Kids v Cancer, Washington, D.C.
U.S. Food and Drug Administration, Silver Spring, MD.
Am Soc Clin Oncol Educ Book. 2020 May;40:1-10. doi: 10.1200/EDBK_278995.
Patients and their families, physicians, drug companies, and regulatory agencies have common goals: to find effective therapies for life-threatening conditions. In oncology, the lines between clinical research and treatment are often blurred; parents and physicians of patients who have exhausted standard-of-care treatments and cannot participate in a clinical trial are likely to consider seeking compassionate use access to investigational drugs; however, knowledge and perspectives about compassionate use may differ among these groups. There are unique considerations associated with providing compassionate use to children diagnosed with cancer, including evaluation for potential developmental toxicities, the need for pediatric-specific dosing and formulations, informed consent, and, when appropriate, patient assent. Positive impacts of providing access to investigational therapies to children include potential treatment benefits to patients who obtain investigational agents as well as benefits to future patients if data from expanded access support drug development for childhood cancer. Challenges for physicians seeking compassionate use access to investigational drugs for their patients include obtaining the drug sponsor's agreement to provide the investigational drug as well as lack of knowledge about the process and regulatory requirements. Clinical trials in oncology provide the possibility of therapeutic benefit for pediatric patients; when feasible and warranted, these benefits should also be available to patients on a compassionate use basis outside of trials.
患者及其家属、医生、制药公司和监管机构有共同的目标：为危及生命的疾病寻找有效的治疗方法。在肿瘤学领域，临床研究和治疗之间的界限常常模糊不清；已经用尽标准治疗方法且无法参与临床试验的患者的父母和医生可能会考虑寻求同情用药来获得试验药物；然而，这些群体对同情用药的认识和看法可能存在差异。为被诊断患有癌症的儿童提供同情用药有一些独特的考虑因素，包括对潜在发育毒性的评估、儿科特定剂量和配方的需求、知情同意，以及在适当情况下获得患者同意。为儿童提供试验治疗方法的积极影响包括为获得试验药物的患者带来潜在的治疗益处，以及如果扩展用药数据支持儿童癌症药物开发，那么对未来患者也有好处。医生为其患者寻求同情用药的挑战包括获得药物赞助商同意提供试验药物，以及对该过程和监管要求缺乏了解。肿瘤学临床试验为儿科患者提供了治疗益处的可能性；在可行和有必要的情况下，也应在试验之外基于同情用药为患者提供这些益处。
