The 2012 Patient Keynote at the Partnerships in Clinical Trials event was Emily Schaller. She delivered a moving speech on how her participation in the clinical trial for Kalydeco has, for the first time, made a difference in her condition and is allowing her to live life to her fullest. Check out her contribution to the blog she contributed before the event.
Recently at Scripp Intelligence, Lucie Ellis looks at the current situation in Australia, where the drug has been blocked, leaving those suffering from the disease with no way to access the life changing medicine. The Pharmaceutical Benefits Advisory Committee, in charge of recommending drugs for the nation's healthcare, has asked Vertex Pharmaceuticals to submit another price proposal. At it's current price, the PBAC believes that the Quality Adjusted Life Year is is too high and uncertain.
Here is an excerpt from the article about why Kalydeco is so important to those with cystic fibrosis:
According to Vertex, Kalydeco is the first medicine to treat the underlying cause of cystic fibrosis in eligible patients, those with the G551D mutation in the cystic fibrosis trans-membrane conductance regulator (CFTR) gene. It was approved by Australia's Therapeutic Goods Administration in July 2013. Australian approval and reimbursement of a new medicine is a multi-step process. Once a new medicine receives approval from the TGA, it is assessed for effectiveness and cost-effectiveness by the PBAC for listing on the PBS.
In a 28 August statement Cambridge, Massachusetts-based Vertex said it "strongly supports the need for a rigorous and transparent process for the assessment of Kalydeco and is encouraged by discussions that have taken place with the PBAC to date."
But CFA said a resubmission from the company, which has been requested by the PBAC, might not be reviewed until its November 2013 or March 2014 meeting. However, it said it "would not tolerate delaying tactics and will be doing everything in its power to expedite a positive outcome for the cystic fibrosis community." CFA added that this is by no means the end of the road for subsidized access to Kalydeco in Australia.
Vertex met with the PBAC on 28 August to "get more information on the deferral," the company said. "We are encouraged by our discussion, and we remain committed to working with the PBAC to make Kalydeco available to eligible Australians as quickly as possible."
Vertex told Scrip that while discussions with the PBAC were ongoing, Kalydeco is currently available free of charge in Australia via a Patient Access Program (PAP). The PAP was set up by Vertex for those with severe disease and for whom treatment options are limited. It also provides the medicine to patients who participated in Kalydeco Phase III clinical trials.