SMA drug delay 'unacceptable'- Ó Caoláin


Sinn Féin TD Caoimhghín Ó Caoláin has launched a stinging attack on the process for approving so-called orphan drugs in Ireland, particularly relating delay in  assessing of the Spinraza for children with Spinal Muscular Atrophy.

Its claimed 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.

Orphan drugs are synthetic pharmaceuticals which as yet remain commercially undeveloped.
“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,” says the Cavan-Monaghan representative, who again raised the matter in the Dáil Chamber last week.

However, he described the reply from Fine Gael Government Minister Jim Daly as “most unsatisfactory”.

Minister Daly in the Dáil indicated that a decision was close and possibly imminent, and a final consideration pushed out to the next HSE leadership meeting in February.

Deputy Ó Caoláin said: “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,” added Deputy Ó Caoláin, who is calling on the Minister to now make a direct intervention on this matter and fastrack approval. “There is no valid reason for prolonging.”