Discipline of Public Health, University of Adelaide, Adelaide, South Australia, Australia.
Pharmacoeconomics. 2010;28(1):1-17. doi: 10.2165/11318770-000000000-00000.
Strategies to change the existing mix of innovative and 'me-too' drugs are intended to increase societal value of a given investment in R&D by providing an incentive for firms to invest in drugs that are more likely to be clinically innovative. How can financial incentives be used to change this mix? Will a strategy have its intended consequence or will it have the unintended outcome of reducing the rate at which the population burden of disease is reduced? The perspective of this review is a country such as Australia, Canada or the UK that has universal health insurance and a drug reimbursement process that is informed by economic evidence. A review of the literature was performed and the views of both the proponents and the opponents of such strategies and the mechanisms by which they could be implemented were summarized. The debate is based largely on hypothesized responses by firms to changes in incentives rather than empirical evidence. The main point of contention is whether a changed mix of new molecular entities (NMEs) increases or decreases the total amount of clinical innovation launched each year. The argument presented in this article is that, despite the limited empirical evidence, it is possible to improve our assessment of the likely costs and consequences of a proposed strategy by appealing to economic theory and observations about the reimbursement process. First, the empirical evidence supporting the view that changing a mix of drugs will improve clinical innovation is based on the average launched drug, not the marginal innovative drug otherwise not developed, and therefore could be misleading. Second, the dynamic and complex nature of evidence of clinical innovation will reduce the feasibility of using contractually based mechanisms to implement such a strategy. Also, a single country is unlikely to have an impact on R&D decisions, and variation in the per capita economic value of new drugs would make multi-jurisdiction contracts with one firm difficult to implement. Third, the quality of evidence of the clinical innovation of the lead drug could be reduced if there are fewer or no follow-on drugs. Finally, the existing inefficiencies in the process of displacement to finance new technologies from a capped budget reduces the efficiency with which any additional potential clinical innovation from NMEs will be translated to reduced population burden of disease. The article concludes that it is possible that such a strategy could be costly to implement, and the impact on global burden of disease uncertain in both direction and magnitude. This is likely to be the case even if the average clinical innovation content of innovative NMEs is higher than for me-too NMEs and the mechanisms designed to change the mix of NMEs are effective. Other options to improve the effectiveness with which pharmaceutical clinical innovation reduces burden of disease should be explored, including improved efficiency of both firm R&D and the process of disinvestment to finance new technologies.
改变现有创新药物和“me-too”药物组合的策略旨在通过为企业提供投资于更有可能具有临床创新性的药物的激励,来提高研发投资的社会价值。如何利用财务激励来改变这种组合?该策略是否会产生预期的结果,还是会产生降低疾病人口负担的速度的意外结果?本综述的观点是,针对像澳大利亚、加拿大或英国这样的国家,这些国家拥有全民医疗保险和一个由经济证据来指导的药物报销流程。对文献进行了回顾,并总结了支持和反对这些策略的观点,以及实施这些策略的机制。这场辩论主要基于企业对激励变化的假设反应,而不是基于经验证据。争论的主要焦点是新分子实体(NME)的组合变化是增加还是减少每年推出的临床创新总量。本文提出的观点是,尽管经验证据有限,但通过诉诸经济理论和对报销流程的观察,我们可以更好地评估拟议策略的可能成本和后果。首先,支持改变药物组合将改善临床创新的观点的经验证据是基于推出的平均药物,而不是否则不会开发的边际创新药物,因此可能具有误导性。其次,临床创新证据的动态和复杂性质将降低使用基于合同的机制来实施这种策略的可行性。此外,单个国家不太可能对研发决策产生影响,而新药的人均经济价值的差异将使得与一家公司签订多国合同变得困难。第三,如果后续药物较少或没有,则可能会降低主导药物临床创新证据的质量。最后,从一个上限预算中为新技术融资而取代现有技术的过程中的效率低下,降低了任何额外的潜在临床创新从 NME 转化为降低人口疾病负担的效率。文章的结论是,实施这样的策略可能代价高昂,对全球疾病负担的影响无论是在方向还是在程度上都不确定。即使创新 NME 的平均临床创新含量高于“me-too”NME,并且旨在改变 NME 组合的机制有效,情况也可能如此。应该探索其他改善药物临床创新降低疾病负担的有效性的选择,包括提高企业研发效率和为新技术融资而进行撤资的过程效率。