Roberts Rosemary, Rodriguez William, Murphy Dianne, Crescenzi Terrie
Office of Counter-Terrorism and Pediatric Drug Development, Center for Drug Evaluation and Research, Food and Drug Administration, Rockville, Md 20855, USA.
JAMA. 2003 Aug 20;290(7):905-11. doi: 10.1001/jama.290.7.905.
Approximately 50% to 75% of drugs used in pediatric medicine have not been studied adequately to provide appropriate labeling information. In 1997, Congress passed the Food and Drug Administration Modernization Act (FDAMA), which encouraged pediatric drug development by providing an incentive in the form of additional marketing exclusivity.
To identify new drug labeling information from pediatric studies submitted to the FDA in response to written requests.
Between July 1998 and April 1, 2002, the FDA requested studies on 242 drugs, and 53 drugs were granted exclusivity. As of January 2003, 49 drugs have new labels. Data from the studies of the first 33 drugs with new pediatric information on the label as of April 2002 are included. Significant labeling information was analyzed along with baseline data and types of studies requested.
Safety data and pediatric information for labeled drugs.
There were 53 studies for 33 drug products, 12 (23%) were evaluated for safety only; 23 (43%), safety and efficacy; and 18 (34%), pharmacokinetics and/or pharmacodynamics. Significant new dosing and/or safety information was identified for 12 (36%) drugs. New dosing information was determined for 7 of these drugs. Safety information was defined for gabapentin, propofol, sevoflurane, the combination of ribavirin and interferon alfa-2b, and various betamethasone-containing dermatologic preparations. There was a higher percentage of deaths reported with patients who received propofol compared with controls in the pediatric intensive care unit. Seizures were seen in patients administered sevoflurane. Patients receiving a combination of ribavirin and interferon alfa-2b experienced an increased incidence of suicidal ideation when compared with adults. An unexpectedly high percentage of those receiving betamethasone-containing dermatologic preparations had documented hypopituitary-adrenal axis suppression.
The FDAMA has stimulated pediatric clinical studies resulting in improved understanding of the pharmacokinetics of drugs prescribed in pediatric medicine, important dose changes, and improved safety for children taking certain drugs.
儿科用药中约50%至75%的药物尚未得到充分研究以提供适当的标签信息。1997年，国会通过了《食品药品管理局现代化法案》（FDAMA），该法案通过提供额外市场独占权的激励措施来鼓励儿科药物研发。
从应书面请求提交给美国食品药品监督管理局（FDA）的儿科研究中识别新的药物标签信息。
1998年7月至2002年4月1日期间，FDA要求对242种药物进行研究，53种药物获得了独占权。截至2003年1月，49种药物有了新标签。纳入了截至2002年4月标签上有新儿科信息的前33种药物的研究数据。对重要的标签信息以及基线数据和所要求的研究类型进行了分析。
有标签药物的安全性数据和儿科信息。
对33种药品进行了53项研究，其中12项（23%）仅评估了安全性；23项（43%）评估了安全性和有效性；18项（34%）评估了药代动力学和/或药效学。为12种（36%）药物确定了重要的新给药和/或安全性信息。其中7种药物确定了新的给药信息。确定了加巴喷丁、丙泊酚、七氟烷、利巴韦林与干扰素α-2b联合用药以及各种含倍他米松的皮肤科制剂的安全性信息。在儿科重症监护病房中，接受丙泊酚治疗的患者与对照组相比报告的死亡百分比更高。接受七氟烷治疗的患者出现了癫痫发作。与成年人相比，接受利巴韦林和干扰素α-2b联合用药的患者自杀意念发生率增加。接受含倍他米松的皮肤科制剂治疗的患者中，垂体-肾上腺轴抑制的记录比例意外地高。
FDAMA刺激了儿科临床研究，从而增进了对儿科用药药代动力学的了解、进行了重要的剂量调整，并提高了服用某些药物儿童的安全性。
