The Wonder Drug!!

A New Start?

So about 2 months ago, on 12th Januay 2018, I was an inpatient at The Royal Papworth Hospital, Cambridgeshire, England. I sat in my room with my consultant, Dr Helen Barker and a specialist nurse, Lyndsey. In front of me was the orange Patient Information Pack for a brand new drug. I was about to sign the consent forms for me to start this AMAZING new treatment. I was scared. I was worried. I had some questions. Who wouldn't feel like that, right?

Let me tell you about:

ORKAMBI

So What Is It?

Orkambi (ivacaftor/lumacafter) is the second of drugs like this to be licensed for use in people with Cystic Fibrosis. Although it is not currently provided by the NHS, you can only get the medicine on 'compassionate grounds'.

• Orkambi treats the DeltaF508 mutation, which around 50% of people with CF in the UK have.
• Data has shown that Orkambi can slow the decline in lung function by 42%!
• Orkambi is a combination medicine, made up of ivacaftor and lumacaftor. Lumacaftor helps get more proteins to the surface of cells in the body, and ivacaftor helps the chloride channels in the cells to operate more effectively. The combination of these two things helps to keep a healthy balance of salt and water in the organs – particularly the lungs. Orkambi is manufactured by a company called Vertex Pharmaceuticals.

Why Isn't It Available On The NHS?

• According to the 2014 UK CF Registry Report, there are around 3,000 people across England, Scotland, Wales Northern Ireland who could benefit from the use of Orkambi.
• Orkambi has been licensed for use in the UK for people with CF over the age of 12 who have two copies of the F508del mutation. The National Institute for Health and Care Excellence (NICE) has rejected its use on the NHS on grounds of cost-effectiveness and a lack of long-term data. Although they do recognised Orkambi as an important treatment.
• Orkambi is currently only prescribed to patients who fulfill a number of criteria.

5th March 2018

I am currently waiting to be sent to Addenbrookes Hospital, to have a PICC (Peripherally Inserted Central Catheter)  line put in. I will then be admitted to The Royal Papworth Hospital again by the end of this week. There I will start a course of Intravenous Antibiotics... 

Towards the end of the course I will start taking the Orkambi. It really does scare me. Without a single bit of doubt, I WILL feel absolutely awful. I will be more breathless. I will be tight chested. I'm going to feel so worn out.

BUT! If I can get through the first little bit, then this medication will almost certainly make my life a bit better!

I'll put up a new post when I am in the hospital!

K💖