Integrating the Voice of the Patient Into the Medical Device Regulatory Process Using Patient Preference Information

The US Food and Drug Administration is one of several US and global agencies making strides to incorporate patient preference information (PPI) into its decision making. PPI has been included in 5 completed medical device marketing decisions to date. Its usage is not more widespread because of uncertainty about how to design “fit-for-purpose” patient preference studies and a lack of standards for the choice of preference elicitation methods, among other reasons. To advance the application of PPI to decision making about medical devices, the Food and Drug Administration has published a guidance document, “Patient Preference Information—Voluntary Submission, Review in Premarket Approval Applications, Humanitarian Device Exemption Applications, and De Novo Requests, and Inclusion in Decision Summaries and Device Labeling.” This article discusses key concepts in the guidance document, in addition to providing lessons learned from the use of PPI for medical device regulatory applications to date and identifying new opportunities to leverage PPI to elevate the patient voice in the medical device product life cycle.     

The medical visit context of treatment decision‐making and the therapeutic relationship

The ascendance of the autonomy paradigm in treatment decision‐making has evolved over the past several decades to the point where few bioethicists would question that it is the guiding value driving health‐care provider behaviour. In achieving quasi‐legal status, decision‐making has come to be regarded as a formality largely removed from the broader context of medical communication and the therapeutic relationship within which care is delivered. Moreover, disregard for individual patient preference, resistance, reluctance, or incompetence has at times produced pro forma and useless autonomy rituals. Failures of this kind, have been largely attributed to the psychological dynamics of the patients, physicians, illnesses, and contexts that characterize the medical decision. There has been little attempt to provide a framework for accommodating or understanding the larger social context and social influences that contribute to this variation. Applying Paulo Freire’s participatory social orientation model to the context of the medical visit suggests a framework for viewing the impact of physicians’ communication behaviours on patients’ capacity for treatment decision‐making. Physicians’ use of communication strategies can act to reinforce an experience of patient dependence or self‐reliance in regard to the patient‐physician relationship generally and treatment decision‐making, in particular. Certain communications enhance patient participation in the medical visit’s dialogue, contribute to patient engagement in problem posing and problem‐solving, and finally, facilitate patient confidence and competence to undertake autonomous action. The purpose of this essay is to place treatment decision‐making within the broader context of the therapeutic relationship, and to describe ways in which routine medical visit communication can accommodate individual patient preferences and help develop and further patient capacity for autonomous decision‐making.     

Integrating Family Visitation and Risk Evaluation: A Practical Bonding Model for Decision Makers

The number of children in foster care remains high, and most disturbing are the numbers of children whose abuse continues after system intervention. Faced by these challenges, child welfare legislation and policy have shifted away from the earlier standard of “reasonable efforts” toward reunification for children in foster care to a standard in which “the best interests of the child” have priority. The new goal calls for greater use of assessment tools in locating those families deemed to be “at risk” for further abuse. Here, we apply a family bonding perspective to address the practical side of risk assessment for those children already in foster care. We present a conceptual model that attaches numerical value to the various components of the parent–child bond and demonstrate how the model can be operationalized during supervised visitations. Using data from a family visitation center, we demonstrate the model's potential for interagency collaboration in the permanency placement decision‐making process.     

Governing through lifestyle—Lalonde and the biopolitical management of public health in Canada

