Medicine’s Collusion with False Hopes: Rights to try, false hope, and the spine of the profession

Bioethics in the News logoThis post is a part of our Bioethics in the News series

By Marleen Eijkholt, JD, PhD

Imagine your loved one is dying. You have heard about an intervention out there, somewhere, that could help—you think. Not trying this intervention for and on your loved one would seem insane, as who knows, there could be a chance for a cure. Without trying it, your loved one’s death is certain. But by trying it—who knows—this intervention might be the miracle for your beloved. Why not try?

Though death ultimately takes all, most of us resist a final exit for both our loved ones and ourselves. Pursuits to forestall death can take the shape of family’s quests for experimental treatments, like in the Charlie Gard case, or they can come by insisting on a “full code” for a terminally ill loved one who is on their deathbed. This rescue impulse (by unbridled hope) encourages health care professionals as well to marshal all available resources in attempts to circumvent the inevitable.

Where “right to try” creates false hope, I submit, however, that that the health care profession (HCP) has an obligation to avoid collaborating in, participating, propagating or augmenting false hope. By not speaking up against such requests, health care providers precipitate a harm, i.e., the false hope harm (FHH). As such, the HCP should show courage, and show spine to resist more broadly unreasonable “rights” to try.

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Image description: an illustration depicts a white whale above a sea of orange waves. The whale is in a net that is being held up by orange birds. The background is blue. Image source: Matthias Töpfer/Flickr Creative Commons

I call for an active stance from the HCP against populist legislative initiatives, such as the latest U.S. Senate’s “right to try” bill. In the same way, I call for an active stance against unrealistic patient and family requests for rights to try, including resuscitative efforts in terminally ill patients or other demands for non-beneficial treatments. My position thus supports the providers’ prudent approach in the tragic Charlie Gard case, as described at the end of this post. The FHH can best be countered by courageous advocacy from the profession.

Earlier last month, the U.S. Senate passed a “right to try” bill, and if the House approves this legislation, the bill would allow terminally ill individuals to access experimental drugs not yet available on the regular market. Once a drug has passed Phase I drug testing, an individual could petition the pharmaceutical company for access to that experimental drug. This bill also provides protection for pharmaceutical firms. The legislation restricts the FDA from using right-to-try-related adverse outcomes in determining a drug’s safety. Accordingly, the bill limits “penalizing” pharmaceutical companies for their “generosity” in offering access.

A discouraging stance against “right to try” legislation is tricky. It is telling that “right to try” legislation has been dubbed “feel good” legislation, and thirty-nine states already have such legislation, including Michigan. Although there are proponents of these laws, I contend that these laws are “rhetorical pleasers” more harmful than beneficial. Criticism directed at “right to try” legislation focuses on several concerns, including its potential to undermine the clinical trial system and the belief that ultimately such laws only serve the elite. Some critics address false harm arguments, although these concerns are mostly different from mine. Critics argue that the legislation creates false hope, because pharmaceutical companies are less than eager to share their innovative pharmaceuticals, and are afraid of right-to-try-associated risks and harms that might lead to scientific and drug development setbacks. Patients, therefore, most often are unable to obtain access to the medications, so their hope for a “miracle drug” is in fact unfulfilled. Without actionable pathways, patient access cannot be guaranteed. (Indeed, in Michigan, Bridge Magazine suggests that the legislation has failed to change the status quo, and no one has sought access.)

My proposal that the HCP should stand up against unreasonable rights to try comes from my belief that these laws create FHH instead of real benefit. “Right to try” legislation generates false hope as the sought after experimental interventions are unlikely to benefit the patient. Although the legislation requires the drug to have passed Phase I clinical trial safety testing, this measure does not amount to a guarantee that the medication offers benefit. A Phase I clinical trial is safety focused, conducted in a very controlled environment, and in fact many treatments fail to demonstrate benefit outside that controlled environment.

The federal legislation is therefore an even more troublesome example of “feel good” legislation, as it propagates and augments FHH. It endorses the idea that pursing experimental treatment is both feasible and reasonable. Equally, it supports the notion that as long as one has the means, one should have access to try. Most importantly, by limiting FDA oversight, it undermines safeguards and trust in the system, and creates more room for FHH, giving pharmaceutical companies nearly a free reign.

