CFNSW CEO Michele Adair’s submission to the PBAC on Orkambi.

By Michele Adair, CEO of Cystic Fibrosis New South Wales.

In addition to any physical outcomes it is vital that the assessment of a drugs like Orkambi recognise the whole patient experience.

In the trial, patients experienced fewer exacerbations and needed fewer hospitalisations as well as experiencing the physical benefits reported by their clinicians. This has a positive impact on quality of life and with that, improved mental health and therefor an increased likihood of compliance with other essential therapies and treatments including exercise, diet management etc. these outcomes also directly supports social and economic participation.

In 2013 the NSW Agency for Clinical Innovation published a Financial and Service Utilisation Analysis of admitted patients with Cystic Fibrosis in the NSW Public Hospital System. In summary the analysis concluded

· Historically, the number of separations and beddays remained relatively stable in those patients without complications and comorbidities (CC) slightly declining by around 0.3% per year, whilst the numbers for patients with CC were increasing by approximately 17% each year over the past 7 years.

· The patient cohort for those with complications and comorbidities has an average length of stay (ALOS) three times more than the ALOS for all patients admitted into NSW public acute hospitals, which were 12 days and 4 days, respectively.

· The greatest growth in activity (separations, beddays) was in patients with CC aged 18 and over and this trend is expected to continue and grow by approximately 7% each year for both separations and beddays in the next ten years.

· In 2011/12, the level of funding under Activity Based Funding that would have been provided to NSW public hospitals for this activity was $11.4 M totaling $72.7M over the past 7 financial years. During this period, over two thirds ($52M) of the level of funding were for those patients without CC and the other third ($20.6M) for those with CC. Despite this, the average price per separation was higher for those with CC which was $17,838 compared to $13,528 for those without CC, over the period. The highest average price per separation in 2011/12 was for those aged 18+ with CC, which was $17,851.

· Projecting service utilisation and financial impact over a year 10 period found that for all CF patients, separations and beddays are projected to grow at approximately 2% p.a. over the next 10 years.

· The level of funding is projected to grow at 5.4% p.a., to approximately $18.3M in Yr 10 and total an amount of $145.3M over the 10 year period.

· The average $ per separation in 2011/12 (base year) is $14,505. This is projected to be $20,022 per separation in Yr 10.

· The level of funding for those with CC is projected to grow at 8.6% p.a., to approximately $8.5M in Yr 10, totalling $60.3M over the 10 year period. The level of funding for those without CC is projected to grow at approximately 3.2% p.a., to approximately $9.9M in Yr 10, totalling of $85M over the 10 year period.

· The average $ per separation for those with CC in 2011/12 (base year) is $17,540 this is projected to be $23,495 per separation in Yr 10. The average $ per separation for those without CC in 2011/12 (base year) is $13,168, and this is projected to be $17,765 per separation in Yr 10.

· The expected growth in the number of CF separations and beddays (Figure 3 and 4) is mainly attributed to the growth in those aged 18+ with CC, and the level of funding for this group is expected to grow at approximately10% p.a.

Approval and listing Orkambi on the PBS will positively change this trajectory.

Without affordable access to drugs like Orkambi there is little or no point investing in medical research. In Australia a young person, usually in their 20s, dies of cystic fibrosis every 9 days (2013 Cystic Fibrosis Australia Data Registry Report). The research that leads to break throughs like Orkambi provide hope for a community that has never had it before. I implore Committee to approve this application.

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