创新实践：探秘NICE的HTA实验室

大家好，欢迎收听NICE Talks。我是主持人马修·布朗。本期节目我们将探讨NICE卫生技术评估创新实验室（简称HTA实验室）的作用。参与讨论其成立两年来的成果的嘉宾有：NICE高级科学顾问杰米·埃尔维里奇、NHS英格兰药品准入副主任杰克·特纳，以及英国制药工业协会价值与准入政策总监维多利亚·乔丹。感谢各位的参与。

Hello and welcome to NICE Talks. I'm your host Matthew Brown. In this episode, we're going to be learning about the role of NICE's Health Technology Assessment Innovation Laboratory, otherwise known as the HTA Lab. Joining me to discuss its first two years of success are our guests: Jamie Elvridge, a Senior Scientific Advisor at NICE Jack Turner, who is deputy director for Medicines Access at NHS England, and Victoria Jordan, the director of Value and Access Policy at the Association of the British Pharmaceutical Industry. Thank you all for taking part.

杰米，我的第一个问题想请教你：HTA实验室是什么机构？

Jamie, my first question is for you. What is the HTA lab?

HTA实验室是NICE科学政策与研究团队中相对较新的职能部门。我们的首席科学官尼克·克拉布主导了其创建，他将其宗旨描述为开发卫生技术评估的新方法和流程——这正是NICE评估新药和卫生技术的核心工作。我们知道医疗干预手段的创新速度极快，新方法和颠覆性技术可能挑战NICE现有工作模式，也可能为其提供更高效的新工作方式。但传统上，像NICE这样的机构很难测试新工作方法，既要确保资源合理配置，又要避免影响现行评估或决策风险。

So the HTA lab is a relatively new function within NICE's science policy and research team. Nick Crabbe, our chief scientific officer led its establishment, and he described its aims of creating new methods and processes for health technology assessment. That's essentially what NICE does when we evaluate new medicines and health technologies. We know that the pace of innovation in health interventions is incredibly fast, and new methods and disruptive technologies might challenge how NICE works or potentially provide new and effective ways for NICE to work. But traditionally, it's quite difficult for an organization like NICE to test new ways of working in terms of finding the resources to do that properly and also without potentially jeopardizing ongoing evaluations or risks to decision making.

因此在2023年，作为VPAG投资计划的一部分，HTA实验室正式成立以改善这一状况。它在NICE内部组建了专职团队，在常规技术评估工作之外开展探索性研究。这采用了沙盒工作模式——这种模式在其他领域（尤其是金融科技）已应用多年，但在卫生技术评估领域尚属首次正式应用。在这个实验空间里，我们可以安全地验证方法可行性、测试新途径、与利益相关方合作，而不会影响实际决策或造成现实风险。

So in 2023, as part of the VPAG investment program, the HTA lab was formally set up to improve that situation. It's created a dedicated team within NICE to conduct exploratory work away from the ongoing day to day business as usual of our technology evaluations. And this adopts a sandbox way of working, which is something that's been established in various other sectors for several years now, notably pioneered in financial technologies. But this is the first use formal use in health technology assessment. So in this exploratory space, we can learn what works, what doesn't work, test new methods and approaches, and collaborate with stakeholders without risks to decision making, without risk of getting something wrong for real.

之后我们可以用恰当的方式推进已验证的解决方案。我们的目标聚焦于应对NICE现行方法在评估颠覆性医疗创新技术时面临的挑战，同时发掘或测试可能全面提升评估质量的新工作方式和新方法。

And then we can take forward identified solutions in an appropriate and proportionate way. So we're aiming to focus on addressing challenges that NICE's current methods and processes might face when evaluating new disruptive innovative health technologies and also to identify or test potentially beneficial new ways of working, new methods that might improve our evaluations across the board.

那么杰米，HTA实验室具体开展哪些类型的工作？

And, Jamie, what kind of work is undertaken by the HTA lab?

HTA实验室项目大体分为两类：探索型和实施型。探索型项目会深入研究复杂问题，比如通过识别新型药物研发管线可能带来的评估挑战，论证是否需要采用不同评估方式；或测试尚未纳入NICE常规方法的新评估途径。实施型项目通常基于探索成果，例如试点前景良好的解决方案，具体研究如何将新方法恰当地融入NICE现有流程。我们通过NICE内部渠道和外部利益相关方获取HTA项目的潜在挑战和主题，定期与各团队沟通他们当前或未来可能面临的评估难题。

Broadly, HTA lab projects fall into one of two categories, exploratory and implementation focused. So an exploratory project will seek to understand complex issue in close detail, such as an innovative pipeline of new medicines by identifying the likely evaluation challenges and perhaps identifying a case for evaluating them in a different way, or it might test a new approach to evaluation that isn't currently part of NICE's routine methods. An implementation focused project would typically build on exploratory work by, for example, piloting a solution that looks promising and understanding in real detail how we can appropriately embed a new approach into NICE's methods and processes. There are several ways that we're informed about potential challenges and topics for HTLR projects from both internal NICE sources and external stakeholders. So we regularly speak to various NICE teams to understand the evaluation challenges that they are facing or anticipating in the future.

