Addressing the Complexities of Rare Disease: Treatment Navigators
A single point of contact for educational support throughout the patient journey

It is estimated that one-in-ten Americans suffer from a rare disease.1 Yet, due to its very nature most of these patients face a long and complex journey towards a diagnosis and treatment. Many face up to eight long years or more of worry, stress, and expense before fully understanding the challenges they are facing. Challenges may include being mis-diagnosed, frequently resulting in patients having to travel long distances to the specialists they need and many having to uproot themselves and family members to be closer to medical care. Once diagnosed, they must then deal with the complexities of obtaining their treatment and using it. They, and their care partners, are overwhelmed and exhausted with what they have been through and by what they still face in finding a path to better health.

Enter the Treatment Navigator. In this vital role, Treatment Navigators, who are nurses by background, become a trusted partner to both patients and their care partners, as well as the HCPs and office staff serving them. Treatment Navigators provide much needed support and education throughout the rare disease journey helping those suffering with a rare disease comprehend what they don’t know and what they will face on their journey to treatment. The Treatment Navigator educates and supports product adherence questions and insurance challenges addressing the
main sources of frustration to those suffering with a rare disease.

The Role of the Treatment Navigator

A Treatment Navigator combines the key competencies of a clinical educator with that of the field reimbursement manager who supports the payer reimbursement process. As such, Treatment Navigators can bring comfort to the patients and their care partners, while providing them with a sense of confidence as they navigate both the treatment and insurance requirements in rare disease. One of their most important roles is setting patient and care partner expectations both in the treatment journey and in the insurance adjudication process. This is critical in keeping them both focused on the reality of their situation and affords them the dignity they deserve throughout the process.

HCPs and offices view the Treatment Navigator as a trusted resource in the patient’s care. Because they all draw from a common language, Treatment Navigators are particularly effective in the product appropriate education with the patient and care partner. In addition, they fully understand the site-of-care challenges and are able to educate offices on how to best support patients receiving new, novel therapies.

The Importance of a Single Point of Contact

Treatment Navigators provide patients and their care partners with a single point of contact for educational support during the journey to a diagnosis and treatment on a rare disease product. In doing so, they help alleviate the stress and confusion that the complexity of a rare disease creates, the importance of which cannot be stressed enough. There is not a straightforward path in a rare disease. There are often many services needed such as blood work, scans, and genetic testing. These tests add a layer of complexity both in pre- and posttesting, and providing the patient and care partner with a single point of contact makes navigation of these services that much easier. Treatment Navigators can also be a much more effective and consistent liaison between patients and HCP offices in explaining payer requirements and helping to understand the necessary information for submission. By providing this comprehensive support through a diagnosis and treatment, patients and their care partners know that they can trust them to support the process eliminating the confusion of navigating multiple contacts.

Why Nurses Make Successful Treatment Navigators

Individuals who have a background in nursing are chosen to be Treatment Navigators first and foremost because they are inherently passionate about the appropriate care of patients and bring that passion to educating patients in order to ensure they have access to a needed therapy. Treatment Navigators are able to draw upon their first-hand experience of helping patients with the strain of navigating the journey of a rare disease therapy and use their deeper understanding of the clinical “whys” to allay patient and care partner concerns. With their background in nursing, they are truly sympathetic to those navigating rare diseases as they have generally experienced the requirements imposed by health plans and the frustrations associated with them.

According to the National Institute of Health (NIH) in their “The Future of Nursing 2020-2030: “Charting A Path to Achieve Health Equity” publication, nurses occupy a special role in health care delivery given their interactions with individuals and families to provide a coordination of person-centered care. Their role has also come to include collaborating with social and community support organizations giving them a strong understanding of the social needs of patients which is so necessary in navigating the challenges of a rare disease.2

The aging population and the high degree of health care illiteracy also make nurses acutely aware of a patient and their care partner needs, making them ideally suited to navigate rare diseases.2 As the role of nurses evolves throughout this decade, the NIH envisions nurses supporting care management and practice operations and having a greater understanding of insurance challenges—all of which supports their role as Treatment Navigators in rare diseases.2

The Ultimate Goal—Successfully Navigating Treatment Access In Rare Diseases

Once a patient is diagnosed with a rare disease, the journey to treatment access begins. Many of the medicines used to treat rare diseases are prohibitively expensive. The Institute of Clinical and Economic Review (ICER) experts reported that the average annual cost of an orphan treatment per patient was $32,000 in 2019, with treatments ranging from $6,000 to $500,000 per year. However, there are reports that these numbers can reach as high as $1 million or more per year. Obtaining affordable access to them can be an arduous and stressful experience.

According to a 2019 survey by The National Organization for Rare Disorders (NORD), 61% of patients had been denied or faced delays accessing treatments that required pre-approval from an insurance company (i.e., prescriptions, medical devices, or other treatments).1 Essentially, just because a physician prescribes a therapy, it is not a foregone conclusion that the patient will be covered by insurance. No one actually knows for sure until the prescription is sent for prior authorization.

The process for prior authorization is complicated, however. There are many reasons for this according to a 2023 American Medical Association (AMA) article “What doctors wish patients knew about prior authorization,” but essentially, prior authorizations complicate the reimbursement process as both physician and patient need to be involved. To ameliorate this situation, the Treatment Navigator can liaise with the patient’s HCP office educating the office on the administrative hurdles in enrollment and prior authorization submissions as well as any denials that HCP offices and patients may face when obtaining benefit verification.

The Treatment Navigator will work throughout the process to educate patients and their care partners on the processes of payers so that if and when a therapy is denied coverage, the patient is not blind-sided to the additional time it will take to start treatment. Although being denied coverage is not ideal, this support goes a long way to help the patient and their care partner understand the basis of the decision and support any appropriate next steps. The Treatment Navigator will continue to educate the HCP and office staff to ensure consistent coverage and during open enrollment periods, they can educate patients on their possible options based on their therapies. They can also partner with internal or external HUB providers and specialty pharmacies to expedite access to treatment once it is approved.

Treatment Navigators play a vital role in rare disease patient and care partner support by implementing an efficient and compliant approach to speed up the time to treatment initiation while providing the empathetic support rare disease patients and their care partners deserve. Ultimately, patients and their care partners learn to become their own advocates and better consumers of healthcare.

1. NORD Rare Insights: BARRIERS TO RARE DISEASE DIAGNOSIS, CARE AND TREATMENT IN THE US: A 30-Year Comparative Analysis, November 19, 2020, pg 3.
2. National Institute of Health: “The Future of Nursing 2020-2030: Charting A Path to Achieve Health Equity,” “Chapter 4 The Role of Nurses in Improving Health Care Access and Quality.”
3. Institute for Clinical and Economic Review, “The Next Generation of Rare Disease Drug Policy: Ensuring Both Innovation and Affordability.”
  • Steve Fields
    Steve Fields

    Vice President of Field Access and Reimbursement Inizio Engage

    With over 15 years of leadership experience in field access, Steve has successfully navigated the challenges of both start-up and large-size companies. Throughout his career, he has specialized in implementing effective FRM strategies, developing go-to-market models, and achieving launch excellence Steve can be reached at


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