The role of health technology assessment bodies in shaping drug development.
The role of health technology assessment bodies in shaping drug development.
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DOI：
10.2147/dddt.s49935
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发表时间：
2014
期刊：
影响因子：
--
通讯作者：
Jommi C
中科院分区：
文献类型：
--
作者：
Ciani O;Jommi C
大多数发达国家都建立了利用卫生技术评估为决策提供信息的做法。与许可机构相比，HTA机构的利益不同，因此对证据的要求也不同。药物化合物的覆盖标准或报销决定各不相同；然而，作为HTA的一部分，通常包括比较有效性评估。这种类型的临床数据可能超出市场授权所需的数据，从而在监管和报销提交之间造成额外的证据差距。从制药公司的角度来看，提交给HTA机构的相关性正在不断增加，因为市场前景受到第三方支付者覆盖范围的强烈影响。在这项研究中，我们的目标是描述当前的HTA活动，在整个药物开发过程中具有潜在的影响，比较强调在意大利和英国的系统。基于广泛的文献和网站回顾，我们确定了三种主要类型的HTA活动，除了主流HTA，有可能影响药物开发计划：1)水平扫描和早期HTA；2)制造商、监管机构和HTA评估人员之间的两方和三方早期对话；3)管理市场准入协议。从临床研究的早期阶段到批准后研究，各方之间的合作、市场准入证据收集的预期和上市后风险分担都有增加的趋势。HTA实践的异质性增加了市场准入环境的复杂性。总体而言，有迹象表明，市场准入部门在制药公司中的重要性正在增强，但仍然缺乏证据和报告，说明HTA日益增强的相关性如何重塑了临床开发的设计和管理方式。
The use of health technology assessment (HTA) to inform policy-making is established in most developed countries. Compared to licensing agencies, HTA agencies have different interests and, therefore, different evidence requirements. Criteria for coverage or reimbursement decisions on pharmaceutical compounds vary; however, it is common to include, as part of the HTA, a comparative effectiveness evaluation. This type of clinical data might go beyond that required for market authorization, thus creating an additional evidence gap between the regulatory and the reimbursement submission. The relevance of submissions to HTA agencies is consistently increasing in a pharmaceutical company’s perspective, as market prospects are strongly influenced by third-party payers’ coverage. In this study, we aim to describe current HTA activities with a potential impact throughout the drug development process of pharmaceuticals, with a comparative emphasis on the systems in place in Italy and in the UK. Based on an extensive literature and website review, we identified three major classes of HTA activities, beyond mainstream HTA, with the potential to influence the drug development program: 1) horizon scanning and early HTA; 2) bipartite and tripartite early dialogue between manufacturers, regulators, and HTA assessors; and 3) managed market entry agreements. From early stages of clinical research up to postauthorization studies, there is a trend toward increased collaboration between parties, anticipation of market access evidence collection, and postmarketing risk-sharing. Heterogeneity of HTA practices increases the complexity of the market access environment. Overall, there are signals that market access departments are gaining importance in the pharmaceutical companies, but there is still a lack of evidence and reporting on how the increasing relevance of HTA has reshaped the way clinical development is designed and managed.