OBJECTIVES

With numerous gene therapies in development, the possibility of choosing between several therapies is becoming a reality. This could introduce the concept of warehousing – foregoing treatment with a current gene therapy (GT1) to wait for a newer, potentially more effective or safer gene therapy (GT2). This research aims to assess payer preferences towards applying such a concept in varying scenarios.

METHODS

A total of 14 European stakeholders were surveyed on the likelihood to warehouse GT1 in lieu of a more competitive, future GT2 option under different disease and timeline scenarios. Three timeline scenarios were explored: <1year, 1-3years, and >3years until launch of GT2. The disease scenarios assessed three hypothetical indications: A) ultra-rare high-burden disease with no current specific treatment options, B) non-rare, moderate burden disease with chronic high-cost treatment options available, and C) common disease with lower cost treatments available, but high societal burden.

RESULTS

Disease A had the lowest likelihood to be warehoused in all timeline scenarios (<1year: 36%; 1-3years: 21%; >3years: 14%). Across all three diseases, the likelihood of warehousing reduced with increasing time to GT2. Besides different unmet needs and launch time horizons, country-specific nuances also influence payers’ decision-making. German payers indicated that warehousing would not be feasible due to the immediate access granted upon marketing authorization. In contrast, Italian payers saw warehousing as a potential method to promote price competition, increasing affordability.

CONCLUSIONS

Our results demonstrate that payers are unwilling to delay access to patients in high unmet need indications, even in the short-term. Besides unmet need, respective market nuances may also affect the application of such a method. Striking the balance between when to treat and when to wait will become increasingly important in years to come.