Case Presentation

A 9‐year‐old boy was born with bicuspid aortic stenosis, underwent two unsuccessful aortic balloon valvuloplasty interventions, and eventually developed restrictive cardiomyopathy and diastolic dysfunction caused by LVEFE at 2 years of age [21]. The attending cardiologist referred the patient to a high‐volume, high‐profile congenital heart disease program 1000 miles away with vast experience with LVEFE resection in a handful of patients with good results. The patient was evaluated there and thought to be a reasonable candidate for a Ross operation with concomitant LVEFE resection. Owing to problems with insurance coverage, the parents sought other options, including paying out of pocket to have the operation at the high‐volume program, as well as approaching local programs in their home state for second opinions [21].

During this time, the parents interviewed a well‐respected congenital heart disease surgeon for his opinion and operative experience [21]. The surgeon had considerable experience with the Ross operation, including superior results without mortality and with high freedom from reoperation. He had no experience with LVEFE resection. When specifically asked about this part of the proposed procedure, he described his results with the Ross operation and volunteered that although he had no experience with LVEFE resection, he had considerable experience with right ventricular endocardial fibroelastosis (RVEFE) resection owing to his expertise with arrhythmia surgery for tetralogy of Fallot, which requires RVEFE resection for ventricular tachycardia. In making such a statement, the surgeon implied that there is little difference between RVEFE and LVEFE resection. The surgeon was aware of the recommendation from the high‐profile institution and offered to perform the operation with low risk of mortality based on his overall experience. He engaged in comprehensive informed consent by describing the nature, risks, and alternatives of the operation with special emphasis on the Ross procedure, thinking that the difference between RVEFE and LVEFE is slight. He stressed that the local institution could provide an equivalent surgical procedure with comparable outcomes without the patient having to travel. The surgeon then left the decision to the family [21].

Commentary

A full discussion of this case and the ethical principles associated therewith is dependent on a proper understanding of the major bioethical principles [21]. These principles include respect for patient autonomy, beneficence, nonmaleficence, and justice [1, 21]. Each of these principles can be applied to the present case. The principle of patient autonomy is perhaps the most important, since many of the issues involved with this case are inherent to the process of informed consent. The principles of beneficence and nonmaleficence are often conflated. Acting in a patient’s best interest and ensuring that no harm is caused are often the same. In this case, the surgeon’s selectivity in emphasizing his experience with certain aspects of the case and his ability to offer the same outcomes as the regional center can be assessed in terms of these principles. The principle of justice concerns the equitable distribution of economic, emotional, and societal burdens and benefits. This principle can be applied to several aspects of this case that are not directly associated with the physician–patient interaction but are nonetheless essential to an understanding of the ethical issues in contention. Such components of this case might include the additional financial and emotional hardship that traveling for care might confer on the family, and how such potential hardships might interact with the additional bioethical principles that are in apposition [21].

As a conscientious professional, the surgeon tried to adhere to the fundamental ethical principles that frame responsible medical practice [21]. However, there are times when differing principles can make conflicting demands on the physician, and it is not always clear how these conflicting demands are reconciled.

This particular case is best understood in terms of tension between the imperatives of beneficence and the obligation to respect patient autonomy [16, 18, 21]. The first of these principles tells the surgeon to act in the best interest of the patient. The second principle tells the surgeon to provide the patient with as much information and explanation as needed in order to make an informed decision. As is often the case, these two principles are in some tension with each other [21].

The surgeon in this case has a clear conviction about what is in the best interest of the patient [21]. He is confident that he can perform the Ross procedure as well as anyone in the country (indeed, better than most), and he believes that his experience with RVEFE resection will provide him the expertise to accomplish LVEFE resection. He acknowledges to himself that performing the resection on the left side is perhaps more complex and more difficult than on the right side, but does not think that this will pose a problem. In addition, he considers that the family will be better off staying close to home rather than traveling 1000 miles to the more high‐profile institution. The out‐of‐pocket costs to the young family will be reduced as well. All things considered, the surgeon judges that it is in the patient’s best interest to perform the procedure at the local children’s hospital [21].

Because the surgeon sees it as his obligation to encourage the family to stay locally and allow him to perform the operation, there are various ways he could influence the parents to agree, but not all of them would respect patient autonomy [21]. Following are a few methods that could be used: he lies, uses emotional blackmail, dabbles in willful obfuscation, or pressures by fear‐mongering. These methods might work, but they would clearly violate the surgeon’s obligation to respect patient autonomy, as they do not result in a truly informed, rational decision on the part of the patient. Rather, these methods result in the patient or parents being manipulated by nonrational, in effect coercive, means to secure agreement [21].

