Posted on August 1, 2017
Last week, British infant Charlie Gard died after his parents withdrew their legal bid to obtain experimental treatment for his terminal illness. The case was characterised by hallmark elements of unfathomable personal tragedy, ever-present when it comes to infants and life or death decisions. Concerns revolved around what constitutes ‘life’, who determines “pain” and how experimental procedures can hold out the promise of great hope against exponentially poor odds – especially when spurred on by international figureheads like Donald Trump and the Pope (must admit I never thought I’d have cause to write both their names in the same sentence).
Charlie Gard was born apparently healthy, but over the first month he started to develop muscle weakness and lost his ability to engage with his surroundings. It turned out that he had an exceptionally rare genetic disease with a dire prognostic outcome. Charlie was soon unable to function independent of a ventilator. He was tube fed. He seemed unable to engage with the world in any meaningful way. It was unclear whether he was in pain.
The court case
On one side of the legal battle was Great Ormond Street Hospital, arguing that the only management in Charlie’s best interests was to withdraw life support and provide palliative care till nature took its course. Charlie’s parents argued for an experimental procedure which may hold out hope of improving Charlie’s quality of life – although this is questionable because:
- The US doctor leading the research seemed quite flakey in his responses to – and interaction with – the Gard family and the courts
- The drug had not been tested on anyone with Charlie’s specific condition
The upshot of regulation
As per this previous post, experimental medical treatments are rigorously regulated world-over. This has some benefits in protecting vulnerable human participants in clinical trials. But one apparent disadvantage is that due to such regulation, experimental treatment often cannot be made available on the basis of urgency, when all other management options have failed.
Because the experimental drug was not registered in the UK, a number of regulatory approvals needed to be sought before it could be offered to Charlie. Sadly, negotiating research regulation takes time, and time did not wait for Charlie Gard. Whilst court proceedings were ongoing, his condition deteriorated to such an extent that the treatment no longer held out any promise.
Is this right or wrong or somewhere in-between? How do we justify regulations the practical upshot of which is that we cannot offer a terminally ill infant with a diagnosis of futility one last shot at lifesaving treatment? On the other hand, what happens if we make an exception for one person, aren’t we setting a dangerous precedent?
Research and development in medicine is a process, and we have to always weigh the risks and benefits. Often regulation facilitates these complex deliberations. But given the rarity of Charlie’s condition it’s unlikely that targeting it for research would ever be profitable. This means it might be overlooked, now and in the future. So where does this leave infants like Charlie? Would it have been worth giving him a chance even though there was little prospect of success, to the extent that if he lived it may have been without any meaningful quality of life? We just don’t know. Maybe, in this case, the law is an ass after all. Many seem to think so. I am undecided.
The views published in this blog are entirely my own (informed) opinions. They are open to debate, discussion and disagreement.