The Challenge of Rare Diseases Stoller, James K.
Chest,
June 2018, 2018-06-00, 20180601, Volume:
153, Issue:
6
Journal Article
Peer reviewed
Open access
Rare diseases pose particular challenges to patients who are affected, to the clinicians who care for them, and to the investigators who study their conditions. Although individually uncommon, rare ...diseases are common in the aggregate, with approximately 7,000 described rare diseases affecting 25 to 30 million US adults. Challenges posed to affected individuals and their families largely regard being diagnosed, receiving optimal care, and affording disease-specific medications. Challenges facing clinicians who care for affected individuals include gaining knowledge and experience in caring for such patients, and the availability of local experts and of expert guidelines. Finally, challenges to investigators regard the difficulty and expense of assembling large cohorts of affected individuals for study, and garnering funding for research. Fortunately, in the face of these challenges, the steadfast resolve of patient and clinical/scientific communities to enhance care and generate new knowledge has fostered a large inventory of countermeasures to offset these challenges. Although further progress is surely needed, successes to date include the formation of powerful patient advocacy groups which have brokered collaborations between the patient, scientific communities, the government, and pharma/device communities in service of detection, optimal care, and research; procurement of funds to support research; formation of consortia of clinicians and scientists to collaborate; and general activation of the respective patient communities to perpetuate these successes. Persisting needs include enhanced detection strategies, dissemination of knowledge regarding optimal care, and research to prevent, treat, and cure disease.
The coronavirus pandemic has disrupted clinical practice, health-care organizations, and life. In the context that “a crisis is a terrible thing to waste,” as disruptive as the pandemic has been to ...traditional practices—both clinically and educationally—opportunities have also presented. Clinical benefits have included the propulsion of clinical innovation, including such items as the development of novel vaccines and accelerated understanding of multiplex ventilation. Approaches to educating students and other learners have also changed radically, with the suspension of live teaching in most instances and a precipitous transition to virtual instruction. This perspective considers a SWOT analysis (strengths, weaknesses, opportunities, and threats) associated with the coronavirus pandemic in health care that focuses on the implications for education. Although the obvious disadvantages (weaknesses) regard the loss of face-to-face interaction with all of its consequences (eg, isolation, risks to camaraderie, loss of hands-on training opportunities, and loss of in-person celebratory events like graduations and end-of-training celebrations), there are clearly offsetting strengths. These include growing experience with virtual teaching and virtual learning strategies, the invitation to codify best virtual teaching practices, a tightening of alignment between undergraduate and graduate medical education (eg, around virtual interview strategies), and opportunities for both self-reflection and a commitment to act virtuously. On balance, the pandemic has created the opportunity, indeed the necessity, to innovate in practice and in education, making the landscape ripe for creative practice, new mastery, and the concomitant benefits to learners and to educators.
In the context that leadership matters and that leadership competencies differ from those needed to practice medicine or conduct research, developing leadership competencies for physicians is ...important. Indeed, effective leadership is needed ubiquitously in health care, both at the executive level and at the bedside (eg, leading clinical teams and problem-solving on the ward). Various leadership models have been proposed, most converging on common attributes, like envisioning a new and better future state, inspiring others around this shared vision, empowering others to effect the vision, modeling the expected behaviors, and engaging others by appealing to shared values. Attention to creating an organizational culture that is informed by the seven classic virtues (trust, compassion, courage, justice, wisdom, temperance, and hope) can also unleash discretionary effort in the organization to achieve high performance. Health care-specific leadership competencies include: technical expertise, not only in one's clinical/scientific arena to garner colleagues' respect but also regarding operations; strategic thinking; finance; human resources; and information technology. Also, knowledge of the regulatory and legislative environments of health care is critical, as is being a problem-solver and lifelong learner. Perhaps most important to leadership in health care, as in all sectors, is having emotional intelligence. A spectrum of leadership styles has been described, and effective leaders are facile in deploying each style in a situationally appropriate way. Overall, leadership competencies can be developed, and leadership development programs are signature features of leading health-care organizations.
Because teams can accomplish goals that individuals cannot, teams matter. Indeed, teams especially matter in settings such as health care, where favorable outcomes depend critically on the ...contributions of many different people with diverse skills. As important as effective teambuilding is for health care, how to build teams is often not included in medical curricula, and physicians learn to build teams through “hidden curricula.” In the context that we can do better, this “How I Do It” presents an approach to building a team in a common scenario for the chest physician: picking up the inpatient Pulmonary Consult Service. The approach is informed by considering the attributes of an effective team, knowledge of common team dysfunctions, and best practices for building a team. The importance of teambuilding is underscored by substantial evidence that effective teamwork produces superior clinical outcomes.
