FUNDING FOR TREATMENT OF RARE DISEASES
The Somerset Guardian, 26th June 2019
Some readers may have seen the sad case of my constituent Max Sewart (pictured with his family) on the local news. Max is eight years old and has Batten Disease, a rare, fatal disease of the nervous system that typically begins in childhood. Without treatment, those with Batten Disease eventually become bedridden, require twenty four-hour care and die prematurely.
Whilst there is no cure, a drug called Cerliponase Alfa has recently been developed that slows the onset of the disease. It is available in twenty other countries, including Germany and France. However, the National Institute for Health and Care Excellence (NICE) is currently refusing to fund the treatment on the NHS, saying that although there are substantial short-term benefits it does not represent good value for money.
Undoubtedly, the drug is expensive and the decision-makers at NICE have an unenviable task. Nevertheless, the question remains: how can a price be put on a child’s life?
This situation is, regrettably, not unusual. I have also been trying to help another constituent, Jake, who has spinal muscular atrophy (SMA). A month ago, it seemed that funding for treatment had been granted but it now transpires that there are several limitations meaning that Jake will not get the drug that he desperately needs. An appeal is underway.
Changes were made in 2017, making it harder for drugs for rare diseases to be funded. It has become increasingly clear that these changes need to be reviewed urgently in order to allow fair access to effective treatments for all those affected by rare diseases in the United Kingdom.