Please click on the link above and sign this petitician and share with your friends to sign too.
I do not normally do this, but Sam's situation is different to many asking the NHS to fund drugs.
Why? Because Sam has been on a trial for 2.5 years and so the benefits to him have have been measured and quantified. The drug has now been licensed, which is testament that it is beneficial to children with Morquio Syndrome. The drug has to go to a panel for funding to be agreed for Sam to continue having the drug. The petition is about getting the score card system changed with regards how new drugs are scored in relation to those affected by rare diseases, child or adult. With the current scoring system it seems unlikely that drugs for those with rare diseases will receive funding. Signing the petition raises the profile of this situation and hopefully will reverse the scoring system, so all diseases needing new drugs to limit the effects, will get a fare crack at being funded.
Sam is age 6. He has a rare life limiting disease called Morquio Syndrome, which is a type of MPS - a group of ultra-rare genetically inherited diseases that affect children's development in different ways. Neither Katy nor Simon knew they were carriers of the gene that causes the disease, until they started having tests to get to the bottom of Sam's physical development delays, when Sam was a toddler.
There are only 105 sufferers in the UK. the disease causes progressive physical disability, shortness of stature and significant health problems in adult life often leading to a significant shortening of life expectancy and reduction in quality of life. Sam has been receiving the drug Vimizim (elosulfasealfa) via a clinical trial of treatment at Royal Manchester Children’s Hospital for two and a half years. The drug received licensing approval in the EU in April 2014 – meaning that it is approved as ethical, safe, and that it delivers the proven clinical benefits. A separate decision will be made on the 16th December as to whether the NHS will be willing to fund the drug in the UK. If it is not funded, Sam’s treatment would stop unless the drug company approved a short extension of treatment on ethical grounds whilst it fought that decision. The drug is a man made enzyme, replacing the one Sam doesn’t have. Its aim is to slow, and in the best case stop the progression of the disease. However pre-existing impacts of the disease cannot be reversed ... meaning that if treatment stopped even for a short time, there would be irreparable consequences. The drug is having a significantly positive impact on Sam – he has grown, his posture and stature is better, and physically his abilities have not deteriorated at all, in fact they have improved. Health wise he has been less prone to chest infections, and his hearing has been normal for two years. Sam not receiving the drug is unthinkable. The consequences are unbearable.
Sam could end up immobile within a few years, which would mean he would need significant services involved with managing his care needs. The drug Vimizim could hold the degenerative effects of the disease for many years, giving Sam an increased quality of life that previously could only be dreamt about. Vimizim gives Sam hope for a brighter future.
Being part of the trial has been a huge huge commitment, Sam misses a day a week of school and it causes significant family upheaval. If it didn't work and give Sam significant benefits to the quality of his life, the upheaval would not be worth it.
Thank you for reading this x