COVID-19在全球范围内凸显了快速诊断的力量。据FIND报道，自新冠疫情开始以来，已经进行了65亿次COVID检测。在疫情过后，整个生态系统的参与者（包括医疗技术创新企业、知名全球IVD研发企业、学者、医疗服务提供者和付费者）都致力于寻求快速诊断可以有效发挥作用的其他领域。大家认识到，快速诊断可用于广泛的适应证，以应对当今医疗服务的一些挑战。
因此，即时检测（POCT）和直接面向消费者的检测（DTC）市场预计将迎来强劲增长（直接面向消费者的检测（DTC）由消费者自行采集样本，然后寄送到检测机构进行检测和分析，或由消费者自行完成检测）。到2030年，即时检测（POCT）市场预计将以7.9%的复合年均增长率（CAGR）增长至710亿美元；而到2031年，居家检测市场预计将以10.5%的复合年均增长率（CAGR）增长至456亿美元。
快速诊断市场的增长受新兴护理模式（例如虚拟病房、居家医疗（HAH）、自我护理）的影响。在美国，自2021年4月以来，联邦医疗保险和医疗补助服务中心（CMS）批准的提供居家医疗服务的医院数量增长了118%。2023年1月，英国政府将虚拟病房的月覆盖率从10,000名患者上升到了50,000名患者。我们在最近发布的“Healthier at Home”报告中，探讨了转向居家护理所创造的价值。
要想把握快速诊断的市场机遇，还需应对一系列复杂的挑战，特别是与监管相关的挑战。获得监管机构的批准是产品市场准入的必要条件，然而对监管环境的把握正变得日益复杂：监管机构不断出台新法规，而监管体系难以追平法规更新的速度，从而导致了严峻的监管能力问题。此外，获得监管机构的批准是一个漫长的过程，通常会产生较高的费用。那么IVD研发企业和医疗技术创新企业如何才能成功把握复杂的监管环境并获得产品市场准入资格，从而使医疗保健系统和患者受益呢？我们概述了5个关键要素供IVD企业参考，助其快速顺利获得产品市场准入资格。
1、深入了解市场，精准定位产品价值
COVID-19席卷全球，波及各类人群，相关快速检测试剂也应用于各种场景。展望未来，快速检测试剂将以高度细分的方式用于多种场景、疾病、用户群体和环境。IVD研发企业很可能会受到不同使用场景下检测频次的影响，而检测频次反过来又会决定生产量，并最终决定销量。在考虑更广泛的适应证时，IVD研发企业必须了解其解决方案将要解决的实际问题是什么，以及谁将从这些解决方案中受益。
在时间紧张的情况下，快速检测所带来的临床受益是显而易见的。例如，肌钙蛋白检测可以在紧急情况下排除“静默”心脏病发作的可能性。流感检测等快速检测试剂可以在特定情况下实现分诊，从而控制感染。此外，快速检测可以帮助长期慢性病患者进行自我监测，并可减少患者临床就诊的需求，从而为患者和医护人员节省时间，并提供更好、更方便的检测体验。胆固醇的定期监测就是一个很好的例子。
IVD研发企业必须深入了解市场，对卫生健康状况、当前上市途径、当前使用的检测技术、当前实践中的痛点以及解决方案所能提供的价值进行调研，从而精准定位产品价值并确保市场接受度。
2、洞悉监管机构的观点，与监管机构协作
快速诊断领域的创新为监管机构带来了新的挑战，监管机构现在需要考虑新的复杂性，比如：1）缺乏同类产品；2）目测结果而非定量结果；3）检测结果的校准和质量保证；4）检测由非专业使用者或缺乏诊断检测经验的医护人员进行；5）检测在非临床环境中进行；6）结果的数字化上传。监管机构重点关注的是患者安全，涉及到快速诊断时，其密切关注的是临床和可用性证据、特定检测环境的影响、使用者的资格和培训、以及结果读取和结果记录方法。关键在于，IVD研发企业需要深入了解监管机构的关注点，并与监管机构合作确立上市许可途径，并就可接受的证据基础达成一致。
监管机构也面临着压力，需要通过自身实践的创新来为创新产品的上市许可途径提供支持，确保患者最终能够从行业创新中受益。为此，监管机构也在通过意见征询等方式来获取外部利益相关者的意见，并推出新举措来应对创新带来的挑战。例如，美国FDA启动了“突破性医疗器械计划”，对于符合该计划条件的器械，在研发阶段为企业提供反馈并对其上市许可申请进行优先审查。美国FDA还就“增加患者居家使用医疗技术的可及性”公开征求意见，并指出“临床护理环境之外的器械可及性可将医疗服务直接带给患者（无论患者身在何处 - 家中、工作场所、城市、农村社区），有助于弥合差距。扩大居家使用医疗技术可及性的举措与美国FDA致力于推进健康公平性的战略目标是一致的。”
英国药品和健康产品管理局（MHRA）已经建立了新的“创新器械准入途径”（IDAP），将监管和技术评估相结合，旨在消除准入途径中的不确定性，从而为患者带来能够改善健康结果的创新技术。
