Lantos JD. The Tragic Case of Charlie Gard. JAMA Pediatr. 2017 Aug 11. doi:
10.1001/jamapediatrics.2017.3079. [Epub ahead of print]
The determination of Charlie’s interests focused on 2 major uncertainties. One was whether Charlie was in pain; the other was whether nucleoside therapy offered any hope of benefit.
Those closest to Charlie were unsure if he was in pain. One physician said that it was possible that he felt pain but acknowledged that Charlie did not demonstrate typical signs. A nurse testified that it was “impossible to know whether Charlie suffers pain, pleasure, or comfort.” His mother testified that he responded to head stroking and tickling.5 Clinicians and parents also disagreed about the likelihood that nucleoside therapy would be beneficial. Charlie’s clinicians repeatedly noted that the therapy had never been tried in this particular condition. The disagreement, then, was about whether data from one genetic subtype of MDDS could be generalized to a different MDDS. Were these distinct diseases or variants of a common syndrome? Everybody agreed that the treatment itself was safe.
Neither of the central questions—whether Charlie was in pain and whether the experimental treatment could help—could be answered precisely. The first required a subjective opinion; the second, a probabilistic extrapolation from scant prior data. Charlie’s lawyers argued that even a small chance of benefit from the experimental therapy outweighed the potential harms. More importantly, they argued that parents and a physician of their choice should be allowed make the decision: “(I)t is for the parents, in the exercise of their parental rights and duties, to decide what course of treatment is in the child’s best interests.”4 Charlie’s mother said, “If it is only being a matter of him dying or a matter of him having a chance at life, the parents should have a say.”6 Judges of the High Court of Justice of England and Wales, Her Majesty’s Court of Appeal in England and Wales, Supreme Court of the United Kingdom, and European Court of Human Rights all ruled that nucleoside therapy was not in Charlie’s best interest and thus that his life support should be withdrawn. Eventually, the outside consultant came to agree that Charlie’s disease had progressed to the point where therapy would no longer be beneficial. At that point, the parents gave up their fight, and Charlie was transferred to hospice, where he died.
This case challenges us to think about the limits of parental rights. Physicians, policy makers, and judges will need to articulate clear principles for deciding when parental perceptions and values should guide treatment choices when (1) we are uncertain about whether a patient is in pain, (2) a physician is willing to administer a safe treatment that might be beneficial, (3) the parents want to try that treatment, and (4) the only alternative to experimental therapy is death.
The case has disturbing implications for the process of shared decision making in such circumstances. Generally, when physicians disagree, we defer to parents and allow them to choose which physician’s recommendations to follow. In the United States, this is true even in states that permit unilateral physician decisions to withdraw life support, such as Texas. Under Texas law, when physicians determine that further life support is inappropriate, parents are given 10 days to find another physician. If they do, their child will be transferred to the care of that physician.8 In California, this was even true for a child who was declared brain dead.9 Instead, the precedent set in the Gard case suggests that courts may make an independent evaluation of which medically endorsed treatment is best. This is a significant encroachment on both physicians’ medical authority and parents’ rights.
The case also has implications for the use of genomic information. Charlie’s parents and their chosen physician thought that data from treatment of patients with a related disease was relevant. The courts thought that this prior experience was irrelevant because Charlie’s disease was genetically different. We are entering an era in which every patient and disease will be genetically distinct. In this new era, questions will arise about how much to generalize from experience with each discrete genotype to patients with related diseases but distinct genotypes. Personalized genomics could lead to a world in which all therapies become experimental because each patient is genetically unique. All clinical care will then become N-of-1 research studies.
Ultimately, the cost of treatment may guide such decisions in the future. Considerations of justice may dictate that some expensive treatments will not be offered if cost outweighs the likely benefits. If such decisions are made, they should be explicit.
The powerful public reaction against the court decisions suggests that, in future similar cases, a different approach might be preferable. If there was any hope for Charlie, or for other patients with relentless progressive degenerative diseases, it is only if treatment was given early. As Savulescu10 noted, if the physicians had started nucleoside therapy at the same time that they went to court, we’d have known whether it worked before we had the final legal verdict. Surely that would have been a better result for all concerned.