Finlay Hesketh is a young boy with Duchenne muscular dystrophy (DMD), a cruel muscle-wasting illness that has few treatment options and which sees sufferers becoming wheelchair dependent by the age of 12. Later, they will need artificial ventilation to help them breathe and death usually follows before the age of 30. It’s horrific.
A new treatment is available – ataluren – which has been described by NICE as a “step change” in care that can delay the loss of the ability to walk for up to seven years, offering children the chance of a normal adolescence and allowing them to stay in school for much longer. In addition to delaying disease progression, the drug can lead to improvements in a child’s mobility such as being able to get out of bed without help and going to school independently.
It is, however, extremely expensive. Nonetheless, NICE has recommended it should be available on the NHS for children over five with DMD who are still able to walk. The recommendation comes with a number of conditions: it will run for only five years, during which time a (confidential) financial agreement will operate with the manufacturer, who will supply the drug under a managed access agreement while continuing to collect data on the drug’s efficacy.
The guidance will then be reviewed. It seems an eminently sensible and reasonable approach yet – in Finlay’s case – the Manx Government has refused funding because, according to them, the evidence for clinical effectiveness is currently “inadequate” to justify the cost. It’s a cruel decision.
NICE (which has much more expertise than the Manx Government in evaluating the benefits of a new treatment) is sufficiently satisfied with the clinical evidence to recommend the drug’s use. So it appears that the Manx government is using the question of effectiveness as a smokescreen to hide the true reason for denying Finlay the treatment he desperately needs – cost.
To put this into perspective, DMD is very rare and Finlay is the only child on the island affected. What kind of mentality is it that refuses to help such a vulnerable little boy? It’s shameful. I came into pharmacy because it is a caring profession. It may not be as hands-on as medicine or nursing, but none the less pharmacists make a valuable contribution to patient wellbeing.
This saga should not be about money; it should be about the compassion that we, as a society, have for children like Finlay. Like others, I urge the Manx Government to reconsider its decision, and I ask my pharmacy colleagues on the island to add their voices to mine.
Withering is the pen name of a practising independent community pharmacist. Withering’s views are not necessarily those of ICP.