In 1974, the Liberal government of Pierre Trudeau released a “green paper” known as the Lalonde Report, after the health minister at that time. The report formulated perspectives on health and the main concepts and ideas developed in it, particularly the concept of “lifestyle,” which became the foundation of public health policies in many different European countries and the United States. The concept of “lifestyle” connected personal behaviour and habits to the individual health condition; people were not dying due to a lack of access to medical care but because they lived a life prone to personal risk taking. Furthermore, what is seldom discussed is that this report not only propagated the (neo)liberal view of citizens as autonomous rational actors (homo oeconomicus), with personal responsibility for their health, but it was a first step in the transformation of Medicare and went far beyond the question of health promotion. Health was no longer something that happened to a person but was created through personal choice and, therefore, one had to assume responsibility for one's behaviour. Using Foucault's definition of government as the “conduct of conduct,” we will demonstrate that the Lalonde report must be understood as a specific “technology of government” and contributed to a neoliberal transformation of health care despite the fact that the Canadian system of Medicare was based on the idea of universality, meaning citizens had equal access to health care independent of their socio‐economic situation. As we will demonstrate, the Lalonde report undermined this foundation and initiated a profound reorientation, not only of the healthcare system, but even more importantly, it radically changed the way we think about our behaviour around health‐related issues. We will also discuss how the making of the report contributed to the redefinition of politics and demonstrated a lack of concern with liberal‐democratic decision‐making processes.     

Patients' participation in decision‐making in the medical field – ‘projectification’ of patients in a neoliberal framed healthcare system

This article focuses on patients' participation in decision‐making in meetings with healthcare professionals in a healthcare system, based on neoliberal regulations and ideas. Drawing on two constructed empirical cases, primarily from the perspective of patients, this article analyses and discusses the clinical practice around decision‐making meetings within a Foucauldian perspective. Patients' participation in decision‐making can be seen as an offshoot of respect for patient autonomy. A treatment must be chosen, when patients consult physicians. From the perspective of patients, there is a tendency for healthcare professionals to supply the patients with the information that they think are necessary for them to make their own decision. But patients do not always want to be a ‘customer’ in the healthcare system; they want to be a patient, consulting an expert for help and advice, which creates resistance to some parts of the decision‐making process. Both professionals and patients are subject to the structural frame of the medical field, formed of both neoliberal framework and medical logic. The decision‐making competence in relation to the choice of treatment is placed away from the professionals and seen as belonging to the patient. A ‘projectification’ of the patient occurs, whereby the patient becomes responsible for his/her choices in treatment and care and the professionals support him/her with knowledge, preferences, and alternative views, out of which he/she must make his/her own choices, and the responsibility for those choices now and in the future. At the same time, there is a tendency towards de‐professionalization. In that light, participation of patients in decision‐making can be regarded as a tacit governmentality strategy that shapes the location of responsibility between individual and society, and independent patients and healthcare professionals, despite the basically desirable, appropriate, and necessary idea of involving patients in their own situations from a humanistic perspective.     

Making choices about medical interventions: the experience of disabled young people with degenerative conditions

Background Current western policy, including the UK, advocates choice for service users and their families, taking greater control and being more involved in decision making. However, children’s role in health decision making, especially from their own perspective, has received less research attention compared to doctors and parents’ perspectives. Objective To explore the perspective and experiences of disabled young people with degenerative conditions as they face significant medical interventions and engage in decision‐making processes. Design and methods Findings from a longitudinal qualitative study of 10 young people (13–22 years) with degenerative conditions are reported. Individual semi‐structured interviews were conducted with participants over 3 years (2007–2010); the paper reports data from all three interview rounds. Interviews focused on medical intervention choices the young people identified as significant. Results Although the young people in this study felt involved in the medical intervention choices discussed, findings demonstrate a complex and diverse picture of decision making. Results highlighted different decisional roles adopted by the young people, the importance of information heuristics and working with other people whilst engaging in complex processes weighing up different decisional factors. Discussion Young people’s experiences demonstrate the importance of moving beyond viewing health choices as technical or rational decisions. How each young person framed their decision was important. Recognizing this diversity and the importance of emerging themes, such as living a normal life, independence, fear of decisions viewed as ‘irreversible’ and the role of parents and peers in decision making highlights that, there are clear practice implications including, active practitioner listening, sensitivity and continued holistic family working.     

