This 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.
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.
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.
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References
- Associated Press. Law allows patients to try experimental treatments. Detroit Free Press. http://www.freep.com/story/news/local/michigan/2014/10/17/michigan-experimental-treatments/17458611/. Published October 17, 2014. Accessed September 21, 2017.
- Dresser R. Right to Try Laws and the Power of Stories. The Hastings Center. http://www.thehastingscenter.org/right-to-try-laws-and-the-power-of-stories-2/. Published December 15, 2015. Accessed September 21, 2017.
- Kearns L, Caplan A. Ethical Opposition to Right to Try Laws Is Easy to Articulate. The Hastings Center. http://www.thehastingscenter.org/ethical-opposition-to-right-to-try-laws-is-easy-to-articulate/. Published December 14, 2015. Accessed September 21, 2017.
- Pear R, Kaplan S. Senate Passes F.D.A. Funding and ‘Right to Try’ Drug Bills. The New York Times. https://nyti.ms/2hryyx8. Published August 3, 2017. Accessed September 21, 2017.
- Roelofs T. This law promised medical hope for dying patients. Was it a cruel deception? Bridge Magazine. http://www.bridgemi.com/public-sector/law-promised-medical-hope-dying-patients-was-it-cruel-deception. Published June 8, 2017. Accessed September 21, 2017.
MSU Bioethics 
I am in agreement and, having experienced the rush to rescue, I advocate the use of POLST documents. They need to be available to patients along with the availability of the
physician to have the conversation.
Petra Pohl
Dear Petra, Thank you for your response. I love your reference to the rush to rescue – i might borrow that phreaseology-. I wholeheartedly endorse POLST, MOLST, MOST or POST (in Michigan). I’m not sure that these instruments will solve all the problems as described above, also because they have their own set of issues. But I think they are very valuable initiatives!
An excellent and important essay. It could be strengthened by specifying more precisely what a FHH is, e.g., what kind of harm, and the damage it does, both to individuals, and socially.
Dear Larry,
Apologies for the delay. Thank you so much for your support and interest in this concept. I am working on this and I’ll be in touch.
I agree I think that HCP’s should resist providing false hope. I do think it is important to consider why the HCP is providing false hope. Are they allowing the hope for the family or for avoidance of delivering the fatal diagnosis?
Is there an emotional or psychological toll that could result from refusing to treat the false hope? How will it affect the patient-doctor relationship? Will they look for a different HCP who is willing to collaborate with them?
Dear Karlee, Sorry for the delay in reply. These are very valuable questions. I think it’s a tricky assessment drawing lines between hope and false hope. This difficulty and anticipating that the patient might doctor-shop, probably fuels some of the practices that are not uncommon today.
I acknowledge that I don’t have all the answers, and I really appreciate your contributions in bringing discussion to this field. I will keep developing work in this area.
I’m also in agreement that HCP’s should resist “right to try” legislation initiatives. I also wonder, just because a drug has passed Phase I testing in a controlled environment, this does not necessarily mean that the drug could cause more harm than good. What if a so-called “miracle drug” actually hastened a person’s death, which could be especially painful for parents of young children.
Dear Emalie, I appreciate your observation and I think you make a good point. A dying process that is worsened by the medications, because of side effects would be even more awful. A dying process hastened by the medications would also exacerbate the grieving process, I believe (this is probably an empirical question, that I don’t have an answer to). In an ideal world, the miracle drug would be a ‘cure’, but especially in Charlie’s case, as far as I understood, the inevitable dying process would just be prolonged, and the grieving process too. The right to try seemed just to be invoked as a mitigation for the parent’s grieving process. Where rights to try are invoked to mitigate the grieving process, I’m not sure this is a good use of medicine.
While there are parts of the “right to try” initiative that I have concerns about, ultimately I believe in self-determination of the patient. I think there are ways health care professionals can educate patients on their options, while still being realistic about odds for improvement. If I were a patient in charge of my care, I would want to know what all of my options are so I could accurately decide for myself what I want to pursue. I do agree that there is a risk with this bill in prolonging suffering, especially if a patient is no longer able to make decisions for themselves. But ultimately, I believe in honoring a patients wishes and educating them fully on the repercussions.
Dear Zoie, thank you for your response. Self-determination is one of the cornerstones of treatments, and I agree with your emphasis on this principle. I still think, however, about self-determination as one of many competing interests in health care.