Emotional intelligence (EI) has become widely appreciated as an important leadership attribute, in business, education and, increasingly, in health care. Defined as "the capacity to understand your ...own and others' emotions and to motivate and develop yourself and others in service of improved work performance and enhanced organizational effectiveness," EI is correlated with a number of success attributes in several sectors; for example, in business, with enhanced business performance and enhanced personal career success, and in health care, with enhanced patient satisfaction, lower burnout, lower litigation risk, and enhanced leadership success. While multiple models of EI have evolved, perhaps the most popular model is framed around four general rubrics with component competencies. The general rubrics are: self-awareness, self-management, social awareness, and relationship management. EI can be measured by using available instruments, and it can be learned and taught. Indeed, teaching EI has become increasingly common in health-care organizations in service of improving health care and health-care leadership. Although more research is needed, ample evidence supports the notion that EI is a critical success element for success as a health-care leader, especially because EI competencies differ markedly from the clinical and scientific skills that are core to being a clinician and/or investigator. This review of EI presents evidence in support of the relevance of EI to health care and health-care leadership, discusses how and when EI can be developed among health-care providers, and considers remaining questions.
Change is a fact of life; the absence of change creates stagnation. This is perhaps especially true in health care, where progress in treating disease depends on innovation and progress. At the same ...time, change is often uncomfortable. Thus, it is helpful to model the change process to optimize the chances of successfully effecting change. Furthermore, how to lead change is a critical leadership competency. Three models for leading change are reviewed: the first-the eight stages of change-which was not designed for health care; the second called "switch"; and the third called Amicus, which was uniquely designed for health care. The models share many common features, with the explicit reminder in the third model that physicians should be involved in the change effort early. Although sparse, the evidence does suggest the applicability of these models to health care. Beyond having a roadmap for leading change, it is helpful to assess the worthiness of undertaking a change effort and of predicting the phasic response to change efforts, given that humans are often change-averse. In this regard, both the "payoff matrix" and the change curve, derived from the work of Kübler-Ross on grieving, are offered as tools. Finally, physicians' avidity for change is framed by two opposing vectors. On the one hand, physicians share in the general human aversion to change. On the other hand, physicians are data-reverent and also wish to do their best for patients, which encourages their embrace of ever-increasing evidence and change.
Rationale Area under expiratory flow-volume curve (AEX) has been shown to be a valuable functional measurement in respiratory physiology. Area under inspiratory flow-volume loop (AIN) also shows ...promise in characterizing upper and/or lower airflow obstruction. Objectives we aimed here to develop normative reference values for AIN, able to ascertain deviations from normal. Methods We analyzed AIN in 4,980 spirometry tests recorded in non-smoking, healthy individuals in the Pulmonary Function Testing Laboratory. Results The mean (95% confidence interval, CI), standard deviation and median (25 th -75 th interquartile range) AIN were 16.05 (15.79–16.31), 9.08 and 14.72 (9.12–21.42) L 2 ·sec -1 , respectively. The mean (95% CI) and standard deviation of the best-trial measurements for square root of AIN (Sqrt AIN) were 3.84 (3.81–3.87) and 1.14; 4.15 (4.12–4.18) and 1.03 in men, and 2.68 (2.63–2.72) and 0.72 L·sec -1/2 in women. The mean (standard deviation) of pre- and post-bronchodilator Sqrt AIN were 3.71 (1.17) and 3.81 (1.19) L·sec -1/2 , respectively. The mean (95% CI), standard deviation and lowest 5 th percentile (lower limit of normal, LLN) of Sqrt AIN/Sqrt AEX (%) were 101.3 (100.82–101.88), 18.7, and 71.8%; stratified by gender, it was 102.2 (101.6–102.8), 18.6, and 72.8% in men, and 98 (96.9–99.2), 18.8, and 68.6% in women, respectively. Conclusions The availability of area under the inspiratory flow-volume curve (AIN) and the derived indices offers a promising opportunity to assess upper airway disease (e.g., involvement of larynx, trachea or major bronchi), especially because some of these measurements appear to be independent of age, race, height, and weight.
Background
The many challenges in health care today create a special need for great leadership. However, traditional criteria for physicians’ advancement to leadership positions often regard academic ...and/or clinical accomplishments rather than the distinctive competencies needed to lead. Furthermore, physicians’ training can handicap their developing leadership skills. In this context, an emerging trend is for health-care institutions to offer physician-leadership programs.
Methods and Results
This paper reviews the rationale for developing physician-leaders. Factors that underscore this need include: (1) physicians may lack inclinations to collaborate and to follow, (2) health-care organizations pose challenging environments in which to lead (e.g., because of silo-based structures, etc.), (3) traditional criteria for advancement in medicine regard clinical and/or academic skills rather than leadership competencies, and (4) little attention is currently given to training physicians regarding leadership competencies.
Conclusion
Definition of these competencies of ideal physician-leaders will inform the curricula and format of emerging physician leadership development programs.
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