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Access to medicines: an update from Scotland

Lynsey Beswick, Public Affairs Manager at the Cystic Fibrosis Trust, gives an update on the fight for access to medicines for people with cystic fibrosis (CF) in Scotland. Read on to find out more about the Peer Approved Clinical System and an upcoming Ultra Orphan Status decision.

I want to provide an update on the access to medicines campaign in Scotland as there is a great deal going on behind the scenes.

Questions about PACs Tier Two

As many of you will be aware, the Trust met with representatives from the Medicines and Pharmacy division over the summer. This meeting was secured following a letter to the newly appointed Cabinet Secretary for Health Jeane Freeman.

At the meeting we discussed the Peer Approved Clinical System (PACs Tier Two), introduced in June 2018, which is an individual application for patient access to drugs not recommended by the Scottish Medicines Consortium (SMC).

PACs Tier Two has publicly and repeatedly been referenced as a potential access route to Orkambi for over 400 people with cystic fibrosis by the Scottish Health Minister. 

Last week we were advised that the Medicines and Pharmacy division have almost completed the process of collating data from NHS Health Boards across Scotland around the PACs Tier Two system. We are waiting for a further update on the outcome of this review, which will look at whether or not the system is being utilised by cystic fibrosis clinicians in Scotland.

We have been informed that the Medicines and Pharmacy division have recently held a series of meetings with CF clinicians in Scotland to discuss some of the possible challenges of and barriers to using the PACs Tier Two system to access Orkambi for their patients. 

The Trust understands that the uptake of the PACs Tier Two scheme has not been utilised by CF centres due to significant concerns about the system, and whether equitable access to Orkambi for eligible people with cystic fibrosis across Scotland can be really be achieved.

Ultra Orphan Status decision in the pipeline

Meanwhile, we have been told that the outcome of a decision on the consideration of Orkambi being granted Ultra Orphan Status is imminent. Usually this would only be granted to a drug with a much smaller patient cohort, but at a debate in Holyrood before the summer recess, it was announced that the government would discuss if this treatment could be considered under the new system, which was rolled out at the beginning of October.

Under the Ultra Orphan Status, this could potentially allow more flexibility in the assessment of new and high-cost drugs like Orkambi.

In the meantime, we have met with a number of MSPs at Holyrood over the autumn who are committed to keep the pressure on the Scottish government to help move things forward and have tabled a number of Parliamentary questions on this issue.

Find out more about the fight for access to precision medicines in your area, or join our campaigning mailing list for the latest updates.