Research ethics and assent in paediatric research — formative SAQs

SAQ 1 — Non-beneficial physiology study in children (10 marks)

A team proposes a non-beneficial study of disease physiology in school-age children. It requires two venepunctures the children would not otherwise undergo, and it cannot ethically be done in adults. The knowledge would be valuable to understanding the disorder. [1] [3]

Questions

1. Under the Subpart D risk-benefit categories, into which tier does this study fall, and what must the ethics committee find to approve it? (4 marks) [1] [3]
2. Distinguish parental permission, child assent and consent in this scenario, and state the age at which assent is typically sought. (3 marks) [2]
3. How should the team handle a child's dissent, and when (if ever) could assent or permission be waived? (3 marks) [2] [3]

Model answer

Risk-benefit tier and approval (4). Because the study offers the child no prospect of direct benefit and carries more than minimal risk, it falls into the Subpart D category of a minor increase over minimal risk with no direct benefit. The ethics committee may approve it only if the intervention presents experiences reasonably commensurate with the child's situation, the risk is a minor increase over minimal risk, and the knowledge is of vital importance to understanding or ameliorating the participant's disorder and cannot be obtained otherwise. Parental permission alone cannot make a greater-than-minimal-risk non-beneficial study acceptable. [1] [3]

Permission, assent and consent (3). The parents give informed permission (authorisation) for the child to participate; the child, from about age 7 and adapted to development, gives assent (affirmative agreement). Consent is the legal authorisation a competent older adolescent may give for themselves, which a younger child cannot. The terms must not be conflated. [2]

Dissent and waiver (3). A child's dissent to non-beneficial research is generally binding and must be recorded. An ethics committee may waive assent or parental permission only if the research is minimal risk, it could not practicably be carried out with permission, and participants' rights and welfare are protected. This study is greater than minimal risk, so a waiver would not apply. [2] [3]

SAQ 2 — Emergency resuscitation trial and oncology misconception (10 marks)

A trial of a neuroprotective agent must be given within minutes of return of spontaneous circulation after paediatric cardiac arrest; prospective parental permission is impossible. Separately, a family with a child in relapsed cancer describes a phase I trial as "the treatment that will cure him". [5] [4]

Questions

1. Describe the ethically appropriate consent mechanism for the resuscitation trial, naming the safeguards. (4 marks) [5]
2. Identify the ethical problem in the oncology family's statement and explain how to address it before enrolment. (3 marks) [4] [6]
3. State two outcomes of an ethically sound trial that matter to participants after the study ends. (3 marks) [5]

Model answer

Deferred consent and safeguards (4). The appropriate mechanism is a deferred-consent pathway approved in advance by the ethics committee, with community consultation and public disclosure before and after the study. The child is enrolled at the moment of crisis; once the family is reachable they are notified and asked about ongoing participation, with the option to withdraw data per protocol. This was the design of the therapeutic hypothermia after paediatric cardiac arrest trials. The mechanism is a specific, overseen exception, not the absence of consent, and research must never delay emergency care. [5]

Therapeutic misconception (3). The family has conflated an early dose-finding study (whose primary aim is to find a safe dose for future patients) with curative treatment. This therapeutic misconception means the permission is not informed. The corrective is careful re-education about the purpose of the study, randomisation and realistic likelihood of benefit, while preserving reasonable hope and confirming understanding with teach-back before enrolment. [4] [6]

Post-trial outcomes (3). Two outcomes that matter: the results are returned to families and the child in plain language, regardless of whether the findings were favourable; and the study is published and registered publicly (ANZCTR/ClinicalTrials.gov/ISRCTN) so that the risk the child carried is not wasted by hidden or selective reporting. [5]