanded access. We propose that the psychiatric community, with pharmaceutical industry, should consider establishing and optimizing expanded access programs.
一些精神科患者已经用尽了所有批准的治疗方案。目前正在临床试验中开发和测试许多研究药物。然而，并非所有患者都可以参加临床试验。扩展获得途径计划可能为那些无法参加临床试验的患者提供机会，使他们可以在临床试验之外将研究药物作为治疗选择。目前尚不清楚在精神病学中扩展获得途径的程度。我们在 PubMed、Embase 和 PscyInfo 上进行了系统的文献检索，并从 ClinicalTrials.gov、世界卫生组织国际临床试验注册平台和 FDA/EMA 批准中获取了额外信息，以查找全球范围内在精神病学领域开展的所有扩展获得途径计划。这总共导致了在精神病学中开展的十四个扩展获得途径计划。鉴于精神障碍的流行程度、精神病学临床研究的活跃度、使扩展获得途径成为可能的监管框架以及精神障碍对患者、他们的家人和社会的影响，我们原本期望更多地利用扩展获得途径。我们建议精神病学界与制药行业一起考虑建立和优化扩展获得途径计划。
