PRM17 - Strategies To Overcome Hurdles In Hta Appraisals Amid Limitations Resulting From Single-Arm Trial Data

Abstract

The purpose of this study was to evaluate the barriers that arise in constructing strong pharmacoeconomic (PE) submission based on evidence from single-arm clinical trials, and to evaluate strategies to overcome them. A set of therapeutics were selected based on the following criteria: oncology indication with a PE submission for health technology assessment (HTA) review, utilising single-arm trial data. HTA appraisals in five major markets were considered – GBR, CAN, SCT, GER, and FRA. Modelling approaches used in each appraisal were enumerated, and HTA body comments on each employed strategy were collected. Each identified modelling strategy was evaluated for its likelihood in overcoming a negative appraisal across the selected major markets. The PE model barriers associated with single-arm trial data included: lack of direct comparison with comparator, reliance on surrogate endpoints, and lack of long-term follow-up. The techniques employed to address these drawbacks included: comparison with historical control, comparison with self-control, comparison with non-responder control, comparison with placebo-arm of a published trial, correlation of surrogate endpoints to hard outcomes, survival extrapolation methodology (including parametric extrapolation and cumulative survival approach). These methods were most successful in FRA and CAN, where positive HTA appraisals were awarded 7/8 and 2/2 times, respectively; and were least successful in GBR and SCT, where positive HTA appraisals were awarded 1/4 and 2/8 times, respectively. Manufacturers may face difficulties when modelling a product’s economic benefit based on data from a single-arm trial. While securing a positive HTA appraisal for such PE submissions remains difficult, there exist strategies that can help to overcome identified barriers, which have led to success in major HTA markets – most notably FRA and CAN.