For some, it is 12 steps that stand between them and hope for the future. For Sophie Basma it was a mere five.
Sophie is 10 years old and has Type 3 Spinal Muscular Atrophy, a rare neuro-muscular disease which leads progressively to an inability to walk or sit unaided. The Trusts involved in her care – one in Manchester and one in London – decided that Sophie was not eligible for treatment with a recently NICE-approved drug, Nusinersen. Why? Because they concluded that they could not be satisfied that, at some point in the 12 month period before she became entitled to be considered for the drug, she remained “able to walk 5 steps in an upright position, with a straight back and with no contact with a person or object”.
There was no dispute that the drug was clinically appropriate for her. There was some evidence of clinical effectiveness in the short to medium term. Long term effectiveness was less clear but there was a possibility that, with Nusinersen, she might regain the ability to walk and maintain significant levels of upper body strength. Without treatment, there would be an inevitable decline into complete immobility.
Under NICE guidance (TA588), Nusinersen is to be provided to Type 3 SMA sufferers provided that seven criteria are met. There was no disagreement over six of the criteria but whether or not she satisfied the seventh – the “5 steps criterion” – remained a matter of dispute. Because SMA sufferers commonly have “off days”, it was recognised that the 5 steps criterion was neither fixed by a single assessment on a particular day nor by reference only to a formal clinical assessment. But what made the issue more difficult in Sophie’s case was the dearth of formal assessments during the critical period (October 2018 – October 2019), together with the fact that, as the drug was not a treatment option during this period, no one in Sophie’s immediate circle (HCPs, family, friends, teachers) understood the importance of noting and recording how far she could walk.
By the time the matter reached court, seven lay witnesses (family and friends, a teaching assistant and Sophie’s yoga teacher) had produced witness statements testifying to Sophie having satisfied the 5 steps criterion in the relevant period, referring to some memorable incident or event that had made the occasion stick in their minds. Clinical reluctance to confirm that Sophie fulfilled the seventh criterion persisted, however, because such evidence as there was was not in the form of a formal assessment or video recording. The decision was made, therefore, that Sophie was not eligible to receive the drug.
At first instance, the court granted Sophie’s mother permission to bring the judicial review on her behalf, but dismissed the substantive application on the basis that the need to weigh evidence from competing sources required the exercise of clinical judgment and that clinical judgment had concluded that Sophie had lost the ability to walk independently before the critical period. Ms Basma appealed to the Court of Appeal.
The Court of Appeal concluded that:
- The decision to be reached on the seventh criterion was not one of expert clinical judgment alone. A very substantial part of the task was to ascertain the facts by taking account of all relevant evidence.
- The more clinically complex the decision challenged, the less likely the court is to interfere with it. But this case involved largely factual decisions, albeit with a clinical element, which had a considerable impact upon the claimant. The court could therefore be robust in the deference it accorded to the decision-maker and there was no need to adopt a ‘kid glove’ approach.
- The evidence of family and friends was relevant because there was no clinical evidence from the relevant period. The decision-maker was therefore bound to consider that evidence and to decide what weight it should carry.
- The treating consultants’ decisions were, accordingly, irrational for failure to take account of relevant evidence.
The Court of Appeal therefore quashed the decisions to refuse Sophie the drug and ordered the Trusts to reconsider their decisions in light of the judgment, evidence submitted by Ms Basma to date and any further evidence she might choose to submit.
We are used to high cost drug cases focusing on whether or not the commissioner should have commissioned the drug at all, either for a cohort of patients or for a particular individual. In Basma, by contrast, funding was not in issue and the debate was about eligibility for treatment by fulfilment of a set of criteria. Had there been a vast array of formal assessments, no doubt the informal evidence of family and friends would have carried far less weight. In the absence of formal clinical evidence, however, decision-makers should take care to ensure they do not adopt a blinkered stance which excludes informal but still relevant material. If they do, they could find their conclusions declared irrational and unlawful even where, as here, the court acknowledged that the individual clinicians had each been “conscientious and well-intentioned”.