What Is Cystic Fibrosis?

A rare illness, cystic fibrosis, is caused by an unusual gene mutation. Patients suffers from thick mucus clogging the individual’s organs causing discomfort trying to digest food, declining lung capacity and performance, scarring, increased sweat chloride levels causing skin wrinkling and overall deteriorating health.

The condition is an inherited disease caused by a faulty gene and the effects of the clogging mucus typically impacts breathing as it settles in the individual’s lungs and digestive system. Impact of this life threatening disease, if remained untreated can lead sufferers to their death in their early forties. On average cystic fibrosis sufferers have a life expectancy of 38 years.

Kalydeco Drug Helping Patients Live

With the assistance of the drug Kalydeco, many of the symptoms disappear and sufferers can hope to return to leading a normal life. It is also the first medicine that has the potential to successfully target the defective gene essentially enhancing the quality of life of cystic fibrosis sufferers. With the symptoms gone, individuals suffering from cystic fibrosis can resume many of their daily life’s routines without the discomfort associated with the condition.

In Australia there are about 3000 cystic fibrosis sufferers out of which 200 are afflicted with the gene mutation and are in need of Kalydeco to help them recover their health. Kalydeco also promises to pave the way for future medicines that can prolong the life expectancy of cystic fibrosis sufferers.

What Is The Cost Of Kalydeco?

However, the life changing drug comes at an overwhelming price of $300,000 a year.

With a price tag that astronomical, many Australians cannot afford to pay for the much needed medication. The drug is needed to be taken twice a day with one single pill costing $400.

Currently the Australian government provides no subsidy for Kalydeco and all expenses for treatment need to be met with out of pocket expenses. For most patients this is virtually impossible to do so with only a handful of Australians able to procure the mediation through private health insurance.

Some have considered selling their homes to collect the funds for Kalydeco supplies while others have considered moving overseas to locations where the drug is subsidised.

Some patients have reported halving the dosage due to cost constraints.
The extremely expensive drug is considered unlikely to be subsidised by the PBAC according to Cystic Fibrosis Australia, the reason being the limit of $50,000 a year that it has for approving various drugs.

The cost of Kalydeco substantially exceeds these budget constraints and appeals have been made to the Australian health minister to think about creating an independent fund to finance expensive drugs such as Kalydeco.

Patients under six years of age and older can benefit from subsidising Kalydeco under the government’s Highly Subsidised Drugs Program. So far subsidy for Kalydeco has only been approved by PBAC with conditions with attempts at negotiating a more reasonable price for the medication are underway.

Moreover, the federal Cabinet will also have to consent to providing funding for the drug. Worldwide Kalydeco is subsidised in Scotland, Germany, UK, USA, France, Austria, Norway, and the Netherlands.

References:

1. Wonder drug Kalydeco out of reach for Ellie Haikalis’ parents: http://www.news.com.au/lifestyle/health/wonder-drug-kalydeco-out-of-reach-for-ellie-haikalis-parents/story-fneuzlbd-1226754434200

2. Cystic fibrosis ‘breakthrough’ pills have $300K price tag: http://www.cbc.ca/news/canada/nova-scotia/cystic-fibrosis-breakthrough-pills-have-300k-price-tag-1.2453058

3. Bureaucrats delay subsidy approval of cystic fibrosis drug Kalydeco: http://www.ntnews.com.au/news/national/bureaucrats-delay-subsidy-approval-of-cystic-fibrosis-drug-kalydeco/story-fnjbnxug-1226700941494

4. Kalydeo: http://www.cysticfibrosis.org.au/media/wysiwyg/CF-Australia/Advocacy/Kalydeco/Kalydeco_reco

5. Subsidy for Cystic Fibrosis kalydeco approved by pbac with conditions: http://www.news.com.au/lifestyle/health/subsidy-for-cystic-fibrosis-kalydeco-approved-by-pbac-with-conditions/story-fneuzlbd-122678768