Amyloidosis group devastated at HSE's rejection of life-saving treatment

In what has been described as a “completely devastating decision” the HSE Drugs Group has rejected Patisiran for the treatment of hereditary transthyretin-mediated amyloidosis (hATTR).

The HSE Drugs Group is the body which decides whether or not the Government will fund medicines through the public health system.

Patisiran was considered at the organisation's January 2021 meeting, after which the All Ireland ATTR Amyloidosis Awareness group, which is campaigning for the “life-saving” drug to be made available, received an email outlining the rationale for the decision.

The email said: “The HSE Drugs Group considered in detail the clinical information in relation to Patisiran for the treatment of hATTR amyloidosis.

“While the HSE Drugs Group recognised that hATTR amyloidosis is an inherited, rare, life-limiting, multi-systemic disease, where there are limited treatment options currently available, it found itself unable, at this juncture, to recommend in favour of reimbursement of Patisiran for the treatment of hereditary transthyretin-mediated amyloidosis (hATTR amyloidosis) on its review of all criteria it had responsibility to consider.”

Speaking to Donegal Live regarding the HSE Drugs Group's rejection of Patisiran, amyloidosis awareness campaigner, Rosaline Callaghan said: “First and foremost, everyone in the All Ireland ATTR Amyloidosis Awareness group is utterly devastated at this decision.”

Rosaline who is currently undergoing treatment for amyloidosis and has lost three close family members to the disease said: “However, the thing which really baffles us is the HSE Drugs Group's assertion that once it gets the new material from Alnylam Pharmaceuticals it will be presented 'afresh to the HSE Drugs Group at a later date'.

“We are not happy about this. Why does new material have to be presented 'afresh' if there is just a final tweak? It is almost like running a jury trial, which needs to stop to allow new evidence to be admitted, and then starting the trial all over again, from the very beginning. It just would not happen. Surely, there is a more streamlined process to do this?

“Everything seems to be really slow and laborious with the HSE Drug's Group. Even on a small point, the minutes of January 2021's meeting will be published at the end of February, five weeks after the meeting took place.

“Every other country in Europe had to go through a similar process and managed to do it in half the time or less. Patisiran had to go through the whole process in the North and was approved in October 2019. So to be told in early 2021, a decision in the Republic of Ireland could be months away, is absolutely devastating to us,” said Rosaline Callaghan.

Rosaline Callaghan said people with an amyloidosis diagnosis also had great difficulty with the HSE's Corporate Pharmaceutical Unit's (CPU's) “lack of transparency”.

She added: “All of the work we are having to try and tell a Government what is actually happening is also absolutely exhausting. Most of us are patients and most of us are ill.

“The applicant company, Alnylam Pharmaceuticals, has told us it will have the necessary information with the CPU in the matter of weeks. If that is to be the case, why is it going to take months for Patisiran to be approved. We would call of the CPU to make sure this is time-tabled in March.

“We thank the public for its support. We are calling on everyone in Donegal to contact their local politician as a matter of urgency, to register their objection to the delay in providing Patisiran to people with an amyloidosis diagnosis.

“We desperately need the public's help on this. In different times we would be organising public action to highlight this issue but we are conscious now of the Covid-19 restrictions and wish to be respectful of these. We ask the public to contact all elected representatives and advocate on our behalf,” said Rosaline Callaghan.