PHARMAC’s new approach getting real traction for people with rare disorders

A new approach PHARMAC has used for funding medicines for rare disorders is gaining real traction, says chief executive Steffan Crausaz.

PHARMAC has now approved funding for four medicines from a request for proposals, specifically aimed at medicines for rare disorders.

Steffan Crausaz says the approach succeeded in attracting bids for medicines not previously seen in New Zealand, some of which were now available and funded for people with rare disorders.

“The PHARMAC model has always been flexible enough to consider funding medicines for rare disorders, as well as those for more common conditions,” says Steffan Crausaz.

“This new approach has enabled us to find new funding solutions for rare disorders medicines. We’ve used our evidence-based approach to make decisions on the best value medicines from bids received, and at the same time maintained our ability to fund other medicines as well.

“This really is the PHARMAC model in action – it has enabled us to improve health outcomes for people with rare disorders, and for other New Zealanders, from within the funding available.”

Steffan Crausaz says one of the medicines approved for funding this month – bedaquiline – has important implications for public health.

Bedaquiline treats extensively multi-drug resistant tuberculosis, a disease which, while rare, could have a significant effect if it became established in the community.

“Funding bedaquiline gives clinicians a new treatment to combat this disease, and help prevent its spread into the community.”

Steffan Crausaz says he is confident of further medicines being funded as a result of the rare disorders contestable fund process.

PHARMAC has made $5 million per year available, for five years, to fund the rare disorders medicines. Funding is ongoing once they are listed on the Pharmaceutical Schedule.

Medicines approved for funding from the rare disorders contestable funding pilot

Icatibant (Firazyr): Icatibant treats the rare blood disorder hereditary angioedema (HAE). It was approved for funding in late 2015 and listed on the Pharmaceutical Schedule from 1 January 2016, following approval by Medsafe.

Galsulfase (Naglazyme): Galsulfase treats the enzyme deficiency disorder MPS VI, and is the second enzyme replacement therapy listed on the Schedule. Galsulfase was listed from 1 May 2016.

Siltuximab (Sylvant): Siltuximab treats a rare form of immune system disorder called HHV8-negative idiopathic multicentric Castleman’s disease (iMCD). Siltuximab will be listed on the Pharmaceutical Schedule from 1 June 2016.

Bedaquiline (Sirturo): Bedaquiline is used to treat a rare extensively multi-drug resistant form of tuberculosis (XDR-TB). While XDR-TB is rare in New Zealand, there’s a risk of it spreading into the community which can be reduced by using medicines like bedaquiline. Bedaquiline would be listed on the Schedule once Medsafe registration occurred.

Source: Scoop Media