Exploring patient and public perspectives on recruitment and consent in cluster randomised trials involving children
Lead Research Organisation:
University of Liverpool
Department Name: Public Health Policy & Systems
Abstract
Clinical trials provide important evidence to improve patient care. There are two main types of trials, patient randomised controlled trials (RCT) and cluster randomised trials (CRT). The main difference between the two is that in an RCT individuals are randomly allocated to receive an intervention (e.g. drug A or drug B). However, in a CRT a whole group of people (e.g. hospital ward) are randomised, for example, one ward receives drug A and another receives drug B. In an RCT a patient is provided with information about the trial, including potential risks, with their informed consent being sought before taking part. Existing CRT guidance make provision for waiver of consent for the intervention in trials where consent would not be feasible and pose no more than minimal risk to participants.
Approaches to consent in CRTs depend upon what is being investigated and at what level. For example, when the intervention is a public health message shown on media formats (e.g. TV, Radio, newspaper adverts, social media) informed consent is not sought because as all members of the community would receive the intervention anyway. A waiver of consent is therefore used. Health care interventions given to an individual person (e.g. a drugs or vaccine) could in principle be evaluated using a RCT with informed consent sought. However, a CRT involving a waiver of consent may be designed due practical, or cost concerns. Although a CRT with a waiver of consent may seem an appropriate design, both the choice of a cluster randomised design and waiver of consent must be justified; particularly as CRTs involve larger patient samples than RCTs, are less efficient and more prone to bias.
Historically CRT guidance has been derived from 'expert' professional opinion without insight into patient and public perspectives. This is an oversight as without such insight there is a risk that current recruitment and consent processes may not be in line with the needs and perspectives of patients and family members. For example, we don't know if patients find it acceptable to be entered into an individual CRT without their consent.
The need for insight into patient and public perspectives is particularly important in CRTs involving vulnerable populations, such as children and neonates. CRTs in these populations are relatively new and have specific ethical and consent issues related to patient and parental decision making capacity, which may vary by setting (e.g. surgical or emergency CRTs). There is a need to explore patient/family and public perspectives on CRTs involving such vulnerable populations to ensure future CRTs are ethically appropriate and conducted in a way that considers the needs of children and their families. The proposed study is novel. In CRTs involving children and neonates we urgently need to know:
1. What are the most appropriate approaches to recruitment and consent in CRTs?
2. How should researchers consult with patients and the public about recruitment and consent (or waiver of consent) in CRTs and adapt such processes for different types of trials or patient populations?
3. Given these views, how should guidance for CRTs be formulated?
Our 30 month project will use established social science research methods, including scoping review, interviews, focus groups and an online survey to help answer these important questions. Focussing on paediatric and neonatal settings, our research will include those who do and do not have trial participation experience. We will analyse data collected with consideration of ethical principles (such as respect for persons, respect for persons, beneficence and justice) and the research ethics literature to make recommendations on approaches to recruitment and consent in future CRTs in these populations. To help ensure this work is internationally relevant, the final stages will include an evaluation of draft guidance involving the public and international experts on CRTs.
Approaches to consent in CRTs depend upon what is being investigated and at what level. For example, when the intervention is a public health message shown on media formats (e.g. TV, Radio, newspaper adverts, social media) informed consent is not sought because as all members of the community would receive the intervention anyway. A waiver of consent is therefore used. Health care interventions given to an individual person (e.g. a drugs or vaccine) could in principle be evaluated using a RCT with informed consent sought. However, a CRT involving a waiver of consent may be designed due practical, or cost concerns. Although a CRT with a waiver of consent may seem an appropriate design, both the choice of a cluster randomised design and waiver of consent must be justified; particularly as CRTs involve larger patient samples than RCTs, are less efficient and more prone to bias.
Historically CRT guidance has been derived from 'expert' professional opinion without insight into patient and public perspectives. This is an oversight as without such insight there is a risk that current recruitment and consent processes may not be in line with the needs and perspectives of patients and family members. For example, we don't know if patients find it acceptable to be entered into an individual CRT without their consent.
The need for insight into patient and public perspectives is particularly important in CRTs involving vulnerable populations, such as children and neonates. CRTs in these populations are relatively new and have specific ethical and consent issues related to patient and parental decision making capacity, which may vary by setting (e.g. surgical or emergency CRTs). There is a need to explore patient/family and public perspectives on CRTs involving such vulnerable populations to ensure future CRTs are ethically appropriate and conducted in a way that considers the needs of children and their families. The proposed study is novel. In CRTs involving children and neonates we urgently need to know:
1. What are the most appropriate approaches to recruitment and consent in CRTs?
2. How should researchers consult with patients and the public about recruitment and consent (or waiver of consent) in CRTs and adapt such processes for different types of trials or patient populations?
3. Given these views, how should guidance for CRTs be formulated?
Our 30 month project will use established social science research methods, including scoping review, interviews, focus groups and an online survey to help answer these important questions. Focussing on paediatric and neonatal settings, our research will include those who do and do not have trial participation experience. We will analyse data collected with consideration of ethical principles (such as respect for persons, respect for persons, beneficence and justice) and the research ethics literature to make recommendations on approaches to recruitment and consent in future CRTs in these populations. To help ensure this work is internationally relevant, the final stages will include an evaluation of draft guidance involving the public and international experts on CRTs.
Technical Summary
Although CRTs help provide evidence where group effects or groups interventions are relevant, they raise important methodological and ethical issues. Whilst randomisation takes place at the cluster level, the units of randomisation, intervention, data collection may vary. When a waiver of patient consent is proposed, there is question as to whether, to what degree and how patients should be notified about the research and provided with a debrief. Even when interventions are administered at the individual level, and informed consent may be possible, a waiver of consent may be preferred due practical or cost concerns. Article 30 of European clinical trials legislation states that consent for CRTs should be sought through 'simplified means' yet there is a lack of clarity about what this means.
The need for insight into patients and public perspectives is particularly important in CRTs involving vulnerable populations, such as children and neonates. There may be different ethical and consent considerations, which patients/parents may have different perspectives about. These include: consent for the intervention, or samples, linkage with routine data, how or whether patients/parents should be notified about the research; and what should happen when a child dies after enrolment into an CRT.
Our 30-month project will use established social science research methods to explore public and patient/family views on:
1. What are the most appropriate approaches to recruitment and consent in CRTs involving children?
2. How should researchers consult with patients and the public about recruitment and consent (or waiver of consent) in such CRTs and adapt processes for different types of trials or patient populations?
3. Given these views, how should guidance for CRTs be formulated?
We will analyse data with consideration of ethical principles and the research ethics literature to make recommendations for international guidance to inform future CRTs involving children.
The need for insight into patients and public perspectives is particularly important in CRTs involving vulnerable populations, such as children and neonates. There may be different ethical and consent considerations, which patients/parents may have different perspectives about. These include: consent for the intervention, or samples, linkage with routine data, how or whether patients/parents should be notified about the research; and what should happen when a child dies after enrolment into an CRT.
Our 30-month project will use established social science research methods to explore public and patient/family views on:
1. What are the most appropriate approaches to recruitment and consent in CRTs involving children?
2. How should researchers consult with patients and the public about recruitment and consent (or waiver of consent) in such CRTs and adapt processes for different types of trials or patient populations?
3. Given these views, how should guidance for CRTs be formulated?
We will analyse data with consideration of ethical principles and the research ethics literature to make recommendations for international guidance to inform future CRTs involving children.