Since 2016, The FoCUS consortium has brought together diverse system stakeholders to identify challenges and design solutions for financing innovative cell and gene therapies (CGTs). We have worked collaboratively to address the need for innovative financing and reimbursement models for CGTs that ensure patient access and sustainability for all stakeholders. This is a multi-stakeholder effort of developers, providers, regulators, patient advocacy groups, and payers from all segments of the US healthcare system, along with academics working in healthcare policy, financing, and reimbursement.

In FoCUS “Design Lab” workshops, the FoCUS participants identified challenges from varying perspectives. Operational changes may be required regarding how and where care is delivered. Reimbursement processes and payment terms impacting therapy access will need to be changed for providers, patients, and payers. Government regulators and legislators will need to ensure regulatory policies continue evolving along with innovations in science and payers’ approaches to reimbursement. Stakeholders will need to work together to address coverage and reimbursement issues.

The FoCUS Project has developed this toolkit as a resource for understanding the financial challenges of delivering and financing cell and gene therapy (CGT). We acknowledge our coverage of CGT challenges in the toolkit is not all-inclusive. Other organizations specifically provide education on rare diseases and/or on the science, technology, and potential clinical benefits of CGT, including National Association of Rare Disease (NORD), the Cancer Support Community, and the American Society of Gene and Cell Therapy. This section provides stakeholder-specific insights into the financing challenges of coverage for CGT, and the impact of potential innovative financing solutions.

The Stakeholders’ Shared Journey

Access to, treatment with, and payment for CGTs will impact various stakeholders differently. The FoCUS consortium works collaboratively to ensure patient access and sustainability for all stakeholders. By understanding these differences, we are better able to collaborate on effective solutions for all parties.

Concerns roughly fall into three categories: financial challenges, market adoption pressures, and implementation issues related to administering Precision Financing Solutions. Some issues pose strategic challenges while others can be addressed with resources.

Financial challenges: These challenges arise from high upfront payments for uncertain benefits that are to be received over time.

• For payers – Concerns about budget impact and treatment’s positive/negative financial value to the organization
• For developers – Concerns about recovery of development costs and risk of underperformance
• For patients – Disease burden with and without new treatment

Market adoption pressures: Once a product is approved, various factors outside of the financial influence market penetration and adoption.

• For payers – Disenrollment of treated patients; resources to manage any contracts used to mitigate loss of value from providing access to therapy
• For developers – Maximizing use of tools, financial or otherwise, to support market penetration and adoption
• For patients – Maneuver health system in a timely manner for best care

Implementation issues: Strategies to address financial challenges and market adoption concerns may result in implementation issues.

• For payers – Establishing new provider networks, payment terms, developer contracts, outcome metrics
• For developer – Facilitation of provider, patient, FDA, and payer needs for access and outcomes tracking
• For patients – Navigating the healthcare system while minimizing its personal impact on care preferences, privacy, and expense