Background
It is estimated that there are 187,000 adults known to intellectual disability services in the UK [
1]. Clinical research with this population has historically been problematic as it raises severe and at times intractable ethical concerns [
2,
3]. Inexperience with clinical research and randomised controlled trials (RCTs) in particular, may hinder the development of effective interventions for people with intellectual disabilities and mental disorders and/or challenging behaviour.
RCTs that include individuals with learning disabilities are rare, and only a small proportion of all papers published in specialist journals for intellectual disabilities concern RCTs [
4‐
6]. The RCTs that have taken place have been limited by under-recruitment [
7‐
11] or high drop-out rates [
12]. Researchers have previously reported numerous barriers to conducting RCTs that include difficulties in communication, accessing participants through gatekeepers such as paid carers, lack of understanding about clinical trial processes amongst care agencies, and obtaining informed consent from service users [
13‐
15]. The latter is particularly important in the UK in light of the Mental Capacity Act (2005) [
16] which not only presumes capacity from the outset but also seeks to ensure that every effort is made for service users to receive appropriate support in order to make an informed decision.
There has been suggestion that stakeholders have limited tolerance for this kind of research and may be hostile towards RCTs therefore decreasing the likelihood of them participating in research trials [
15,
17]. In response to this notion we reviewed the literature available on participant experiences of RCTs [
17] and found that stakeholders may be better able to understand the clinical equipoise than was previously thought and that stakeholder opinions may be changing. For example, parents of children with autism involved in drug RCTs showed that they had understood the various elements of a clinical trial such as the use of placebo, the need to test the medication efficacy, potential harm and benefits and the right to withdraw at any time [
18]. Another questionnaire survey reported high levels of satisfaction with the practicalities relating to the use of drugs in autism and the conduct of the trial [
19‐
21]. Importantly, the level of satisfaction reported was not related to participant's clinical outcome after the trial [
21]. Furthermore, service users with mild intellectual disabilities may have greater understanding of RCT methods than might be expected [
22]. These examples illuminate a potential discrepancy between carers and service users, and researchers who believe that RCTs in this population are inherently difficult.
Additionally, the Mental Capacity Act (2005) [
16] presents a statutory framework which enables service users the opportunity to be involved in research and sets out the legal duties of researchers who wish to investigate any vulnerable population who may lack the ability to make a voluntary, informed decision. In cases where service users are deemed to lack capacity, 'best practice' states that proxy decision makers are essential, but that any decision made on behalf of someone without capacity must reflect the best interests of the individual concerned. The Act states that 'informed' consent should be gained for any individual to participate in research. Therefore service users and proxy decision makers should have a richer understanding of RCTs than has previously been assumed; else this would highlight an ethical issue and a problem relating to the RCT recruitment process.
It has been suggested that individuals are more likely to participate in a trial if they deem the research as being meaningful [
21] but little work has been carried out to explore the "social validity" [
23] of RCTs in this population. The concept of social validity originates in Applied Behaviour Analysis and focuses on whether the treatment goals of the intervention employed and the outcomes achieved are acceptable, relevant, and useful to the individual in treatment [
19]. In order to enhance ethical quality and future research activity, whilst improving the evidence base for interventions for people with intellectual disabilities, it is important to explore stakeholders' perceptions of RCTs and the procedures that they may be asked to be involved in. Stakeholders may include service users, paid carers, family carers and professionals from health and social care background. Using qualitative, semi-structured interviews we aimed to investigate the opinions of stakeholders who were involved in a recent pragmatic RCT in intellectual disabilities concerning a psychological intervention for challenging behaviour. Pragmatic RCTs evaluate the comparative efficacy of two types of treatment in real life settings, i.e. not under experimental conditions [
24]. Furthermore, pragmatic trials require wide inclusion criteria in order to gain results that can be generalised to the wider population. This is therefore particularly useful when including individuals with intellectual disabilities in RCTs as the population of people labelled as having intellectual disability includes a wide variety of individuals, with and without additional diagnoses as well as, a wide range of other individual differences.
