Pediatric Home Assistance and Palliative Care, Department of Pediatrics, AAS5 Friuli Occidentale, via Montereale 24, 33170, Pordenone, Italy.
Department of Pediatrics, S. Maria delle Croci Hospital, AUSL della Romagna, Ravenna, Italy.
Ital J Pediatr. 2018 Nov 29;44(1):144. doi: 10.1186/s13052-018-0584-8.
Paediatric palliative care (PPC) aim to ensure the control of symptoms and the best possible quality of life for patients whose underlying disease, characterized by an unstoppable evolution and negative prognosis, no longer responds to specific treatments. The scientific evidence in this context are very deficient and, in order to obtain welfare objectives consistent with the situation, in the overwhelming majority of cases the prescription of drugs is off-label for indication of use and/or for age and/or for way of administration and/or formulation. The Agenzia Italiana del Farmaco - AIFA and the Italian Society of Palliative Care (Società Italiana di Cure Palliative - SICP), under a dedicated working group, wrote a document that collects the scientific evidence available to support the off-label use of medicines more frequently used in PPC. The goal is to certify the consolidated off-label use of these drugs and propose their use under the Law 648/96, in the absence of data from its pivotal clinical trials. Aim of the commentary is to report the conditions for this important work and to present the 10 drugs, usually used off-label in PPC and in pain therapy, now included in Law 648/96.
This work is deemed essential to resolve, at least in part, the lack of availability of medicines researched and approved.
儿科姑息治疗（PPC）旨在控制症状并为那些潜在疾病的患者提供最佳的生活质量，这些患者的疾病具有不可阻挡的发展和负面预后，对特定治疗已不再有效。在这种情况下，科学证据非常缺乏，为了实现与实际情况相符的福利目标，在绝大多数情况下，药物的处方是超适应证使用，或者是超年龄、超给药途径或超剂型使用。意大利药品管理局（Agenzia Italiana del Farmaco - AIFA）和意大利姑息治疗学会（Società Italiana di Cure Palliative - SICP）在一个专门的工作组的指导下，编写了一份文件，收集了支持在 PPC 中更频繁使用的药物进行超适应证使用的现有科学证据。其目的是证明这些药物的惯常超适应证使用，并在缺乏关键临床试验数据的情况下，根据第 648/96 号法律建议使用这些药物。本文的目的是报告这项重要工作的条件，并介绍目前已被纳入第 648/96 号法律的 10 种药物，这些药物通常在 PPC 和疼痛治疗中被超适应证使用。
这项工作对于解决药品研究和批准的不足问题具有重要意义。
