Life-saving amyloidosis treatment not on Government agenda

Patisiran, the proven life-saving amyloidosis treatment, has been left off the agenda of the National Drugs Group by the HSE's Corporate Pharmaceutical Unit (CPU), despite the efforts of the All Ireland ATTR Amyloidosis Awareness group.

Speaking to Donegal Live, amyloidosis advocate, Rosaline Callaghan, who has been diagnosed with the illness said she was “completely frustrated and driven to distraction” by the CPU's “lack of transparency and accountability”.

Rosaline, who is originally from Inishowen and has lost her father, aunt and cousin to amyloidosis, said: “People may have become aware of the dire situation of Pat Tinsley from Newbridge in County Kildare through the national media. Pat has been diagnosed with amyloidosis and he is one of the reasons why the All Ireland ATTR Amyloidosis Awareness group have been pushing the Patisiran issue so hard. Pat is seriously unwell.

“It took us quite a while to get the HSE Corporate Pharmaceutical Unit (CPU) to engage with us. A few months ago, the CPU issued a statement to say it was talking with amyloidosis patients as part of its consideration of Patisiran. I responded to that claim by saying, the All Ireland ATTR Amyloidosis Awareness group comprises 175 people and CPU has not spoken to one of us.

“I sent the group letter to the CPU to say, we have 175 people here and no-one has spoken to us. In fairness to CPU, it came back and asked us to submit a patient submission. Within 14 days, the All Ireland ATTR Amyloidosis Awareness group sent the CUP a considerably-sized submission. We had collated information from patients. CPU then agreed to speak to Pat Tinsley and his brother, Brian, by Zoom.

“A number of weeks ago CPU informed us it had received the submission and we would hear from it 'next week'. I thought, 'brilliant, brilliant, we are getting a bit of movement'. However, we heard nothing at all from CPU and then, last Friday, in the afternoon, we heard on the grapevine, that the CPU recommendation on Patisiran was now with the National Drugs Group, who say 'yes or no'. The National Drugs Group met yesterday (Tuesday) and, devastatingly, Patisiran was not on its agenda,” said Rosaline.

Rosaline said she was “completely frustrated and driven to distraction” by the CPU's “lack of transparency and accountability”.

She added: “Over the past weekend, we asked All Ireland ATTR Amyloidosis Awareness group to contact as many TDs as possible and beg them to put Patisiran on the National Drugs Group's agenda.

“The important thing to say is, we understand that other people are waiting on other drugs. We do not think that we are any more special than anyone else but the nature of amyloidosis is the progression and it is a rapid progression. There is such a sense of urgency. We tried absolutely everything we could think off. And then we heard on Monday evening, patisiran was not going to be on Tuesday's agenda.

“It is absolutely desperate. The other 20 patients potential patisiran candidates and Pat Tinsley is beyond urgent. What is most frustrating is that the CUP would not keep a patient group of our number updated or advised about what is happening. Are we just shouting into the wind here? Is anyone really listening?

“It was so disrespectful, so discourteous. Many in our group are patients. We are trying to do this work while coping with our own not insignificant health difficulties and A one line email to keep us up to date would have been acceptable and welcome. All we can do is press everybody we can thing off to make sure that Patisiran is on the first agenda of the New Year 2021,” said Rosaline.

The patisiran application in the Republic of Ireland is now two years old.

Rosaline added: “Every other country in Europe and Britain has approved patisiran for the treatment of amyloidosis, in half that time or less, in spite of having to go through the same stringent testing regime as Republic of Ireland. We are just about to go into the third year of the application.

“We are asking everyone who can to lobby their local TD or a TD of choice to keep the pressure on. In reality, Pat Tinsley needs patisiran as a matter of urgency. He has waited for two and a half years since his diagnosis with hATTR Amyloidosis, to learn that the HSE Corporate Pharmaceutical Unit (CPU) did not include Amyloidosis and the purchasing of Patisiran at next week’s scheduled meeting of the HSE Drugs Group.

“To say Pat and similar patients who live with the degenerative and debilitating illness are gutted, is an understatement,” said Rosaline.

Pat Tinsley said: “My brother and I were asked to participate in a Zoom call with the rare disease committee. We had an intensive discussion, and relayed the horrendous quality of life I exist with, from bed to bathroom, some days I sleep up to 21 hours, and when I’m up, I’m dizzy, vomiting, generally weak and miserable.

“We know that the producer of patisiran has made several discussions with the HSE in the past two years but the HSE still has not made a decision regarding the reimbursements, despite knowing that I and circa 30 other people live with this dreadful illness.

“The rare disease committee compiled a detailed case of my medical history in recent years including the appalling lack of services, the lack of any continuity for me and other patients. I will die without the drug and now it’s not even on the agenda?

“Please Minister Donnelly, get this back on the agenda! Are none of the hATTR Amyloidosis sufferers in Ireland entitled to any consideration here?” said Pat Tinsley.

hATTR amyloidosis (or familial Amyloidosis) is a genetic chronic disease that affects multiple organs and body systems, such as the heart, nervous system, gastrointestinal tract, and kidneys. As the disease progresses, it causes loss of sensation, limb weakness and pain plus alternating episodes of diarrhoea, constipation, nausea, and vomiting.