UK Court of Appeal declares young girl must be reconsidered for drug for rare genetic disorder

The UK Court of Appealhas ruled that Sophie Basma, a young girl suffering from type 3 Spinal Muscular Atrophy (SMA), should be reconsidered for the drug Nusinersen and the NHS Trust had acted unlawfully when deciding not to administer the drug to Sophie.

SMA is a rare, genetic, neuromuscular disease which progressively leads to sufferers being unable to walk or sit unaided with devasting consequences on their quality of life. The drug would provide SMA sufferers with the opportunity to regain their ability to walk. However, in order to qualify for it, a SMA sufferer has to satisfy a number of criteria.

Sophie Basma passed six out of the seven criteria; the criterion that was in dispute was whether she could walk five steps unaided in the 12-month period preceding the treatment decision, which was scheduled for October 2019. Sophie was able to walk unaided up until September 2019, after which she needed assistance. Based on this, Sophie’s physician determined that she was not eligible for the drug. A consultant reached the same conclusion in December 2019 and referred the case to the NHS England Clinical Panel. The panel concluded that “family recollection” was unreliable and gave its opinion that Sophie was not eligible.

This decision was upheld in the High Court, however the Court of Appeal held, unanimously, that the Judge erred in categorising the five-step criterion as one of “expert clinical judgement”. The decisions under appeal were largely factual, but with a clinical element. The Judge placed undue weight on the fact that the decision-maker was a clinician. A substantial part of the task was to ascertain the facts, which required the decision-maker to take into account all relevant evidence. For one, the Court held that the evidence provided by Sophie’s family that she was able to walk during the relevant period of time was important as there was an absence of clinical evidence during the same period.

The Court determined that the clinician and the consultant acted unlawfully in treating the panel’s decision as determinative and not advisory. This, in effect, was an unlawful delegation of decision-making power to the panel.

In her judgement Lady Justice King said, "I have concluded that decisions made by each of the doctors were both 'unlawful' and, 'irrational'; I should make it absolutely clear that in reaching those conclusions and by the use of those term, I am not to be taken as doing other than having the highest regard for the expertise and dedication of each of these two consultants, nor should it be thought that their decisions were influenced by any financial considerations.

"It is clear each of them had a strong desire to prescribe Nusinersen for Sophie, but each concluded that the interpretation they felt compelled to adopt of the '5 Steps Criterion' meant that they were unable to do so."

Click here to read the full decision.



Sustaining Partners