Apr 11, 2023, 12:45 PM
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Rare Disorders NZ is extremely disappointed to learn from Pharmac’s final response to the independent Pharmac Review that the drug-buying agency is waiting for Manatū Hauora to develop its Strategy for Rare Disorders rather than take immediate action to improve assessment pathways for modern rare disorder medicines to save lives.

Pharmac’s full response to the Pharmac Review was finally released on 3 March after the sector has been waiting months for the report to be made public. It made for disappointing reading for those affected by rare disorders.

“It is frustrating that despite the Review dedicating an entire chapter to how the rare disorder community is being failed, Pharmac’s response is that it will wait and see what comes out of Manatū Hauora’s work to develop its Strategy for Rare Disorders,” says James McGoram, Rare Disorders NZ Chair.

The independent review of Pharmac, commissioned by the Government in 2021 and released to the public in June 2022, found the current model within which rare disorder medicines are assessed at Pharmac contributes to inequitable health outcomes for people living with a rare disorder.

Currently, there are only two assessment pathways for rare disorder medicines – through the pharmaceutical schedule along with all other medicines, or through the exceptional circumstances framework, under which individuals, with exceptional clinical circumstances, can seek to have access to a medicine not on the pharmaceutical schedule. Neither of these pathways provide reasonable assessment conditions for rare medicines to be approved.

“We have long been calling for separate assessment pathways for rare disorder medicines. The current one-size-fits-all model under the pharmaceutical schedule does not work for low volume, high-cost medicines for rare disorders, and there are too many inconsistencies under the exceptional circumstances framework. There is nothing in Pharmac’s response to the review which suggests the shift change required for our community,” says McGoram.

“We will continue our dialogue with Pharmac, and to provide the lived experience voice, but if the agency is truly committed to centering equitable health outcomes in all of their work, it is time to start showing it.”

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