Research Centre for Pharmaceutical Care and Pharmaco-economics, Faculty of Pharmaceutical Sciences, Katholieke Universiteit Leuven, Leuven, Belgium.
J Med Econ. 2009 Sep;12(3):211-8. doi: 10.3111/13696990903260094.
This article discusses health economic challenges of research and development, registration, pricing and reimbursement of biopharmaceuticals and biosimilars. A literature search was carried out of PubMed, Centre for Reviews and Dissemination databases, Cochrane Database of Systematic Reviews and EconLit up to March 2009.
The development process of biopharmaceuticals is risky, lengthy, complex and expensive. Registration is complicated by the inherent variation between biopharmaceuticals. Also, as biopharmaceuticals are likely to be efficacious in a subgroup of the patient population, there is a need to select the most responsive target population and to identify biomarkers. To inform pricing and reimbursement decisions, the development process needs to collect comparative data to calculate the incremental cost effectiveness and budget impact of biopharmaceuticals. There is a role for innovative mechanisms such as risk-sharing arrangements to reimburse biopharmaceuticals.
Given that biosimilars are similar, but not identical to the reference biopharmaceutical, the development process needs to generate clinical trial data in order to gain marketing authorisation. From a health economic perspective, the question arises whether inherent differences between biopharmaceuticals and biosimilars produce differences in safety, effectiveness and costs: to date, this question is unresolved. The early inclusion of health economics in the process of developing biopharmaceuticals and biosimilars is imperative with a view to demonstrating their relative (cost) effectiveness and informing registration, pricing and reimbursement decisions.
本文讨论了生物制药和生物类似药的研发、注册、定价和报销所面临的健康经济挑战。对 PubMed、Cochrane 系统评价数据库、中心评价和传播数据库以及 EconLit 进行了截至 2009 年 3 月的文献检索。
生物制药的开发过程具有风险大、周期长、复杂且昂贵的特点。由于生物制药之间存在固有差异，注册程序变得复杂。此外，由于生物制药很可能对患者群体中的亚组有效，因此需要选择最敏感的目标人群并识别生物标志物。为了为定价和报销决策提供信息，开发过程需要收集比较数据，以计算生物制药的增量成本效益和预算影响。需要创新机制，如风险分担安排，来报销生物制药。
鉴于生物类似药与参照生物制药相似，但并不完全相同，因此开发过程需要生成临床试验数据以获得市场授权。从健康经济学的角度来看，出现了一个问题，即生物制药和生物类似药之间的固有差异是否会导致安全性、有效性和成本方面的差异：迄今为止，这个问题尚未得到解决。从展示其相对（成本）有效性并为注册、定价和报销决策提供信息的角度来看，在开发生物制药和生物类似药的过程中尽早纳入健康经济学至关重要。
