Office of Hematology and Oncology Products, Center for Drug Evaluation and Research, U.S. Food and Drug Administration, Silver Spring, Maryland 20901-0002, USA.
Pediatr Blood Cancer. 2013 Apr;60(4):531-7. doi: 10.1002/pbc.24346. Epub 2013 Jan 17.
The Food and Drug Administration (FDA) Modernization Act, enacted in 1997, created a pediatric exclusivity incentive allowing sponsors to qualify for an additional 6 months of marketing exclusivity after satisfying the requirements outlined in the Written Request (WR). This review evaluates the impact of the WR mechanism on the development of oncology drugs in children.
A search of the FDA document archiving, reporting, and regulatory tracking system was performed for January 1, 2000 to December 31, 2010. Drugs were identified and pediatric-specific labeling information was obtained from Drugs@fda.gov and FDA Pediatric Labeling Changes Table.
Fifty WRs have been issued for oncology drugs. Pediatric studies have been submitted for 14 drugs. Thirteen received pediatric exclusivity. As of December 31, 2010, labeling changes have been made for 11 drugs. Three drugs were approved for pediatric use.
WRs have provided a mechanism to promote the study of drugs in pediatric malignancies. Information from studies resulting from the WRs regarding safety, pharmacokinetics, and tolerability of oncology drugs has been incorporated into pediatric labeling for 11/14 of the drugs. Earlier communication and collaboration between the FDA, National Cancer Institute, clinical investigators, and commercial sponsors are envisioned to facilitate the identification and prioritization of emerging new drugs of interest for WR consideration. Since this is the only regulatory mechanism, resulting from specific legislative initiatives relevant to cancer drug development for children, efforts to enhance its impact on increasing drug approval for pediatric cancer indications are warranted.
1997 年颁布的《食品和药物管理局现代化法案》设立了儿科独占激励机制，规定满足书面申请（WR）中概述的要求后，申办方可获得额外 6 个月的市场独占期。本研究评估 WR 机制对儿童肿瘤药物开发的影响。
对 FDA 文件归档、报告和监管跟踪系统进行了 2000 年 1 月 1 日至 2010 年 12 月 31 日的检索。从 Drugs@fda.gov 和 FDA 儿科标签变更表中获取药物和儿科特定标签信息，确定药物。
WR 已针对 50 种肿瘤药物发布。已提交了 14 种儿科研究药物。13 种获得儿科独占权。截至 2010 年 12 月 31 日，11 种药物的标签已更改。3 种药物已获准儿科使用。
WR 为研究儿科恶性肿瘤药物提供了一种机制。WR 研究产生的有关肿瘤药物安全性、药代动力学和耐受性的信息已纳入 14 种药物中的 11 种药物的儿科标签。预计 FDA、国家癌症研究所、临床研究者和商业赞助商之间将更早地进行沟通与合作，以确定和优先考虑新出现的、有 WR 考虑价值的新药。由于这是唯一一种与儿童癌症药物开发相关的特定立法举措产生的监管机制，因此有必要努力增强其对增加儿科癌症适应证药物批准的影响。
