A new treatment for children with Duchenne muscular dystrophy will not be made available in the Republic, the Health Service Executive has decided.
Muscular Dystrophy Ireland (MDI) says the HSE has informed it that the treatment, Translarna, will not be approved for reimbursement under its drugs schemes.
This is in spite of the fact that the drug is available in Northern Ireland and 22 European countries.
Families of children with Duchenne’s have been campaigning for the past two years for it to be approved.
Translarna is used to treat boys with a particular type of Duchenne’s, whose condition is caused by a particular genetic defect and who are aged five years and older and able to walk.
It is estimated about five boys a year would have been eligible for the drug if it had been approved.
MDI described the news as very disappointing. "It's like being hit by a brick wall. We've been round the houses on this over the past few years," said Lisa Fenwick, a family support worker with the charity.
Last year, the National Centre for Pharmacoeconomics (NCPE) found the drug would not be cost effective, given the annual cost of about €411,000 a patient. The NCPE estimated the five-year cost of the drug at €5.8 million.
The HSE subsequently entered into talks with the manufacturer, PTC Therapeutics International Ltd, but these failed to result in a price acceptable to the State health body.
Genetic cause
Translarna, which is approved by the European Medicines Agency, is the first drug to deal with the underlying genetic cause of this type of the disease, which causes muscle degeneration.
In a statement on Tuesday, the HSE confirmed it “has written to the manufacturer of Ataluren [Translarna] advising them that the HSE will not be funding this drug”.
“The HSE is very much aware that this decision is upsetting to patients who are affected by this condition. It will also be a disappointment to their families and the treating clinicians who support these patients. However, it is of the utmost importance to recognise that the HSE Drugs Group who reviewed the effectiveness of the drug did not consider the evidence for the clinical benefit of Ataluren [Translarna] to be sufficiently strong in the context of the proposed cost and budget impact.
“There is an onus on the HSE to ensure that any new drug is cost effective. New drugs can provide improvements in quality of life, but may not provide a cure. Nevertheless, the prices sought by drug companies for such products can run to hundreds of thousands of euro per patient per annum. The HSE must have regard to its wider obligations to the 4.7 million population it serves and needs to maintain the full range of health services to all of the other patient groups within the finite resources at its disposal.”
Approvals
Last week, the HSE approved nine new treatments for other conditions following months of controversy over delays in the approval process. The health service lacks money to fund the high cost of new treatments, particularly after the Government approved new cystic fibrosis drugs costing over €700 million earlier this year.
Duchenne muscular dystrophy is one of more than 30 types of muscular dystrophy and affects about one in 3,500 male births.
MDI said the treatment would make a huge difference in slowing the progression of the condition and in keeping young patients on their feet for longer and out of a wheelchair.
