Orphan Drug Award

Summary of work

Two years after receiving marketing authorisation as the only licensed treatment to reduce the number of new digital ulcers, and with no supporting NICE guidelines (due to its orphan drug status), restricted PCT funding meant Tracleer was being prescribed to very few appropriate patients. Lack of prescriber understanding about the funding process for orphan drugs and poor communication of patient need to PCTs was resulting in few successful treatment funding applications.

The delivery of a funding support strategy and toolkit gave prescribers the confidence and understanding to apply for funding effectively – the resulting increase in appropriate prescriptions has empowered prescribers and given patients access to the only licensed treatment for their condition, providing them with an improved quality of life.

Judges comments

“Good insight into the specific healthcare environment relating to digital ulcers and a much deeper understanding of the process around Rx’ing orphan drugs. The two-pronged approach to increase successful individual funding requests and secure formulary status on PCTs worked well with eight PCTs demonstrating effective market access. The well-designed pack of support materials worked well in solving the barrier to access.”

Stakeholder comments

“The agency team that worked on this project has done a fantastic job; creating tools that help prescribers understand how they can access on-going funding for Tracleer when they feel it is appropriate for their patients. DU is a horrible condition that affects a very small number of patients. For prescribers to have to deny patients access to the only licensed therapy was really difficult for them. Insights on the issue and, critically, how they have helped prescribers communication with PCTs, have made this project a great success for Actelion, prescribers and patients.”
Head of Market Access, Actelion