Australian patients mobilise to fight Kalydeco block


Cystic fibrosis (CF) patients in Australia are becoming increasingly frustrated - and vocal - as negotiations for access to Vertex' new therapy Kalydeco drag on.

A social media campaign (#YesToKalydeco) is being waged to try to persuade Australia's Health Minister Peter Dutton to agree a deal with Vertex so that the 200-or-so patients in the country who are eligible for treatment with the drug can receive it.

Kalydeco (ivacaftor) has been approved in Australia since July 2013 and was the first drug to reach the market that targets one of the underlying mutations in the CF transmembrane regulator (CFTR) gene, rather than the symptoms of the disease. Prior to Kalydeco the only pharmacological treatment options for CF patients were enzyme therapy to thin down the mucus that clogs the lungs of patients and antibiotics to tackle lung infections.

The Pharmaceutical Benefits Advisory Committee (PBAC) - a government-appointed body in charge of vetting treatments for reimbursement - recommended shortly after approval that Kalydeco should be included in the state Pharmaceutical Benefits Scheme (PBS). However, it baulked at the A$300,000-a-year Vertex was asking for Kalydeco and deferred a decision on subsidisation, leaving patients with the G551D mutation in limbo.

The PBAC finally recommended in April that the cost of Kalydeco should be 100 per cent covered for patients, but only if they fully respond to treatment. If the patient does not respond at all the cost of the drug should be repaid by the sponsor, while for partial responders only a portion of the total cost would be subsidised, said the panel.

Ahead of the weekend, Vertex published an update on negotiations, saying it is now offering Kalydeco to the Australian government at a price that is "equivalent to the best in the world".

The April recommendation by the PBAC "includes conditions that would impose strict eligibility criteria that exclude the sickest patients from being treated, and discontinuation criteria that would result in patients experiencing benefits being taken off treatment," said the firm in a statement.

The onus is now on the Australian Department of Health to reach a decision on Kalydeco, and the CF community is pulling no punches as it tries to mobilise public support behind the access campaign.

A flood of tweets to Dutton and Australian Prime Minister Tony Abbott - along with Vertex itself - accuses them of abandoning patients to their disease while G551D-positive CF patients in other countries have had access to the drug for months.

"USA did it, England, Ireland, Scotland & Wales did it, Germany & France did it. When is it Australia's turn?" asked Naomi Nichols in a tweet posted last week.

Patients and their families are incensed that they are still waiting for Kalydeco even as Europe is on the brink of approving use of the drug in a broader range of CF patients, following the lead of the US, which backed it for eight additional mutations in February.

The broader use could drive sales from $371m last year to around $500m in 2014, according to analysts at Zacks.

Meanwhile, CF patient and Kalydeco access campaigner Alex Parker, who has been able to get back onto therapy with the drug thanks to being enrolled into a clinical trial, is in no doubt that Kalydeco - known in the CF community as 'K' - has the ability to transform lives.

"My Kalydeco supply ran out in October last year and my health had begun to gradually fall back into its old ways," she wrote last week. "I've now been back on K for 3.5 weeks and I'm a new person again."

"Over the past couple of months I have felt extremely overwhelmed by the Kalydeco campaign, especially when I was feeling so unwell. I do feel a great sense of responsibility to speak on behalf of so many people in desperate need for this miracle medication, but sometimes I have to take a step back and focus on maintaining my own health.

"Now that my health is getting back on track I feel stronger physically and mentally and I will continue to fight for those that so desperately need access to Kalydeco," says Parker.

Vertex said it was continuing to meet with the Department of Health in Australia and is 'working to reach an agreement as quickly as possible'.


Wider use of Kalydeco recommended in Europe

Advances in our understanding of Cystic Fibrosis: Interview with Dr Katrine Whiteson



Linda Banks

7 July, 2014