Funding system for costly drugs ‘unfair’

Access to costly but life-saving treatment for rare diseases comes down to looking cute on TV or knowing a politician, sufferers say.

Allyson Lock is one of only eight Kiwis diagnosed with Pompe disease, an incurable degenerative disorder. Untreated, it can be fatal, but Mrs Lock of Masterton and other sufferers have been refused treatment because of its cost.

A medication called Myozyme is available, but it is expensive and gets no funding from government drug-buying agency Pharmac. Costs vary according to a patient's age, weight and severity of illness, but in Mrs Lock's case have been estimated at $575,000 a year.

She said patients with similarly unusual illnesses had obtained funding for expensive treatment, which was unfair. "They say it's too expensive, but there are other treatments that cost more. If your family can get you on TV, if you're a little kid, if the public gets into an uproar, you get funded. That's what it comes down to."

Mrs Lock had an application for "exceptional circumstances" funding for Myozyme turned down in 2011 and said the funding system was flawed and inconsistent.

"They've put a price on our life, and they don't want to pay for it . . . you're made to feel guilty for needing an expensive treatment."

Mrs Lock, 49, a mother of three, suspected that support from politicians had helped sufferers of similar conditions requiring expensive treatment, such as Gaucher's disease and Hunter syndrome, to get funding.

But Health Minister Tony Ryall said Pharmac was advised by senior doctors when making funding decisions, and politicians were not involved.

Since gaining a place on a treatment trial funded by drug giant BioMarin, Mrs Lock has flown to Brisbane every fortnight for almost two years to receive the medication intravenously.

With husband Steve's navy job allowing him to work from home and care for sons Josh, 10, and Ben, 12, she had become such a well-known traveller that Auckland hotel staff gave her a cookbook to mark her 50th trip away. She still had another 80 of the 5500km round trips to go, which she said was a strain on the family.

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Funding system for costly drugs 'unfair'