Murteira Susana, Millier Aurélie, Toumi Mondher
University of Lyon, University Claude Bernard Lyon I, UFR d'Odontologie, 11 rue Guillaume Paradin, 69372 Lyon, Cedex 08, France; Lundbeck SAS, 37-45, Quai du Président Roosevelt, 92445 Issy-les-Moulineaux, Cedex, Paris, France.
Creativ-Ceutical S.A., 215, rue du Faubourg St-Honoré 75008 Paris, France.
J Mark Access Health Policy. 2014 Feb 25;2. doi: 10.3402/jmahp.v2.22814. eCollection 2014.
Drug repurposing is a group of development strategies employed in order to overcome some of the hurdles innate to drug research and development. Drug repurposing includes drug repositioning, reformulation and combination.
This study aimed to identify the determinants of successful market access outcome for drug repurposing in the United States of America (USA) and in Europe.
The case studies of repurposing strategies were identified through a systematic review of the literature. Price information and reimbursement conditions for all the case studies were collected mainly through access of public datasources. A list of attributes that could be associated with market access outcome (price level and reimbursement conditions) was developed, discussed, and validated by an external expert group. Detailed information for all attributes was researched and collected for each case study. Bivariate regression models were conducted to identify factors associated with price change for all repurposing cases. A similar analysis was performed for reformulation and repositioning cases, in the USA and in Europe, separately. A significance level of 5% was used for all analyses.
A total of 144 repurposing case studies were included in the statistical analysis for evaluation of mean price change. Combination cases (the combination of two or more individual drug components) were excluded from the statistical analysis due to the low number of cases retrieved. The main attributes associated with a significant price increase for overall repurposing cases were 'change in administration setting to hospital' (374%, p<0.0001), 'addressing unmet needs' (69%, p<0.05), 'reformulations belonging to Group 3'-that is, change in administration route (117%, p<0.001), and being a repurposed product with the 'same brand name' as the original product (65%, p<0.05).
We found that the ability of the repurposed product to address unmet needs, a reformulation where the target product had a different administration route than the source product, and having a similar brand name for repurposed and original products, were variables that impacted a positive price change for repurposed drugs overall. Our research results also suggested that orphan designation could have a positive impact for repositioning in the USA, in particular. Although a change of administration from ambulatory to hospital setting seemed to be significantly correlated with a price increase for the target product, only one case was retrieved for this parameter; as such, it was not possible to infer a firm correlation between this parameter and a change in price.
药物重新利用是为克服药物研发过程中固有的一些障碍而采用的一组研发策略。药物重新利用包括药物重新定位、重新配方和联合用药。
本研究旨在确定在美国和欧洲药物重新利用成功进入市场的决定因素。
通过对文献的系统回顾确定重新利用策略的案例研究。所有案例研究的价格信息和报销条件主要通过访问公共数据源收集。由一个外部专家组制定、讨论并验证了一份可能与市场准入结果（价格水平和报销条件）相关的属性清单。为每个案例研究研究并收集所有属性的详细信息。进行双变量回归模型以确定所有重新利用案例中与价格变化相关的因素。分别在美国和欧洲对重新配方和重新定位案例进行了类似分析。所有分析的显著性水平均为5%。
总共144个重新利用案例研究纳入了评估平均价格变化的统计分析。联合用药案例（两种或更多种单一药物成分的联合）因检索到的案例数量较少而被排除在统计分析之外。与总体重新利用案例价格显著上涨相关的主要属性是“给药环境改为医院”（374%，p<0.0001）、“满足未满足的需求”（69%，p<0.05）、“属于第３组的重新配方”，即给药途径改变（117%，p<0.001），以及作为与原产品“同名”的重新利用产品（65%，p<0.０5）。
我们发现，重新利用产品满足未满足需求的能力、目标产品与源产品给药途径不同的重新配方，以及重新利用产品和原产品具有相似的品牌名称，这些变量总体上对重新利用药物的积极价格变化产生影响。我们的研究结果还表明，孤儿药指定可能对在美国的重新定位产生积极影响，尤其是。虽然从门诊给药改为医院给药似乎与目标产品价格上涨显著相关，但该参数仅检索到一个案例；因此，无法推断该参数与价格变化之间的确定相关性。
