Doos Lucy, Ward Derek, Stevens Andrew, Packer Claire
NIHR Horizon Scanning Research and Intelligence Centre Institute of Applied Health Research University of Birmingham Birmingham UK.
Institute of Applied Health Research University of Birmingham Birmingham UK.
J Pharm Health Serv Res. 2016 Jun;7(2):117-122. doi: 10.1111/jphs.12132. Epub 2016 Mar 22.
To determine the accuracy of pharmaceutical companies' predictions of drug licensing timeframes for their products in late stage clinical development.
We compared predicted licensing dates provided to the National Institute for Health Research Horizon Scanning Research and Intelligence Centre by pharmaceutical companies against actual marketing authorisation application (MAA) and marketing authorisation (MA) dates published by the European Medicines Agency for drugs granted authorisation between 2009 and 2013.
One hundred and twenty-three drugs met our inclusion criteria. About 78% were new drugs and 16% had orphan designation. Less than half (44%) and less than a quarter (24%) of MAA and MA predictions respectively were considered accurate (same month or 1 month either side of the actual date). Pharmaceutical companies were significantly more accurate in predicting MAA dates than MA dates ( < 0.001). For accurate predictions, the mean duration between the prediction being made and the actual MAA and MA dates were 17.5 and 18.7 months respectively. Out of the total 108 MA predictions, almost two-thirds (65.4%, 16/26) of short-term predictions (made in the 2 years prior to the actual MA) were accurate. For predicted dates that were earlier than the actual MA date, there was a positive relationship between accuracy and the time between the prediction and authorisation.
Even in predicting near events from well-informed sources, accuracy is imperfect. There appears to be an optimum time for the provision of accurate information on predicted MAA and MA dates for drugs. This information is crucial for effective early awareness and alert activities.
确定制药公司对处于临床后期开发阶段产品的药物许可时间框架预测的准确性。
我们将制药公司提供给英国国家卫生研究院地平线扫描研究与情报中心的预测许可日期，与欧洲药品管理局公布的2009年至2013年期间获批药物的实际上市许可申请（MAA）和上市许可（MA）日期进行了比较。
123种药物符合我们的纳入标准。约78%为新药，16%有孤儿药认定。MAA和MA预测分别只有不到一半（44%）和不到四分之一（24%）被认为是准确的（实际日期当月或前后1个月）。制药公司在预测MAA日期方面比预测MA日期的准确性显著更高（<0.001）。对于准确的预测，做出预测与实际MAA和MA日期之间的平均时长分别为17.5个月和18.7个月。在总共108次MA预测中，近三分之二（65.4%，16/26）的短期预测（在实际MA前2年做出）是准确的。对于早于实际MA日期的预测日期，准确性与预测和获批之间的时间存在正相关关系。
即使是对来自消息灵通来源的近期事件进行预测，准确性也并不完美。似乎存在一个提供药物预测MAA和MA日期准确信息的最佳时间。该信息对于有效的早期认知和警报活动至关重要。
