IQVIA Ltd., 210 Pentonville Rd, London, N1 9JY, UK.
National Institute for Health and Care Excellence, London, UK.
Appl Health Econ Health Policy. 2021 Sep;19(5):625-634. doi: 10.1007/s40258-021-00654-4. Epub 2021 May 24.
The arrival of precision oncology is challenging the evidence standards under which technologies are evaluated for regulatory approval as well as for health technology assessment (HTA) purposes. Several key concepts are discussed to highlight the source of the challenges in evaluating these products, particularly those impacting the HTA of histology-independent therapies. These include the basket trial design, high uncertainty in (potentially substantial) benefits for histology-independent therapies, and the inability to identify and quantify benefits of standard of care in daily practice when the biomarker is not currently used in practice. There is little precedent for a technology with the unique mixture of challenges for HTA of histology-independent therapies and they will be evaluated using standard HTA, as there currently is no evidence suggesting the standard HTA framework is not appropriate. A number of questions proposed to help guide HTA bodies when assessing the appropriateness of local processes to optimally evaluate histology-independent therapies. Pragmatic solutions are further proposed to decrease uncertainty in the benefits of histology independent therapies as well as fill gaps in comparative evidence. The proposed solutions ensure a consistent and streamlined approach to evaluation across histology-independent products, although with varying strengths and limitations. Alongside these solutions, sponsors should engage early with HTA bodies/payers and regulatory agencies through parallel/joint scientific advice to facilitate the integration of both regulatory and HTA perspectives into one clinical development programme, potentially reconciling evidence requirements.
精准肿瘤学的出现，给基于监管审批和卫生技术评估（HTA）目的而对技术进行评估的证据标准带来了挑战。本文讨论了几个关键概念，以突出评估这些产品（尤其是针对组织学独立疗法的 HTA）所面临挑战的根源。这些挑战包括篮子试验设计、组织学独立疗法潜在的（可能较大的）获益的高度不确定性，以及当生物标志物尚未实际应用于临床实践时，无法识别和量化标准治疗的获益。对于一种具有独特的组织学独立疗法 HTA 挑战组合的技术，几乎没有先例，因此将使用标准 HTA 对其进行评估，因为目前尚无证据表明标准 HTA 框架不适用。本文提出了一些问题，旨在帮助 HTA 机构在评估优化组织学独立疗法评估的本地流程的适宜性时提供参考。此外，还进一步提出了一些实用的解决方案，以降低组织学独立疗法获益的不确定性，并填补对比证据的空白。所提出的解决方案确保了对所有组织学独立产品的评估方法具有一致性和精简性，尽管它们具有不同的优缺点。除了这些解决方案之外，申办方还应通过平行/联合科学咨询，尽早与 HTA 机构/支付方和监管机构接触，以促进将监管和 HTA 视角整合到一个临床开发项目中，从而有可能协调证据要求。
