A US drugs firm has begun legal action against the Health Service Executive for refusing to fund the treatment of two young boys with a rare genetic condition, Duchenne muscular dystrophy.
The case taken by PTC Therapeutics is believed to be the first time a mechanism that allows companies to appeal drug funding decisions to the High Court has been used.
The company says it is taking the unprecedented action because the window of opportunity for treating the boys is fast running out. The therapy, Translarna, can only be given to children while they are still able to walk.
The drug is available in Northern Ireland and 22 European countries but was rejected by the HSE a month ago because of concerns over cost and clinical effectiveness.
"We feel we have no option on behalf of the two boys but to appeal the HSE's decision," said Adrian Haigh, the company's European director. The legal action will cost more than any revenue that can be expected from the product this year, he pointed out. It may also cost the HSE more than it would have to pay for the drug.
Translarna is used to treat boys with a particular type of Duchenne’s, whose condition is caused by a particular genetic defect, who are aged five years and older and are able to walk.
Approved by the European Medicines Agency (EMA), it is the first drug to deal with the underlying genetic cause of this type of the disease, which causes muscle degeneration.
Dr Declan O’Rourke, consultant paediatric neurologist at Temple Street children’s hospital, said the drug keeps patients on their feet longer and slows the progress of the disease.
“The families involved are disappointed and distressed that it is not available. For them, it is a very time-sensitive matter. Once their boy can no longer walk, he cannot avail of the treatment.”
An analysis for the HSE last year found the drug would not be cost effective, at an annual cost of about €411,000 per patient. Subsequent talks between the company and the HSE failed to result in an agreement.
The company believes the process of considering the drug was flawed, with little engagement by the HSE with doctors and patients as happens in other countries. It also says the HSE was wrong to rely on the fact that Translarna has not yet been approved by the Food and Drug Administration in the US, when the relevant regulatory body is the EMA.
Mr Haigh declined to say how much the company proposed to charge the HSE but he said it had offered a “confidential discount” in negotiations. The number of treatable children in Ireland will rise to five by 2019.
PTC runs its international operations out of a base in Dublin, where it plans to increase staffing from 24 to 40. Mr Haigh said the refusal for Translarna here would not affect these plans but “it is not a positive signal for other companies wanting to come here”.
The HSE said its drugs group did not consider the evidence for the clinical benefit of Translarna to be sufficiently strong in the context of the proposed cost and budget impact.