I think no one operates or should operate in a vacuum when it comes to health (care). When it comes to experimental treatments and rights to try, a main concern is that by allowing an individual access outside the trial system, this system towards safe and efficacious medicine is under threat, potentially compromising future benefit and risking a setback in science. This would affect individuals and the public at large. We should avoid this. We are all benefactors of sound (and unsound) scientific endeavors from the past, and we should carry this forward. Especially where individuals pursue unreasonable demands on medicine, we are all affected in this way.
The question that one may raise is if the individual is responsible for this. Does the sole individual’s pursuit for an experimental treatment matter? Does the sole individual’s claim on resources, monetary or otherwise, really matter in the big scheme (the costs of this individual’s pursuit are probably negligible). I think they do. Individuals’ pursuits may therefore be curtailed, in my opinion, especially where they are unreasonable. There is a system in place for individuals to access unapproved drugs: via compassionate use access. The right to try laws are just a less regulated avenue that does not have the oversight that we need to maintain integrity of the system.
Thank you for an insightful post and introduction to an important conversation.
Just yesterday, I actually found myself wondering why discussion of the Charlie Gard case has so quickly escaped from mainstream media. Of course, there are obvious reasons for this, but I thank you for continuing the discussion.
I agree that HCPs should push back on the “right to try” legislation, but I am curious about your qualification: “Most importantly, by limiting FDA oversight, [the legislation] undermines safeguards and trust in the system”.
Is this fear of losing “trust in the system” a justifiable and rational fear among HCPs?
It is my opinion that patients and families are far too trusting of said “system” and should be emboldened to question and search for answers that may fall among the entirety of wellness, though still valuing input from HCPs and weighing personal and cultural beliefs in the determination of critical health care decisions. For example– as you too have mentioned, I would advocate for the weighing of personal and cultural pros and cons of the resuscitation of elderly family members before trusting and following a certain HCPs quick advice to resuscitate (or not).
That said, I am still in agreeance of your position against “right to try” for reasons of FHH and the “undermining of safeguards”, but I am critical of the “most important” reason (being undermining trust in the system) for doing so.
Dear Terah, thanks very much for your reply. In my answer to Zoie, I’ve explained a little bit about how ‘rights to try’ are seen as a threat to the integrity of clinical trials system. I hope this addresses your concern too. If you desire more info, please let me know. My main problem with the ‘right to try’ laws and the FHH are primarily that unreasonable requests for ‘experimental treatments’, probably mostly serve to mitigate grief. Unreasonably postponing imminent death, to great costs ($, emotional, social etc) I don’t think falls within the goals of medicine. I’m working on elaborating the ideas around the FHH, so keep an eye out for this in case you are interested.
I must admit that I was unaware of the Charlie Gard case and after reading your blog I took some time to read up on the case. I agree with you in your statement that the “HCP has an obligation to avoid collaborating in, participating, propagating or augmenting false hope”. I believe that it is important for health care providers to be honest with patients and families who ultimately have an illness or disease that is untreatable. However, I also find myself struggling to come to terms with not letting a patient or patient’s family decide what they want to do with their body and their physical health. I believe that individuals have a right to know all their options and make decisions based on what is available to them but I understand that health care providers don’t want patients to partake in something that could cause harm and ultimately not help them with their health. Questions I have is how do people find out about these drugs that are just in phase 1 testing? Is this information found through patients and their families doing research? Or is information about these treatments given by health care providers? If health care providers do give out the information about these treatments do they also tell the patients if it is a possible option for them or do they just tell them it is ruled out?
Dear Eva, I empathize with you and physicians who have to do this ‘challenging work’, i.e. balancing conflict, disappointment, grief, expertise, hope and sadness. I only have to write about it, while sitting behind my desk, and now and then talk about this in my clinical ethics practice. ClinicalTrials.gov has information about phase I clinical trials, but I doubt that many individuals obtain information about Phase I tests this way. I suspect more individuals browse the internet by diagnosis. In Charlie Gard’s case it was exactly like that: parents browsed the internet and found an expert. His ‘treatment’ was not in Phase I.
Practitioners are care continuously navigating hope, clinical realities and ‘wisdom’ of the Internet. Practitioners are navigating different levels of ‘expertise’, with the patient/family often having become an expert via the Internet. I don’t think that many providers offer suggestions that they haven’t heard about and are not (yet) evidence based, unless they have somehow studied these ‘alternative/ experimental’ interventions more in depth.
Practitioners have tough jobs. Sometimes I worry that interventions are just offered ( complied with requests) because patients/families ask for it, without good reason. Providers comply just to avoid conflict, to make life easier, to make the job easier, and under a specific interpretation of what it means to be compassionate: i do it because you want it, i do not want to fight you, to elevate trust etc etc. But distrust won’t go away by mere compliance, also the grief experienced with ‘false hope’ isn’t helpful. Trust and acceptance is hard to accomplish in a fragmented system, but allowing patients to live into False Hope isn’t the answer.