If the surgeon is correct and bases his judgment on rational considerations, the parents do not need to be manipulated into agreeing [21]. They need to be provided with only the relevant facts and allowed to draw the right conclusions. If the parents are provided with the same information that persuaded the surgeon, and if they are helped to see this information in the same light that he sees it, then the parents will agree. The process therefore will have been an ideal case of informed consent and thus maximal respect for patient autonomy [21].

Rarely are these ideals actually fulfilled in practice, especially in complicated cases [21]. The parents are informed individuals with internet skills and intellectual curiosity, but short of taking anatomy, embryology, and pathology courses, the nuances of the anatomic and functional asymmetry of the two sides of the heart may be lost in translation. Except for the drawings that the surgeon shows the parents, the Ross operation is an abstraction to them, and LVEFE resection does not actually register complete understanding despite all metaphors used to explain the operation [21].

And herein lies the crux of this case [21]. The surgeon holds that his obligation to do what is in the best interest of the patient requires that he convince the parents to authorize him to perform the operation. But the facts and evidence that convince him are not really available to the parents, nor can he, with the very best of intentions, make these facts available to them. He does not want to be overtly manipulative or disrespectfully paternalistic, but he does want to encourage the parents to agree with him. How can he convince them to act in the best interests of their child while respecting their autonomy [21]?

Rhetoric, Persuasion, and Psychology

Philosophers from Ancient Greece had thought about ways in which rational and nonrational factors influence a person’s health and decisions [21]. Early teachers in classical Athens (often called – nonpejoratively – “Sophists”) claimed to be able to teach the art of persuasion, the art of convincing someone else to agree or share your beliefs. Occasionally this art of persuasion relies on strictly rational factors, such as logic and evidence. But nonrational factors can be brought to bear in the effort to persuade as well. In the ancient world, the art of persuasion was sometimes called rhetoric and was rightly thought to be of great importance in politics, education, commerce, and even family life. In Aristotle’s study of the subject, Rhetoric [33], he defines it as “the faculty of observing in any given case the available means of persuasion” [21].

An interesting passage from Plato’s Gorgias [34] highlights the importance of intense persuasion over informed consent [21]. Gorgias uses persuasion techniques to help his brother, who is a physician, convince his patient to “lie down and undergo the knife” for therapeutic reasons (Gorgias, 456b). Gorgias, when questioned, infers that his technique is applied not necessarily for the good of the patient, but for Sophistic principles of persuasion [21].

Gorgias replies, “Ah, if only you knew all, Socrates, and realized that rhetoric includes practically all other faculties under her control. And I will give you good proof of this. I have often, along with my brother and with other physicians, visited one of their patients who refused to drink his medicine or submit to the surgeon’s knife or cautery, and when the doctor was unable to persuade them, I did so, by no other art but rhetoric. And I claim too that, if a rhetorician and a doctor visited any city you like to name and they had to contend in argument before the Assembly or any other gathering as to which of the two should be chosen as doctor, the doctor would be nowhere, but the man who could speak would be chosen, if he wished” [21, 34].

In the modern world, volition, motivation, and cognition have been studied in great depth to ascertain what factors play a role in influencing decision‐making and belief formation [21]. Psychologists, sometimes in the spirit of pure research and sometimes in the service of high‐paying advertisers or politicians, have studied the methods by which people can be persuaded to embrace a certain belief, buy a certain product, or vote for a certain candidate. While this is hardly an exact science, significant progress has been made, and we have now a better understanding than ever before of the ways in which we can influence the beliefs of others and, in turn, how they can influence us [21].

Surgeon Responsibility and Psychological Techniques of Persuasion

After the issues of informed consent were discussed, the surgeon in the case being discussed asked his nurse practitioner to stay with the parents in his absence to answer further questions the family might have [21]. It was during this time that the nurse practitioner confirmed the surgeon’s expertise, emphasized his favorable reputation, and allowed the family to ask questions that perhaps they were uncomfortable asking him directly. She also noted the long‐distance travel of 1000 miles to the high‐profile institution and how their family support system and geographic familiarity would help during the recuperation period. The family confirmed that they liked the surgeon and found comfort that a member of the team, namely his nurse practitioner, thought so highly of him. They decided to have the operation at the local institution [21].

