By Sarah Alawi
There has long been a debate about the potential ethical conflict between the role of the clinician and that of the clinical researcher. On the one hand, the overriding ethical obligation of a clinician is to maximize the individual patient’s health; on the other, the overriding ethical principle of the researcher is to produce socially useful research, with the consequence that, at times, it may require subordinating the best interests of the individual to achieve the greatest public good.
Although some have called into question the utility of this framing, the concept of ultra-personalized medicine is pushing this debate even further. The idea of ultra-personalized medicine – or “custom drugs” – is not new. As NHS England puts it, although “clinicians have been working to personalize care… throughout the history of medicine,” technological and scientific advances mean that never before has it been possible to move to an era of “truly personalized care.”
Last week, the New York Times reported that – for the first time in history (it is believed) – a new drug has been created to treat just one patient: “The drug is believed to be the first ‘custom’ treatment for a genetic disease. It is called milasen, named after the only patient who will ever take it.” Milasen involved the development of a custom piece of RNA to block the effects of an extraneous piece of DNA in Mila that caused an unusual case of Batten’s disease.
The scientific possibilities represented by Mila’s case engage the old debate on ethics in an entirely new way. The reality of the health care industry is that there are scarce resources. And given that it is impossible for custom drugs to be made available to everyone, the issue is whether it is ethical for them to be made available to anyone. Put another way, it represents the inverse of the classical ethical problem of the clinical researcher. The problem for the researcher has traditionally been phrased as whether it is ethical to subordinate the good of the individual to benefit the common good. By contrast, the problem of custom drugs is whether to subordinate the common good for the interests of the individual by dedicating scarce medical resources to research that stands to benefit only one person. Then again, the life of an individual is inherently valuable, and a crude utilitarian calculus is unlikely to provide any easy answers.
The other ethical conundrums raised in this context include the wider systemic issues of wealth inequality. Realistically, custom drugs are only an option for “the very wealthy, those with the skills to raise large sums of money, or those who gain the support of foundations.” In Mila’s case, it was mostly paid for by a foundation run by her mother. Custom drugs also raise important questions about the role of the FDA. As Dr. Janet Woodcock notes, this is uncharted territory for the FDA: what type of evidence is needed before exposing a human to a new drug? How do we evaluate a custom drug’s efficacy? How should regulators weigh the urgency of the patient’s situation in this context? Milasen was approved by the FDA. If there is an ethical dilemma in ultra-personalized medicine, one wonders whether these ethical issues ought to be special considerations the FDA should take into account in deciding whether to approve custom drugs.