我们期待看到全球监管机构与外部利益相关者（如卫生技术评估机构、IVD研发企业、临床医生、学者和患者）之间的更高程度的合作，共同努力开发监管途径的创新方法。
3、以用户为中心，尽可能降低或消除风险
许多快速诊断解决方案是为受过医学培训的专业人员在传统临床环境中使用而设计的。若直接将这些检测方案应用到其他环境中（包括社区检测中心、零售环境、养老院、学校和其他家庭环境），会增加使用错误的风险。
检测频次在使用者行为和检测准确性方面起着重要作用。例如，每天进行的检测可以成为一种习惯，重复检测可以提高熟练程度。另一方面，针对不常进行或一次性进行的检测，可能需要适当设计特定的操作说明。
使用说明书并不总是用户友好的。IVD研发企业可通过以下措施设计出可以降低和缓解这些风险的检测解决方案，包括：1）减少组件数量；2）减少检测步骤；3）设计适用于一系列样本类型（不止血液样本）的检测方案；4）设计用于特定的非临床环境；5）改进包装和处理步骤；6）改进结果读取和记录方法（包括借助数字解决方案）；7）为非专业人员提供用户友好、容易理解的说明。在新环境下，以用户为中心的设计可以减轻监管机构对患者安全的担忧（上文第2点有提及），从而为上市许可申请提供支持。不过，这需要IVD研发企业在以用户为中心的设计和人因工程方面具备世界一流的能力。
4、按需实现综合临床决策
在适用的情况下，快速诊断检测能够以数字化的形式连接到更广泛的卫生系统，以确保做出适当的临床决策。例如，患有长期慢性疾病的最终用户可能需要针对特定分析物进行定期自测。对于护理人员来说，了解检测频次和结果以做出最佳临床决策可能很重要。鉴于初级和二级医疗中已建立的数字系统的异质性，这给IVD研发企业带来了巨大挑战。这是IVD研发企业需要具备的另一种能力。对于最终用户和医疗服务提供者来说，创建另一种专有的结果记录工具（如专用的电子设备）并不总是会增加价值。利用无处不在的消费者设备（如iPad和智能手机），而不是专用设备，可能更方便，因此更有可能鼓励采用且具有可扩展性。但另一方面，使用个人设备来执行诸如记录、分析、存储和传输患者结果的任务可能并不总是被医疗保健系统所接受。
5、进行卫生技术评估，证明价值并支持采用
在快速诊断解决方案旨在作为医疗保健系统的一部分的情况下，卫生技术评估（HTA）是证明价值和支持采用新检测技术和现有技术新用途的宝贵工具。卫生技术评估（HTA）的主要目的是为政策制定者提供基于证据的信息，以便其能够制定出安全、有效、以患者为中心、成本效益高的卫生政策。国家当局还通过卫生技术评估（HTA）来帮助决定在国家层面应该对哪些技术进行补偿。卫生技术评估（HTA）是补偿决策途径中的重要一环。
最近，人们开始重新关注卫生技术评估（HTA）。例如，一项新的欧洲法规将从2025年1月开始施行，该法规将取代目前由欧盟资助的成员国之间基于项目的卫生技术评估合作体系。该法规将为联合工作引入持久性框架，该框架还将涵盖联合科学磋商、新兴卫生技术的识别、自愿合作以及联合临床评估工作。英国国家卫生与临床优化研究所（NICE）最近与来自三大洲的其他五家全球HTA机构建立了合作伙伴关系，就一系列主题展开合作，旨在帮助世界各地的人们获得医疗保健服务。这些合作伙伴将就共同优先事项协同努力，针对一些共同面临的挑战确定解决方案。优先事项包括：1）面向未来的HTA体系；2）与监管机构合作；3）可能使用彼此的HTA信息；4）探索联合临床评估试点。
结论
虽然快速诊断技术的应用有望强劲增长，但我们预计，要确保成功推广和广泛采用，还需要克服多重障碍。为了克服这些障碍，需要整个生态系统中的多个参与者的参与，特别是与监管机构建立密切的工作关系，以设计出具有明确风险缓解要求和证据基础的审批途径，以确保产品能够获准上市。这需要一种全新的产品设计和开发方法，且需要在技术文件中和上市许可申请时加入新的内容。至关重要的是，快速诊断器械制造商之间需要建立合作伙伴关系，且需要具备新的能力，以支持这种上市许可批准的新方法。
考虑到预期的市场红利规模，我们预计会有高水平的竞争、一些创新合作以及一些新进入者在这个充满活力的市场领域建立份额。
英文原文
5 Key Factors To Gain Regulatory Approvals for Innovative Rapid Diagnostic Devices
By Valerie Phillips, PA Consulting; and Ben Jacoby, Cambridge RA Ltd.