The surgeon knew that he had the support and loyalty of his nurse practitioner, who had witnessed his excellent results over a five‐year period [21]. He was comfortable leaving his nurse practitioner alone with the family, knowing that she would underscore the benefits of staying at the local institution. It is of some interest that the nurse practitioner is also financially dependent on the continued clinical volume and well‐being of the surgical program. Many provocative ethical questions arise. Was the informed consent comprehensive enough? Was there selective emphasis on the Ross operation over the LVEFE resection? Did the surgeon consult the literature to explore the potential differences between LVEFE and RVEFE resection? Does he, in fact, have the expertise to perform this part of the operation? Was there willful or unwillful deception on the part of the surgeon or his nurse practitioner? In his mind, perhaps he determined that too much information would confuse and scare the parents at a time when he sensed that they wanted confidence. What techniques of persuasion are permissible that do not step over the line into coercion and manipulation [21]?

Selective Emphasis

During the process of informed consent, the surgeon does not give all aspects and every nuance equal weight [21]. Certain conditions or potential complications seem more salient and are emphasized. Others are very rare [16, 21] and are mentioned but not emphasized. There is no intention of concealment per se, but the intent is to give proper weight to the various factors at hand. In this case, the surgeon believes that the more difficult part of the procedure is the Ross operation, which will, in his mind, make the LVEFE resection easier owing to the increased exposure after native aortic valve resection. He therefore stresses the former over the latter, even though he has no experience with LVEFE resection. He truly believes that he is capable of performing this operation and has employed persuasive measures to convince the parents to have the operation at his institution [21].

Beneficent Persuasion

Beneficent persuasion permits physicians to employ decision‐making psychology to influence patient behavior in a manner that will favor the patients’ long‐term health [21, 35]. Physicians have a moral duty to enhance and improve patient well‐being while respecting patient autonomy. Beneficent persuasion is ethically justified under these circumstances. Swindell and associates note that beneficent persuasion through empathy, respect, and negotiation includes several techniques, such as introducing vivid depictions of possible negative outcomes, providing default options to the patient, encouraging patients to think about regret for lost opportunities if medical recommendations are not followed, as well as framing and refocusing [35]. Framing is a technique that can be implemented by noting the benefits of the procedure first, then discussing the risks or side effects, and finally concluding the interaction by once more repeating the benefits. Refocusing reminds patients of past physical and emotional challenges that they have overcome by stressing the end result rather than the temporary effects of pain and suffering during recuperation [21, 35].

Nudging and Informed Consent

Recent reports have highlighted libertarian paternalism as a way of influencing individual decision‐making that “makes choosers better off while preserving freedom of choice” [21, 36–39], thereby concurrently merging patient autonomy and medical beneficence. Libertarian paternalism acts as a nudge, helping to “alter people’s behavior in a predictable way,” and is “paternalistic in that it aims to influence people through means other than rational persuasion to make choices perceived as good for them without threatening their liberty” [36]. Based on a report by Cohen [37], prompting individual decision‐making or nudging allows the chooser to benefit without affecting his or her free choice. Nudging, if performed correctly and ethically, can bridge the gap between the duty to respect patient autonomy and beneficence. This posture resembles paternalism and can be appropriate as long as it is ethically legitimate [39]. Nudging, like libertarian paternalism, recruits the use of “choice architects” [38] who construct contexts in which people make decisions by changing the default choice. In the case of the surgeon and family, the default choice is to have the operation; the optout choice, which requires some destructive action, is not to have the operation or to have it at another institution. Either choice must be easy and transparent [39]. In reports by Cohen [37] and Thaler and Sunstein [38], nudging must not be used to coerce people into making a specific decision, but rather must adhere to three guiding principles: all nudging must be transparent and never misleading; it must be easy to opt out of the nudge; and nudging must be with the purpose of improving the welfare of those being nudged [21, 39].

Shared Decision‐Making

Decision‐making is profoundly influenced by an individual’s sentiments, spirituality, personal beliefs, society’s tenets, and the law [18, 21]. Whether by life‐long learning, societal teaching, or providential influence, humans are capable of discerning moral choices by perceptual acuity, patterns of attention, capacity for affective resonance with others, and ingrained tendency to do what the individual knows to be the right thing to do [18]. Charles and colleagues [40–42] crafted a context for shared treatment decision‐making with reference to the doctor–patient relationship. This was developed in the context of a “life‐threatening disease where several treatment options were available with different possible outcomes” in a specialist oncology practice for early‐stage breast cancer. The doctor–patient interaction model contained characteristics of paternalism and also left room for the three components of shared decision‐making: information exchange, deliberation, and negotiation on treatment plan implementation [21, 40–42].

