Securitization as a means to pay for cell and gene therapies for orphan diseases: a simulation study

Cell and gene therapies may provide life-extending treatments for patients. However, paying for these therapies using a single upfront payment will be challenging because of uncertainty about long-term clinical effectiveness and affordability. Developers, recognizing the challenges of paying for these therapies, have offered payers 5-year outcomes-based installment plans. The short length of these plans, however, does little to address uncertainties about the cost-effectiveness of paying for these therapies. Instead, we propose to offer 30-year performance-based annuities that shift payments to match the expected accrual of clinical benefits more closely. Using securitization techniques combined with long-term performance-based annuities, we demonstrate that in the case of the gene therapy Zolgensma, this mechanism is effective at mitigating concerns over value and affordability for payers. In summary, our proposal for financing cell and gene therapies creates a viable incentive for developers, while also balancing long-term effectiveness and budget impact concerns from payers and access challenges for patients.