Academic Physician Specialists' Approaches to Counseling Patients Interested in Unproven Stem Cell and Regenerative Therapies-A Qualitative Analysis.

OBJECTIVE: To explore the experiences, approaches, and challenges of physicians consulting patients about experimental stem cell and regenerative medicine interventions (SCRIs). PARTICIPANTS AND METHODS: From August 21, 2018, through July 30, 2019, semistructured interviews of 25 specialists in cardiology, ophthalmology, orthopedics, pulmonology, and neurology were conducted and qualitatively analyzed using modified grounded theory. RESULTS: All specialists used informational approaches to counsel patients, especially orthopedists. Informational approaches included explaining stem cell science, sharing risks, and providing principles. Several specialists also used relational counseling approaches including emphasizing that physicians want what is best for patients, acknowledging suffering, reassuring continued care, empathizing with patients and families, and underscoring that patients have the final decision. Many specialists reported being comfortable with the conversation, although some were less comfortable and several noted challenges in the consultation including wanting to support a patient's decision but worrying about harms from unproven SCRIs, navigating family pressure, and addressing stem cell hype and unrealistic expectations. Specialists also desired that additional resources be available for them and patients. CONCLUSION: Physicians relied more heavily on providing patients with information about SCRIs than using relational counseling approaches. Efforts should be directed at helping physicians address the informational and relational needs of patients, including providing tools and resources that inform physicians about the unproven SCRI industry, building skills in empathic communication, and the creation and dissemination of evidence-based resources to offer patients.

The Importance of Being Earnest.

Attempting to effect change in modern medicine, particularly as trainees or junior faculty, is often an overwhelming undertaking. While early-career physicians are in close contact with patients, they often lack the credibility or access to resources that are necessary to implement new ideas at large institutions. Although there may be scientific evidence to support new models of care, existing cultural patterns of practice can foster resistance to these interventions. The authors describe their own experience as residents reforming the management of endocarditis patients at an academic medical center, emphasizing the important role that residents can play in changing medical practice. Starting with a devastating patient case, the authors share their story of creating a multidisciplinary endocarditis team while navigating the many obstacles, some unseen, that can derail innovative ideas. Ultimately, through a combination of new perspectives, data-driven analysis, determination, and-most importantly-hope, the authors were able to dramatically improve outcomes for patients. Moving forward, their experience can serve as a model for young physicians and inspire them to effect change in their own institutions.

Harnessing the Power of Residents as Change Agents in Quality Improvement.

Residency training represents a unique period when learners begin to personally experience the patient safety and quality-of-care issues that affect health care systems and increasingly take responsibility to address them. Their integration into the clinical workflow in clinics, wards, and operating rooms positions them perfectly to observe and characterize the underlying processes that contribute to patient safety and health care quality problems. Residents' practices and perspectives are less entrenched than those of their faculty counterparts, which enables them to offer fresh ideas on the quality improvement (QI) process. Their creativity and ingenuity serve as assets when coming up with new and innovative changes to test using rapid change cycles. As such, they are ideally suited to serve as health systems change agents. Training programs and clinical institutions typically see residents as frontline care providers whose primary role is to treat the patient in front of them. Yet, by enabling residents to "treat the system" through QI work, they can take on the role of residents as change agents, which has the potential to have long-lasting effects on patient care on a much wider scale. However, training programs must do more than simply harness residents' enthusiasm and root them on from the sidelines. Instead, they must create an environment that is conducive to successfully implementing changes at the curricular, institutional, and health systems levels.

Ethical aspects in the care of a child with infantile spinal muscular atrophy (SMA).

The pediatrician has a privileged relationship with a child with infantile spinal muscular atrophy (SMA). At all times, he/she must be the child's mentor, promoting a comprehensive approach and support in order to ensure the best possible solution for the patient's autonomy. In all circumstances, an ethical stance is essential. After a reminder on the notions of ethics of care, we will address various ethical questions encountered through three critical situations during the care of a child with infantile spinal muscular atrophy: the announcement of the diagnosis, the transmission of information on innovative therapies, and palliative care and end-of-life support. © 2020 French Society of Pediatrics. Published by Elsevier Masson SAS. All rights reserved.

Is ovarian cancer surgery stuck in the dark ages?: a commentary piece reviewing surgical technologies.

