Clinical Trials Involving Minors: The Role of the Ethics Consultation in Avoiding Therapeutic Misconception

"Therapeutic Misconception (TM) occurs when clinical trial participants believe that the central purpose of the trial is therapeutic and that they will personally benefit from participation. If individuals who are entitled to consent to participation in a specific clinical trial do not understand that the defining purpose of clinical research is to produce generalizable knowledge, regardless of whether the subjects enrolled may potentially benefit from the intervention under study, this false belief may motivate them to participate, and in extreme cases may disqualify their consent. TM is especially frequent in fields in which the patients are highly vulnerable, such as when they are children and require parental consent. The informed consent is an essential ethical prerequisite before enrolling each and every participant in research that should protect patients through a process of dialog about a planned course of action. We argue that Ethics Consultant’s competencies may be crucial in avoiding TM: The Ethics Consultant should be involved in neonatal and paediatric clinical trials in order to face the unique vulnerability of children as research subjects, and to ensure that parental consent procedures are rigorously managed, enhancing recruitment in research trials in the context of fully understood consent. "

Caregiver reactions to neuroimaging evidence of covert consciousness in patients with severe brain injury: a qualitative interview study

Background Severe brain injury is a leading cause of death and disability. Diagnosis and prognostication are difficult, and errors occur often. Novel neuroimaging methods can improve diagnostic and prognostic accuracy, especially in patients with prolonged disorders of consciousness (PDoC). Yet it is currently unknown how family caregivers understand this information, raising ethical concerns that disclosure of neuroimaging results could result in therapeutic misconception or false hope. Methods To examine these ethical concerns, we conducted semi-structured interviews with caregivers of patients with PDoC who were enrolled in a concurrent neuroimaging research program designed to detect covert consciousness following severe brain injury. Caregivers held surrogate decision-making status for a patient. Interviews were conducted at two time points for each caregiver. The first interview occurred before the disclosure of neuroimaging results. The second occurred after disclosure. Descriptive analysis was applied to the data of four interview topics: (1) expectations for neuroimaging; (2) reactions to evidence of preserved cognition; (3) reactions to null results; and (4) understanding of the results and study. Results Twelve caregivers participated in the study; two caregivers shared surrogate decision-making status for one patient with PDoC. Twenty-one interviews were completed; one caregiver declined to participate in the post-disclosure interview. Three patients with PDoC associated with the study displayed evidence of covert consciousness. Overall, caregivers understood the neuroimaging research and results. Caregivers who received results of covert consciousness were generally pleased. However, there was some variation in expectations and reactions to these data and null results. Conclusion This study, for the first time, reveals caregiver expectations for and reactions to neuroimaging evidence of covert consciousness in patients with PDoC. Caregivers understood the neuroimaging research and results, casting doubt on speculative ethical concerns regarding therapeutic misconception and false hope. However, disclosure of neuroimaging result could be improved. Pre-disclosure consultations might assist professionals in shaping caregiver expectations. Standardization of disclosure might also improve comprehension of the results.

Consent to research participation: understanding and motivation among German pupils

Background The EU’s 2006 Paediatric Regulation aims to support authorisation of medicine for children, thus effectively increasing paediatric research. It is ethically imperative to simultaneously establish procedures that protect children’s rights. Method This study endeavours (a) to evaluate whether a template consent form designed by the Standing Working Group of the German-Research-Ethics-Committees (AKEK) adequately informs adolescents about research participation, and (b) to investigate associated phenomena like therapeutic misconception and motives for research participation. In March 2016 a questionnaire study was conducted among 279 pupils (mean age 13.1 years) of a secondary school in northern Germany. Results A majority of participants showed a general good understanding of foundational research ethics concepts as understood from the AKEK consent form. Nevertheless, our data also suggests possible susceptibility to therapeutic misconception. Own health concerns and pro-social considerations were found to be significant motivational factors for participating in research, while anticipation of pain lessens likelihood of participation. Advice from trusted others is an important decisional influence, too. Furthermore, data security was found to be a relevant aspect of adolescents’ decision-making process. Conclusion Bearing in mind adolescents’ generally good understanding, we infer the lack of knowledge about medical research in general to be one source of therapeutic misconception. To further improve the quality of consent we propose a multi-staged approach whereby general research education is completed before an individual becomes a patient or potential participant. To the best of our knowledge this is the first German questionnaire-study addressing issues of informed consent in a large under-age sample.

Treatment Decisions and the Therapeutic Process

This chapter considers the challenges of treatment decision-making for patients with incurable disease and the benefits of having a neutral space for reflection. Though respect for patients’ autonomy, shared decision-making, and informed consent are considered to be central to modern healthcare, adherence to these principles may seem illusory when patients are desperate and when the risks and benefits of treatment are ambiguous. The potential challenges to patient autonomy and informed consent are explored in this chapter, with particular reference to the context of phase one trials. In this setting, the intent of the treatment may easily be misconstrued by patients (therapeutic misconception) and the chances of benefits versus risks incorrectly estimated by them (therapeutic misestimation). This chapter explores the value of Managing Cancer and Living Meaningfully (CALM) in providing a neutral space for reflection and in allowing patients to explore their preferences regarding treatment while also considering the views of their healthcare providers and family members.

Stakeholder perspectives on clinical research related to therapies for rare diseases: therapeutic misconception and the value of research

Background For many rare diseases, few treatments are supported by strong evidence. Patients, family members, health care providers, and policy-makers thus have to consider whether to accept, recommend, or fund treatments with uncertain clinical effectiveness. They must also consider whether and how to contribute to clinical research that may involve receiving or providing the therapy being evaluated. Objective To understand why and how patients and families with rare metabolic diseases, specialist metabolic physicians, and health policy advisors choose whether to participate in studies and how they use and value research. Methods We conducted separate focus group interviews with each stakeholder group (three groups in total); two groups were conducted by telephone and the third was held in-person. Participants were recruited using purposive sampling. We analyzed each interview transcript sequentially using a qualitative description approach to inductively identify key themes. Several strategies to ensure credibility and trustworthiness were used including debriefing sessions after each focus group and having multiple team members review transcripts. Results Four patients/caregivers, six physicians, and three policy advisors participated. Our findings did not support conventional perspectives that therapeutic misconception (gaining access to treatment) is the main motivating factor for patients/caregivers to participate in clinical research. Rather, patients’/caregivers’ expressed reasons for participating in research included advancing science for the next generation and having an opportunity to share their experiences. Patients/caregivers and physicians described the difficulties in weighing risks versus benefits of accepting treatments not well-supported by evidence. Physicians also reported feeling conflicted in their dual role as patient advisor/advocate and evaluator of the evidence. Policy advisors were primarily focused on critically appraising the evidence to make recommendations for the health system. Conclusions Stakeholders differ in their perspectives on rare disease research but share concerns about the risks versus benefits of therapies when making individual- and population-level decisions.