Distinctive aspects of consent in pilot and feasibility studies

Abstract

Prior to a main randomized clinical trial, investigators often carry out a pilot or feasibility study in order to test certain trial processes or estimate key statistical parameters, so as to optimize the design of the main trial and/or determine whether it can feasibly be run.Pilot studies reflect the design of the intended main trial, whereas feasibility studies may not do so, and may not involve allocation to different treatments. Testing relative clinical effectiveness is not considered an appropriate aim of pilot or feasibility studies.However, consent is no less important than in a main trial as a means of morally legitimizing the investigator's actions. Two misperceptions are central to consent in clinical studies-therapeutic misconception (a tendency to conflate research and therapy) and therapeutic misestimation (a tendency to overestimate possible benefits and/or underestimate possible harms associated with participation). These phenomena may take a distinctive form in pilot and feasibility studies, owing to potential participants' likely prior unfamiliarity with the nature and purposes of such studies. Thus, participants may confuse the aims of a pilot or feasibility study (developing or optimizing trial design and processes) with those of a main trial (testing treatment effectiveness) and base consent on this misconstrual. Similarly, a misunderstanding of the ability of pilot and feasibility studies to provide information that will inform clinical care, or the underdeveloped nature of interventions included in such studies, may lead to inaccurate assessments of the objective possibility of benefit, and weaken the epistemic basis of consent accordingly. Equipoise may also be particularly challenging to grasp in the context of a pilot study. The consent process in pilot and feasibility studies requires a particular focus, and careful communication, if it is to carry the appropriate moral weight. There are corresponding implications for the process of ethical approval.

Feasibility of a Food Delivery Intervention during Pregnancy in a Rural US Population: The PEAPOD Pilot Study

“…In Table 4 we present selected nutrient and food group data from the ASA-24 dietary data system for those participants who had at least one valid dietary recall at both the pre- and post-intervention data collection time points to show descriptive evidence of feasibility of outcome measurement. Following best practices for pilot studies with small sample sizes, we are not including statistical testing between study arms [ 15 , 16 ], although we did assess differences within each study arm pre- vs. post-intervention. The only difference that approached statistical significance was an increase in the dietary intake of fiber in the intervention arm between the pre- and post-intervention time periods.…”

Humans, machines and decisions: Clinical reasoning in the age of artificial intelligence, evidence‐based medicine and Covid‐19

“…The section includes papers on EBM but extends beyond EBM to broader philosophical discussions concerning clinical knowledge, causal reasoning, research and consent-discussing issues of person-centred care and the purpose of medicine, and challenges to clinical judgement presented by the need for virtual care, a need generated by the global pandemic. [17][18][19][20][21][22][23][24][25][26][27] The first three papers in the section [17][18][19] take up the issues that were the topic of the concluding papers in the preceding 'Humanities' section, 15,16 though in this case the author outlines the history of the EBM debate from the perspective of medical philosophy and the history of ideas. The papers provide an extremely helpful account and critical analysis of the debate over the last 30 years, concluding with arguments concerning the possibilities for future development and the relationship between EBM and complexity theory, providerpatient decision making and person-centred care.…”

“…21 The issues of person-centred care, the value of knowledge and the disconnect between currently dominant theoretical models of reasoning on the one hand, and real-world reasoning on the other, are brought out in a fascinating discussion of 'transdisciplinary generalism'. 22 The paper provides a rich alternative to opposing 'reductionist (positivist) biomedical measures and social science (post-positivist) constructivist theories of knowledge' and it is followed by a similarly rich discussion of causal reasoning with application to specific cases 23 The focus on the need to develop theories of knowledge and judgement that make sense in the context of practice continues with papers on consent and the purpose of medicine 24,25 and a detailed and instructive account of the transition 'from inquiry to evidence to actionable clinical knowledge'. 26 The section concludes with a discussion of the transition from 'traditional in-person care into a new reality of virtual care for patients with complex chronic disease' precipitated by the Covid-19 pandemic, and the specific challenges to clinical judgement this transition presents.…”