Guiding the Gene Therapy Journey: How Clinical Program Managers Deliver Coordinated, Compassionate Care

The promise and complexity of gene therapy

Gene therapy is transforming the lives of individuals with rare and complex diseases. For individuals and families managing chronic conditions, the potential of a one-time treatment brings both hope and uncertainty.

However, the journey to gene therapy is far from simple. It is an unfamiliar, emotional, and high-stakes process that demands precise coordination and consistent support. Clinical Program Managers at Accredo® Specialty Pharmacy play a vital role in this process, ensuring that patients are surrounded by a team of experts who understand both the scientific and human aspects of care.

These professionals serve as essential connectors—bridging clinical and logistical gaps between prescribers, manufacturers, payers, and treatment centers. Their goal is to ensure that patients and their families feel informed, supported, and confident at every stage of their gene therapy journey.

Advocacy and Connection: The role of a Clinical Program Manager

Patients with rare and complex conditions, such as bleeding disorders, often spend their lives managing various treatment regimens, juggling appointments, and advocating for themselves. When gene therapy becomes a possibility, the opportunity can feel both hopeful and overwhelming.

Clinical Program Managers help translate the complex science and intricate logistics of gene therapy into a clear, manageable process. Their responsibilities span clinical, financial, and logistical domains:

• Clinical Support: All Clinical Program Managers are specialty trained pharmacists and provide direct support to patients and families. They reinforce physician guidance, review medications to prevent interactions, and answer clinical questions that arise outside scheduled visits.
• Financial Coordination: In collaboration with the GeneAXS™ team—a group of eligibility specialists dedicated to high-touch, gene therapy-specific support—Clinical Program Managers help navigate insurance approvals, coordinate prior authorizations with prescribers, and explore manufacturer copay assistance or other financial support programs.
• Logistical Oversight: Acting as liaisons between pharmaceutical manufacturers and the specialty pharmacy, this role helps ensure that gene therapies are dispensed to the appropriate Qualified Treatment Center (QTC) with the correct documentation and timing.

While orchestration is complex; the goal remains simple: to provide patients with confidence, clarity, and hope throughout their gene therapy journey.

Translating hope into action

One example involved an 18-year-old patient with a bleeding disorder who had been receiving factor IX infusions through Accredo for years. Over time, a strong relationship developed between the patient’s family and their dedicated support team. When the family decided to pursue gene therapy, the Clinical Program Manager and Patient Advocate worked in tandem to coordinate insurance coverage and logistics —each contributing their expertise to ensure a smooth transition.

The Clinical Program Manager led clinical and logistical coordination, while the Patient Advocate helped connect the family to available support services through Accredo and the broader community. Eventually, the patient’s mother received news she never expected: their financial responsibility for the gene therapy treatment would be $0. The relief was immediate and deeply felt.

Support continued beyond that moment. The care team remained in close contact with the family, providing updates, answering questions, and offering reassurance before and after the gene therapy infusion. This level of engagement—helping patients understand their therapy, navigate insurance, prepare for administration, and feel supported long after treatment—is what defines the role of Clinical Program Managers every day.

Extending clinical reach

Clinical Program Managers serve as trusted partners to healthcare providers, acting as an extension of the care team. Their role includes reinforcing physician guidance between visits, supporting the prior authorization process, and ensuring patients remain informed and on track toward treatment.

Behind the scenes, this coordinated care model—anchored by Clinical Program Managers and supported by the GeneAXS™ team and Patient Advocates—manages the complex logistics and communications required to deliver gene therapy successfully. By integrating clinical expertise, financial navigation, and patient advocacy, the model allows prescribers to focus on clinical decision-making while patients receive seamless, compassionate support throughout their gene therapy journey.

Providers can refer patients directly to Accredo at: https://www.accredo.com/prescribers/manage-referrals.

Elevating patient access

Accredo offers fully customizable gene therapy programs designed to meet the unique needs of each patient, providing highly personalized support services that adapt to the specific requirements of the therapy, prescriber, and manufacturer. While customization occurs at the therapy level, the coordinated care model—anchored by Clinical Program Managers and supported by the GeneAXS™ team and Patient Advocates—remains agile and responsive to each patient’s journey.

Through close, ongoing contact with gene therapy patients, Clinical Program Managers gain valuable insight into real-world challenges such as insurance delays and care coordination obstacles. These insights are shared with manufacturer partners to enhance education and help optimize patient support programs.

By collaborating with Accredo, manufacturers gain a clearer understanding of what’s working, where improvements are needed, and how to help more eligible patients receive timely, effective care. The result is improved access to gene therapies, better outcomes, and higher satisfaction across the care continuum.

Raising the bar for gene therapy access

Accredo is committed to a higher standard of care—one where patients are supported holistically, not treated in isolation. This commitment is reflected in the collaboration between the GeneAXS™ team and Clinical Program Managers, who work together to streamline access and remove barriers for patients pursuing gene therapy.

Since February 2023, 44% of gene therapy shipments have been completed without a formulary Letter of Agreement (LOA)—a document typically required by payers to authorize coverage. This is made possible by Accredo’s ability to contract directly with payers for their members, allowing the team to bypass traditional formulary restrictions and accelerate time to treatment.

Financial coordination is equally critical. Through early engagement with payers and thorough benefit investigation, Accredo helps ensure that patients receive coverage with minimal disruption. This proactive approach reduces financial burden and allows families to focus on what matters most—their health and recovery.

These outcomes reflect the strength of Accredo’s coordinated care model, built on deep payer relationships, hands-on advocacy, and a shared commitment to delivering timely, life-changing therapies.

A steady guide toward hope

As gene therapy continues to transform the treatment landscape for complex conditions, the need for coordinated, compassionate patient support is more important than ever. Accredo’s integrated care model—Clinical Program Managers supported by the GeneAXS™ team and Patient Advocates—ensures that patients and families are never alone on their journey.

By combining clinical expertise with logistical precision, the program helps patients access life-changing therapies with confidence and clarity.

To learn more about how Accredo supports patients through the gene therapy journey, visit our GeneAXS™ program page.