Ovarian cancer surgery endeavours to remove all visible tumour deposits, and surgical technologies could potentially facilitate this aim. However, there appear to be barriers around the adoption of new technologies, and we hope this article provokes discussion within the specialty to encourage a forward-thinking approach to new-age surgical gynaecological oncology.

Unrealistic parental expectations for cure in poor-prognosis childhood cancer.

BACKGROUND: Many parents of children with advanced cancer pursue curative goals when cure is no longer possible. To the authors' knowledge, no pediatric studies to date have prospectively evaluated prognosis communication or influences on decision making in poor-prognosis childhood cancer. METHODS: The authors conducted a prospective cohort study at 9 pediatric cancer centers that enrolled 95 parents of children with recurrent or refractory, high-risk neuroblastoma (63% of those who were approached), a condition for which cure rarely is achieved. Parents were surveyed regarding the child's likelihood of cure; their primary goal of care; the child's symptoms, suffering, and quality of life; and regret concerning the last treatment decision. Medical records identified care and treatment decisions. RESULTS: Only 26% of parents recognized that the chance of cure was <25%. When asked to choose a single most important goal of care, approximately 72% chose cure, 10% chose longer life, and 18% chose quality of life. Parents were more likely to prioritize quality of life when they recognized the child's poor prognosis (P = .002). Approximately 41% of parents expressed regret about the most recent treatment decision. Parents were more likely to experience regret if the child had received higher intensity medical care (odds ratio [OR], 3.14; 95% CI, 1.31-7.51), experienced suffering with limited benefit from the most recent treatment (OR, 4.78; 95% CI, 1.16-19.72), or experienced suffering from symptoms (OR, 2.91; 95% CI, 1.18-7.16). CONCLUSIONS: Parents of children with poor-prognosis cancer frequently make decisions based on unrealistic expectations. New strategies for effective prognosis communication are needed.

In Experimental Hand Transplantation, Whose Views About Outcomes Should Matter Most?

Consent to any experimental procedure, even when offered as therapeutic, involves extensive discussion between patient-subjects and clinician-researchers. Decision making should be shared with a focus on potential risks and benefits of enrolling in a protocol. Just as patients who underwent nonexperimental interventions might experience regret or reconsider autonomously made choices, patient-subjects who are undergoing or who have undergone experimental therapies should be afforded latitude to reconsider their decisions. Although clinician-researchers tend to be deeply invested in gathering data about patient-subjects' experiences, they are obligated to express respect for patient-subjects' fundamental right to stop being enrolled in research.

What do patients with unmet medical needs want? A qualitative study of patients' views and experiences with expanded access to unapproved, investigational treatments in the Netherlands.

BACKGROUND: Patients with unmet medical needs sometimes resort to non-standard treatment options, including the use of unapproved, investigational drugs in the context of clinical trials, compassionate use or named-patient programs. The views and experiences of patients with unmet medical needs regarding unapproved, investigational drugs have not yet been examined empirically. METHODS: In this qualitative study, exploratory interviews and focus groups were held with patients with chronic or life-threatening diseases (n = 39), about topics related to non-standard treatment options, such as the search for non-standard treatment options, patients' views of the moral obligations of doctors, and the conditions under which they would or would not wish to use non-standard treatment options, including expanded access to unapproved, investigational drugs. RESULTS: Respondents had very little knowledge about and/or experience with existing opportunities for expanded access to investigational drugs, although some respondents were actively looking for non-standard treatment options. They had high expectations of their treating physicians, assuming them to be aware of non-standard treatment options, including clinical trials elsewhere and expanded access programs, and assuming that they would inform their patients about such options. Respondents carefully weighed the risks and potential benefits of pursuing expanded access, citing concerns related to the scientific evidence of the safety and efficacy of the drug, side effects, drug-drug interactions, and the maintaining of good quality of life. Respondents stressed the importance of education and assertiveness to obtain access to good-quality health care, and were willing to pay out of pocket for investigational drugs. Patients expressed concerns about equal access to new and/or non-standard treatment options. CONCLUSION: When the end of a standard treatment trajectory comes into view, patients may prefer that treating physicians discuss non-standard treatment options with them, including opportunities for expanded access to unapproved, investigational drugs. Although our respondents had varying levels of understanding of expanded access programs, they seemed capable of making well-considered choices with regard to non-standard treatment options and had realistic expectations with regard to the safety and efficacy of such options. Dutch patients might be less likely to fall prey to false hope than often presumed.

