Analysis and strategy

Client Situation

Our client planned to submit a marketing authorization application for a haematological drug to the EMA. It was questionable whether the drug would be granted orphan designation. Our client wanted to plan the strategy with regard to the benefit assessment as early as possible – before submitting the marketing authorisation application.

Our Solution

Based on the study reports of two studies – a single-arm Phase II study and a randomised controlled Phase III study (RCT) – we prepared a strategy workshop. We considered several scenarios for the likely label, which was covered to varying extent by the studies. In this full-day workshop with our client, we discussed the options for presenting the benefits of each of these scenarios. The key challenge we identified was that in none of the scenarios the comparison arm of the RCT would correspond to the probable appropriate comparator. Since no adequate data had been published that would be suitable for an indirect comparison, an additional benefit was hardly to be achieved. We advised our client to seek orphan designation if possible, as this was the only way to achieve at least a non-quantifiable additional benefit.

Client Benefit

Our client gained an approval with orphan designation. In the G-BA decision, a non-quantifiable additional benefit was granted, which helped our client to gain a satisfying price in the price negotiations.