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July 20, 2017

The United Kingdom Sets Limits on Experimental TreatmentsThe Case of Charlie Gard

Author Affiliations
  • 1Harvard Medical School, Boston Children’s Hospital, Boston, Massachusetts
JAMA. Published online July 20, 2017. doi:10.1001/jama.2017.10410

The case of Charlie Gard in London, England, has been the focus of international attention, generating polarized views about the use of experimental treatments. On one side are those who hold that patients should be able to purchase whatever treatments they desire and can afford; on the other are those who maintain that governments must play a regulatory role in protecting patients from harm and that unproven therapies must meet a threshold of scientific validity before they are offered, regardless of the ability of the patient to pay.

Charlie Gard is an 11-month-old boy who, according to court records, “suffers from a rare inherited mitochondrial disease called infantile onset encephalomyopathic mitochondrial DNA depletion syndrome.”1,2 During the several months he has been hospitalized at Great Ormond Street Hospital his condition has steadily deteriorated, and he is now dependent on life support and mechanical ventilation. His physicians believe he has no reasonable remaining treatment options, and they have recommended palliative care and withdrawal of the ventilator. Charlie’s parents, however, are asking that he be transported to the United States, where an unnamed US physician has offered to treat Charlie with nucleosides, an experimental treatment that has been used on a few children with a less severe form of the disease caused by a different mutation. The parents have raised more than $1.6 million in contributions to fund his transport and treatment.3

Several experts in various specialties, including pediatric intensive care, neurology, and mitochondrial diseases, have argued against treating Charlie with this therapy, pointing out that the treatment has not been tried on humans or even animals with the mutation causing Charlie’s disease.1 The US physician acknowledged in his testimony that his recommendation was theoretical and based on very limited evidence from a related but less severe condition caused by a different mutation. After reviewing the records, the physician noted that the damage to Charlie’s brain was more severe than he had thought. He said that the chances of meaningful brain recovery would be small, which he agreed he could not distinguish from vanishingly small. He conceded that to a large extent, if not entirely, the brain damage was irreversible. Nevertheless, he concluded that if Charlie were in the United States, he would treat him if the parents so desired and could pay for it: “I would just like to offer what we can. It is unlikely to work, but the alternative is that he will pass away.”1

The case has been heard by 3 courts in the United Kingdom (the High Court,1 the Court of Appeal,4 and the Supreme Court5) as well as the European Court of Human Rights.6 All of the courts have affirmed the initial ruling that palliative care and withdrawal of life support are in Charlie’s best interest. In an unusual development, however, both Pope Francis and President Donald Trump have expressed support for the parents, and the Vatican has offered to accept Charlie “for the time it will take for him to live.”7

At least 3 issues are involved in this case that are at the core of many of the worldwide debates regarding health care delivery: the best interest of the patient, financial considerations, and scientific validity. First, the judge in the trial court explained that the law required him to base his decision solely on what he deemed to be the child’s best interest.1 Based on testimony from the physicians, the judge ruled that continued treatment would cause Charlie pain and suffering and that, in the absence of a realistic chance of benefit, continued treatment would not be in Charlie’s best interest.1 The problem with this decision is 2-fold. First, this decision depends on an objective opinion about a subjective phenomenon, namely pain and suffering. Although the clinicians caring for this child apparently believe that he is experiencing pain and discomfort, his parents disagree and have said that they would not be insisting on continued treatment if they thought that he was. In addition, medical treatments in the intensive care unit are almost always associated with some degree of discomfort, but intensive care unit clinicians are well trained and equipped to effectively manage these symptoms. If pain and suffering were the only issue, then an alternative to treatment withdrawal would be to use a standard regimen of analgesia and sedation.

Second is the question of whether health care is a commodity that should be distributed based on the preferences of patients and their ability to pay, or is a communal resource that governments have an obligation to regulate in accord with principles of distributive justice. Advocates of the former view are outraged at the efforts of the hospital to block the parents’ request for transfer to the United States. What gives the hospital the right or the authority, they argue, to say that the chances of success are too small to be worth the effort, particularly since the parents are willing to pay for it with donations they have raised?

If Charlie’s continued treatment were to be funded through either governmental or private insurance, then a strong case could be made that acceding to the parents’ request would be an irresponsible use of limited resources. But the notion that Charlie’s parents are paying out-of-pocket for this treatment is misleading. The very existence of tertiary care medical centers that are able to do the research and provide the care requested by Charlie’s parents depends on the vast communal investment that has been made by society in the infrastructure of our health care system over many decades. Paying the incremental expenses for the care of 1 patient in a hospital no more covers the cost of that care than paying the incremental costs for a fire truck to come to a home covers the cost of the fire department that makes the trip possible. In other words, society always has a financial stake in how communal services, and in this case health care resources, are used and has a legitimate claim in insisting that these resources be used wisely and for the benefit of all. No one can demand nonbeneficial treatments simply by claiming that they are paying out-of-pocket.

