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Work in funding medicines for rare disorders progressing

PHARMAC work in funding medicines for rare disorders progressing

PHARMAC is progressing work aimed at improving funded access to medicines for rare disorders.

Chief Executive Sarah Fitt says the latest step is the appointment of a clinical subcommittee with expertise in evaluating medicines for rare disorders. The subcommittee, which includes leading New Zealand and Australian experts in managing patients with rare disorders, will provide advice to PHARMAC’s main clinical committee, PTAC.

Earlier in the month, PHARMAC published updated policy settings for assessing medicines for rare disorders, and opened a commercial process seeking funding applications from pharmaceutical suppliers, specifically for medicines for rare disorders.

“We’re pleased to have made this progress following on from the commitments we made at the end of last year,” says Sarah Fitt. “The subcommittee will provide expert clinical advice on applications we receive from the commercial process we are currently running. This involves actively seeking funding proposals for medicines for rare disorders.”

“The updated policy settings help define which medicines we will consider in this process, and provide certainty to stakeholders of the sort of medicines we are looking for.

“Ultimately, we hope this work will improve access to funded medicines for people with rare disorders.”

PHARMAC has also made a decision to change, and widen access to, the funded enzyme replacement therapy for the rare disorder Gaucher disease. Taliglucerase will be phased in from 1 August 2018 to replace the currently funded treatment, imiglucerase.

PHARMAC had earlier run a dedicated contestable process specifically for medicines for rare disorders, which resulted in decisions to list 10 medicines on the Pharmaceutical Schedule.


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