Standing up to false hope is painfully hard, especially in the face of persuasive, emotionally vivid stories. Who would want to deny anyone hope for a miracle? Charlie Gard’s parents wanted to try a new treatment for their son, but their providers refused access. While their son was tragically dying, they were helpless to avert the inevitable. They heard about a treatment in the U.S. and appealed for access. But the UK doctors refused to endorse their appeal and the Court eventually prevented Charlie Gard’s parents from taking their son to the U.S. for treatment. A compassionate intuition could be: provided Charlie did not feel pain, why would there be anything wrong with them pursing this treatment in the U.S.? His parents had sufficient funds to cover related costs, so their pursuit would not further tax the social system. Experts suggested that there was a zero percent chance of health benefits for Charlie, but who can really know zero percent if you haven’t tried? We can never exclude miracles, can we? Even though this drug was not past Phase I, it had shown marginal benefit in other kids with illness that were related to Charlie’s, so safety wasn’t hard to prove.

I propose that if providers would have allowed Charlie to leave for the U.S., their endorsement would have exploited vulnerable individuals; they would have collaborated in FHH. The providers took the right and courageous action. Not speaking out against FHH passively supports initiatives that are more harmful than beneficial, and speaking out requires an active positioning of individual providers—optimally supported by the larger body of HCPs. I submit that this should extend to participating in requests for non-beneficial treatment requests like resuscitative efforts on dying patients. The medical profession rests on elements of trust, guidance, expertise and collaboration. Absent health care providers’ courage to deny treatment, we are doomed to see a further erosion of that profession. Without provider action against “right to try,” under legislation or requests in the clinic, medicine is at risk of further drifting into consumerist medicine, where money, fear, and privilege turn health care (and providers) into hostages of patient demands.

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Marleen Eijkholt is an Assistant Professor in the Center for Ethics and Humanities in the Life Sciences and the Department of Obstetrics, Gynecology and Reproductive Biology in the Michigan State University College of Human Medicine. Dr. Eijkholt is also a Clinical Ethics Consultant at Spectrum Health System.

Join the discussion! Your comments and responses to this commentary are welcomed. The author will respond to all comments made by Thursday, October 5, 2017. With your participation, we hope to create discussions rich with insights from diverse perspectives.

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Drs. Eijkholt and Cabrera published in ‘Neuromodulation: Technology at the Neural Interface’

Marleen Eijkholt photoLaura Cabrera photoCenter Assistant Professor Dr. Marleen Eijkholt and Assistant Professor Dr. Laura Cabrera are co-authors of a new article in Neuromodulation: Technology at the Neural Interface. The article, “Shaking Up the Debate: Ensuring the Ethical Use of DBS Intervention Criteria for Mid-Stage Parkinson’s Patients,” was written by Dr. Eijkholt, Dr. Cabrera, Adolfo Ramirez-Zamora, MD (University of Florida), and Julie G. Pilitsis MD, PhD (Albany Medical Center).

Abstract
Objectives
Deep brain stimulation (DBS) is a well-established treatment for the management of severe motor fluctuations in advanced Parkinson’s disease (PD). Until recently, device regulation, medical, and insurance practices limited DBS to patients with advanced stages of PD. In February 2016 this changed, however, when the US Food and Drug Administration (FDA) granted formal approval for the use of brain stimulator in mid-stage PD patients. In this article, we examine whether DBS in mid-stage PD can be ethically justified beyond the FDA approval.

Materials and Methods
We scrutinize the current risk-benefit profile, the costs-benefit profile, and the capacity for informed consent requirement, to ask if use of subthalamic nucleus (STN) in mid-stage DBS is ethically appropriate.

Results
We propose that mid-stage DBS decisions could be appropriate under a shared decision-making model, which embraces a broad quality of life perspective.

Conclusion
Although it might be too premature to know how the FDA decision will affect medical and insurance practices, we conclude by arguing that revisions to persisting guidelines seems justified both on scientific and ethical grounds.

The full text is available through Wiley Online Library (MSU Library or other institutional access may be required to view this article).

Dr. Eijkholt presents at 13th Annual International Conference on Clinical Ethics Consultation

Marleen Eijkholt photoThe 13th Annual International Conference on Clinical Ethics Consultation (ICCEC) was held on May 25-27 in Singapore. Center Assistant Professor Dr. Marleen Eijkholt attended and presented on a neuroethics panel, “A Cross Cultural Examination of Pain: Conceptual, Practical and Clinical Ethical Challenges.”

Pain is a relevant clinical problem, but also a social construct impacted by culture, environment, and gender. As a result, pain is perceived, framed and treated differently across cultures. However, minimal literature exists on cross-cultural examinations of pain.

Dr. Eijkholt and collaborators Nneka Sederstrom (U.S.), Daniel Fu-Chang Tsai (Taiwan), and Yonghui Ma (China) explored various challenges in the framing of pain across cultures. By focusing on a specific case, the panel examined the meaning of pain, the various challenges of the pain discourse, and the varying approaches toward pain through a cross-cultural lens. They delved into the ethical, legal, and social issues around pain. Asking: what is the relevance of pain in the clinical setting, what (in)abilities do patients and surrogates have to address pain in the clinical setting, how is pain treated, and what concepts frame the importance of pain, they sought to compare and contrast different approaches to draw lessons for working in a cross-cultural context.