Results
A number of common themes arose between the stakeholders groups. There was a perception amongst carers and professionals that insufficient financial resources were available to optimally support this population. However, despite this, views on research were almost unanimously positive especially when the research was designed to have a practical purpose. Professionals often expressed the view that service users' communication skills and ability to provide informed consent made the conduct of research ethically complicated. Below, we report on the most common themes that arose from each stakeholder group but a full list of all the themes we encountered in the narratives can be seen in table
2.
Table 2
Emerging themes and frequency per participant group
Safeguarding | 1 | 1 | 17 | 19 |
Motivation | 3 | 14 | 3 | 20 |
Disadvantage/labelling | 3 | 6 | 12 | 21 |
Fairness | 5 | 16 | 13 | 34 |
Access | 4 | 9 | 24 | 37 |
Funding/resources | 2 | 13 | 23 | 38 |
Seeking help and support | 4 | 16 | 19 | 39 |
Research outcomes | 3 | 15 | 23 | 41 |
Research within services | 1 | 15 | 27 | 43 |
Communication/understanding | 5 | 13 | 25 | 43 |
Preferences | 3 | 15 | 25 | 43 |
Perception of clinical research | 4 | 16 | 24 | 44 |
Method | 5 | 14 | 27 | 46 |
Informed consent | 5 | 16 | 26 | 47 |
Benefits | 5 | 16 | 27 | 48 |
The work environment | 3 | 18 | 27 | 48 |
Terminology | 5 | 17 | 26 | 49 |
Approach | 5 | 18 | 27 | 50 |
Opinions about research | 6 | 18 | 27 | 51 |
1. Service users
Irrespective of group allocation, a minority of the interviewed service users demonstrated some understanding of basic concepts and at least one reported that trials can be used to test the effectiveness of the service.
"[You're doing this research] just to see how well the service is run from a scale of one to ten" (Service user #6)
"Research is finding out stuff" (Service user #3)
Most service users focused on the fact that research required to "ask for people's opinions". Only one service user appeared unable to explain what research was in any way, describing it as "something that gets you around".
Service users were aware of the importance of communication skills in relation to their daily lives. One service user indicated that people with intellectual disability can develop complex skills, such as sign language, in order to compensate for communication deficits. They suggested the use of pictorial information to aid understanding.
"A couple of my friends are Down's and they can use sign language, they can lip-read as well. There's two of them, they're both very good at signs, very good. It's not easy to do or understand, I can't do it. Some people with learning disabilities can be very good at signs; it depends on how your brain works." (Service user #1)
"Using pictures might help" (Service user #1)
One service user was particularly positive about research and described it as a platform by which they could contribute. Another was particularly interested in finding out about the outcomes of the research.
"I think research is good because you get input off everyone else who...you have input in the fact that you can get your point across...to the researcher, then they can do the research on any information at the end of it, it's really quite a good input. You feel like you're giving something to the researcher." (Service user #5)
"I'd like to know where does this go, what becomes of this when it is completed. I just wondered really." (Service user #6)
Procedures such as chance allocation and comparison groups appeared to cause two service users worry about the 'fairness' of a procedure in which some participants were seen by a service before others.
"I don't think it should be chosen by a computer I think people should actually go through more...who needs the help the most and then put them at the top. [...] I think everyone should really get the help, I don't think you know, otherwise it isn't fair." (Service user #3)
"Six months is too long to wait for help. Within that time, those other ten that didn't get the help six months before were now probably...in and out of hospital, lost, don't know where they're going." (Service user #5)
"It really depends on if they see one party before the other, because it could be unfair to do the other people if they didn't get that treatment. That's just my opinion. I think everyone has got the right to equal opportunities and get services." (Service user #6)
One service user associated the allocation of participants to the treatment and TAU groups to the lack of resources in the local area, which was a perception shared across the participant groups.
"I think it's because there isn't enough nurses out there...there isn't enough nurses, there isn't enough...help groups, there isn't...to manage, there's a lot of patients out there that need help." (Service user #5)
2. Family carers
As with the service users, family carers were positive about research irrespective of the trial arm the service user was assigned to. There was greater emphasis on how the research would be followed up with practical action and dissemination.
