Yesterday, the first of 17 patients who had, until recently, been receiving the drug Respreeza was laid to rest.
The patients, who have a progressive disease known as Alpha-1, are in their third week of not having access to Respreeza, because neither its manufacturer, CSL Behring, nor the HSE, is willing to pay distribution and administration costs, estimated at about €120,000 per annum.
It means the drug, which is meant to be infused weekly, is sitting in a warehouse while patients forego therapy. A meeting took place last Thursday between the HSE and CSL Behring in an effort to end the standoff, but neither side has reported any progress.
However correspondence seen by the Irish Examiner suggests neither the HSE nor CSL Behring put forward solutions to break the impasse. In a letter to patients, their respiratory consultant, Gerard McElvaney, said it was “basically a reiteration of the positions of the HSE and CSL”. He said the HSE repeated that they will not reimburse the administration costs, which until recently had been borne by the company.
He said the HSE was “unhappy about directly delivering the drugs to our patients in their hospitals or clinics”. Furthermore, CSL said while they would continue to supply Respreeza free of charge until June, when they are due to commence another clinical trial, potentially involving a number of the Irish Alpha 1 patients, they would not pay for the administration cost, “which they felt was exorbitant”.
The cost of the drug itself is around €84,000 per patient per annum, but is currently free of charge to patients who took part in a clinical trial and who subsequently had access under a compassionate use programme.
Prof McElvaney put forward some solutions at the meeting, which he was not invited to, but was eventually permitted to do so after lobbying by the Alpha One Foundation. His presence at the meeting was to represent his patients’ interests.
He told patients that he “asked the HSE for solutions and offered suggestions” and they told him they would come back to him this week.
“I reiterated that time was of the essence and we needed a decision quickly and they said they would try to accommodate that,” said Prof McElvaney.
He said the possibility that Alpha-1 patients might have to forego six months of free therapy (until the next trial) because of the administration stand-off was “absolutely not acceptable”, especially as some of his patients were “at significant risk of hospitalisations”.
Prof McElvaney, who is based at Beaumont Hospital and is Prof of Medicine at the RCSI, also expressed concern that not all 16 of his patients who had access to Respreeza would continue to do so as part of the new CSL trial.
“I will try my best to get as many as possible on the trial but my hands will be tied by the inclusion/exclusion criteria,” he said.
Alpha-1 antitrypsin is a protein whose main job is to protect the lungs from infection. Deficiency of this protein can lead to severe lung, liver and skin problems.
Respreeza works by replacing the protein that is lacking and is the only therapy shown to slow the progress of genetic emphysema. The State has refused to reimburse it on cost grounds.