As many as 40 cell and gene (CGT) therapies will launch in the next decade. In addition to clinical benefits to patients, these therapies have steep price tags. Our objectives were to (1) understand payer attitudes towards CGT reimbursement and (2) categorize payment models that payers and manufacturers have pursued to address reimbursement challenges. This study examines CGT launches to understand manufacturer pricing and reimbursement approaches.

We performed a literature review of recent developments in the CGT market access landscape to identify solutions to challenges. Recent CGT launch approaches were analyzed by their product clinical profile, epidemiology, and pricing (via the Pricentric® database). Interviews with medical directors at payer institutions were performed to estimate the relative impact of challenges and feasibility of implementation of solutions across payer channels: 1) commercial health plans, 2) IDNs, 3) Medicaid FFS, 4) managed Medicaid, 5) Medicare Part D, 6) Medicare FFS, and 7) Self-funded employers.

We identified two key challenges facing value assessment and reimbursement for CGTs: the temporal gap between the cost and benefit of a durable therapy, and the volatility in annual budget impact. The impact of these challenges was quantified among payer segments. We identified four archetypical solutions that US health plans have pursued to better align the risks and benefits of covering CGTs: amortization, outcomes-based agreements, reinsurance, and payment pools. The proposed solutions are segmented by feasibility and potential to overcome CGT access challenges. Implications for CGT manufacturers are provided.

Potential solutions for achieving alignment among US stakeholders – including manufacturers, health plans, and policy-makers – require changes to the US health system. Potential solutions may require adoption of features of the single-payer systems widespread in Europe. Solutions require outcomes tracking and research to appropriately set premiums (i.e., for reinsurance or shared payment pools) or negotiate terms of outcomes-based contracts.