Competency, Transparency, and Informed Consent

A fundamental dilemma that often arises in clinical surgical practice concerns assessing and performing new procedures, especially in rare cases, in which the collective global experience is scant [21]. General principles dictate that when confronted with such a challenge, practitioners consult the known literature, visit other programs with more experience, and prepare their proposed operation with forethought and comprehensive planning. The looming question in this case remains; does the surgeon have the expertise to perform LVEFE resection based on his stellar experience with RVEFE resection, and is he adequately prepared? To make a comparison to another surgical subspecialty, does a board‐certified general surgeon who has proven expertise in colon surgery possess the expertise to perform complex pancreatic surgery? This is a common dilemma and is attended by surgeon age, clinical experience, and technical expertise. At some point, in order to attain surgical experience, one has to perform independent surgery. Our value system calls for board certification, peer‐reviewed hospital privileging, outcomes analysis, and reputation to establish perceived competency. These achievements notwithstanding, the assessment of skill when it comes to rare lesions and previously not performed operations remains problematic [21].

At first blush the right ventricle is thinner, expected to produce pulmonary pressure (not systemic pressure), and is probably amenable to reparative procedures if the RVEFE resection proves to cause iatrogenic injury to papillary muscles, cords, valve leaflets, and the ventricular wall [21]. The left ventricle is thicker, expected to support the systemic circulation with higher pressures, and less amenable to reparative procedures in the event of unwanted iatrogenic injuries that are noted for the RVEFE resection [21].

As expected, a comprehensive review (PubMed) of the differences between RVEFE and LVEFE resection proved fleeting [21]. Most literature citations concerning endocardial resection, other than what was published by the high‐volume institution [43, 44], were found in relation to ventricular tachycardia/fibrillation treatment in the left ventricle that occasionally required papillary muscle translocation, localized ventriculotomy, and localized cryoablation [45]. The approach to the left ventricle in these patients was transaortic and through the mitral valve. Both exposures are challenging for an extensive endocardial fibroelastosis resection and raise the possibility of unwanted complications. References to RVEFE were mostly confined to postoperative tetralogy of Fallot patients with pulmonary regurgitation, right ventricular dysfunction, and ventricular tachycardia [46]. A recent study abandoned RVEFE resection in these patients, preferring instead placement of selected cryoablation lesions between the ventricular septal defect patch and the pulmonary annulus [47]. Some, however, still perform RVEFE resection in addition to the cryoablation lesion [46], and under these circumstances it is performed through the existing right ventriculotomy with superior exposure, making unwanted surgical mishaps less prevalent than those for the left ventricle. The surgeon therefore is left to review the comparative anatomy of the right and left ventricles, recognize the contrasting exposures, and be prepared to perform the LVEFE resection taking into consideration the team’s experience with RVEFE and making the necessary adjustments to perform a successful LVEFE resection [21].

This cursory literature review and the intuitive complexity of the LVEFE resection over the RVEFE resection would leave the surgeon with the idea that the LVEFE resection will be a more difficult operation with the potential for far more serious complications [21]. The surgeon does not explain this difference to the family, perhaps because he has not considered the possibilities, he has not consulted the literature, or he truly believes that there is no material difference between the two procedures. Is this hubris or is it confidence? Should he be more circumspect about the LVEFE resection and offer epistemological modesty? After all, he could be wrong about how easy the procedure in the left ventricle will be. Let us not forget that the Ross procedure in which he has demonstrated expertise is probably the more difficult part of this combined procedure and affords very good exposure for the LVEFE resection. However, unsubstantiated confidence, while comforting to the patient and parents, may not be appropriate. On the other hand, overly cautious comments that emphasize all the differences in the procedures may erode patient confidence. In the end, is the surgeon competent enough to perform the operation? The studied answer has to be “yes,” but the potential complications were not explained sufficiently, as evidenced by absence of a focused literature search and biased informed consent [21].

