With over 30 million individuals in the United States alone affected by 7,000 rare diseases,1 it is crucial to provide these patients with timely access to diagnosis and prompt treatment where it is available.

仅在美国就有超过3000万人受到7000种罕见疾病的影响1，为这些患者提供及时的诊断和及时的治疗至关重要。

Empowering patients with a deeper knowledge of their disease and the available treatment options is the primary goal of patient engagement, which helps to reduce the often prolonged periods of uncertainty following diagnosis and speed up the path to starting treatment.

赋予患者更深入的疾病知识和可用的治疗方案是患者参与的主要目标，这有助于减少诊断后常常长时间的不确定性，并加快开始治疗的途径。

Fortunately, recent years have seen the landscape of rare and orphan disease patient engagement undergo a transformative shift, with comprehensive support for patients and their families prioritised from the beginning of treatment.

幸运的是，近年来，罕见和孤儿疾病患者参与的格局发生了转变，从治疗开始就优先考虑患者及其家属的全面支持。

In this article, Nareda Mills, Global President, Patient Solutions at Inizio Engage, explores the unfolding evolution of patient engagement models by examining the challenges facing patients and healthcare providers (HCPs), and the strategies pharma is putting in place to revolutionise rare disease care..

在本文中，Nizio Engage患者解决方案全球总裁Nareda Mills通过研究患者和医疗保健提供者（HCP）面临的挑战，探讨了患者参与模型的发展演变，以及制药正在制定的彻底改变罕见疾病的策略关心。。

Breaking down treatment barriers for patients

打破患者的治疗障碍

Following the diagnosis of a rare or orphan disease, patients often encounter the challenge of finding a suitable specialist who can initiate the appropriate treatment plan. In many cases, the HCP delivering the initial diagnosis may have limited experience in the specific disease area, which can lead to potential delays in connecting the patient with the right specialist for their unique needs..

在诊断出罕见或孤儿疾病后，患者经常遇到寻找可以启动适当治疗计划的合适专家的挑战。在许多情况下，提供初步诊断的HCP在特定疾病领域的经验可能有限，这可能导致潜在的延误将患者与针对其独特需求的合适专家联系起来。。

The longer these delays persist, the greater the risk of patient disengagement. Consequently, patients may turn to unreliable sources found online and across social media in search of alternative treatment methods. This poses significant health risks, underscoring the importance of ensuring patients receive prompt and comprehensive information about the available treatment options. .

这些延误持续的时间越长，患者脱离接触的风险就越大。因此，患者可能会转向在线和社交媒体上发现的不可靠来源寻找替代治疗方法。这带来了重大的健康风险，强调了确保患者获得有关可用治疗方案的及时全面信息的重要性。 .

To mitigate the risk of disengagement, it is crucial to establish clear expectations and provide comprehensible patient education. This will make patients more likely to remain committed to their prescribed treatment throughout its course.

为了减轻脱离接触的风险，建立明确的期望并提供可理解的患者教育至关重要。这将使患者更有可能在整个疗程中继续致力于他们的处方治疗。

Helping patients navigate the challenges of securing healthcare coverage

帮助患者应对确保医疗保险的挑战

Obtaining access to and securing coverage for prescribed treatments is a critical obstacle facing many US patients after their diagnosis. Navigating complex treatment options and patient assistance programmes can become a daunting task, especially when treatments come with a high price tag.

获得并确保处方治疗的覆盖范围是许多美国患者诊断后面临的关键障碍。导航复杂的治疗方案和患者援助计划可能成为一项艰巨的任务，特别是当治疗价格高昂时。

Fortunately, dedicated patient engagement partners have emerged to provide crucial support in overcoming these challenges. They offer services that encompass verifying patients’ insurance and benefit coverage to ensure alignment with their specific plan, whether through commercial or public health insurance..

幸运的是，专门的患者参与合作伙伴已经出现，为克服这些挑战提供关键支持。他们提供的服务包括核实患者的保险和福利范围，以确保与他们的具体计划保持一致，无论是通过商业或公共健康保险。。

The spectrum of services offered by these providers extends to  assisting patients in determining their eligibility for co-pay assistance programmes offered by drug manufacturers. This assistance becomes particularly crucial for patients requiring immediate treatment, as it expedites the process of acquiring the financial support necessary to proceed with their treatment promptly..

这些提供者提供的服务范围扩展到协助患者确定他们是否有资格获得药品制造商提供的共同支付援助计划。这种帮助对于需要立即治疗的患者尤其重要，因为它加快了获得及时进行治疗所需的财务支持的过程。。

Across both sides of the patient experience, dedicated Field Reimbursement Managers (FRMs) play a pivotal role in bridging the gap, offering valuable insights into the payor landscape. Armed with this knowledge, HCPs are empowered to guide patients through smoother treatment journeys from the outset.

在患者体验的双方，专门的现场报销经理（FRM）在弥合差距方面发挥着关键作用，为付款人提供有价值的见解。有了这些知识，HCP有权从一开始就通过更顺畅的治疗旅程指导患者。

Informed HCPs armed with a clear understanding of the intricate cost and coverage aspects associated with specialised therapies ultimately pave the way for improved patient experiences. .