中国临床医师对子宫肌瘤药物治疗的认识及其潜在分类研究

目的：了解我国临床医师对子宫肌瘤药物治疗的认知、需求、选择依据及其对药物治疗子宫肌瘤的期望。方法在国内7个经济发达地区的2级和3级公立医疗机构对妇产科临床医师进行问卷调查,应用多水平Logistic回归模型及潜在类别分析对数据进行深入探讨。结果共在7个省市的57家医院对314名临床医师进行了问卷调查,半数以上受访者拥有硕士或博士学历,职称以主治和副主任为主。受访者均曾使用药物治疗子宫肌瘤,以孕激素受体调节剂（67．52％）和促性腺激素释放素类似物（66．88％）最为常用。选择药物治疗时受访医师对患者年龄（64．01％）、肌瘤大小（59．24％）、患者的意愿（57．32％）、生育功能保留（51．91％）和经济承受力（50．64％）考虑较多。多水平Logistic回归模型分析提示,受访医师药物治疗子宫肌瘤的决定在同一家医院有相似性。经潜在类别分析表明,受访医师子宫肌瘤药物治疗的思维模式可以分为“药物治疗关键因素考量型”（45．86％）、“药物治疗排斥型”（34．06％）以及“药物治疗谨慎型”（20．06％）三种,且硕士及以上学历的医师以第一种类型为主,本科学历者以第三种为主,上述模式与医师的职称无显著相关性（χ2＝8．000,P＝0．237）。结论我国经济发达地区临床医师使用药物治疗子宫肌瘤的比例广泛,但所在医院以及临床医师教育程度对选择药物治疗的决策影响较大。     

How is medical decision‐making shared? The case of haemophilia patients and doctors: the aftermath of the infected blood affair in France

Objective This article looks at how users and doctors in France have rethought the question of shared decision‐making in the clinical field of haemophilia following a major crisis – that of the infected blood affair. Design We did a qualitative survey based on semi‐structured interviews in three regions of France. Setting and participants The interviews covered 31 clinical doctors of haemophilia and 31 users: 21 adult males with severe haemophilia (21/31), infected (14/21) or not (7/21) with HIV, the infected wife of one of the latter (1/31) and nine parents of young patients with severe haemophilia (9/31), either HIV positive (6/9) or negative (3/9). Results and conclusions The results show the infected blood affair to be a major individual and collective ordeal. It has caused users and doctors to rethink their roles within clinical relationships and to develop new ways of sharing medical decision‐making. Prior to the crisis, the dominant model was based upon a distinction between the medical aspect, governed by the doctors, and the psychosocial aspect, which involved the patients and their families. Since the crisis, medicoscientific knowledge has been shared between users and doctors. This general trend nevertheless permits the existence of different patient, family and doctor profiles which in turn correspond to different notions of what a clinical decision should be. Some users remain attached to the idea of complementarity between doctors and patients (new partnership model), whilst others put doctors and patients on an equal footing (negotiation model). On the doctors’ side, whilst some still prefer the initial model for therapeutic decision‐making, the majority have reassessed their perceptions and viewpoints. A certain number believe that decisions should be made by both doctor and patient in accordance with scientific procedures (decision‐making controlled by scientific standards) or regulatory procedures (decision‐making controlled by legal standards). Yet others feel that multiple points of view are acceptable within the decision‐making process (decision‐making model as interactivity).     

Is “end of life” a special case? Connecting Q with survey methods to measure societal support for views on the value of life‐extending treatments

Preference elicitation studies reporting societal views on the relative value of end‐of‐life treatments have produced equivocal results. This paper presents an alternative method, combining Q methodology and survey techniques (Q2S) to determine the distribution of 3 viewpoints on the relative value of end‐of‐life treatments identified in a previous, published, phase of this work. These were Viewpoint 1, “A population perspective: value for money, no special cases”; Viewpoint 2, “Life is precious: valuing life‐extension and patient choice”; and Viewpoint 3, “Valuing wider benefits and opportunity cost: the quality of life and death.” A Q2S survey of 4,902 respondents across the United Kingdom measured agreement with these viewpoints; 37% most agreed with Viewpoint 1, 49% with Viewpoint 2, and 9% with Viewpoint 3. Regression analysis showed associations of viewpoints with gender, level of education, religion, voting preferences, and satisfaction with the NHS. The Q2S approach provides a promising means to investigate how in‐depth views and opinions are represented in the wider population. As demonstrated in this study, there is often more than 1 viewpoint on a topic and methods that seek to estimate that averages may not provide the best guidance for societal decision‐making.     