我们还定期与英国制药工业协会（ABPI）举行会议，了解药物研发者面临的挑战。同样，我们也会定期与其他医疗系统合作伙伴交流。英国国家健康与临床卓越研究所（NICE）每年都会征集对其评估方法进行针对性模块化更新的建议。如果某些建议方法尚不成熟，无法立即应用于NICE的评估体系，这些建议就可能成为HCA实验室项目的合适候选，以便进行更深入的研究。NICE还参与了多个多方利益相关者研究项目。

We're also setting up regular meetings with the ABPI to understand what challenges medicines developers face. And similarly, we regularly speak with other health care system partners. NICE annually requests suggestions for targeted modular updates to our evaluation methods. And if some of the suggested approaches aren't really mature enough to support an immediate change to NICE's methods, then those things might be suitable candidates for an HCA lab project to investigate them in a bit more detail. NICE is also involved in several multistakeholder research projects.

这些项目通常聚焦于支撑我们工作的核心技术方法——卫生技术评估，因此它们可能为我们指明方向，揭示那些对HTA实验室有益的新方法和新途径，以便在NICE的背景下进行更细致的考察。

These are often focused on the technical methods that underpin the work we do, health technology assessment, and so they might point us in the direction of potentially beneficial new methods and approaches for HTA lab to examine in the NICE context in a bit more detail.

杰克，现在请你谈谈。能否从你的角度概述NICE的HTA实验室带来的好处？

Turning to you, Jack. Can you outline the benefits of NICE's HTA lab from your perspective?

从支付方的角度来看，我们认为像HTA实验室这样的举措极具价值，因为我们看到进入市场的产品管线日益复杂。其中一些治疗方法极具创新性，可能是同类首创，或是针对极少数患者的n=1疗法，上市时仅有非常有限的临床试验数据，这使得价值评估极具挑战性。另一方面，我们也看到越来越多的人口健康干预措施进入市场，这些措施有望带来显著的总体质量提升和大规模患者获益。这些治疗带来的机遇与其快速大规模实施所需的复杂服务转型相匹配——要让这些治疗在实践中真正发挥作用，这种转型是必要的，但也是挑战，因为这些治疗往往最能满足未满足的临床需求。

From a payer perspective, we find initiatives like the HTA lab incredibly valuable because we're seeing an increasingly complex pipeline of moving to market. Some of these are treatments that are incredibly novel. They might be first in class, or they might be n f one therapies that have very small patient populations and therefore very limited clinical trial data when they come to market, which makes the assessment of value incredibly challenging. On the other extreme, we are seeing increasingly a number of population health interventions that are coming to market, which have the potential to offer significant aggregate quality gains, significant patient benefit at scale. The opportunity that they provide is also matched with the complexity of delivering such treatments at pace and scale in terms of the service transformation that's required to actually make these things work in practice, which is a challenge because often these treatments are the things that demonstrate the highest potential to deliver on unmet clinical needs.

谢谢。维多利亚，从行业的角度，能否请你解释为什么HTA实验室很重要？

Thank you. Victoria, from an industry standpoint, could you explain why the HTA lab is important?

行业一直主张NICE在完成极其繁忙、占用大量时间和资源的指南制定工作之外，还应具备足够的能力来改进其方法和流程。实际上，这也是NICE转向成本回收模式并引入评估费用时向行业解释的关键理由之一。为什么这一点如此重要？主要是因为持续改进的方法有助于支持创新医疗技术的及时获取，可以最大限度地减少新型治疗进入NICE工作流程时出现延误甚至无法获得准入决定的风险。同类首创药物，尤其是那些与NICE以往评估经验截然不同的药物，往往面临艰难而漫长的评估过程，我们已经看到延误发生，患者不得不等待这些非常重要的治疗。

So the industry has always advocated for NICE to have sufficient capacity, be able to evolve its methods and processes on top of delivering its guidance development work programs, which we know are extremely busy and take a lot of their time and resource. And, actually, this was one of the key rationale that was given to the industry when NICE moved to a cost recovery model and fees for appraisals were introduced. Why is it so important? Primarily because an approach that enables continuous improvements can help support timely access to innovative health care technologies, can minimize the risk of delays or even no access decisions when a new type of treatment enters the NICE work program. First in class medicines, especially those that are quite different from what NICE has previous experience in evaluating have often faced challenging and lengthy appraisals, and we've seen delays happen and and patients waiting for really important treatments.

