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Ongoing delay in assessing approval of Spinraza (Nusinersen) drug for ill children completely unacceptable – Caoimhghín Ó Caoláin TD

25 January, 2019 - by Caoimhghín Ó Caoláin TD

Sinn Féin spokesperson on Disability Rights Caoimhghín Ó Caoláin TD has launched a stinging attack on the process for approving orphan drugs in Ireland and particularly relating to the ongoing delay in making a final decision on the assessment of the Spinraza drug for ill children, and others, with Spinal Muscular Atrophy (SMA).

SMA is a disease that robs people of physical strength by affecting the motor nerve cells in the spinal cord, taking away the ability to walk, eat, or breathe. It is the number one genetic cause of death for infants.

There are approximately 25 children across the State with SMA and their parents are desperately seeking access to this drug which is deemed to be potentially lifesaving in many cases.

Deputy Ó Caoláin raised the matter again in the Dáil Chamber yesterday and received a most unsatisfactory reply from Fine Gael Government Minister Jim Daly this morning (see below).

Deputy Ó Caoláin said;

“I have been constantly raising this matter on the floor of the Dáil since last September. At this stage I feel that we are being absolutely strung along.

“It is quite clear that the current process for approving orphan drugs is not fit for purpose.

“We have seen this situation with Spinraza develop in far too many times for persons with rare diseases which, in many cases, are life limiting.

"Patients seeking access to Ocrevus, Translarna, Orkambi and other such drugs are left fighting and sometimes protesting outside the gates of Leinster House while seeking access to drugs that will prolong their lives.

“The HSE argue that the cost ratio balance must be right. Of course cost is a factor. But it cannot and must not be the primary consideration. Lives are more important.

“The parents of these ill children were led to believe that a decision on the approval of Spinraza would be made in December.

"This did not happen. It was then strung out to the HSE leadership meeting which happened last Tuesday.

"Yesterday, Minister Daly in the Dáil indicated that a decision was close and possibly imminent. Disappointingly, from his reply this morning, this has now been pushed out to the next HSE leadership meeting in February.

“I am calling on the Minister to now make a direct intervention on this matter and fastrack it. There is no valid reason for prolonging.

"Parents need certainty. Once a decision is made, and hopefully the right one, we as Oireactas members need to re-examine the entire process and make it as transparent as possible and with the involvement of appropriate clinicians and parents."
Editor's note: 

Reply from Minister Daly;
The HSE has statutory responsibility for medicine pricing and reimbursement decisions, in accordance with the Health (Pricing and Supply of Medical Goods) Act 2013. The Act specifies the criteria for decisions on the reimbursement of medicines. 

Nusinersen (Spinraza) is indicated for the treatment of 5q spinal muscular atrophy (SMA), a disorder characterised by progressive muscle atrophy and weakness.

An application for Nusinersen is currently undergoing assessment by the HSE.

The statutory assessment process involves a Health Technology Assessment followed by detailed consideration by the HSE expert groups on new Drug therapies, including the Technology Review Group for Rare Diseases and the HSE Drugs Committee.

The assessment focus is twofold and particularly centred on reviewing the evidence of the clinical effectiveness of this new drug therapy i.e. the benefits for patients undergoing clinical trials; and on the cost effectiveness of the product in view of the prices being charged for the drug.

The application is currently going through the final stages of assessment, and the various reports are due to be considered by the HSE Leadership Team in February, following which the final decision will be notified.

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