COVID-19 demonstrated the power of rapid diagnostics on a global scale. FIND reported that 6.5 billion COVID tests have been performed since the beginning of the pandemic. In the aftermath of the pandemic, players across the ecosystem, including medtech innovators, established global in vitro diagnostic (IVD) developers, academics, and healthcare payers and providers, are committed to finding other areas where rapid diagnostics can be effectively deployed. All recognize that rapid diagnostics can be deployed for a wide range of indications to meet some of the challenges of healthcare delivery today.
Consequently, strong growth is anticipated in both point of care testing and direct to consumer testing, which includes a consumer self-collecting a sample, which is then processed remotely, or a consumer undertaking and completing the test themselves. The point of care testing market is estimated to grow at a CAGR of 7.9% to $71 billion by 2030, while the at-home testing market is forecast to grow at a CAGR of 10.5% to $45.6 billion by 2031.
Growth in rapid diagnostics is influenced by the deployment of new care models, such as virtual care wards and hospital at home schemes, as well as a shift toward self-care. In the U.S., the number of Centers for Medicare and Medicaid Services (CMS)-approved hospitals offering hospital at home care has increased by 118% since April 2021. In the U.K., the government increased its coverage of virtual wards to 50,000 patients a month in January 2023, up from 10,000. Creating value from this shift to home care is explored in our recently published Healthier at Home report.
A range of complex challenges need to be overcome to realize the market opportunity for rapid diagnostics, especially in the regulatory space. Gaining regulatory approval is imperative for market access, yet navigating the regulatory landscape is becoming increasingly complex: regulators are bringing in new regulations and the system is finding it difficult to play catch-up, leading to huge capacity problems. Moreover, gaining regulatory approval is a lengthy and often expensive process. How can IVD developers and medtech leaders successfully navigate this complex territory and gain market access for the benefit of the healthcare systems and patients? We outline five key factors for leaders to consider enabling efficient, smooth regulatory approval pathways.
1. Obtain Deep Market Understanding To Inform “Value”
COVID-19 was a worldwide problem, across all demographics and use cases. Going forward, use of rapid tests will be deployed in a highly segmented manner across multiple use cases, diseases, user groups, and settings. IVD developers are likely to be influenced by the frequency of testing across use cases, which in turn can determine manufacturing volumes and, ultimately, sales. When considering a wider range of indications, it is key for developers to understand the actual problem their solution will address and who will benefit from their proposed solutions.
A rapid test can provide a clinical benefit in a situation where time is key. For example, a troponin test can rule in/out a “silent” heart attack in an emergency, while a rapid test such as flu testing can support triage in a specific setting and enable infection control. Rapid testing also enables patient self-monitoring for long-term chronic conditions and may reduce the requirement for in-person clinical visits, thereby saving time for both patients and healthcare professionals, as well as providing an enhanced, more convenient testing experience. An example is regular cholesterol monitoring.
It is imperative that IVD developers gain a deep market understanding of the health conditions, current pathways, current testing technologies in use, pain points in the current practices, and value their solutions can provide to understand “value” and ensure market acceptance.
2. Recognize The Regulator’s Point Of View And Collaborate With The Regulator
Innovations in the rapid diagnostics space are introducing new challenges for regulators, who now need to consider new complexities, such as lack of predicates, visual rather than quantitative results, calibration and quality assurance of test results, tests being performed by lay users and healthcare professionals with less experience with diagnostic testing, tests being used in non-clinical settings, and digital uploads of results. The regulators’ key concerns are for patient safety and when it comes to rapid diagnostics, they will have a keen eye on clinical and usability evidence, impact of specific test settings, the qualification and training of users, and methods for results reading and results recording. It is key that IVD developers gain an in-depth understanding of the regulators’ concerns and work with the regulators to establish pathways and agree on an evidence basis that is acceptable.
Regulators are under pressure themselves to innovate their practices to support innovation pathways and ensure that, ultimately, patients can benefit from industry innovations. In response, the regulators are launching consultations to obtain external stakeholder input and are bringing in new initiatives to respond to the pace of innovation. For example, in the U.S., the FDA has launched the Breakthrough Devices Program, which offers feedback to the developer during the development of the device and offers prioritized submission review, once the device qualifies for the program. The FDA has also announced a public consultation on Increasing Patient Access to At-Home Use Medical Technologies, stating that “access to devices outside a clinical care setting may help bridge the gap by bringing health care directly to patients, wherever they are – at home, at work, in cities, in rural communities. The effort to expand access to home use technologies is consistent with the FDA’s strategic aim focused on advancing health equity.”