Autonomy and Transparency

Pediatric cardiac surgery clinical outcomes continue to improve [21, 48]. Transparent public reporting of pediatric cardiac surgical results is becoming increasingly common [49–57]. The justification for this transparency is based on the following principles:

• Variation in pediatric cardiac surgical outcomes exists [53, 54].
  
• Patients and their families have the right to know the outcomes of the treatments that they will receive [49–52].
  
• It is our professional responsibility to share this information with patients in a format that they can understand [49–52].

The surgeon is faced with the unusual challenge of caring for a patient with a rare lesion [21]. This challenge is associated with multiple conundrums, including the moral dilemma of how public reporting can help regarding an operation that is so rare that only a few people in the world have actually performed it. In other words, although public reporting and transparency are virtuous, these qualities are complicated in the setting of a rare diagnosis and avant‐garde surgery [21].

Surgeons are confronted with a moral dilemma when asked to transparently share their expected outcomes of a rare operation [21]. The principles of honesty and transparency help solve this dilemma. The logical solution is to share with the parents and family the rare nature of the proposed operation and then to extrapolate the expected outcomes based on the known outcomes of operations of similar complexity and expected risk. This approach capitalizes on available data and respects the principles of honesty and transparency [21].

Clinical Outcome

In the end, the patient was taken to the operating room with preoperative and intraoperative assessment that showed aortic stenosis/regurgitation, left ventricular restrictive cardiomyopathy (left ventricular end diastolic pressure 37 torr), and diminished systolic function [21]. The preoperative plan was to perform a Ross operation in conjunction with LVEFE resection. Preparations were made for transaortic and transmitral exposure, retrograde cardioplegia, and moderate systemic hypothermia. After aortic transection, the bicuspid aortic valve showed a well‐formed raphe that was supported by a fused commissure and two deep and competent leaflets on either side of the raphe. The fused raphe was incised, thereby repairing the aortic stenosis and forming a trileaflet aortic valve that appeared competent. At this point, it was determined that a Ross operation would not be necessary and that a mild degree of postoperative stenosis and regurgitation would be preferable to a Ross operation. The index operation therefore was changed to LVEFE resection. The LVEFE resection was performed through the aortic valve and the mitral valve orifice after proper exposure was attained through the interatrial groove. The fibrous peel was approached at the apex of the left ventricle and carefully resected by deliberate and precise scissor dissection to delineate the plane between the fibrous peel and the viable myocardium. The resection was accomplished in stages, with frequent changes of transaortic and transmitral operative exposure. The papillary muscles were preserved, the integrity of the ventricular wall was maintained, and the chordae were left undisturbed. Nevertheless, the operation proved to be longer, more involved, and more challenging than any RVEFE resection performed in the past. Postoperatively, ventricular function and left ventricular end diastolic pressure improved. The left ventricular end diastolic pressure was measured to be 12 torr and compared favorably with 37 torr as measured preoperatively. Intraoperative transesophageal echocardiography revealed minimal aortic stenosis and insufficiency, excellent relaxation of the left ventricle, and a considerably smaller left atrium. The patient had an uncomplicated postoperative course with significantly improved symptoms and functional status [21].

Government‐Level Funding

In 2014, the United States allocated $2 billion to research in cardiovascular disease and $5.4 billion to research in cancer [19, 63]. Considering that these two disease processes accounted for roughly 600,000 deaths each in 2013, and each accounted for more deaths than the next four most common causes of death combined, this prioritization of healthcare resource allocation is unsurprising [64]. By contrast, $3.6 billion were allocated to the category of orphan diseases [19, 63].

Implicit in this government‐level resource allocation are recognition and prioritization of the major threats to public health based on disease prevalence [19]. Indeed, a further screening of the National Institutes of Health Research Resource Allocation Report yields a correlation between disease prevalence and funding amount for research [63]. Other funding mechanisms do exist on both the micro scale (hospital‐wide resource allocation, insurance company reimbursement for disease burden) as well as the macro scale (philanthropic organizations, professional societies, etc.) that supplement government resource allocation [19].

Acquired cardiovascular disease healthcare costs totaled approximately $444 billion in 2010 [65], a paradigm of high incidence and high cost [19]. The significant resource allocation to this disease is thus easy to justify, because it is a common disease that kills a large percentage of Americans and represents a significant fraction of healthcare spending. It is difficult to assess the healthcare costs of the 30 million Americans affected by disease, because there is considerable variation in healthcare costs. Neonatal surgery for some of the rarer forms of congenital heart disease such as hypoplastic left heart syndrome and transposition of the great arteries carries the most expensive hospitalizations among birth defects [66]. Furthermore, 6 out of the top 10 most expensive birth defect–related hospitalizations are congenital heart defects [67, 68]. From a resource allocation standpoint, congenital heart disease represents a significant problem because of the small population affected and the high cost of disease treatment [19].