Characteristics and behaviours of formal versus informal nurse champions and their relationship to innovation success.

AIMS: To identify the sociodemographic attributes, project characteristics and champion strategies that differentiate formal from informal nursing champions, and to test their success in terms of project spread and novelty. BACKGROUND: Champions spread innovation in healthcare organizations. Empirical research has not explored the differences between formal and informal champions in terms of their antecedents and success. DESIGN: A quantitative cross-sectional design. METHOD: Data were collected on 93 nursing champions in three hospitals from 2015 - 2016. Champions were identified according to a validated approach; data on their sociodemographic attributes, project characteristics and strategies were assembled through interviews and validated questionnaires. Their success in terms of novelty and spread was assessed via expert ratings and validated questionnaire. FINDINGS: Informal champions had longer tenure and were involved mainly in bottom-up projects aimed mostly at improving human resources and services; formal champions were mostly involved in top-down projects aimed at quality control. Informal champions expressed more enthusiasm and confidence about the innovation; formal champions tended to use more online resources and peer-monitoring strategies. Projects of informal champions were more novel than those of formal champions. Project spread did not differ between the two groups. CONCLUSION: Formal and informal champions differ in their characteristics and implementation strategies. To encourage project's innovation, the organizational climate should encourage the emergence of informal champions; formal and informal champions should be chosen wisely, assuring that they possess enough organizational resources; and coaching programmes for junior champions should be planned to equip them with championing behaviours.

Charlie Gard and the weight of parental rights to seek experimental treatment.

The case of Charlie Gard, an infant with a genetic illness whose parents sought experimental treatment in the USA, brought important debates about the moral status of parents and children to the public eye. After setting out the facts of the case, this article considers some of these debates through the lens of parental rights. Parental rights are most commonly based on the promotion of a child's welfare; however, in Charlie's case, promotion of Charlie's welfare cannot explain every fact of the case. Indeed, some seem most logically to extend from intrinsic parental rights, that is, parental rights that exist independent of welfare promotion. I observe that a strong claim for intrinsic parental rights can be built on arguments for genetic propriety and children's limited personhood. Critique of these arguments suggests the scope of parental rights remains limited: property rights entail proper use; non-personhood includes only a small cohort of very young or seriously intellectually disabled children and the uniqueness of parental genetic connection is limited. Moreover, there are cogent arguments about parents' competence to make judgements, and public interest arguments against allowing access to experimental treatment. Nevertheless, while arguments based on propriety may raise concerns about the attitude involved in envisioning children as property, I conclude that these arguments do appear to offer a prima facie case for a parental right to seek experimental treatment in certain limited circumstances.

Understanding Treatment Burden and Quality of Life Impact of Participating in an Early-Phase Pediatric Oncology Clinical Trial: A Pilot Study.

PURPOSE: Early-phase clinical trials (EPTs) have led to new, more effective treatment options for children with cancer. Despite the extensive use of EPTs in pediatric oncology, little is known about parent and child experiences during EPT participation. The purposes of this pilot study were to assess the feasibility and preliminary results of having children with cancer and their parents complete measures of treatment burden and quality of life (QOL) concurrent with EPT participation. METHODS: In this descriptive, longitudinal, pilot study, parents and children were followed for the first 60 days of an EPT. Feasibility was assessed by participant enrollment and retention and completion of measures. Measures completed included the following: demographic form (completed at baseline); Diary of Trial Experiences to capture treatment burden (completed ongoing); and PedsQL™ Quality of Life Inventories, Cancer Modules, and Family Impact Module (completed at baseline, post-first disease evaluation, and off-study). Data were analyzed using descriptive statistics. RESULTS: Feasibility goals of enrollment, retention, and measure completion were partially met. Preliminary treatment burden and QOL results are provided. CONCLUSIONS: While QOL assessments may provide insight into EPT experiences, future studies need to be conducted at multiple sites and enrollment goals must account for participant attrition.

Feasibility of placebo-controlled trial designs for new CFTR modulator evaluation.