知情的医务人员清楚地了解与专业治疗相关的复杂成本和覆盖范围，最终为改善患者体验铺平了道路。 .

Moreover, these services have expanded to assist patients at earlier stages of their journey, covering aspects such as seeking treatment, obtaining prescriptions, and addressing their payment concerns. The expanded service offerings include investigating the benefits available, verifying coverage, and providing prior authorisation support and submissions — often carried out electronically to streamline and accelerate the process. .

此外，这些服务已经扩展到协助患者在其旅程的早期阶段，涵盖寻求治疗，获得处方和解决他们的支付问题等方面。扩展的服务产品包括调查可用的福利，验证覆盖范围以及提供事先授权支持和提交-通常以电子方式执行以简化和加速流程。 .

It is common for the newer, typically more expensive treatment plans to face initial denials, with payors often requiring patients to have already undergone failed alternative treatments or specific biomarker testing.

对于较新的，通常更昂贵的治疗计划而言，通常会面临最初的否认，付款人通常要求患者已经经历了失败的替代治疗或特定的生物标志物测试。

To avoid such denials, it is crucial to educate both patients and HCPs on the prior authorisation process, ensuring that all necessary clinical notes are submitted to the payor to secure treatment qualification.

为避免此类否认，至关重要的是对患者和HCP进行事先授权过程的教育，确保将所有必要的临床笔记提交给付款人以确保治疗资格。

Future-proofing patient engagement services

未来校对的患者参与服务

As we look to the future of patient engagement services, advanced technologies are increasingly being leveraged to accelerate the diagnosis and care of patients with rare diseases.

当我们展望患者参与服务的未来时，越来越多地利用先进技术来加速罕见疾病患者的诊断和护理。

The potential of artificial intelligence (AI) and natural language processing (NLP) could provide significant value in these areas, from diagnosis to treatment — and beyond.

人工智能（AI）和自然语言处理（NLP）的潜力可以在这些领域提供重要价值，从诊断到治疗-以及其他。

One example is utilising AI technology that harnesses medical records, patient-reported data, and genetic information to assist in symptom recognition, resulting in an impressive diagnostic accuracy rate of 79%.2

一个例子是利用AI技术，利用医疗记录，患者报告的数据和遗传信息来帮助症状识别，从而使诊断准确率达到79%.2

After diagnosis, this technology can also empower patient engagement partners to listen to patient interactions and derive valuable insights from them. With the combined capabilities of AI and NLP, these powerful tools can detect confusion during calls made to HCPs and equip agents with the most appropriate actions to address issues effectively..

诊断后，该技术还可以使患者参与合作伙伴能够倾听患者互动并从中获得宝贵的见解。凭借AI和NLP的综合功能，这些强大的工具可以在向HCP呼叫期间检测混乱，并为代理商提供最适当的行动来有效解决问题。。

Additionally, these tools can aid in identifying patients at high risk of discontinuing treatment, enabling HCPs to offer timely and tailored support to ensure uninterrupted access to the necessary treatment.

此外，这些工具可以帮助识别处于停止治疗高风险的患者，使HCP能够提供及时和量身定制的支持，以确保不间断地获得必要的治疗。

Embracing partnerships for a patient-focused future

拥抱以病人为中心的未来的伙伴关系

As the healthcare landscape continues to evolve, it brings regulatory, technological, and treatment changes that pharmaceutical companies must adapt their patient engagement services to.

随着医疗保健领域的不断发展，它带来了制药公司必须调整其患者参与服务的监管，技术和治疗变化。

By teaming up with the right partner dedicated to supporting patients, companies can effectively tackle these challenges by providing comprehensive support throughout the patient’s diagnosis and treatment journey.

通过与致力于支持患者的正确合作伙伴合作，公司可以通过在患者的诊断和治疗过程中提供全面支持来有效应对这些挑战。

Beginning with disease understanding and finding appropriate specialists, this type of partner provides a service where individuals can seek guidance for their specific disease state and the treatments that may be available to them.

从对疾病的理解和寻找合适的专家开始，这种类型的合作伙伴提供一种服务，让个人可以为他们的特定疾病状态和可能对他们可用的治疗寻求指导。

Whether it is clarifying information found on unverified websites or locating the right specialists to provide treatment, this service helps patients navigate their options and understand the necessary tests for treatment eligibility.

无论是澄清未经验证的网站上发现的信息还是找到合适的专家提供治疗，本服务都可以帮助患者浏览他们的选择并了解治疗资格的必要测试。

By engaging with these experts, pharmaceutical companies can enhance patients’ treatment adherence, facilitate better care throughout their journey, and contribute to improved healthcare outcomes.

通过与这些专家的接触，制药公司可以提高患者的治疗依从性，促进整个旅程中更好的护理，并有助于改善医疗保健结果。

References

工具书类

1. https://www.fda.gov/patients/rare-diseases-fda

1. https://www.fda.gov/patients/rare-diseases-fda

2. https://rarediseases.org/wp-content/uploads/2020/11/NRD-2088-Barriers-30-Yr-Survey-Report_FNL-2.pdf

2.https://rarediseases.org/wp-content/uploads/2020/11/NRD-2088-Barriers-30-Yr-Survey-Report_FNL-2.pdf