OBJECTIVES

To explore the heterogeneity of payment schemes of cell and gene therapies across the EU-4 and the UK.

BACKGROUND

Potentially live transformative cell and gene therapies often exhibit high costs, which can be challenging to reimburse due to many data uncertainties and potentially high budget impact (e.g. limited long term and epidemiology data).

Therefore, payers and manufacturers are increasingly managing these risks by applying innovative contracting approaches to share the risk of such treatments.

METHODS

A targeted literature review was executed to identify the different payment schemes associated with one-time cell and gene therapies in the UE-4 and the UK.

Key secondary research findings were validated with payers, clinical and economic advisors, and industry market access experts.

RESULTS

Earlier approved (2012-2017) one-time cell and gene therapies were fully reimbursed at administration. The therapies that have become available more recently tend to follow alternative payment schemes linked to outcomes following administration of the treatment.

The building blocks these schemes vary across the EU-4 and the UK. Outcomes used to define success range from binary measures that apply to all patients, such as survival, to an improvement from baseline scores in a condition-specific metric, set individually for each patient. Duration of the schemes is also variable, ranging from 1.5 to 5 years. In addition, the payment mechanisms underlying the scheme vary including annual payment instalments and rebates when outcomes are not met.

CONCLUSIONS

Innovative payment schemes for cell and gene therapies seem to become increasingly popular, addressing some of the key reimbursement challenges for these therapies, such as financial risks for the payer due to lack of data. In addition, there is high variability in the application and design of these schemes, as well as the level of success of each mechanism, in terms of facilitating reimbursed access to patients.