A Mullingar man is to walk from his home to Leinster House this week in protest at the refusal of the Government and the HSE to treat his wife with one of the world’s most expensive drugs.
Declan Connolly, whose wife Charlotte is the only person in Ireland with a rare blood vessel condition known as Degos disease, leaves the Co Westmeath town on Monday and hopes to reach Leinster House at 4pm on Tuesday.
He is undertaking the 84km march after failing to make progress in a two-year campaign to allow his wife be treated on an experimental basis with eculizumab, which is licensed in Ireland for the treatment of other blood diseases.
Degos is a disease of the blood vessels in which cells in the linings of the walls of the small veins and arteries under the skin swell when they become inflamed. This causes restricted blood flow. In some cases, blood vessels in the gut, central nervous system or eyes are affected, and skin lesions appear.
There is no known cure for the disease, and Ms Connolly has the more serious systemic version, which affects the internal organs and has a high mortality rate. However, patients in the US have been treated on an experimental basis with eculizumab, known commercially as Soliris, and the condition of some has improved.
Soliris, which costs up to €430,000 per patient annually, was approved by then minister for health Leo Varadkar and the HSE in 2015 to treat patients with two unrelated blood diseases. This was in spite of the HSE saying the cost was exorbitant and Mr Varadkar accusing the manufacturer, Alexion Pharmaceuticals, of aggressive pricing.
"To say that we are disappointed with the support of the HSE and Minister [Minister for Health] Simon Harris is an understatement," Mr Connolly said. "The Minister has consistently refused to meet with me or to sanction the use of this drug in Charlotte's case. It is appalling that we have had to go to such lengths in order to access this drug."
Soliris is not licensed for treating Degos disease but such off-licence use of a drug is not unknown, when approved by doctors treating the patient.
However, doctors at St James’s Hospital have questioned the scientific justification for using the drug in Ms Connolly’s case.
Mr Harris has said existing arrangements for the provision of Soliris do not permit the drug to be used for the treatment of a condition “beyond the terms of the marketing authorisation and where evidence of clinical benefit has not been demonstrated”.
Ms Connolly began to suffer numbness in her lower body, loss of weight and poor appetite four years ago. Doctors initially thought she had multiple sclerosis but this was ruled out after tests. Eventually, Degos disease was diagnosed.