In the U.K., the MHRA (the Medicines and Health products Regulatory Agency), has announced a new Innovative Devices Access Pathway, iDAP, combining regulation and technology assessment that aims to take uncertainty out of the route to access, thereby bringing innovative technologies to patients that can transform health outcomes.
We expect to see a higher degree of collaboration between global regulatory bodies and external stakeholders such as health technology assessors, IVD developers, clinicians, academics, and patients, all of whom will work together to develop innovative approaches for regulatory pathways.
3. Design User-Centric Solutions To Reduce Or Eliminate Risk Where Possible
Many rapid diagnostic solutions have been designed for use by medically trained professionals and in traditional clinical settings. By moving these tests into alternate settings, including community testing hubs, retail settings, care homes, schools, and other home settings, there is a higher risk of use error.
Frequency of testing plays a significant role in user behaviour and accuracy of testing. For example, tests that are performed daily can become routine and repetition may encourage proficiency. On the other hand, specific tailored instructions may need to be designed appropriately for tests that are undertaken infrequently or on a one-off basis.
Instructions for use are not always user-friendly. There is an opportunity for IVD developers to design testing solutions that reduce and mitigate these risks, including reducing the number of components, reducing the number of testing steps, designing tests that incorporate a range of sample types (beyond blood), designing for use in specific non-clinical settings, improving packaging and handling steps, providing improved reading and recording of results, including via digital solutions, and providing user-friendly, easy to follow instructions for laypersons. As we mentioned in point 2, user-centric design will support regulatory submissions by allaying regulators’ concerns for patient safety in new settings. However, this places a demand on IVD developers to develop world-class capabilities in user-centric design and human factors engineering.
4. Enable Integrated Clinical Decision-Making Where Required
Where applicable, rapid diagnostic tests can be digitally connected to the wider health system to ensure appropriate clinical decision-making is enabled. For example, an end user suffering from a long-term chronic condition may have a requirement to self-test for a specific analyte on a regular basis. It may be important to the caregiver to have visibility of testing frequency and outcomes to enable optimal clinical decision-making. Given the heterogeneity of established digital systems in primary and secondary care, this poses a huge challenge to IVD developers. This is another capability that IVD developers will need to develop. Creating yet another proprietary result recording tool such as a dedicated electronic device will not always add value for the end user and care provider. Utilizing ubiquitous consumer devices, such as iPads and smartphones, rather than dedicated devices, can be convenient and therefore more likely to encourage adoption and scalability. On the other hand, the use of personal devices to carry out tasks such as recording, analyzing, storing, and transmitting patient results may not always be accepted by healthcare systems.
5. Undertake A Health Technology Assessment To Evidence Value And Support Adoption
In instances where rapid diagnostic solutions are intended to be used as part of the healthcare system, health technology assessments (HTAs) are valuable tools in evidencing value and supporting adoption of novel testing technologies and new uses of existing technologies. The main purpose of HTAs is to provide policymakers with evidence-based information, so they can formulate health policies that are safe, effective, patient-focused, and cost-effective. They are also used by national authorities to help make decisions on which technology should be reimbursed at national levels. HTAs form a critical component of reimbursement pathways.
Recently, there has been a renewed focus on HTAs. For example, a new European regulation will apply from January 2025 that supersedes the current system of EU-funded project-based cooperation between Member States on health technology assessments. It introduces a permanent framework for joint work that will also cover joint scientific consultations, the identification of emerging health technologies, and voluntary cooperation, as well as work on joint clinical assessments. In the U.K., NICE recently established partnerships with five other global HTA bodies from three continents to collaborate on a range of topics that will benefit people accessing healthcare around the world. These partners will work together on shared priorities to identify solutions to some of the common challenges. Their priorities include future proofing HTA systems, collaborating with regulators, and potentially using each other’s HTA information and exploring pilots for joint clinical assessments.
Conclusion
While strong growth is widely anticipated in the uptake of rapid diagnostics, we anticipate there are several barriers to overcome to ensure successful rollout and widespread adoption. To overcome these, there will need to be participation by several players across the ecosystem and, in particular, close working relationship with regulatory bodies to design approval pathways with clear requirements for risk mitigation and evidence basis to ensure approval. This will require a fresh approach to product design and development as well as new content built into technical files and regulatory submissions. Critically, rapid diagnostic manufacturers will need to build partnerships and acquire new capabilities to underpin this fresh approach to market approval.
Given the anticipated size of the prize, we anticipate a high level of competition, some innovative collaborations, and some new entrants establishing a share in this dynamic market sector.
https://www.meddeviceonline.com/doc/key-factors-to-gain-regulatory-approvals-for-innovative-rapid-diagnostic-devices-0001
来源：MED DEVICE ONLINE
作者：Valerie Phillips（PA Consulting）、Ben Jacoby（Cambridge RA Ltd.）
