OBJECTIVES: Gene therapies challenge the status quo in value assessment partially because they have the potential to generate larger and broader value than typically considered by Health Technology Assessment (HTA) agencies, e.g. lifetime productivity gains; however, data uncertainty exists. To estimate the implications of this, we explored the gap between potential value generation and current value recognition for a future gene therapy targeting X-linked retinitis pigmentosa (XLRP) – an inherited retinal degenerative disorder which causes progressive vision loss.

METHODS: We reviewed HTA guidelines in England (ENG), the Netherlands (NL) and France (FR) to identify which value elements are likely to be fully captured and compared these against 13 elements relevant to XLRP based on ISPOR’s “value flower” and gene therapy value frameworks. We used literature and expert judgement to assess for which elements an XLRP gene therapy may generate value, to describe the potential value-recognition vs. value-generation gap.

RESULTS: An XLRP gene therapy may potentially generate value on eight of the 13 value elements identified. HTA agencies are currently very likely to consider half or less of these (2, 4 and 1 respectively for ENG, NL and FR) while up to 3 elements are unlikely to be considered in FR and NL, and 2 in ENG. The remaining elements (4 for ENG and FR and 1 for NL) are somewhat likely to be considered, which provides an opportunity but also uncertainty as to how much of this value would be recognised. The value-generation vs. value-recognition discrepancy seems most pronounced regarding productivity losses and disease severity.

CONCLUSIONS: There is a substantial value-generation vs. value-recognition gap for gene therapies targeting XLRP in three leading European HTA systems. This may lead to undervaluation of such technologies with potential detrimental health and societal effects due to delayed or limited patient access.