New information from New Zealand’s Port CF patient data registry is expected to be one of the best weapons in the fight for new medicines and improved treatment.

The 2016 Port CFNZ report will be released in the next few months and Auckland University Paediatrics Associate Professor Cass Byrnes says this quality data, which shows where New Zealand sits in terms of health of persons with CF nationwide, is what is needed to convince health authorities of the importance of new precision medicines that are available in other countries.

“This registry is one of the most important resources we have in cystic fibrosis care. If there are gaps in care, or if we can show how many are eligible for any new treatment that is being considered, then we can argue for new medication or better resources. No decisions are made without data.

“Every time Pharmac is considering a new medication for this population, they will do nothing without facts and numbers to assess cost and effect.

“Furthermore, if we can compare the health and availability of medicines with other countries national registries - and we see gaps - we can fight for what they have that we don’t.”

Parents need to give their consent to have children’s health information included on the registry with these young people re-consenting when they reach 16 years of age. The number of New Zealanders who provide this anonymous data is around 98 percent of all persons with CF in our country.

Dr Byrnes has noted some areas where we could improve access to treatment according to the 2016 figures. “You can see poor uptake of some of the treatment options – for example while many people are appropriately on inhaled hypertonic saline to improve secretion clearance, there are fewer on pulmozyme. And given the numbers that have chronic Pseudomonas infection, we should see more on inhaled antibiotic therapy. Is that individual choice? Or are some people not being offered some medications, as they don’t realise they are eligible for them?”

“There are other areas in the 2016 report that we should drill into to find out what’s happening. An example of that is physiotherapy. A lot of people are ticking ‘no physiotherapy’ as their primary airway clearance technique – and is that right? A lot of people are ticking sport as their primary airway clearance technique and it would be really nice to drill into that to find out how much exercise they are doing.

“Sport might be appropriate for some who are really, really well and do a lot of activity but we’ve only got one tick and it could mean anything.

“We have gaps in our treatment compared to places overseas but in addition to that we can probably do better with what we have,” she says.

Dr Byrnes believes one of the main reasons for these differences is the number of New Zealanders receiving care in different places throughout the country.

“The total number of New Zealand people with cystic fibrosis would make up one single clinic in the States for example. The same in Australia – they have an outreach programme that is just more rigorous than ours. We do have an outreach programme and we have very good paediatricians and specialists around the country managing children and adults with cystic fibrosis but we don’t have enough outreach from our centres sometimes to support them.”

Dr Byrnes says the genetic make-up of New Zealanders could also be changing with the country’s evolving population which may inform changes to the newborn screening programme and which CF genes are tested for with the heel prick test.

“In future we are going to look at whether we are doing the correct screening,” says Dr Byrnes who is also on the Ministry of Health’s Newborn Screening Programme’s Technical Committee.

“It’s important for people to know what their genes are for the new drugs coming through, so anyone who doesn’t know their genes should retest, especially if their original test was done some time ago,” she advises.

For the 2016 report, there was extra emphasis on getting all the data on all persons with CF around New Zealand into the database with CFNZ engaging Jan Tate to support clinics from around the country to input data. “This 2016 report will be the most accurate snapshot of where all people with cystic fibrosis are. It will be the best one so far to compare to how we’re doing with international registries – Australia, Canada, the States, UK, France and Germany.” Port CF data suggests that the median age of people with CF in New Zealand is a few years behind other countries which is another reason why it’s important to have this information available. She says life expectancy data isn’t available because the New Zealand Port CF registry hasn’t been going long enough to make this calculation.

There are some positive NZ figures compared to other countries that have emerged from the data.

“Our chronic Pseudomonas rate is actually lower and our lung function at the time of transition to adults is better. And nutrition (weight) seems very good compared to other places as we have a high and early use of nutritional supplements. So we do some things very well.”