Utility, Economics, and Resource Allocation

While the specific issue of government‐directed healthcare resource allocation is a relatively recent ethical and public policy problem and would have been foreign to its initial proponents, the philosophical school of utilitarianism offers key insights and appears to have had a significant impact on modern healthcare economics [19, 69]. The utilitarian school of thought was first described by Jeremy Bentham and later John Stuart Mill, and its central thesis is defined in Mill’s 1863 work Utilitarianism as “actions are right in proportion as they tend to promote happiness, wrong as they tend to produce the reverse of happiness … happiness … is not the agent’s own happiness, but that of all concerned. As between his own happiness and that of others, utilitarianism requires him to be as strictly impartial as a disinterested and benevolent spectator” [19, 70].

Utilitarianism espouses a kind of detached maximization of utility, or happiness, of the many even if it is at the expense of the few or the individual [19]. The resulting utilitarian ethics become strictly democratic and provide little, if any, consideration of the needs of the minority or of groups who may require more resources to achieve the same level of utility as the majority. The ethical issues associated with this school of thought have been debated since its initial publication [19].

However, while the origins of utilitarianism and modern healthcare economics are separated by roughly a century, the kind of detached maximization of societal benefit that utilitarianism espouses represents one of the fundamental goals of healthcare economics [19]. Healthcare economics starts off with the familiar preface that resources are finite and must be rationed. Three main questions follow: what goods and services shall be produced; how shall they be produced; and who shall receive them? While basic economics might defer to the market to answer these questions, one of the most important problems and the basis of inquiry for both economics and healthcare economics is how to manage so called “market failures.” Having market forces determine healthcare resource allocation has several potential problems stemming from information asymmetry (physicians have greater knowledge than patients): healthcare should be provided based on need and not ability to pay, certain treatments may not be profitable, and others. These problems form the basis for the myriad economic models that attempt to answer the three main questions while maximizing the wellness of society [19, 71].

The conflicts associated with prioritizing the interests of the many over the interests of the few while maintaining equitable access to healthcare form the basis of market failures, and the science and practice of healthcare economics involve creating economic models to describe and ultimately address these market failures [19]. Examples of such economic models include cost–benefit analysis, cost‐effectiveness analysis, needs assessment, marginal analysis, and quality‐adjusted life years. These models are designed to inform policy decisions by quantifying aspects of resource allocation that are often difficult to quantify, such as the benefits to society of treating certain diseases, the value of an individual life, the difference in a human life’s worth in the case of significant morbidity, or the point at which resources should no longer be allocated to a disease process treatment because of futility. These topics may seem more apropos to an ethical discussion and the approaches of ethicists and economists may seem completely different at first, but the strongly utilitarian ethos behind healthcare economics places the resulting economic arguments within an ethical framework. Quantification and evaluation of seemingly ethical variables and problems become just another way to inform the fundamental ethical problem of resource allocation [19, 71].

Healthcare Economics

Because it is easier to quantify treatment costs rather than more abstract concepts such as quality of life, many of the initial and simple healthcare economic models involve an assessment of healthcare costs [19]. In its most basic form, a cost of illness study is just that, and it is used for this purpose. Treatments can then be assessed based on both the direct costs of treatment as well as any decreased healthcare resource utilization after treatment. While these are among the more straightforward measures of healthcare resource allocation, they inform additional models that balance cost of disease with either strictly monetary benefit to either individuals or society, as is the case in cost–benefit analysis, or with nonmonetary measures of treatment efficacy, as is the case in cost‐effectiveness analysis. In presenting an objective assessment of the costs associated with a treatment, cost‐based analyses can help to supplant sometimes subjective and political decisions with mathematical and economic decisions. Opponents of these models argue that these analyses are rarely thorough and comprehensive, and that, depending on the data they use, have the potential to be just as subjective and political as the decisions they are meant to replace [1, 19].

