Submission on proposal to fund nitisinone for people with rare inherited metabolic disorders

Rare Disorders NZ supports nitisinone being listed on the Pharmaceutical Schedule and funded for people in New Zealand who need it. We note that nitisinone is currently funded for people with tyrosinemia type 1 or alkaptonuria, two rare inherited metabolic disorders, through Pharmac’s exceptional circumstances framework (NPPA).

It is positive to see Pharmac making changes to processes that reduce the administrative burden for health care professionals, provide transparency around criteria for access and provide certainty of ongoing supply for the people who need it.

We are pleased to see flexibility built into this proposal with an Alternative Brand Allowance proposed to be available for people who are unable to change to the Nitisinone (LogixX Pharma) brand of nitsinone capsules for clinical reasons.

Rare Disorders NZ encourages Pharmac to ensure adequate consultation has occurred with clinicians who have expert knowledge in these inherited metabolic disorders and who prescribe nitisinone to ensure that the changes being made meet their and their patients’ needs.

While this change is welcome, we note that this is not a case of a new medicine being funded for rare disorders, simply a shift in the process to obtain an already funded medicine.

Read our full submission here.