Moreno Lucas, Pearson Andrew D J
a 1 CNIO-HNJ Clinical Trials Unit, Hospital Niño Jesús , Madrid, Spain.
b 2 Instituto de Investigación Sanitaria La Princesa , Madrid, Spain.
Expert Rev Clin Pharmacol. 2015;8(6):665-7. doi: 10.1586/17512433.2015.1077699. Epub 2015 Aug 24.
Despite numerous advances with the incorporation of multimodal treatment, children with cancers have a major unmet need to access new drugs. Still, a reduced number of new targeted drugs is evaluated in pediatric patients and very few of them progress into late phase trials and clinical use. Changes required include: increased collaboration between all stakeholders, improved understanding of disease biology and its incorporation into early clinical trials, faster and more efficient early and late clinical trials, better incentives for pharmaceutical companies and improving access to new drugs across the globe.
尽管在采用多模式治疗方面取得了诸多进展，但癌症患儿在获取新药方面仍有重大未满足需求。然而，在儿科患者中评估的新靶向药物数量有所减少，其中很少有药物能进入后期试验和临床应用阶段。需要做出的改变包括：所有利益相关者之间加强合作，更好地理解疾病生物学并将其纳入早期临床试验，加快早期和后期临床试验的速度和效率，为制药公司提供更好的激励措施，以及改善全球范围内新药的可及性。
