Advisory Boards & Workshops – Orphan Disease Pricing Case Study


Appropriate price bands had been defined through earlier payer research with our client. The objective for this project was to understand how to achieve the optimal price for an orphan drug which had lost its orphan designation. A key objective was to understand how to negotiate with key stakeholders within HTA agencies and, specifically, pricing authorities.


The project was delivered in the form of a workshop, with Payers representing the five key European markets on a panel, answering questions from over 35 delegates from the client company. The second part of the session was series of parallel workshops where delegates from different markets were split into groups to work with Payers on mock negotiations and submissions to HTA agencies.


The client was able to gain valuable insights into what drives negotiations for drugs with small target populations. From a pricing perspective, the relevance of orphan designation was deemed to be relatively unimportant compared to actual patient numbers, which were more significant. There were, however, other benefits that orphan drug designation would have afforded the product, such as fast tracking, patent protection and some financial breaks.
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