While Rare Disorders NZ will be celebrating this news with the SMA community, frustration remains that it has taken so long to approve this life-changing medicine, having long called for a separate assessment pathway for medicines for rare disorders.
Rare Disorders NZ is pleased to learn that Pharmac’s Pharmacology and Therapeutics Advisory Committee (PTAC) has recommended the life-saving drug Trikafta be funded for eligible people for the age groups 6 years and older and 12 years and older.
Rare Disorders NZ applauds the Government for accepting the Pharmac Review Panel’s recommendation to ‘develop a strategy that will lead to better, more timely services and more equitable support and outcomes for people and whānau with rare disorders’.
Rare Disorders NZ is incredibly disappointed about the inadequate funding allocated to Pharmac in this year’s Budget announcement, as it means life-saving medicines that many in the rare disorder community are desperately awaiting access to will not be funded for another year, and lives will be lost as a result.
We are honoured to have received confirmation that Her Excellency, The Right Honourable Dame Cindy Kiro, GNZM, QSO, Governor-General of New Zealand, has accepted our invitation to become patron of Rare Disorders NZ.
Rare Disorders NZ is extremely frustrated to hear the Minister of Health this week dismiss the call to increase funding for Pharmac to fund the medicines on its Options for Investment list due to budget constraints.