现代药物发现包括以下几个环节:苗头化合物(Hit compound)的筛选与发现、 [3]药物化学研究即从苗头化合物至先导化合物(Hit to Lead)[4]和先导化合物优化(Lead optimization)。其中优化过程涵盖了:增加与靶点的亲和力(提高活性)、增加配体选择性(减少副作用)、提高效力或活性、提高化合物代谢稳定性(以增加生物半衰期 )和提高药物的口服生物利用度。只要找到某化合物可满足以上所有条件,药物开发过程就可以继续推进,如若开发过程成功结束,将使用开发的新药开展临床试验,以在人体上测试该新药的安全性和有效性。[5]
现代新药发现过程属于资本密集型产业,这些资本涉及制药企业的大量投资以及政府提供的政策补助和贷款担保。尽管药物科技和现代生物理论的不断进步和对药物发现颇有帮助,但迄今为止药物发现仍然是一个漫长、昂贵、困难且低效的过程,发现全新疗效的成功药物的数量很少。[6]2010年统计每个新化学实体的研发费用约为18亿美元。[7]21世纪针对药物的基础研究工作主要由政府和慈善组织资助,而药物的后期开发主要由制药公司或风投资本资助。[8]药品的获准上市前,必须经历几个临床试验阶段,若成功才可通过新药批准程序并上市销售,在美国称为新药申请(New Drug Application,NDA)。
当一种药物在其整个研究过程中证明其在预计的治疗用途中是安全且有效时,药品研发公司可以向药监部门提交申请,即所谓的新药申请(New drug application,NDA)。[125][126]药监部门会检查并研究药物研发企业递交的NDA相关资料,根据该药物在临床前研究和人体临床实验中的安全性、药物作用的特异性,药物的剂量,药物的有效性等信息,做出是否批准候选药物科通过该申请的决定。通过NDA的药物即可商业化上市并销售。[127]
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