Rare and expensive disease resource allocation provides a significant challenge to such analyses, particularly in the case of treatment equipoise and uncertain prognosis [19]. Sample size is paramount to establishing the assumptions and basis for cost necessary for analysis; in the case of complex congenital heart disease, sample size is generally low, and there are often significant discrepancies with healthcare costs, as evidenced by recent analyses on the subject [66]. Postoperative morbidity suffered by patients can also be highly variable in the case of complex congenital heart disease, further confounding analyses of outcomes to provide any prospective, normative guidance regarding preferred or economically beneficial treatment options. Furthermore, the time frame of such an analysis may confound analysis of disease processes that require timely, early, and expensive surgical intervention if it does not sufficiently take into account the years of hospitalizations and untimely death of a patient who does not undergo corrective surgery [19].

Because the alternative for critical congenital heart disease is often death, cost‐based analyses and healthcare economics in general must at least implicitly place a value on a human life for the purposes of analysis [19]. The most common form of so‐called cost–utility analysis is quality‐adjusted life years. The basic premise of this metric is that, if a year of additional life in good health is worth 1, then a year of additional life in poor health must be worth less than 1 [72]. Varying degrees of disability and morbidity are associated with a decimal value (utility value) that corresponds with how studied individuals would rate quality of life associated with a given condition. If a degree of human suffering carries with it a detrimental cost to quality of life, it is implied that the healthy life also has a value associated with it [19].

Such implicit valuation of human life becomes problematic when it is made explicit, as is the case in the debate over the value of a single quality‐adjusted life year and whether treatments for it should be offered or paid for [19]. To be sure, there is a point of healthcare resource spending beyond which no further benefit can be achieved, either because the patient has been cured or because futility of care has been reached. Marginal analysis, the healthcare economic model dedicated to defining such a point, has been very helpful in establishing guidelines and informing medical decision‐making [71]. The debate over what constitutes an appropriate amount of healthcare resource allocation to an individual in a given year is contentious and the origins of what are generally considered acceptable figures for quality‐adjusted life years are somewhat murky. A common figure cited is $50,000 as an appropriate amount per year to justify treatment for an individual with a good quality of life [73]. The origin of this figure is said to have arisen from the per annum cost of hemodialysis as it was in the 1970s, but this point is debated. The amount of $50,000 per quality‐adjusted life year gained widespread acceptance in the early 1990s in the world of healthcare policy, and it has endured as a benchmark figure for healthcare policy decisions [73]. It should be noted that this figure is used primarily as a model, and that the favorable economic environment of the US healthcare system has not forced the issue of using such a value to deny treatments to individual citizens to this point. That the $50,000 per quality‐adjusted life year has not been formally adjusted for inflation as it remains a benchmark of ethical and economic inquiry is mathematically perplexing, but different organizations have proposed higher quality‐adjusted life‐year values of $100,000 and even $300,000 [19, 73].

The explicit valuation of human life, while perhaps ethically troubling, is nonetheless an important aspect of healthcare resource allocation in a society with finite resources [19]. Such valuation is important in evaluating different treatment modalities for common diseases, but its use is much more challenging in the case of rare and expensive medical problems such as critical congenital heart disease. Maximizing quality‐adjusted life years places a premium on preserving life years, not necessarily individual lives. Critics of quality‐adjusted life years argue that there is a fundamental conflict between a cost‐effectiveness approach similar to the maximization of quality‐adjusted life years and the “‘Rule of Rescue’ – people’s perceived duty to save endangered life when possible” [19, 74].

Many forms of congenital heart disease treatment fall within the scope of rescue [19]. Patients can be in extremis and treatment initiation is often time sensitive. Failure to initiate treatment may result in patient death and, thus, deprivation of their life plan. Hyry et al. argued that quality‐adjusted life years should not be used in cases where the debate is treatment versus no treatment, because of the ethical issues associated with withholding treatment from a certain group for strictly economic reasons [19, 69].

