All by Myself! Day 6 on Strensiq (Asfotase Alfa) and 'Metabolic Support UK Conference'
I used my new MediGenix Cool Meds case to transport the medication for the first time. It was fantastic and I found the temperature display so reassuring. A great buy and the temperature gauge was still reading 3 degrees 14 hours later. I travelled to Birmingham to the Metabolic Support UK conference, a little bit more about that later.
This was the first day I was on my own to inject the medication. I was surprised at how nervous I was. This wasn't helped by the fact that I am still using the wrong drawdown needle attachment and so was worried about it getting stuck again. In the event it all was fine of course and the new needles arrive early next week. My injection sites are becoming obvious after 3 days. There is a red patch about 2.5cm in diameter. There is a mild itch so far. One thing I am noticing though it extreme tiredness.
I was very keen to be part of the 'Metabolic Support UK' conference as much of the agenda was to do with the Access Environment and there were to be talks from NHS Commissioner Joan Ward and Sheela Upadhyaya from NICE. In the afternoon there was to be a breakout session to discuss the 'Managed Access Agreement for Asfotase Alfa - Strensiq'. Now I couldn't miss that could I?
The welcome you receive from the team at Metabolic Support is always warm. They are wonderful people who work extremely hard on behalf of all metabolic matters and conditions. The conference was no exception either. It it always a revelation to meet other adults, carers and parents of children with HPP. We are so rare that there were only 5 of us at the conference!
Through our breakout session in the afternoon it immediately became very clear that access to specialists looking after patients with HPP is not working properly. We discovered that many patients are still being referred to local hospitals and not the specialist centres. As the condition is so rare potentially the local Rheumatologist or Endocrinologist may not know about HPP. Obviously they are keen to learn about the condition but potentially this could hold up access to care. Therefore it is essential to be referred to a specialist who already looks after patients with HPP. There is a care pathway in place in the UK and to find the route speak to 'Metabolic Support UK' or 'Soft Bones UK' who will be able to advise on the closest Consultant. Local hospitals can be educated by asking the HPP specialist to send copies of letters through. This also gives patients the safety net of a local point of call in case of emergency. I travel all the way to Sheffield from Wales but copies of my letters have been sent to my specialists locally in Wrexham. Once a referral is made the consultant knows the steps to be taken for prescribing Asfotase alfa (Strensiq) through the Managed Access Agreement for treating Hypophosphatasia if patients are symptomatic. There is a wonderful fact sheet, once again provided by 'Metabolic Support UK', all about the Managed Access Agreement in the UK. See the easy to understand PDF - Managed Access Agreement for treating Hypophosphatasia.
It was fantastic to be able to speak to Sheela Upadhyaya from NICE. She was very engaged with us as a group and very keen to hear our responses. I feel that she left the meeting with extremely useful feedback from us all and that she is very keen to hear more from us whether is it about Access to Strensiq via Managed Access Agreement or on other aspects of care.
'Metabolic Support UK' were our advocate during the initial stages of getting 'Asfotase Alfa' approved via the Managed Access Agreement. They are always there to help us whatever rare metabolic condition we have. If you are not already a member please consider joining up. Their slogan is 'Your rare condition. Our common fight'. They are a registered charity in the UK since 1981 and they support patients and families, signposting to the best experts, working closely with medical professionals worldwide, raising awareness, funding research and providing much needed hope for our future. 'Metabolic Support UK' .
Make sure that anyone living with a rare or undiagnosed condition is also registered on the 'Rudy Study' as they are improving research into rare diseases. Rudy is a national network of doctors, researchers, patients and families working together to improve our understanding of rare diseases and develop new tests and treatments.