Govt to study access to high cost drugs

People with rare disorders are hoping for greater access to high-cost medicines after a Government review.

Access to Medicines Coalition chairman John Forman, who made a submission to the high-cost medicines review panel, said people with rare disorders had been missing out on specialised medicines for too long.

Expensive drugs to treat just a few people could never stand up to central funding body Pharmac's cost assessments, Forman said.

While the treatments were very high cost for one or two patients, they cost substantially less than the total amounts spent on more prevalent diseases such as diabetes.

"There needs to be an appropriate budget and system to cope, and we previously had neither of these things," he said.

"Everyone washed their hands of it, and said it's a bit too hard."

Forman said Pharmac's ability to fund high-cost medicines was constrained by its budget, which had risen by just $137 million over the past seven years – from $516m to $653m.

Australia had set up a special programme with a separate budget to assess the benefits of high-cost drugs.

Forman believed this approach could be replicated in New Zealand.

To help ease such pressures, the Government announced in its May Budget $185m in new funding for medicines over four years.

Pharmac has since added 11 medicines to its funding list and increased access to a further four.

Among those to benefit is Jack Peacock.

The Palmerston North boy has Hunter syndrome, a rare enzyme deficiency. When Jack was born, only one other boy in New Zealand had the disorder.

That boy died at 11, and Jack was expected to live to a similar age.

However, after extensive lobbying of the Government by his family and supporters, Jack could now lead a reasonably normal life.

Jack's treatment, which started in August, was approved under Pharmac's exceptional circumstances policy.

Pharmac does not reveal how much it pays for pharmaceuticals, but the market rate for the treatment is about $500,000 a year.

At the time Jack was born, there was no treatment available. Within a few years, his family had read about an enzyme-replacement therapy being trialled overseas.

When the treatment became available 18 months ago, the family then had to convince Jack's specialist.

Jack's mother, Kirsty Peacock, said the specialist believed Pharmac would never accept the high treatment cost.

However, the Peacocks contacted politicians – along with other families of children with lysosomal disorders – and lobbied for better access to treatment.

Kirsty Peacock said it seemed unfair that someone could get drunk and drive, cause an accident, and have costly treatment and rehabilitation, while innocent children with rare disorders were denied life-saving drugs.

After just a few months of treatment, Jack was more active and alert, she said.

He needed to use his wheelchair less and his extended belly was decreasing.

REVIEW PANEL

The high-cost medicines review panel:

* Set up in May this year.

* Will review access to highly specialised medicines and exceptional-circumstances funds.

* Will recommend ways to improve access and will report to Minister of Health Tony Ryall in mid-2010.

* Panel members are former Pegasus Health managing director Paul McCormack, and former MP Joy Quigley, and Paul Hansen, of Otago University's economics department.

- © Fairfax NZ News