OBJECTIVES

: In the last decade, the relapsed refractory multiple myeloma (RRMM) treatment landscape has changed, with high-cost combination therapies becoming more prevalent and showing limited value differentiation versus existing treatments. Comparative evaluation of these regimens is a challenge for HTA bodies given the saturated area; manufacturers can avoid potential pitfalls by understanding HTA criticisms. Our research evaluates initial pricing/market access outcomes and payer critiques in RRMM across 8 EU markets for triplet combination therapies of daratumumab, elotuzumab, panobinostat, carfilzomib and ixazomib, with the aim of identifying common challenges and evidentiary requirements to guide future sponsor access activities.

METHODS

: Published initial pricing/market access reports from nine national HTA bodies in Spain (n=3), Italy (n=4), France (n=4), Germany (n=5), UK (n=6, NICE and SMC), Ireland (n=4), Sweden (n=3), and Netherlands (n=1) were identified from 2016-2018 (including local language reports). HTA outcomes and rationales were extracted, including the use of clinical access restrictions to the EMA label and economic restrictions (financial agreements specified in the HTA reports).

RESULTS

: Of the 30 pricing/market access outcomes identified 47% resulted in non-recommendation, 23% in restrictions, and 30% in positive recommendations. A primary reason for non-recommendations was economic uncertainty, often associated with immature efficacy data (particularly OS). Label restrictions and simple discounts were the most common access conditions, with innovative contracting or requirements for further evidence generation being less common.

CONCLUSIONS

: The RRMM space for triplet combination therapies is extremely challenging for manufacturers, with over 70% of initial access outcomes resulting in non-recommendations or restrictions. Given the lack of curative treatment for MM, manufacturers and HTA bodies need to work closely together, and harmonize through early scientific advice to ensure alignment on evaluation requirements and goals of treatment. Access outcomes and negotiations may increasingly reflect the evidence available at the time of marketing, to mitigate economic uncertainty.