By 2030, as many as 75 new cell and gene (C&G) therapies may be approved by the FDA. These therapies offer the potential of life-altering cures of sometimes previously untreatable conditions. This paper brings together several related areas of literature concerning C&G therapies to examine their implications for managing population health versus individual patient incentives, access, and outcomes.

METHODS:

We examined four areas of the C&G therapy literature--(1) payer concerns about coverage and reimbursement, (2) financing and reimbursement models, (3) health insurance design, and (4) dimensions of value specific to C&G therapies--with a focus on the extent to which they prioritize population health management versus the patient perspective.

RESULTS:

Based upon the experience with orphan drugs, insurance financing models may increasingly shift to debt-like instruments, such as annuity payments with outcomes guarantees. Patient access to C&G therapies may transition to insurance benefit designs using increased utilization review and steerage of patients; such approaches have historically exposed patients to high out of pocket costs. One-time, curative treatments raise unique issues of value and risk that must be addressed in a broader context and across multiple payers to equitably support access and reimbursement. For example, as patients transition from commercial health insurance or Medicaid plans to Medicare, a sizeable potential social surplus could result as Medicare inherits patients who have been previously cured by commercial insurance or Medicaid programs. How should such a social surplus be reallocated among patients, pharmaceutical firms, and payers to ensure optimal access to C&G therapies from a societal perspective?

CONCLUSIONS:

All proposed or currently utilized benefit models focus on financing issues to address C&G therapies. These approaches may create access barriers for C&G therapies due to coinsurance and related out-of-pocket costs. Current approaches also do not account for patient or physician perspectives as part of the coverage and reimbursement process.