Department of Pharmacy Services, Michigan Medicine, Ann Arbor, MI, USA.
Am J Health Syst Pharm. 2022 Nov 22;79(23):2118-2127. doi: 10.1093/ajhp/zxac242.
The process of providing treatment with investigational drugs through expanded access is explained. Roles and informational resources for pharmacists are discussed.
Expanded access is a regulatory pathway for the treatment of serious or life-threatening diseases or conditions with investigational agents outside of clinical trials. In the setting of no available therapies or ineligibility for clinical trials, a patient and their treating physician may pursue therapies that are not approved by the Food and Drug Administration (FDA). The drug manufacturer, FDA, and institutional review boards are required stakeholders in the expanded access process. Other pathways for obtaining investigational agents outside of clinical trials, including federal Right to Try and emergency use authorization, exist but differ in their level of involvement of these key stakeholders. Pharmacists are equipped to be involved in therapy identification, risk vs benefit evaluations, therapy preparation and administration, supportive care, transitions of care, and regulatory compliance. Specific websites, publications, and organizations can aid in navigating expanded access.
Combining elements of traditional clinical care and research, expanded access involves direct treatment with non-FDA-approved agents outside of a clinical trial. Healthcare providers should be aware of the possibility of providing investigational treatments after all approved options have been exhausted.
解释通过扩大准入途径提供研究药物治疗的过程。讨论药剂师的角色和信息资源。
扩大准入是一种监管途径，用于在临床试验之外用研究性药物治疗严重或危及生命的疾病或病症。在没有可用疗法或不符合临床试验条件的情况下，患者及其治疗医生可能会寻求未经食品和药物管理局 (FDA) 批准的疗法。药品制造商、FDA 和机构审查委员会是扩大准入过程中必需的利益相关者。临床试验之外获得研究性药物的其他途径，包括联邦“尝试权”和紧急使用授权，虽然存在，但在这些主要利益相关者的参与程度上有所不同。药剂师有能力参与治疗方案的确定、风险与获益评估、治疗准备和管理、支持性护理、治疗过渡以及法规遵从性。特定的网站、出版物和组织可以帮助扩大准入途径。
扩大准入将传统的临床护理和研究的元素结合在一起，涉及在临床试验之外直接使用未经 FDA 批准的药物进行治疗。在所有已批准的治疗方案都已用尽的情况下，医疗保健提供者应该意识到提供研究性治疗的可能性。
