OBJECTIVES: Upfront combination therapy has been shown to be clinically beneficial in certain patient groups in rare diseases. However, the potentially high economic burden associated with combination therapy in the rare disease space maybe restricting patient access. This research aims to explore innovative pricing and contracting options that have already been implemented in the cancer space which may also be considered within rare diseases with a view to increase patient access to combination therapies whilst minimising cost burden.

METHODS: Primary research with payers in the UK, France, Germany, Spain and Italy were probed on market dynamics that will affect the pricing of combination therapy in their markets. Potential contracting strategies were evaluated in each market and the feasibility of launching fixed dose combinations in rare diseases were explored.

RESULTS: Over the next 5 years, innovative pricing and contracting options are expected to be increasingly adopted in the EU5 markets to reduce the increasing economic burden associated with combination therapy within the rare disease space. Particular value was seen in simple price discounting along with more complex patient outcomes related contracting measures. Fixed dose combinations offering a 5-30% reduction compared to standalone drug prices given in combination was seen as a crucial component to success in all markets.

CONCLUSIONS: The increasing burden of combination therapy in rare diseases maybe restricting access; this burden maybe eased by the introduction of innovative contracting. Furthermore, the introduction of a fixed dose combination could prove to be successful if priced at a discount in comparison to the cost of standalone drugs given in combination.