OBJECTIVES: The argument of socially unacceptable high costs of Gene therapy (GT) and health care decision uncertainty have led to restricted or no GT access. Outcomes Based Agreements (OBAs) can minimize uncertainties and risks and are considered a promising solution. Still the absence of a broadly supported definition for socially acceptable cost (SAC) can block implementation. This survey assesses the elements that may play a role in SAC and can be addressed in OBAs.

METHODS: To determine what key elements impact the social acceptableness of (high) costs for GT, an online survey amongst experts in the field was held. The survey was distributed by mail, direct contact and LinkedIn. Respondents could choose a top-4 of elements.

RESULTS: In this convenience sample, all relevant stakeholders are represented, the main groups are industry (40%), payers (14%), patients (14%) and science (10%). Valid respondents (n=50) are international, 64% is Dutch.

The most important elements determining the SAC for GT are related to treatment outcome: 92% of respondents (CI=84-100%) ticked one or more of the three outcome elements in their top-4. Treatment outcomes related elements indicated are: potential (duration) of effect (68% respondents, CI=55-81%), effect size (40%, CI=26-54%) and improvement in Quality of Life (26%, CI=14-38%).

Trends are seen that industry also highly values level of innovation, while payers also value the cost elements expected profit on the product and budget impact. For patients this was unmet medical need and burden of disease.

CONCLUSIONS: Treatment outcomes are the common main driver for SAC and can be included in OBAs. However, there is ambiguity about other elements. This should be addressed. With more alignment on SAC amongst all stakeholders, OBAs can be an important step towards access to GT.