Within the valuation of human life proposed by the limits of quality‐adjusted life years and their use in healthcare economic policy are numerous ethical debates beyond the obvious valuation of human life [19]. Depriving a patient of treatment because of cost violates patient autonomy, and depriving a healthcare worker of the right to initiate treatment to a patient violates the bioethical principle of beneficence. The bioethical principle of justice becomes a matter of debate regarding the proper distribution of healthcare resources, but it is difficult to fathom denying expensive treatments for treatable conditions such as congenital heart disease based solely on economic concerns. Moreover, having limitations on expensive treatment distribution would stifle further development of these treatments that might ultimately lower the cost of treatment. The cost of sequencing the human genome has fallen precipitously owing to technological advances, as have costs for once experimental medications and complicated surgical procedures. Setting cost limits on treatment for otherwise life‐threatening conditions not only commits those afflicted to death, it also prevents research and development of better and perhaps less expensive treatments. Worse still, research into currently expensive technologies and treatments has potential ramifications to benefit more than just those afflicted with the disease by improving medical knowledge and research methods. A society that prioritizes quality‐adjusted life years and uses them as a basis for healthcare resource allocation may allow for the current majority to have access to the currently most economic treatment for the currently most prevalent disease processes. However, by limiting resources for research into rare, expensive, or difficult‐to‐treat disease processes, such a society has the potential to be ill‐prepared to deal with new disease processes and may ultimately be unable to accommodate the future needs of the many by having ignored the past needs of the few [19].

Healthcare cost is becoming increasingly important as demand for and access to medical care increase both domestically and internationally [19]. The debate on healthcare resource allocation and resulting healthcare economics has its roots in utilitarianism through the shared telos of prioritizing the utility of the many over the utility of the few. While healthcare economic models can provide a framework by which to make difficult decisions regarding healthcare resource allocation, the treatment of rare and expensive diseases such as congenital heart disease presents a significant challenge to current healthcare economic models. The shortcomings of various healthcare economic models to inform the healthcare resource allocation for rare diseases debate are similar to the shortcomings of their “philosophical parent” [75]. Putting aside the needs of the few for the needs of the many is a simple idea in concept, but becomes significantly more challenging when it is often difficult to quantify the quality and quantity of life gained through the typically urgent surgical interventions necessary to correct critical congenital heart disease in infancy. Further attempts by economists, ethicists, and clinicians alike will be needed to best inform the practice of treating rare and expensive disease processes such as congenital heart disease. Such policies might combine the objective quantification of healthcare costs and human morbidity that healthcare economics provides while maintaining the bioethical principles of justice, beneficence, nonmaleficence, and autonomy. Growing populations and increasing access to healthcare resources will continue to push this and other important healthcare resource allocation debates to the fore, and a multidisciplinary approach will be vital to ensure that the needs of the many are satisfied, but not always to the detriment of the needs of the few [19].

Respect for Patient Autonomy as a Medical Virtue

There is nearly universal agreement among ethicists and physicians that respect for patient autonomy is an important, even indispensable principle in the ethical practice of medicine [1, 18]. US law recognizes the centrality of patient autonomy by prohibiting, in ordinary circumstances, the imposition of medical treatment on a mentally healthy adult patient without his or her free and informed consent [18, 22]. Of course, there can be conflict, in specific cases, between the physician’s obligation to respect patient autonomy and the physician’s other ethical obligations (e.g., the imperative to do no harm or to respect justice). In the course of everyday medical practice, challenging cases result in ethical dilemmas owing to the many different and sometimes conflicting responsibilities that physicians have to patients, society, and themselves. The field of medical ethics is charged with the study of such conflicts, with the promise that better and more just solutions may be achieved by defining the problems and applying the principles that result in successful and morally just patient care: patient autonomy, nonmaleficence, beneficence, and justice. The purpose of this discussion is to examine the medical ethical principle of patient autonomy by exploring the philosophic origins and underscoring the importance of more formalized and structured ethical training in medical practitioner training [18].

Leaving aside, for the moment, moral perspectives that rely entirely and explicitly on a particular religious faith, the two most influential philosophic approaches to ethics (utilitarian and deontologic theories) agree on the centrality of the principle of respect for patient autonomy [18]. It is interesting to note, however, that they do so for different reasons, which will be examined briefly with the intent to deepen our understanding of the principle and highlight the difficulties that may arise as physicians seek to apply the principle in daily professional practice [18].

Utilitarian and deontologic approaches will be addressed from their respective theoretical perspectives and contrasted with ethical theories that focus on the virtues and vices that characterize people as good or bad [18]. The ethical theories, so‐called virtue ethics, have their origins in the writings of Plato [76, 77] and Aristotle [31], and offer insight both into the debate on patient autonomy and on how ethical thinking can be taught [18, 78]. In the process of this exegesis, a theoretical and clinical basis for the importance of patient autonomy as an ethical tenet and for its incorporation into medical practitioner training will be considered [18].

Centrality of the Principle of Respect for Patient Autonomy

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