Measuring catastrophic medical expenditures: Reflections on three issues

In the “basic” approach, medical expenses are catastrophic if they exceed a prespecified percentage of consumption or income; the approach tells us if expenses cause a large percentage reduction in living standards. The ability‐to‐pay (ATP) approach defines expenses as catastrophic if they exceed a prespecified percentage of consumption less expenses on nonmedical necessities or an allowance for them. The paper argues that the ATP approach does not tell us whether expenses are large enough to undermine a household's ability to purchase nonmedical necessities. The paper compares the income‐based and consumption‐based variants of the basic approach, and shows that if the individual is a borrower after a health shock, the income‐based ratio will exceed the consumption‐based ratio, and both will exceed the more theoretically correct Flores et al. ratio; whereas if the individual continues to be a saver after a health shock, the ordering is reversed and the income‐based ratio may not overestimate Flores et al.'s ratio. Last, the paper proposes a lifetime money metric utility (LMMU) approach defining medical expenses as catastrophic in terms of their lifetime consequences. Under certain assumptions, the LMMU and Flores et al. approaches are identical, and neither requires data on how households finance their medical expenses.     

Questioning context: a set of interdisciplinary questions for investigating contextual factors affecting health decision making

Objective To combine insights from multiple disciplines into a set of questions that can be used to investigate contextual factors affecting health decision making. Background Decision‐making processes and outcomes may be shaped by a range of non‐medical or ‘contextual’ factors particular to an individual including social, economic, political, geographical and institutional conditions. Research concerning contextual factors occurs across many disciplines and theoretical domains, but few conceptual tools have attempted to integrate and translate this wide‐ranging research for health decision‐making purposes. Methods To formulate this tool we employed an iterative, collaborative process of scenario development and question generation. Five hypothetical health decision‐making scenarios (preventative, screening, curative, supportive and palliative) were developed and used to generate a set of exploratory questions that aim to highlight potential contextual factors across a range of health decisions. Findings We present an exploratory tool consisting of questions organized into four thematic domains – Bodies, Technologies, Place and Work (BTPW) – articulating wide‐ranging contextual factors relevant to health decision making. The BTPW tool encompasses health‐related scholarship and research from a range of disciplines pertinent to health decision making, and identifies concrete points of intersection between its four thematic domains. Examples of the practical application of the questions are also provided. Conclusions These exploratory questions provide an interdisciplinary toolkit for identifying the complex contextual factors affecting decision making. The set of questions comprised by the BTPW tool may be applied wholly or partially in the context of clinical practice, policy development and health‐related research.     

Who’s afraid of EBM? Medical professionalism from the perspective of evidence-based medicine

Evidence-based medicine (EBM) and medical professionalism are two prominent notions in current medical debates. However, proponents of professionalism fear a restriction in doctors’ freedom to make their best decisions for individual patients caused by the influence of EBM and highly standardised decision procedures. The challenge which EBM allegedly poses to physicians’ discretion forms the starting point for an analysis of the relationship between professionalism, as an inherent value system of medical practice, and EBM, as an approach to optimise the decision-making for individual patients. The analysis starts with a brief conceptual clarification of the ambiguous term “professionalism”. It then focuses on three key aspects of medical professionalism which may come into conflict with the basic tenets of EBM. The potential tensions between (a) professional autonomy and clinical practice guidelines, (b) individualised care and standardisation, and (c) esoteric authority and public accountability are analysed and a suggestion for reconcilement regarding each point is made. The article closes with a summary on how a better reflection on medical professionalism may help towards a fuller understanding of EBM and vice versa.     