Dr. Eijkholt also presented a case study titled “Clinical neuroethics: Cracking brains and health care systems.”

Transplant Headaches: Head transplant on the horizon

Bioethics-in-the-News-logoThis post is a part of our Bioethics in the News series

By Marleen Eijkholt, PhD, LL.B/LL.M

Introduction
On May 1, 2017, the Daily Mail reported on a successful head-transplant involving rats, as a “practice run for [a] controversial human experiment.” Indeed, this run would seem timely and perhaps minimal pertaining only to rats, given the proposed timeline toward the first-ever human head-transplant (HHT). Some reports suggest December 2017 as the date for the HHT, while others report that December is unlikely, and that the procedure would have to be delayed.

With the first HHT on the horizon, guttural yuck-reactions, and references to the ethical questionability of this procedure abound. A famous ethicist suggested that the surgeon is “out of his mind.” But I am unsure if this rejection is fair. If we have allowed the development of kidney, heart, hand, face, uterus, and penis transplants, why would this new area of transplant medicine raise prohibitive concerns?

Several areas of ethical concerns have been identified, and I want to address three reflections briefly here. Although many more could be identified, including the concern that a ‘body transplant’ also means that its other organs cannot be used for other purposes, I will not go into these issues due to word limitations. I offer thoughts for further discussion, drawing on parallels from other controversial (surgical) interventions, i.e. stem cell interventions for spinal cord injury (SCI) and deep brain stimulation (DBS). Often ethics is seen as a prohibitive ‘science’ but I do not want to endorse this idea. Instead, I hope to ask how we can ensure that this development proceeds as reflectively as possible.

Background

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Image description: two surgeons are shown in an operating room while performing a transplant procedure on a patient who is out of frame. Image source: Scott & White Healthcare/Flickr Creative Commons

The history of HHT up to this point is described in many places. In 2013, an Italian surgeon, Canavero, first suggested his willingness to do a HHT, causing outrage and interest. Canavero has given a TED talk in the meantime, published several manuscripts [3, 19] and protocols [1, 2] on the procedure. Initially, one Russian individual, Valery Spiridonov, came forward to undergo the HHT and is now considered as potentially the first patient, while others have started to line up. Spiridonov has a degenerative and incurable muscle disease (Werdnig-Hoffmann) and is confined to a wheelchair. Cost-estimates of the procedure include a 7.5-20 million dollar, 36-hour operation, with a team of 150 people. The community has expressed serious reservations about the scientific feasibility. Still, we read that the first head-transplant in monkeys occurred in 1970 and that several dogs and mice have undergone the procedure. These were mostly as proof of principle, and not focused on long-term survival. Currently, there is no substantial proof that the recipient can be ‘kept alive’ long-term.

Informed Consent (IC)
A primary concern is whether Spiridonov can offer informed consent. Given his vulnerability, suffering Werdnig-Hoffmann disease, and based on reports about what Spiridonov said, these concerns are real. Spiridonov’s desire is to be like the “first man on the moon,” generate knowledge for future generations, and that the HHT will help him. Hence, he seems vulnerable to therapeutic misconceptions and risk-mitigation biases, especially in the light of a surgical learning curve that will challenge the first HHT [10]. Spiridonov’s narrative refers to the first man on the moon—who successfully made it—failing to mention the others who did not make it. Another part of this concern relates to identity problems that transplant individuals might experience with an alien body (part), where HHT concerns a whole body and an irreversible transplant [17].

These areas of concern have been dealt with at length, in reference to transplants and other emerging (surgical) interventions. I do think these concerns can be addressed, provided a thorough shared decision-making process, like in DBS [7]. Why would it be different in the HHT context?

Necessary Evidence
It seems an incredible leap to move from rat experiments to human interventions, without bigger animal models. However, such leap is not unprecedented, and might not automatically be condemned. Geron, a company that injected stem cell derived products in the spinal cord of SCI patients, moved from rat trials to humans. They skipped large animal models in assessing feasibility. Their decision was met two ways. One set of arguments stated the unacceptability of this practice as humans would now be exposed to unacceptable risks, based on insufficient knowledge. Another set of arguments was perhaps less outraged, suggesting that animal rights arguments might favor such direct application. Sacrificing large animals could not always be justified, especially if evidence in large animals may still not apply to humans [9]. Would it be in the HHT case?

