T he or she NHS is to fund a very expensive treatment for an unusual but terrible childhood disorder that leaves babies ruined to life in a sterile bubble. This is a success for medical science but it should also provoke some strong and careful thought. The treatment in question, strimvelis, qualifies because the second most expensive drug ever put on the market (the just one more expensive was withdrawn due to lack of demand). A single dosage costs nearly £ 500, 000 plus VAT, and may only be administered in Milan, where the preparation is created. On the other hand, that one dose is literally life-saving, so that as far as we now know, is the only treatment the particular disorder will ever need. At the rate that the Nationwide Institute for Health and Care Excellence (Nice), is ready to pay for treatments of rare diseases – £ a hundred, 000 a year of good life – this represents value for money. The calculation may seem heartless, but it is unavoidable. Investment property on one patient is unavailable for all others. Someone, in some way, must decide who benefits.
Looked at solely as a medical advance, this is great. The babies delivered with this syndrome have two defective copies of a gene essential to the functioning of white blood cells. They may be therefore unable to defend themselves against infection and unless of course kept in wholly sterile surroundings will die of the variety of horrible diseases and developmental disorders before they will reach school age. Until now the only treatment had been with a stem cell transplant, which was only effective within about three quarters of the cases, and relied on uncovering matched donors, who are rare. The new treatment removes faulty stem cells and replaces their genes with correctly functioning versions before returning these to the patients. As soon as these are incorporated into the bone marrow, they produce healthful white blood cells and the immune system functions as it need to. This is how genetic medicine is supposed to work, but has not carried out reliably until now.
The decision to make the treatment accessible is clearly correct. But it is also irrelevant to the much deeper and more urgent problems of the NHS, which are not the result of a shortage of miracle cures for rare and spectacular diseases. It is the common ills that almost all flesh is certainly heir to that cost most when you add them upward. Sorting out social care, making things work far from the headlines: those are the measures that will relieve one of the most suffering, but compared with medical miracles, political ones are usually harder.