OBJECTIVES

To understand the impact overall survival maturity has on HTA decision-making in Germany, France, England and Italy.

METHODS

The research reviewed publicly available HTA reports for a set of oncology drugs approved by the EMA in 85 indications over a 5-year period (2013-2017).

RESULTS

Across markets, almost half of the indications investigated included median OS evidence at the time of their review by the local HTA agency. The rest of the indications had either immature OS, not reached OS, non-estimable OS or OS without statistical significance. There was no correlation between availability of statistically significant median OS data and a shorter time to reimbursement in HTA markets compared to the other forms of OS evidence. Further, the availability of median OS data did not serve as a guarantee for a more positive HTA outcome. For example, about 25% of products with statistically significant median OS data received a “no additional benefit” score from the G-BA. When the full spectrum of the trial outcomes was considered, a poor safety profile, use of an inappropriate comparator, and lack of QoL measures overrode the benefit of OS availability at launch. Lastly, indications with a higher absolute OS improvement were more likely to receive a favorable HTA outcome, with this association becoming less prominent when a relative magnitude of OS benefit was considered.

CONCLUSIONS

Although median OS data availability has been historically associated with an improved opportunity to achieve positive HTA outcomes, its relevance in European HTA decision-making highly depends on the maturity of evidence and the magnitude of absolute OS benefit, rather than the existence of OS per se. Across all HTA decisions, the absolute magnitude of OS benefit is found to be given greater influence for pricing and market access authority decision-making rather than the relative benefit, that frequently reflects the extent of unmet need.