OBJECTIVES: To explore the concept of option value with regards to cell and therapies and to evaluate the current and future trends in utilizing the concept in health technology assessment (HTA) and in pricing and reimbursement settings.

METHODS: We conducted a targeted online literature review to identify articles reporting on option value and further refined the search for cell and gene therapies. We abstracted the results and summarised key themes before a working session to uncover current utilization of the concept and generate hypotheses on the future outlook.

RESULTS: Option value is the concept that by receiving a certain medicine at an initial time, opportunity opens up to receive other medicines later on. Most of the literature focuses within oncology, where increased survival outcomes are compounded through successive lines of treatment. The option value concept for cell and gene therapies was given prominence by the Institute of Clinical and Economic Research (ICER), who included it in their Adapted Value Assessment Framework (November 2019). One element of option value that is enhanced for cell and gene therapies is the potential disadvantage for therapies that, if not successful, could reduce or even preclude the potential effectiveness of future treatments. This has pricing and reimbursement implications since, provided patients could have foregone the first treatments to wait for subsequent better ones, the value obtained would be less overall, for the patient and for the payer or healthcare service.

CONCLUSIONS: Option value related to cell and gene therapies is an as yet underappreciated concept that could be utilized proactively by payers, as well as manufacturers of alternative chronically administered or bridging treatments, to ‘devalue’ current options through horizon scanning and lobbying. The inclusion of foregone option value in HTA and pricing models may, however, be an opportunity for more sustainable pricing and requires further exploration.