Integrated stakeholder views and challenges

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





Payment risk –impact of high, upfront cost

New costs versus offsetting existing costs; Prevalent population backlog for treatment

Reimbursement required for innovation costs with small treatment-eligible populations

Potential for:

  • prolonged time to approve treatment
  • no coverage
  • premium increases

Performance uncertainty

Non-performance risk

FDA label versus clinical trial data; long-term durability

Value of treatment versus current standard of care

Actuarial risk

Unpredictable high costs, and impact on budgeting

Unpredictability of demand and reimbursement

Treatment eligibility

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





Treatment value assessment

Mismatch of payment and realized benefit over time

Market penetration and adoption impact

Treatment risk versus current standard of care (see patient views and challenges)

Adoption of innovative financial solutions

Resources for contract administration

Medicaid Best Price— impact on reporting and price points for Medicaid

Patient HIPAA protections

Optimize access

Innovative payment models to protect assets

Diagnosis for treatment; coverage for treatment with customized payer contracts

Available providers and payment coverage; diagnosis for treatment

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





Out of pocket costs

Benefit changes to address patient costs

Demand for free goods or patient support programs

Affordability concerns

Outcome tracking

Performance-based contracts; patient mobility and HIPAA; agreement on outcome metrics; administrative capabilities

Long term follow-up required by FDA; agreement on outcome metrics for risk sharing contracts

Meaningful metrics; patient cost of continued follow-up

Out of area treatment

Provider billing; negotiations of out of network charges

Development of Centers of Excellence; provider capacity

Ability to travel; travel costs; loss of time at work

Care navigation

Coordination of patients and providers with internal and external network resources, and with developer hubs.

Need for patient assistance hubs or specialized care management

Variable knowledge to navigate health system; variable resources


Billing challenges

Creation of COEs; training and certification

Need to change providers; Out-of-area providers; provider availability