I definitely agree that HCP’s should oppose the “right to try” bill. It seems like there is an exploitation of patient’s hope that an experimental drug MIGHT work when in reality, it might cause more harm than good if a patient is on their last breath. As a medical professional, you should be truthful when discussing different options that patient’s have in terms of treatment. I thought it was great how there was a mention of if this bill passed their would be a potential to undermine the clinical trial system. This is a great point.
Thank you for your comment. Acts undermining the clinical trial system, and risking a setback in science would affect both individuals and society as a whole. But these concerns are hard to grasp for a patient him/herself, or for an individual whose loved one is on their death bed. Talking to the individual about these concerns is maybe asking too much, but I think it needs to be a concern on the providers’ mind.
I am also in agreement that HCP should resist the “right to try” legislation. This blog discussion reminded me of Grand Rounds at Sparrow last week. Sparrow’s board was introducing/implementing a new policy, withholding/withdrawing non-beneficial treatment. This policy states that healthcare providers have the right to withhold/withdraw non-beneficial treatment to patients, particularly patients who are terminally ill. Previously individual physicians had the right to do this but did not have support from the hospital. Now in an effort to make physicians more comfortable with the policy, Sparrow is supporting their physicians’ decisions.
I am completely supportive of this policy, but what was shocking was that during Grand Rounds some of the medical staff did not grasp the concept of withholding/withdrawing non-beneficial treatment and instead discussed other ways they could have helped the patients in the case examples, similarly to a morbidity and mortality review. How do you think healthcare providers/social workers should proceed in educating people about these policies?
This is a great question: How can healthcare providers/social workers proceed in educating people about ‘Non-Beneficial Treatment policies’ or, ‘Potentially Inappropriate Treatment Policies’ (http://www.atsjournals.org/doi/abs/10.1164/rccm.201505-0924ST)?. I think your question asks for a lengthy reply, some of which I can write here. Please feel free to contact me if you like to brainstorm more.
Let me say from the outset, that I wholeheartedly agree with these policies and a procedural justice approach to intractable conflicts about ‘futility’* disputes. I also agree that providers may need such policies in a litigious society, where there is not a lot of trust in medicine/providers. Finally, I also agree that such policies are an expression of support from the system (state legislator or local institution/hospital system) for the provider.
Still, I worry that my answer to your question is not going to be popular, or make things easier:
I do not think it is the role of providers to teach people, i.e. patients/providers, about ‘policies’. Policies should not be used as a tool to avoid conversation, to set up barriers, or stand in the way of a compassionate interaction/ patient-provider relationship. Providers’ roles are to take care of patients and to work within the goals of medicine. I would hope that by explaining and communicating that a provider’s function is to do no harm!, that medicine has limits to what it can offer and that one is sorry for the patient, there is no need to reference policy. By communicating compassionately, I hope that providers can establish patient trust.
I think it is the role of providers to show moral courage. I hope that providers feel enough courage to engage in a conversation with the patient that ‘the patient’s wishes’ are important, but that demands can’t force ‘miracles’. Policies can help to support ‘moral courage’ to explain that certain interventions are more harmful than beneficial, and that demands are unreasonable, but they cannot substitute it.
If there is anyone that needs to educate patients, I believe it is the system’s responsibility, not the providers’. I think the system, i.e. government or local institution need to educate patients about the need to trust providers, commitment to respectful conversation and communication, and that provider-patient relationships are about shared decision-making. They will also have to communicate, in a compassionate way, that patient demands cannot hold the providers hostage.
To conclude, I believe that all of us in education, in health care, in government have a role to communicate (and educate) everyone about the following: Commitment to autonomy and hearing the voice of patients is an important principle in health care, but it is not the only one. We cannot just rely on policy to explain that commitment.
*I don’t like the word ‘futility’, and prefer alternatives like Non Beneficial, ‘Potentially Inappropriate’. However, I acknowledge that the word ‘futility’ is still very often used as a shorthand, and an easy term to use.
I’m also in agreement that HCP’s should resist “right to try” legislation initiatives. This sounds like it is just another way of taking advantage of a person during their most vulnerable stages of life, it should be the doctor ethical decision to protect the comfort of the patient, specially because the doctors are in the position of power. Ethically, I do not think it is right to sale hope to people when they are desperately trying to live.
Thank you for your comment. I completely agree with your observations.