Paroxysmal Nocturnal Haemoglobinuria (PNH) is a very rare and life-threatening blood disorder where the patient’s red blood cells are destroyed, leading to anaemia, fatigue and increased risk of blood clots. An estimated one-third of patients do not survive for more than five years after diagnosis.
Following a request from The Irish Times, the Health Services Executive provided the following statement about the PNH drug Eculizimab, which reportedly costs about €350,000 a year: “Following consideration of Eculizimab by the HSE’s Products committee in 2007, it was decided that in view of the particular nature and use of this produce, it is appropriate for hospital use only.
“The decision to use this product is a clinical one. The provision of this drug to patients in hospitals is currently a matter for decision by the hospital concerned.”
However, the cost of “orphan” drugs to treat such rare conditions puts them beyond the scope of most, responds Avril Daly, chairwoman of the patient group the Genetic and Rare Disorders Organisation.
“The success of clinical trials for Eculizimab involving Irish patients at St James’s Hospital, Dublin has resulted in the continuation of its provision post-trial at the drug company’s expense. It is unfair that patients who were diagnosed too late to participate in this trial have no access to a treatment that could ultimately save their lives.
“This fact that people’s lives are at stake here seems to be forgotten. As conditions like PNH are so rare and the treatment costly, it is impossible for smaller hospitals with limited budgets to accommodate the expense, particularly when their patients are being referred to national centres.
“We hope that the costs of this and other therapies for rare disease can be captured by the establishment of a national fund for high technology and orphan drugs in the shortest possible time frame.”
The cost of treatments also needs to be considered in the light of savings in the long run, she adds.
“When a drug can improve the quality of life for a person affected by a rare disorder, whether it is classed as a containment of that condition or a cure for it, the consequences of not administering it will in itself result in increased costs.
“Without such treatments, patients can not fully participate in society and therefore contribute – they will require sustained services on a regular basis over a prolonged period.
“This makes it difficult for us as patient representatives to see potential savings.”