此外，还需要持续测试NICE的方法是否允许充分考虑并回报这些技术的全部价值——我们认为目前这一点尚未完全实现。识别和探索可能挑战现有工作方式的技术还有另一个好处，那就是可以早期发现评估之外的其他潜在问题。从支付方和实施的角度来看，还需要考虑哪些因素？是否建立了正确的筛查和诊断路径？例如，是否需要调整护理路径以支持患者的最佳治疗？

There's also a need to continue to test whether NICE's methods are allowing for the full value of these technologies to be considered and rewarded, something we would argue that is not yet being fully done. Another benefit of identifying and exploring technologies that will likely test the existing ways of working is it allows for early identification of other potential issues beyond the evaluation. What else might need to be considered from a payer and an implementation perspective? Are the right screening and diagnostic pathways set up? Does the care pathway need altering to support optimal treatment of the patients, for example?

继续在HTA领域创新的另一个重要原因，是支持NICE内部更高效地利用资源，同时也惠及参与评估流程的各方。要知道，NICE的容量有限，其委员会、参与的临床与患者专家以及参与流程的企业也同样如此。若能在NICE开展评估前预见挑战并采取应对措施，将为所有参与者带来更顺畅快速的流程。最后我想强调，真正有意义的创新——不是小修小补，而是引入全新方法——能够彰显NICE在全球舞台上的思想领导力。

Another good reason to continue to innovate in HTA is to support a more efficient use of resources within NICE, but also those who contribute to the evaluation process. You know? We know NICE's capacity is limited, but so is that of its committees, clinical and patient experts that are involved, and the companies that are engaging with the process too. So challenges can be preempted and something can be done about them ahead of NICE doing the evaluation, then it will lead to a smoother and quicker process for all that are involved. And lastly, I'd say that meaningfully innovating, and I don't mean just tweaking around the edges here, but actually introducing new approaches can demonstrate NICE's thought leadership on the global stage.

众所周知，多个市场都在关注NICE的动向。因此，相关改革能清晰反映出英国在支持国民医疗服务体系（NHS）创新方面的实际表现。需要说明的是，我们承认苏格兰拥有独立的HDA评估流程。

You know, we know that several markets look to NICE and what it's doing. So changes that are made really shine a light on how supportive The UK is or isn't being introduced in innovation into the NHS. And I say The UK, they're acknowledging that Scotland has its own HDA process.

回到你这边，Jack。HDA实验室在让NHS患者获得最具创新性药物方面扮演着什么角色？

Switching back to you, Jack. What is the role the HDA lab plays in getting the most innovative medicines to NHS patients?

我们发现英格兰每年上市的药物中约有5%缺乏广泛使用的充分证据。但我们有机会将这些产品纳入管理准入期，在收集额外数据的同时确保患者适当获取。HTA实验室将持续为Laborate提供机会，让我们优化管理准入方案。此外，在证据不确定时，我们可以通过哪些协作来放宽经济模型中的假设以加速患者获取？如何利用医疗系统的真实世界证据来支持有效且一致的决策？这将是未来发展的关键。

About 5% of medicines launched in England every year, we find, don't have sufficient evidence for widespread use. But instead, we have the opportunity to recommend those products into a managed access period where there is appropriate patient access while additional data is collected. There'll continually be opportunities under the HTA lab for Laborate to think about our managed access positions correct. But, also, usually, where evidence is uncertain, what can we do together that will allow for greater assumptions we made in economic modeling to accelerate access to patients, and how can we use real world evidence from the health system to really support effective and consistent decision making. That will be key moving forward.

我们看到越来越多个体化疗法涌现。值得欣慰的是，在HTA实验室框架下，NICE正在制定评估这类疗法的实用方法，确保评估既全面又能反映现实世界的复杂性。与此同时，NHS英格兰正积极探索建立实施这类治疗的运营框架，包括委托服务的考量，以确保配套基础设施到位来支持安全有效使用。但对于极少数患者群体，这些疗法对基础设施、评估和支付流程都提出了额外挑战。

Individualized therapies, we're seeing more of those coming through. And we're really grateful that under the HTA lab, NICE are developing practical approaches for assessing those individualized therapies that ensure that evaluations remain comprehensive and reflective of real world complexities. In parallel to that, NHS England are actively exploring the development of an operational framework for delivering such treatments to the NHS. That's going to include considerations for commissioning services to ensure the necessary infrastructure is in place to support the safe and effective use of these therapies. But where you have very, very small patient populations, these things challenge additional infrastructure, challenge additional assessment and payer processes.

