Pharmac’s Rare Disorders Advisory Committee makes new recommendations for funding

Te Pātaka Whaioranga – Pharmac has published the record of the March Rare Disorders Advisory Committee meeting, sharing details on a number of funding applications including those for spinal muscular atrophy (SMA).

The Rare Disorders Advisory Committee meeting followed a call for applications in 2022.

“We are continuing to provide more visibility of the treatments we’re considering that could impact the rare disorders community.” says Pharmac’s Director Advice and Assessment/Chief Medical Officer, Dr David Hughes.

Following the funding decisions for nusinersen and risdiplam for spinal muscular atrophy (SMA) Pharmac committed to seeking further advice about funding the treatments for people over 18 years and over, and for people with different types of the condition. The Committee supported wider access to these treatments.

“The next step is to assess and rank these on our options for investment list. As we must work within our fixed budget, we’re unable to give a definitive timeframe for if, or when, funding decisions would be made,” says Dr Hughes.

At the March meeting, the Committee also reaffirmed its recommendations for agalsidase alfa and migalastat for Fabry disease, and recommended teduglutide be listed with a high priority for the treatment of children with short bowel syndrome and intestinal failure.

The Committee reviewed new evidence for elosulfase alfa for the treatment of Morquio syndrome-Type A. It gave a recommendation with a high priority for children under the age of two years and a medium priority people aged two years and over – both applications previously having received a recommendation for decline.

Two further applications the Committee considered included the gene therapy for SMA where the recommendation is deferred until further evidence is available, and the risdiplam application that received a high recommendation for use in pre-symptomatic SMA (which was funded from May 2023).

“The applications that were reviewed at this Committee meeting provide examples of Pharmac’s assessment processes in action. We are pleased that many applications received positive recommendations, and that an application with new evidence had its recommendation changed.”

Dr Hughes shares a special thanks to the people from the rare disorders community who attended the Committee meeting and presented, sharing their lived experience.

“Hearing how medicines impact the lives of New Zealanders is really important in helping us understand what medicines we should be funding. We will continue to work with the rare disorders community to continue the momentum in this space.”