Access to medicines for rare diseases
O'Sullivan welcomes a pilot scheme to improve early access to new medicines, noting long waits for rare-disease patients. Carroll MacNeill says delays can depend on pharmaceutical companies submitting documentation and points to recent approvals.
I welcome a line in this year’s budget that got very little attention, namely, the pilot scheme to support early access to new medicines. For the 300,000 people in this country living with a rare disease, that budget line is incredibly important.
As the Minister knows, Irish patients still wait longer than most of our European counterparts for access to these medicines. It is often in excess of 800 days. For a person with Duchenne muscular dystrophy or Friedreich's ataxia, those 800 days or so are time they do not have. This summer, we saw Skyclarys approved for Friedreich's ataxia after a long and exhausting campaign, so I welcome the early access scheme that the Department is devising. How much has been allocated to the pilot scheme? Which medicines and conditions will it cover? There are rumours that it might exclusively be aimed at cancer, so I ask that the Minister clarify. When can we expect the first patients to benefit from the scheme?
Comment on this
This year is evidence that we have prioritised drugs for rare diseases and the speed with which they are approved. What we cannot control is the conduct or participation of the drugs companies. The Deputy will be aware, particularly in relation to Skyclarys, that we were waiting for an extremely long time for the drugs company to submit the different pieces of documentation, including its commercial proposal. I am very glad that it got resolved in a way that was cost effective and, therefore, possible for the State. There are patients now getting that medication, as there are patients getting givinostat. Indeed, I have met many who have now got it. We have been able to execute getting that to patients very quickly.
We will continue with that focus but we are not going to prejudge what those applications may be or what those medicines that come to the Irish market will be. As the Deputy is aware, we had to convince and invite Italfarmaco to bring givinostat to Ireland because, as a small market, we compete in terms of attractiveness. We had to work very hard to even have the application made.