Intended for healthcare professionals


Doctors unlawfully denied drug to girl with spinal muscular atrophy, appeal court rules

BMJ 2021; 372 doi: (Published 05 March 2021) Cite this as: BMJ 2021;372:n641
  1. Clare Dyer
  1. The BMJ

Decisions made by two consultants to deny a 10 year old girl with type 3 spinal muscular atrophy the chance of treatment with a potentially life changing drug were “unlawful” and “irrational,” three Court of Appeal judges have ruled.1

Imelda Hughes, a consultant paediatric neurologist at Manchester University Hospitals NHS Foundation Trust, and Mariacristina Scoto, a consultant in neuromuscular translational research at Great Ormond Street Hospital for Children NHS Foundation Trust, decided that Sophie Basma did not meet the criteria for treatment.

The drug nusinersin (Spinraza), which has a list price of £450 000 (€520 000; $630 000) for the first year and £225 000 for subsequent years but is supplied to the NHS at a discount, is the only potentially disease modifying treatment available in the UK for Sophie’s condition. The UK National Institute for Health and Care Excellence issued guidance in October 2019 stating that it should be provided to patients with type 3 spinal muscular atrophy provided that seven criteria were …

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