'Why won’t they help him?': 'Broken' mother pleads for Pharmac funding for toddler's rare disorder

Three-year-old Tama.
Three-year-old Tama. Photo credit: Supplied

The mother of a young boy is pleading with Pharmac to fund a drug that could stop her son's suffering.

Three-year-old Tama has a degenerative disease called spinal muscular atrophy (SMA), and though a drug that treats it - called Spinraza - is available overseas, it is not funded in New Zealand. 

Tama's mother, Lisa Geddes says she is "just broken" knowing that help for her son is out there but that she can't access it.

"Until Pharmac is reformed to cater for sufferers of rare disorders, children like my son Tama, will suffer," says Geddes. 

"Why won’t they help him?  His life is not worth saving here according to decision-makers and I am powerless." 

'Why won’t they help him?': 'Broken' mother pleads for Pharmac funding for toddler's rare disorder
Photo credit: Supplied

Pharmac's rare disorder subcommittee met on September 24 to consider funding, though a decision has yet to be made as to the outcome of that meeting.

Pharmac said a record of the subcommittee's advice and recommendations will be available online when it is available.

Fiona Tolich, a trustee of Patient Voice Aotearoa, also suffers from SMA. She says that getting medicines such as Spinraza funded by Pharmac is almost impossible.

"Last week Pharmac presented to the Health Select Committee," says Tolich. "It was demoralising to hear that Pharmac, contrary to what the Minister of Health had told the same Committee earlier in the year, no longer has a ring-fenced fund for rare disorders and that all applications to fund drugs that treat such disorders will be part of the bigger pool that is assessed by Pharmac." 

Tolich says the result of such a policy is that unless drugs that help rare disorders are proven to cater for a significant portion of the population they will fail to get funding.

The Government needs to accept that the Pharmac model is "broken", Tolich said.

However, a spokesperson for Pharmac said there was never a separate pool of funding for rare disorders and that many medicines catering to only a small group of patients are funded.

"Over 100 medicines are funded for treating rare disorders through the combined pharmaceutical budget," Pharmac's operations director Lisa Williams said.

"Pharmac works to get the best health for New Zealanders, by funding medicines from within the available budget.  Having a fixed budget means we need to make careful and considered funding choices in the interests of all New Zealanders and this can result in medicines being funded that be used by small groups of patients, including patients with rare disorders," she said. 

Tolich says without funding it is better for people suffering from rare disorders to look for help overseas.

"I would encourage people with rare disorders, much like those with SMA, to now make plans to shift to a country that has a much better drug funding regime and that values life," says Tolich. 

For Geddes, though, the wait to find out whether the drug will get funding and Tama will the help he needs is agonising.

"I’m just broken, watching my son deteriorate when I know there is something that can stop this, it is devastating

"I should be celebrating new milestones, not watching him get attached to breathing support, as this disease continues to cruelly weaken him.  I am heartbroken."

In September, Health Minister Dr David Clark told Newshub the Government's hands are tied. 

"I absolutely understand why New Zealanders suffering from spinal muscular atrophy and their families want affordable publicly funded access to the latest treatments for this disease.

"However, the Government respects the independence and impartiality of Pharmac. It is not for us as politicians to second guess the experts at Pharmac about which drugs it purchases."