Decision making about pre-medication to children

Background Inviting the child to participate in medical decisions regarding common medical procedures might influence the child's behaviour during the procedures. We wanted to study nurse decision‐making communication regarding pre‐medication before ear, nose and throat (ENT) surgery. Method In total, 102 children (3–6 years) signed for ENT surgery were video‐filmed during the pre‐medication process. The nurse decision‐making communication was identified, transcribed and grouped in six main categories dependent on the level of participation (self‐determination, compromise, negotiation, questioning, information, lack of communication). Associations between child factors (age, gender, verbal communication and non‐verbal communication) and different nurse decision‐making communication were studied. Associations between the decision‐making communication and verbal hesitation and/or the child's compliance in taking pre‐medication were also studied. Results Totally, information was the most frequently used category of decision making communication followed by negotiation and questioning. To the children showing signs of shyness, the nurse used more negotiation, questions and self‐determination communication and less information. The nurse used more compromise, negotiation and gave less information to children with less compliance. No specific type of nurse decision‐making communication was associated with verbal hesitation. The most important predictors for verbal hesitation were none or hesitant eye contact with nurse (OR = 4.5) and placement nearby or in parent's lap (OR = 4.7). Predictors for less compliance in taking pre‐medication were verbal hesitation from the child (OR = 22.7) and children who did not give any verbal answer to nurse initial questions (OR = 5.5). Conclusion Decision‐making communication could not predict the child's compliance during pre‐medication. Although negotiation, questioning and self‐determination communication were associated with more unwillingness to take pre‐medication. More knowledge is needed about communication to children in medical settings and how it influences the child's behaviours.     

Return to play guidelines cannot solve the football-related concussion problem

BACKGROUND: High school football players are the single largest cohort of athletes playing tackle football, and account for the majority of sport‐related concussions. Return to play guidelines (RTPs) have emerged as the preferred approach for addressing the problem of sport‐related concussion in youth athletes. METHODS: This article reviews evidence of the risks and effects of football‐related concussion and subconcussive brain trauma, as well as the effectiveness of RTPs as a preventative measure. Literature review utilized PubMed and Google Scholar, using combinations of the search terms “football,”“sports,”“concussion,”“Chronic Traumatic Encephalopathy,”“athlete,”“youth,” and “pediatric.” Literature review emphasized medical journals and primary neuroscientific research on sport‐related concussion and concussion recovery, particularly in youth athletes. RESULTS: Sport‐related concussion is a significant problem among student athletes. Student athletes are more vulnerable to concussion, and at risk of neurocognitive deficits lasting a year or more, with serious effects on academic and athletic performance. RTPs do little to address the problem of sport‐related concussion or the chronic damage caused by subconcussive brain trauma. CONCLUSIONS: Emphasizing RTPs as the solution to the concussion problem in tackle football risks neglecting genuine reforms that would prevent concussions. More effective concussion prevention is needed. Eliminating tackling from school football for youth under 16 is recommended to reduce concussions. Additional modifications to football are recommended to enhance safety and reduce brain trauma at all levels of play.     

How Uncertainty Matters Under Risk Neutrality

It is typical in cost-effectiveness analysis to invoke a normative decision-making framework that assumes, as a starting point, that “a quality-adjusted life-year (QALY) is a QALY is a QALY.” The implication of this assumption is that the decision maker is risk neutral and that expected values could be considered sufficiently informative for a given “approve or reject” decision. Nevertheless, it seems intuitive that less uncertainty should be desirable and this has led some to incorporate “real” risk aversion (RA) into cost-effectiveness analysis.We illustrate in this article that RA is not always necessary to justify choosing more over less certain options. We show that for a risk neutral decision maker, greater uncertainty can make the approval of technology less likely in the presence of (1) model nonlinearities, (2) nonlinear opportunity costs, and (3) irreversible costs. We call these cases of “apparent” RA.Incorporating explicit risk preferences into decision making can be challenging; nevertheless, as we show here, it is not necessary to justify caring about uncertainty in approval decisions.     