因此共同深入思考这些问题至关重要。最后我想呼吁的是：将服务成本整合到HDA评估中——无论是个体化疗法还是大规模人群健康治疗，都会带来服务成本方面的挑战。我们正与NICE同事紧密合作，研究如何将新基础设施培训和服务交付模式的成本更直接地纳入HDA评估。

So thinking that stuff through together is vital. The last thing I would call on is integrating service costs into HDAs where you have individualized therapies or large population health treatments that creates a challenge for service costs. And we're working closely with colleagues and NICE to think about how those costs of new infrastructure training and service delivery models might be more directly integrated into HDA assessment.

Victoria，VPAG协议在HDA实验室项目开发中发挥了什么作用？

Victoria, what role has the VPAG agreement played in developing projects for the HDA lab?

业界非常高兴看到NICE设立创新实验室。我们认为这是一项需要优先考虑并充分投入资源才能取得成功并实现那些效益的举措。作为当前VPAG谈判的一部分——对于不熟悉这个缩写的人，VPAG指的是品牌药品定价、准入与增长自愿计划，这是政府、英国国民医疗服务体系（NHS England）与代表制药行业的英国制药工业协会（ABPI）之间的协议，旨在支持患者获得药物、保障NHS财务可持续性以及英国生命科学等领域发展。因此首次达成共识，将设立VPAG投资项目，为所有签约方认为应优先推进的多个项目提供资金支持，以增强英国在健康与生命科学领域的竞争力，推动创新引领的增长。该投资项目将在计划执行期间为英国四个地区的项目提供来自计划成员的额外资金。

Industry is really pleased to see NICE set up the innovation lab. We thought it was an initiative that needed to be prioritized and fully resourced to be successful and to be able to achieve those benefits. So as part of the negotiation of the current VPAG, for those who aren't familiar with the acronym, that's the Voluntary Scheme for Branded Medicines Pricing Access and Growth, which is an agreement between the government, NHS England and the ABPI on behalf of the pharmaceutical industry, which seeks to support patient access to medicines and the financial sustainability of the NHS and The UK life science, etcetera. So it's agreed for the first time that a VPAG investment program would be set up to financially support a number of projects that all of the signatories of the scheme felt should be prioritized to strengthen UK competitiveness in health and life sciences and drive innovation led growth. The VPAG investment program is providing additional funding from scheme members to invest in projects across all four UK nations over the course of the scheme.

这是一个始于2024年的五年计划。所支持的项目涵盖三大领域：跨临床实验、生产制造和卫生技术评估（HTA）。HTA的定义较为宽泛，该领域内部分项目虽不直接关联HTA，但致力于改进与药物准入相关的流程。在HTA项目中，投资计划正提供多年资金以支持NICE的HTA实验室资源建设。

It's a five year scheme that started in 2024. And the projects that are being supported fit within the three buckets, cross clinical trials, manufacturing, and HTA. HTA is a bit of a loose definition. Some of the projects within this bucket aren't directly HTA related, but they are seeking to improve processes that relate to access to medicines. Within the HTA projects, the investment program is providing multiyear funding to support resourcing for the NICE HTA lab.

需要明确的是，这些资金不附带干预条件。我们希望业界被视为项目的重要贡献者，但我们将以与所有利益相关方相同的方式进行合作。这真正展现了利用部分BPAC资金开展政府、NICE、NHS England与行业共同关注项目的绝佳机会。

And I should be clear, this funding is hands off. We hope industry is seen as a valuable contributor to the projects, but we collaborate in the same way as all stakeholders. I think what this really shows, it's a great opportunity to be using some of the BPAC money for projects where there's a shared ambition across government, NICE, NHS England, and the industry.

最后，Jamie，能否透露一下HCA实验室近期可能关注哪些项目？

And finally, Jamie, can you give us a taste of what projects the HCA lab might be looking at in the near future?

我们注意到几个可能对未来HCLR项目产生重大影响的议题。首先，我们计划更广泛地审视人工智能的发展进步如何影响NICE，理解其如何支持我们的评估工作并帮助我们优化流程，同时也关注需要调整的领域。另一个议题是，日益创新的医疗技术越来越可能依赖不同类型的证据支撑，比如实验室数据或计算机模拟数据。这些是目前证据提交中不常见的形式。因此我们希望探索如何评估这类证据，并确定它们在哪些情况下能特别补充传统临床研究证据。

Couple of topics that are on our radar that we've been alerted to for potentially impactful future HCLR projects. Firstly, we are planning to take a broader view of how the growth and advancement of AI might affect NICE and understand how it might support our evaluations and allow us to do things better, but also where we may need to adapt. Another where we think that increasingly innovative health technologies are increasingly likely to be underpinned by different types of evidence, things like laboratory based data or computer simulated data. So those are things we don't typically see in evidence submissions tonight at present. So we'd like to explore how we'd consider those things if they were submitted to us and identify situations where they might be a particularly useful addition to traditional evidence from clinical studies.

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