Advanced therapies such as cell and gene therapies (CGTs) tend to target small patient populations, and treatment settings tend to be confined to a limited number of Centers of Excellence (COEs). These therapies are typically developed for individual patients, have unique distribution requirements (e.g., ultra cold chain management), and are more expensive than traditional specialty products. It is essential to ensure comprehensive care, enhanced patient support, and adequate distribution services. These components influence the flow of products and shape the patient journey, including patient preparation for treatment administration and ongoing post-treatment monitoring. Specialty pharmacies are developing capabilities to effectively support the highly specialized requirements of CGTs.

Specialty pharmacies evolved from traditional retail pharmacies with the introduction of specialty biologics in the 2000s. They support patients in need of specialty products, often requiring a higher level of pharmacist education, support, delivery, and ongoing adherence management.

Specialty pharmacies are increasingly addressing the unique challenges associated with CGT products. At the pre-treatment stage, specialty pharmacies offer:

• Patient scheduling support
• Clinical expertise, including benefits investigation and verification to ensure expedited access to treatment
• Prior authorization support, including coordination of pre-treatment lab draws, drafting an appeal letter if coverage is denied, and support with genetic testing requirements

During treatment, specialty pharmacies provide standard reimbursement support and enhanced patient support services such as patient-facing care managers, financial assistance (e.g., transportation support), and peer-to-peer/emotional support.

For patients that have completed the treatment regimen, pharmacies provide data tracking support such as data inventory, dispenses, and status reports to manufacturers. In addition, specialty pharmacies offer product distribution and third-party logistics services such as:

• Drop-shipping
• Implementing product storage capabilities at ultra-cold temperatures
• Assuming initial financial liability (e.g., offering expanded offerings by optionality for buy-and-bill or pass-through payment)

These capabilities have become the standard for specialty pharmacies and manufacturers have come to rely on these enhanced services.

Manufacturers of CGTs and other rare disease therapies are interested in specialty pharmacies managing the entire patient and product experience.

Specialty pharmacies have begun to meet evolving demands to ensure comprehensive patient support and efficient treatment logistics. Recent developments include:

• Implementing full care management services with trained nurses to assist patients throughout their treatment journey
• Establishing long-term patient tracking systems to monitor patients across different healthcare plans and providers, collect outcomes, and contribute to registries
• Capturing financial and administrative data from multiple sources and integrating it into existing registries
• Taking temporary or “flash” title in order to bill insurance then shipping product to the infusion center
• Enhancing cold chain shipping capabilities to ensure the safe and reliable transport of products between manufacturing sites, pharmacies, and sites of care, while maintaining temperature monitoring, chain of custody, and product tracking

Given the critical importance of seamless gene therapy provision to the site of care, specialty pharmacies must have the capabilities to accommodate a growing pipeline of products with unique distribution considerations. These pharmacies can be a partner to manufactures by taking on a larger role in providing payment adjudication and authorization, patient support services for CGT-treated patients, and distribution logistics support.

Avalere CGT specialized practice area can help manufacturers to develop their channel distribution strategy to support patient access and help prioritize specialty pharmacies that are building capabilities to support the unique nature of these products and patients. To learn more and how Avalere can help, connect with us.