New Medicines in Scotland

On Monday, Cayston, a new nebulised treatment to tackle Pseudomonas in people cystic fibrosis was approved for use in Scotland. Here, Yvonne Hughes, Public Affairs Officer for Scotland tells us more about how such decisions happen.

The decision this week by the Scottish Medicines Consortium (SMC) to approve another drug for cystic fibrosis underlines how the work the Cystic Fibrosis Trust does here in Scotland is making an impact. As Public Affairs Officer, ensuring access to new medicines is a part of my job that may appear a bit boring at first, but is an essential and enjoyable part of my work and allows the Trust to make the representation on behalf of the CF community.

With the help of clinical CF staff across Scotland, I am able to contact patients who are on the trial for the new drugs being assessed. It is crucial that the patient experience and that of their families is captured in papers we submit to the SMC as it could be the difference between a Yes and a No recommendation. Without your help drugs like Kalydeco and Cayston may never have got to clinics, so I am extremely grateful. I have also made a few friends in the process! As someone with cystic fibrosis I understand how isolating it is, so to speak to you and hear your stories is always a pleasure.

The recent Cayston decision fell under changes made last year to the way medicines are appraised to improve access for those at end-of-life or with a rare disease. A Patient and Clinician Engagement meeting (PACE) has been introduced where it is likely a drug may not be approved. Cayston went through this process and we found it to be valuable, particularly as we were able to talk about living with cystic fibrosis direct to people involved in making decisions around new medicines.

The changes to the SMC will be up for review this year and I shall feed in to that process but in the meantime I will continue to ensure that patients with cystic fibrosis are represented when new drugs come online. Please feel free to leave a comment or get in touch with me yvonne.hughes@cysticfibrosis.org.uk.