Curr Med Res Opin. 2010 Sep;26(9):2119-26. doi: 10.1185/03007995.2010.505137.
Abstract The growing number of biosimilars presents challenges to regulatory and health technology assessment (HTA) systems. This paper illustrates these challenges by focusing on biosimilars used in the oncological setting. In particular, discordances between data required by regulatory and HTA authorities potentially deprive patients of effective treatments and hinder optimal resource allocation. Regulatory and HTA authorities need to harmonize requirements to foster the development and widespread use of biosimilars, which potentially release considerable resources. The authors believe that often-inappropriate methodology creates a very real chance that HTA authorities will reject some biosimilars. This would effectively extend patent protection and, in the absence of competitor pressure from biosimilars, result in prices remaining unnecessarily high. The authors propose that HTA organizations should accept pharmacokinetic and pharmacodynamic equivalence between the brand and the biosimilar as a proxy of biological comparability. HTA organizations should then adopt, in the absence of compelling reasons otherwise, cost-minimization analysis (CMA) as the basis of the cost-effectiveness deliberations. In the absence of adequate studies demonstrating equivalent efficacy, a prerequisite of CMA, HTA organizations should require threshold analysis. Once approved, biosimilar manufacturers and regulators should maintain rigorous pharmacovigilance to exclude immunoreactivity or other rare adverse events. Furthermore, cancer centres and trusts should regularly audit and publish the impact of biosimilars on clinical outcomes and resource use. When appropriate, regulatory and HTA authorities should demand revised cost-effectiveness analyses from biosimilar manufacturers. This approach would hone the accuracy of the cost-effectiveness analyses, protect patients and allow health services rapid access to low cost treatments.
生物类似药的数量不断增加，给监管和卫生技术评估（HTA）系统带来了挑战。本文通过关注肿瘤学领域使用的生物类似药，说明了这些挑战。具体而言，监管机构和 HTA 机构所需的数据之间存在差异，这可能使患者无法获得有效治疗，并阻碍资源的最佳配置。监管机构和 HTA 机构需要协调要求，以促进生物类似药的开发和广泛使用，这可能会释放出大量资源。作者认为，不恰当的方法常常会给 HTA 机构拒绝某些生物类似药带来非常现实的机会。这将有效地延长专利保护期，并且在没有生物类似药竞争压力的情况下，导致价格保持不必要的高位。作者建议 HTA 组织应接受品牌药和生物类似药之间的药代动力学和药效学等效性，作为生物学可比性的替代指标。在没有其他令人信服的理由的情况下，HTA 组织应采用成本最小化分析（CMA）作为成本效益审议的基础。在缺乏充分研究证明等效疗效的情况下，即 CMA 的前提条件，HTA 组织应要求进行阈值分析。一旦获得批准，生物类似药制造商和监管机构应保持严格的药物警戒，以排除免疫原性或其他罕见的不良事件。此外，癌症中心和信托基金应定期审核并公布生物类似药对临床结果和资源利用的影响。在适当的情况下，监管机构和 HTA 当局应要求生物类似药制造商进行修订后的成本效益分析。这种方法将提高成本效益分析的准确性，保护患者，并使卫生服务部门能够快速获得低成本治疗。
