Kate Gudsell - @KateGudsell
Pharmac has announced the first drug it will fund for rare disorders will be for treating the life-threatening genetic condition hereditary angiodema.
Last year the country's drug-buying agency started a competitive process to fund $25 million worth of medicines over the next five years to treat different rare disorders.
About 90 people in New Zealand suffer from hereditary angiodema, known as HAE, and about 25 experience the type of extreme attacks which would make them eligible for the funded treatment.
Photo: 123RF
Sufferers of HAE experience swelling in various body parts including the hands, feet, face and airway which can be fatal.
Patients can also experience bouts of excruciating abdominal pain, nausea and vomiting that is caused by swelling in the intestinal wall.
The severe swelling is usually treated in hospital, but icatibant is a filled syringe which can be injected and reduce the swelling.
Icatibant from Shire Pharmaceuticals will be funded and will mean sufferers can keep the medicine for injection in the event of an attack, rather than having to be treated in hospital.
Funding for rare disorders
The funding for the rare disorders comes from Pharmac's budget, and came about after community consultation in 2012 and 2013 into how the agency considers funding treatments when quite often the price is very high and the ability to demonstrate benefits is often very low.
Last year the agency put out the funding provision to the market of suppliers who have products that fit the criteria that it sets out for rare disorders, and Pharmac's chief executive Steffan Crausaz said that encouraged 28 bids for the use of that funding.
"Then what we were able to do is assess each of those and work out which ones really offer the best value for money in terms of meeting a health need, in terms of having evidence that will demonstrate effectiveness for the patients suffering from various conditions."
Mr Crausaz said the process was to introduce competition for the rare disorder funding.
Normally Pharmac introduces competition for a particular drug, but in this case it told the market it had this funding, it didn't know the products it wanted, but it knew the features of the products it was interested in.
"What we wanted to do here was to see if we could use competition for a limited source of funding to get the suppliers to basically provide better terms of supply and that would then help us get access in an affordable and sustainable way."
Once Pharmac has allocated the funding it will go back and review how the process went to make sure it has met all the objectives.
Mr Crausaz said it will look at whether it got proposals from suppliers for treatments Pharmac has not seen before and whether it received prices that were lower than it had been offered previously.
"One of the issues that we've found with some of these treatments that are used in very small numbers of people is that the prices can be very high compared sometimes to the benefits that are demonstrated through the evidence.
What we were hoping to achieve was to be able to introduce competition which would hopefully lower prices and make them more affordable and enable us to give access."
Shire Pharmaceuticals has not been active in New Zealand before and he said the process had encouraged a new supplier to come to the market and given Pharmac access to a product which previously it did not have access to.
Once Pharmac makes a commitment to funding a drug it is long-term, so there is an expectation it will continue to fund icatibant past the five years.
Mr Crausaz said funding icatibant was a commercial arrangement and therefore confidential but there was still space from the $25 million pool for several more drugs.
"It met a health need that was there, that was serviced with some alternate treatments which weren't ideal, and those alternate treatments were actually more expensive than icatibant itself, that's why icatibant stacks up out of all the proposals we got as actually quite a good one."
Pharmac said funding the drug is cheaper than the hospital treatment and several more funding decisions will be made over the next few months.