Developing Endpoints to Facilitate Clinical Trials in Rare Diseases

 描述（由申请人提供）：神经纤维瘤病1（NF 1）、神经纤维瘤病2（NF 2）和神经鞘瘤病是一组相关的神经遗传性肿瘤抑制综合征，具有发生神经鞘肿瘤的倾向。由于NF1、NF2和神经鞘瘤病患者中遇到的大多数肿瘤在组织学上是良性的，因此总生存期不是这些患者的适当临床试验结果。2011年，一组NF研究人员同意定期开会，为这些条件开发新的成果。神经纤维瘤病和神经鞘瘤病（REiNS）国际合作的反应评价由来自多个国家和学科的专家组成，致力于神经纤维瘤病，神经鞘瘤病和临床试验的研究。REiNS在冬季举行年度会议，讨论临床试验结果的建议。每年，REiNS的领导小组都会选择正在讨论的主题的专家，提出每个工作组的建议，供更大的小组讨论。这些会议产生了多篇出版物，这些出版物已被纳入正在进行的临床试验中， 已经发表在同行评审的文献中。年度REiNS会议是一个重要的论坛，为这些罕见的肿瘤抑制综合征产生新的想法和方法。2015年会议的主题是开发终点，以促进罕见疾病的临床试验，并将包括来自食品和药物管理局，癌症治疗评估计划，儿童肿瘤基金会和国家癌症研究所的外部发言人。

 DESCRIPTION (provided by applicant): Neurofibromatosis 1 (NF1), neurofibromatosis 2 (NF2), and schwannomatosis are a group of related neurogenetic tumor suppressor syndromes that share a predisposition to develop nerve sheath tumors. Because most tumors encountered in patients with NF1, NF2, and schwannomatosis are histologically benign, overall survival is not an appropriate clinical trial outcome for these patients. In 2011, a group of NF researchers agreed to meet periodically to develop novel outcomes for these conditions. The Response Evaluation in Neurofibromatosis and Schwannomatosis (REiNS) International Collaboration consists of specialists from multiple countries and disciplines dedicated to the study of neurofibromatosis, schwannomatosis, and clinical trials. REiNS holds an annual conference during the winter in which recommendations for clinical trial outcomes are discussed. Each year, the Leadership Group of REiNS selects experts on the topics under discussion to present the recommendations of each working group for discussion by the larger group. These meetings have led to multiple publications that have been incorporated into ongoing clinical trials and that have been published in the peer-reviewed literature. The annual REiNS meeting is a critical forum for generating new ideas and approaches to these rare tumor suppressor syndromes. The 2015 conference topic is Developing Endpoints to Facilitate Clinical Trials in Rare Diseases and will include outside speakers from the Food and Drug Administration, the Cancer Therapy Evaluation Program, the Children's Tumor Foundation, and the National Cancer Institute.