Therapeutic Misconception: Hope, Trust and Misconception in Paediatric Research

Woods, Simon; Hagger, Lynn; McCormack, Pauline

doi:10.1007/s10728-012-0201-8

Cited by 22 publications

(30 citation statements)

References 49 publications

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“…Yet the parents reported that their expectations originated primarily from sources other than the clinicians—notably, the sponsor and the “community.” This assertion should be cautiously interpreted given the retrospective nature of the study and the multifaceted and subconscious nature of variables that contribute to expectations. The effect of overly-optimistic advocacy communities on DBMD clinical trial participants has been raised by Woods and colleagues, who suggest that a ‘collective therapeutic misconception’ may be propagated by neuromuscular disorder advocacy organizations to patients and families 21 .…”

Section: Discussionmentioning

confidence: 99%

Expectations and experiences of investigators and parents involved in a clinical trial for Duchenne/Becker muscular dystrophy

et al. 2013

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Background The social context of rare disease research is changing, with increased community engagement around drug development and clinical trials. This engagement may benefit patients and families, but may also lead to heightened trial expectations and therapeutic misconception. Clinical investigators are also susceptible to harboring high expectations. Little is known about parental motivations and expectations for clinical trials for rare pediatric disorders. Purpose We describe the experience of parents and clinical investigators involved in a phase II clinical trial for Duchenne and Becker muscular dystrophy: their expectations, hopes, motivations, and reactions to the termination of the trial. Methods This qualitative study was based on interviews with clinical investigators and parents of sons with DBMD who participated in the phase IIa or IIb ataluren clinical trial in the United States. Interviews were transcribed and coded for thematic analysis. Results Participants were twelve parents of affected boys receiving active drug and nine clinical investigators. High trial expectations of direct benefit were reported by parents and many clinicians. Investigators described monitoring and managing parents’ expectations; several worried about their own involvement in increasing parents’ expectations. Most parents were able to differentiate their expectations from their optimistic hopes for a cure. Parents’ expectations arose from other parents, advocacy organizations, and the sponsor. All parents reported some degree of clinical benefit to their children. Secondary benefits were hopefulness and powerful feelings associated with active efforts to affect the disease course. Parents and clinical investigators reported strong, close relationships that were mutually important. Parents and clinicians felt valued by the sponsor for the majority of the trial. When the trial abruptly stopped, they described loss of engagement, distress, and feeling unprepared for the possibility of trial termination. Limitations This was a retrospective study of one clinical trial. We were unable to recruit participants whose children received placebo. The interviews occurred during a time of significant uncertainty and distress for many of the participants. Conclusions This pilot study reflects complex outcomes of strong community engagement. The findings highlight a need for renewed education about, and support for, clinical trial termination and loss of drug access. The primary positive outcome was demonstration of strong relationships among committed parents and study teams. These relationships were highly valued by both parties, and may suggest an ideal intervention opportunity for efforts to improve psychological wellbeing. A negative outcome attributed, in part, to community engagement was inappropriately high trial expectations. More optimistically, high expectations were attributed, in part, to the importance of hope and powerful feelings associated with active efforts to affect the disease course.

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Section: Discussionmentioning

confidence: 99%

Expectations and experiences of investigators and parents involved in a clinical trial for Duchenne/Becker muscular dystrophy

et al. 2013

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“…Children and families living with disability may be particularly prone to therapeutic misconception due to trauma (e.g., if the disability is the result of a recent traumatic injury) or desperation (e.g., if they have exhausted all available treatment options, or, available treatments are largely unproven) [43]. Consequently, researchers and review boards must be particularly attentive to the risks of therapeutic misconception in this context [44].…”

Section: Therapeutic Misconceptionmentioning

confidence: 99%

Ethics in Pharmacologic Research in the Child with a Disability

2014

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This article reviews the unique ethical concerns that face clinicians, researchers, and family members in the realm of research involving children and youth with childhood-onset disabilities. Presented are the contemporary legal and regulatory environments in which we work and a synopsis of relevant articles on bioethics in this sector of the scientific literature. The most important ethical themes that emerged for children with disabilities include justice in research, consent and assent, child-centered communication, child- and family-centered decision making, participation in multiple studies, and therapeutic misconception. Two publicly recorded clinical studies are profiled to illustrate common considerations and concerns that arise during our ethical review of drug studies involving children with disabilities. It is concluded that the balance of access to current research and treatments must be weighed against risk for all involved. Collaborative planning amongst those involved in the development, review, approval, conduct, and oversight of drug study protocols can lead to effective scientific inquiry within the context of core ethical principles and child- and family-centered care.

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“…Patient associations may be involved in trial design and even be signatories of trial contract agreements. Their role should serve to improve dialogue and communication between pharma, patients and their families, contributing to a better understanding of research and a higher quality of informed consent 29 .…”

Section: Broader Issues On Communication Around Clinical Trialsmentioning

confidence: 99%

Guidance in Social and Ethical Issues Related to Clinical, Diagnostic Care and Novel Therapies for Hereditary Neuromuscular Rare Diseases: “Translating“ the Translational

et al. 2013

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Drug trials in children engage with many ethical issues, from drug-related safety concerns to communication with patients and parents, and recruitment and informed consent procedures. This paper addresses the field of neuromuscular disorders where the possibility of genetic, mutation-specific treatments, has added new complexity. Not only must trial design address issues of equity of access, but researchers must also think through the implications of adopting a personalised medicine approach, which requires a precise molecular diagnosis, in addition to other implications of developing orphan drugs. It is against this background of change and complexity that the Project Ethics Council (PEC) was established within the TREAT-NMD EU Network of Excellence. The PEC is a high level advisory group that draws upon the expertise of its interdisciplinary membership which includes clinicians, lawyers, scientists, parents, representatives of patient organisations, social scientists and ethicists. In this paper we describe the establishment and terms of reference of the PEC, give an indication of the range and depth of its work and provide some analysis of the kinds of complex questions encountered. The paper describes how the PEC has responded to substantive ethical issues raised within the TREAT-NMD consortium and how it has provided a wider resource for any concerned parent, patient, or clinician to ask a question of ethical concern. Issues raised range from science related ethical issues, issues related to hereditary neuromuscular diseases and the new therapeutic approaches and questions concerning patients rights in the context of patient registries and bio-banks. We conclude by recommending the PEC as a model for similar research contexts in rare diseases.

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Therapeutic Misconception: Hope, Trust and Misconception in Paediatric Research

Cited by 22 publications

References 49 publications

Expectations and experiences of investigators and parents involved in a clinical trial for Duchenne/Becker muscular dystrophy

Expectations and experiences of investigators and parents involved in a clinical trial for Duchenne/Becker muscular dystrophy

Ethics in Pharmacologic Research in the Child with a Disability

Guidance in Social and Ethical Issues Related to Clinical, Diagnostic Care and Novel Therapies for Hereditary Neuromuscular Rare Diseases: “Translating“ the Translational

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