Thalassaemia patients are hoping they will soon get access to a new drug that will radically change their life after a tortuous four-year wait riddled with bureaucratic hitches.

The drug, Deferasirox, accepted for use in the EU in 2006, replaces a gruelling treatment which sees thalassaemia patients, who suffer from chronic anaemia, being stuck to a drip all night through a needle in their stomach, with a few pills a day.

Finally, a public tender for the purchase of 500mg doses has almost gone through all the stages, which means the treatment could be available within six weeks.

However, the journey has not been smooth.

A call for tenders for the drug was issued early in 2010 after the government decided to include Deferasirox in the free list of medicines. But the bid fell through over an issue of price. The Health Department said that when Deferasirox was approved as a national health service drug, the government established a maximum price it was willing to pay for the two formulations of the drug, in 250mg and 500mg doses. The health authorities are bound to stick within this price when issuing a call for tenders. However, the agent selling the drug in Malta mad a bid above the reference price and the tender fell through, dashing the hopes of patients whose lives would have changed radically with the introduction of the new drug.

Earlier this month, a new tender was issued for Deferasirox in 500mg and 250mg doses. Again, the bid for the 250mg dose exceeded the reference price but the 500mg tender went through and was now in the process of being awarded, the director general for health care services, John Cachia, said.

If things proceeded smoothly, the 500mg dose should be available within six to eight weeks, he said.

Effectively, this would mean the end of the needle treatment for patients because the 500mg drug will do the job as tablets can be split in half and most people would need the larger dose anyway, according to molecular geneticist Christian Scerri.

“The drug will change the patient’s life dramatically,” Prof. Scerri said, adding it would also increase patient compliance, reduce side effects from the infusion and, therefore, lessen the need for hospitalisation for adverse reactions.

However, Labour health spokesman Michael Farrugia argued the wait was still unacceptable. The government should find an interim measure for the patients to have this medication until the tender issue was cleared, he insisted.

“I cannot understand how the patient can be left without the drug until the government debates the price,” Dr Farrugia said, pointing out this was not only a problem for Deferasirox.

Earlier this month, Dr Farrugia had raised in Parliament the issue of sensitive situations that existed in the health sector, giving the shortage of angioplasty stents that occurred earlier in the year as an example. He called on the Finance Ministry to recognise the importance of things related to health, saying he would back the Health Minister if he were to breach the government’s financial regulations for the benefit of patients.

Thalassaemia is an inherited blood disorder where there is abnormal formation of haemoglobin in the oxygen carrying red blood cells, causing anaemia, the typical presenting symptom of the disease.

The drug is needed because many thalassaemia sufferers require repeated blood transfusions due to the anaemia, which, in turn, raises the amount of iron in their blood. The iron toxicity’s elimination is aided by the drug, which binds to the iron, enabling it to be excreted.

In Malta, there are about 30 patients diagnosed with thalassaemia, with ages ranging from two years to over 60.

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