Causes and Consequences of Interrupted Enteral Nutrition

Background: Malnutrition and underfeeding are major challenges in caring for critically ill patients. Our goal was to characterize interruptions in enteral nutrition (EN) delivery and their impact on caloric debt in the surgical intensive care unit (ICU). Materials and Methods: We performed a prospective, observational study of adults admitted to surgical ICUs at a Boston teaching hospital (March–December 2012). We categorized EN interruptions as “unavoidable” vs “avoidable” and compared caloric deficit between patients with ≥1 EN interruption (group 1) vs those without interruptions (group 2). Multivariable logistic regression was used to investigate the association of EN interruption with the risk of underfeeding. Poisson regression was used to investigate the association of EN interruption with length of stay (LOS) and mortality. Results: Ninety‐four patients comprised the analytic cohort. Twenty‐six percent of interruptions were deemed “avoidable.” Group 1 (n = 64) had a significantly higher mean daily and cumulative caloric deficit vs group 2 (n = 30). Patients in group 1 were at a 3‐fold increased risk of being underfed (adjusted odds ratio, 2.89; 95% confidence interval [CI], 1.03–8.11), had a 30% higher risk of prolonged ICU LOS (adjusted incident risk ratio [IRR], 1.27; 95% CI, 1.14–1.42), and had a 50% higher risk of prolonged hospital LOS (adjusted IRR, 1.53; 95% CI, 1.41–1.67) vs group 2. Conclusions: In our cohort of critically ill surgical patients, EN interruption was frequent, largely “unavoidable,” and associated with undesirable outcomes. Future efforts to optimize nutrition in the surgical ICU may benefit from considering strategies that maximize nutrient delivery before and after clinically appropriate EN interruptions.     

Risk Attitudes and Personality Traits Predict Perceptions of Benefits and Risks for Medicinal Products: A Field Study of European Medical Assessors

Background: Risk attitudes and personality traits are known predictors of decision making among laypersons, but very little is known of their influence among experts participating in organizational decision making. Methods: Seventy-five European medical assessors were assessed in a field study using the Domain Specific Risk Taking scale and the Big Five Inventory scale. Assessors rated the risks and benefits for a mock “clinical dossier” specific to their area of expertise, and ordinal regression models were used to assess the odds of risk attitude or personality traits in predicting either the benefit or the risk ratings. Results: An increase in the “conscientiousness” score predicted an increase in the perception of the drug’s benefit, and male assessors gave higher scores for the drug’s benefit ratings than did female assessors. Extraverted assessors saw fewer risks, and assessors with a perceived neutral-averse or averse risk profile saw greater risks. Conclusions: Medical assessors perceive the benefits and risks of medicines via a complex interplay of the medical situation, their personality traits and even their gender. Further research in this area is needed to determine how these potential biases are managed within the regulatory setting.     

Clinical decision‐making in the context of chronic illness

This paper develops a framework to compare clinical decision making in relation to chronic and acute medical conditions. Much of the literature on patient‐physician decision making has focused on acute and often life‐threatening medical situations in which the patient is highly dependent upon the expertise of the physician in providing the therapeutic options. Decision making is often constrained and driven by the overwhelming impact of the acute medical problem on all aspects of the individual’s life. With chronic conditions, patients are increasingly knowledgeable, not only about their medical conditions, but also about traditional, complementary, and alternative therapeutic options. They must make multiple and repetitive decisions, with variable outcomes, about how they will live with their chronic condition. Consequently, they often know more than attending treatment personnel about their own situations, including symptoms, responses to previous treatment, and lifestyle preferences. This paper compares the nature of the illness, the characteristics of the decisions themselves, the role of the patient, the decision‐making relationship, and the decision‐making environment in acute and chronic illnesses. The author argues for a different understanding of the decision‐making relationships and processes characteristic in chronic conditions that take into account the role of trade‐offs between medical regimens and lifestyle choices in shaping both the process and outcomes of clinical decision‐making. The paper addresses the concerns of a range of professional providers and consumers.