BACKGROUND: New CFTR modulators are in development that sponsors anticipate will be comparable or superior to approved modulators. Testing these agents for efficacy will require either placebo-controlled or active-comparator trials. METHODS: We surveyed US CF physicians and their patients eligible to receive approved modulators or their families for willingness to participate in placebo-controlled modulator trials of varying duration. RESULTS: Interest in placebo-controlled trials of short duration (2-4weeks) was greatest, with few respondents, particularly among patient respondents, willing to consider 6month studies. Patients/families with access to approved modulators were consistently less interested in placebo-controlled modulator trials of any duration. CONCLUSIONS: Sample size and interpretability advantages of placebo-controlled trials outweigh alternative active-comparator trials, but must consider physician and patient thresholds for forgoing treatment with approved modulators. Enrollment will be most feasible for short-duration trials and those conducted among populations without access to approved modulators.

Perceptions of Oncologists, Healthcare Policy Makers, Patients and the General Population on the Value of Pharmaceutical Treatments in Oncology.

INTRODUCTION: The purpose of this study was to explore the main factors explaining the relative weight of the different attributes that determine the value of oncologic treatments from the different perspectives of healthcare policy makers (HCPM), oncologists, patients and the general population in Spain. METHODS: Structured interviews were conducted to assess: (1) the importance of the attributes on treatment choice when comparing a new cancer drug with a standard cancer treatment; (2) the importance of survival, quality of life (QoL), costs and innovation in cancer; and (3) the most worrying side effects related to cancer drugs. RESULTS: A total of 188 individuals participated in the study. For all participants, when choosing treatments, the best rated characteristics were greater efficacy, greater safety, treatment adaptation to patients' individual requirements and the rapid reincorporation of patients to their daily activities. There were important differences among participants in their opinion about survival, QoL and cost. In general, oncologists, patients, and the general population gave greater value to gains in QoL than healthcare policy makers. Compared to other participants healthcare policy makers gave greater importance to the economic impact related to oncology treatments. CONCLUSIONS: Gains in QoL, survival, safety, cost and innovation are perceived differently by different groups of stakeholders. It is recommended to consider the perspective of different stakeholders in the assessment of a new cancer drugs to obtain more informed decisions when deciding on the most appropriate treatment to use. FUNDING: Eli Lilly & Co, Madrid (Spain).

Attitudes and Willingness to Assume Risk of Experimental Therapy to Eradicate Genital Herpes Simplex Virus Infection.

BACKGROUND: Current treatment of genital herpes is focused on ameliorating signs and symptoms but is not curative. However, as potential herpes simplex virus (HSV) cure approaches are tested in the laboratory, we aimed to assess the interest in such studies by persons with genital herpes and the willingness to assume risks associated with experimental therapy. METHODS: We constructed an anonymous online questionnaire that was posted on websites that provide information regarding genital herpes. The questions collected demographic and clinical information on adults who self-reported as having genital herpes, and assessed attitudes toward and willingness to participate in HSV cure clinical research. RESULTS: Seven hundred eleven participants provided sufficient responses to be included in the analysis. Sixty-six percent were women; the median age was 37 years, and the median time since genital HSV diagnosis was 4.7 years. The willingness to participate in trials increased from 59.0% in phase 1 to 68.5% in phase 2, and 81.2% in phase 3 trials, and 40% reported willingness to participate even in the absence of immediate, personal benefits. The most desirable outcome was the elimination of risk for transmission to sex partner or neonate. The mean perceived severity of receiving a diagnosis of genital HSV-2 was 4.2 on a scale of 1 to 5. CONCLUSIONS: Despite suppressive therapy available, persons with genital herpes are interested in participating in clinical research aimed at curing HSV, especially in more advanced stages of development.

Committee Opinion No. 674: Guiding Principles for Privileging of Innovative Procedures in Gynecologic Surgery.

New or emerging surgical procedures and technologies continue to be developed at a rapid rate and must be implemented safely into clinical practice. Additional privileging may be required if substantively new technical or cognitive skills are required to implement an innovative procedure or technology. Guiding principles for privileging should include cognitive and technical assessment to ensure appropriate patient selection and performance of the new procedure. Implementation also should include pertinent institutional and staff support as needed. A dynamic process for assessment and maintenance of current competency will enhance the safety of implementation and continued application of emerging procedures and technologies. The number of cases needed to demonstrate cognitive and technical proficiency will vary depending on many factors, including the health care provider's baseline expertise and technical acumen.