Third is the commitment of the medical profession to scientific integrity. Today, some segments of society regard the claims of medical experts, and of scientists in general, not as expressions of efforts to uncover truths about the physical world, but merely as reflections of a particular set of values. Putting aside the dubious credibility of such an opinion, the historical development of the profession of medicine has been characterized by an increasingly strong commitment to grounding practice on evidence generated through research. Today, for example, it would be extremely rare for a hospital in the United States to admit patients for the exclusive purpose of receiving homeopathic therapy or unproven stem cell infusions, regardless of how much the patient paid.

This does not mean, of course, that medicine does not seek to improve through experimentation. But as the concept of evidence-based medicine implies, progress occurs through careful application of the scientific method. While the most trustworthy advances come through the performance of well-designed trials, sometimes experimental treatments based on theoretical considerations alone may lead to major breakthroughs. But such successes are rare, and to be worth trying they must meet a threshold of scientific plausibility that transcends theoretical possibility. Whether the proposed nucleoside treatment meets that threshold is a legitimate matter for debate, but based on the lack of either animal or human data to support the use of this approach, as well as evidence of severe neurologic injury that could not be reversed in any case, the consensus of respected experts in the United Kingdom was that it did not.

The sad and tragic story of Charlie Gard is a window into several of the deep and fundamental debates that are roiling the governance of health care delivery both domestically and abroad. How can the rights of individuals to use their money for whatever purposes they desire be balanced against the obligation of societies and their governments to regulate health care systems in ways that ensure the delivery of beneficial and cost-effective treatments for the good of all? Considerations of the best interest of patients and of fairness are part of the answer, but they need to be bolstered by a reaffirmation of the scientific values at the core of the profession as well as a commitment to upholding the integrity of medical practice by refusing to provide treatments that fail to meet a reasonable threshold of scientific justification.

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Article Information

Corresponding Author: Robert D. Truog, MD, Center for Bioethics, Harvard Medical School, 641 Huntington Ave, Boston, MA 02115 (robert_truog@hms.harvard.edu).

Published Online: July 20, 2017. doi:10.1001/jama.2017.10410

Conflict of Interest Disclosures: The author has completed and submitted the ICMJE Form for Disclosure of Potential Conflicts of Interest. Dr Truog reported serving on data and safety monitoring boards for Sanofi.

References
1.
Great Ormond Street Hospital v Yates & Ors, [2017] EWHC 972 (Fam) (England and Wales High Court [Family Division]). April 11, 2017. http://www.bailii.org/ew/cases/EWHC/Fam/2017/972.html. Accessed July 17, 2017.
2.
El-Hattab  AW, Scaglia  F.  Mitochondrial DNA depletion syndromes: review and updates of genetic basis, manifestations, and therapeutic options.  Neurotherapeutics. 2013;10(2):186-198.PubMedArticle
3.
BBC News. Charlie Gard case explained. July 10, 2017. http://www.bbc.com/news/health-40554462. Accessed July 17, 2017.
4.
Yates & Anor v Great Ormond Street Hospital For Children NHS Foundation Trust & Anor (Rev 1), [2017] WLR(D) 391, [2017] EWCA Civ 410 (Court of Appeal [Civil Division] on appeal from the High Court of Justice [Family Division]). May 23, 2017. http://www.bailii.org/ew/cases/EWCA/Civ/2017/410.html. Accessed July 17, 2017.
5.
In the matter of Charlie Gard—determination of permission to appeal application (UK Supreme Court). June 8, 2017. https://www.youtube.com/watch?v=P6rPmvGlNhA&app=desktop. Accessed July 17, 2017.
6.
Gard and Others v the United Kingdom (ECLI:CE:ECHR:2017:0627DEC003979317) (European Court of Human Rights). July 3, 2017. http://hudoc.echr.coe.int/eng?i=001-175359. Accessed July 17, 2017.
7.
Bilefsky  D, Chan  S. Dispute over British baby’s fate draws in pope and US president. New York Times. July 3, 2017. https://www.nytimes.com/2017/07/03/world/europe/uk-trump-pope-francis-charlie-gard.html?emc=edit_th_20170704&nl=todaysheadlines&nlid=19900389. Accessed July 17, 2017.
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