Medical Need
A final question is: How to go about the risk/benefit assessment for someone whose medical need is not immediately life threatening? Spiridonov does not suffer an immediately life threatening illness, unlike liver failure, so some might argue that the risk/benefit profile of this experiment is disproportionate. Aside from concerns about the patient facing risks, executing novel risky experimental interventions may entail a loss of trust in the field if they go wrong. We have seen this in the tragic story of Jesse Gelsinger, gene therapy trial participant. So, some could argue that Spiridonov’s medical need does not warrant a loss of trust in science. But in response one could propose that for a proper risk/benefit assessment, weighing many factors is required. We see that for many composite allograft transplant areas as well as for DBS, the focus is on improving quality of life, not providing a cure. References to medical need and life-saving interventions are social constructs. If interventions like penis transplants were justified and marked as life-saving, referencing suicide concerns, life-limiting conditions like Spiridonov’s condition could put these concerns into perspective.

Conclusion
Although HHT are a new transplant territory, many of the headaches are familiar ethical concerns to the domain of novel transplant medicine and emerging surgical interventions. I am unsure that HHT is very different than the first heart transplant, which was met with huge skepticism, involving concerns about survival, IC, and identity. Heart transplants have offered huge progress in medicine, by generating new knowledge and applications for patient well-being. What makes us say that this body/human head-transplant is going to be less of a revolutionary intervention, and should be stopped before it even begins?

Marleen Eijkholt photoMarleen Eijkholt is an Assistant Professor in the Center for Ethics and Humanities in the Life Sciences and the Department of Obstetrics, Gynecology and Reproductive Biology in the Michigan State University College of Human Medicine. Dr. Eijkholt is also a clinical ethicist at Spectrum Health System.

Join the discussion! Your comments and responses to this commentary are welcomed. The author will respond to all comments made by Thursday, June 22, 2017. With your participation, we hope to create discussions rich with insights from diverse perspectives.

You must provide your name and email address to leave a comment. Your email address will not be made public.

Click through to view references

Dr. Eijkholt published in ‘Journal of Emergency Medicine’

CMarleen Eijkholt photoenter Assistant Professor Dr. Marleen Eijkholt is the co-author of an article in the April 2017 issue of the Journal of Emergency Medicine. The article, “Enabling Donation after Cardiac Death in the Emergency Department: Overcoming Clinical, Legal, and Ethical Concerns,” is co-authored by Michael Dailey, MD, Sean P. Geary, MD, Stefan Merrill, MD, and Marleen Eijkholt, PhD.

Abstract
Background: In light of the growing gap between candidates for organ donation and the actual number of organs available, we present a unique case of organ donation after cardiac death. We hope to open a discussion regarding organ procurement from eligible donors in the prehospital and emergency department setting.

Case: This case study, involving an otherwise healthy man who, after suffering an untimely death, was able to successfully donate his organs, highlights the need to develop an infrastructure to make this type of donation a viable and streamlined option for the future.

Discussion: Given the departure from traditional practice in United States transplantation medicine, we bring forth legal and ethical considerations regarding organ donation in the emergency department. We hope that this case discussion inspires action and development in the realm of transplant medicine, with the aim of honoring the wishes of donors and the families of those who wish to donate in a respectful way, while using our medical skills and technologies to afford candidates who are waiting for organs a second chance.

Conclusions: We believe that this case shows that donation after cardiac death from the emergency department, while resource-intensive is feasible. We recognize that in order for this to become a more attainable goal, additional resources and systems development is required.

The full article text is available on the ScienceDirect website (MSU Library or other institutional access may be required to view this article).

The Center welcomes Assistant Professor Marleen Eijkholt

m-eijkholt-cropThe Center for Ethics is happy to announce the addition of a new faculty member, Dr. Marleen Eijkholt. Dr. Eijkholt is an Assistant Professor in the Center for Ethics and Humanities in the Life Sciences and the Department of Obstetrics, Gynecology and Reproductive Biology. Dr. Eijkholt is also a clinical ethicist for Spectrum Health System.

Dr. Eijkholt was most recently an Assistant Professor in Medical Humanities at Davidson College in Davidson, NC, and Co-Director of Clinical Ethics Consultation Service & Clinical Ethics at Charlotte Medical Center in Charlotte, NC. She received a JD (or LL.B./ LL.M.) from Maastricht University in The Netherlands, and a PhD in Bioethics & Medical Jurisprudence from University of Manchester in the United Kingdom.

Dr. Eijkholt’s research interests include clinical ethics consultation, reproductive ethics, neuroethics, bioethics and human rights; the broad array of ethical, legal and social issues (ELSI) in biomedicine, including rights in reproduction, stem cells, placebos, deep brain stimulation, and rights to experimental interventions. Visit Dr. Eijkholt’s faculty page to learn more about her work.