"I think it's a good idea, it depends if it's just research or if they're actually gonna do something with it" (Family carer #3)
"It would be nicer to sort of, so that that you knew exactly the end result, what was actually going on with the research." (Family carer #5)
"If we didn't have research we wouldn't be where we were today would we? [...] if research wasn't done the scientists and things didn't do what they've gotta do we'd never...go out...you know get further on in medicine and whatever." (Family carer #6)
Family carers appeared to have a varied understanding of the trial procedures and were concerned that it might prevent access to services that they needed. Those who did not understand the trial procedure were unsure about why their relative had been allocated into the TAU group. However, one carer was able to describe the need for comparison groups in order to evaluate the service.
"Otherwise what have you got to compare it with? You've got no comparison. If everyone gets the same...service, if everyone got say both, if everyone just got the behaviour therapy service, then what have you got to compare it against? You've got nothing." (Family carer #2)
"It seems very random to pick them by computer as to say one...Group 1 or Group 2. I don't quite see the point of it [...] I don't understand. Why do they have two groups? I mean why are they not all assessed the same?" (Family carer #1)
"I didn't understand what it meant, I thought that once [the nurse] had put her information over to them that they would automatically get involved with X...and it didn't work out that way in the end." (Family carer #4)
The family carers were aware of the process of informed consent and questioned how the service users' communication problems might impact on that process. Mostly they felt it was natural to provide permission to participate on behalf of someone who lacked capacity. However, one family carer reported discomfort in taking responsibility for agreeing on behalf of a relative; she argued that her views were only one of many including other stakeholders (e.g. professionals) whose input was equally valid, but might contradict her own views.
"We are and were his consent on this, but we knew that it was for his good, and that why we at the...but whether it was the behaviour therapy, whether it was the community learning, that meant no difference to X...himself, it wouldn't have mattered whether it was the Pope." (Family carer #2)
"I wouldn't just go by the carer and parent because again it's a very, very sticky and a very awkward situation. I think you've got to have an input from everybody, psychiatrists, doctors, consultants but I do feel the perhaps 60-70% of the results should come through the carers." (Family carer #5)
3. Paid carers
Paid carers thought of research as important for the improvement of future service delivery.
"If you don't do research how are you going to learn? And make it better for the next lot that come along" (Residential care manager #1)
"Without research you're not finding out new information and you can't make your way forward can you?" (Paid carer #1)
"I have a positive attitude towards research on people with learning disabilities because I read a book where there were very many positive examples of how the research changed the work practice for the better of this client group." (Paid carer #2)
Paid carers had better understanding of the trial than family carers and service users. Several were aware of the use of placebo as control in drug trials, that usually there is a comparison group and, within this context, our trial procedures were seen as acceptable. More than family carers, some paid carers expressed doubt whether quantitative measurements used within RCTs including REBILD were subtle enough to measure the complex nature of each service user's difficulties.
"How else would you do it?...Because you've got to get...a different perspective haven't you? For the group that has been helped and the group that hasn't, it's a bit like taking a placebo innit?" (Residential care manager #2)
"As I say the questionnaires they're very good but they're questionnaires and questionnaires are never, never accurate because you can only say 'yes', 'no', 'maybe', 'sometimes'." (Paid carer #3)
However, two paid carers criticized the research process. For example, for one of the carers assessments carried out by the researcher were seen as potentially influencing the service user's allocation to the treatment group while the other likened the trial procedure to a lottery.
"My understanding was that you came here, took back as much details about the incident of the concerns we had with X, then you would then feed it back to the main core, the main centre then it was from that that they would decide whether the input or who would be selected." (Residential care manager #3)
"I really don't know, I've no idea...it's like picking your numbers out of the lottery...and, it's just not right." (Paid carer #3)
A foremost concern for the paid carers was the way in which consent was gained to enter a service user without capacity into a trial; paid carers liked a shared decision to be reached for those individuals. Subsequently, paid carers often believed that a decision should be made in the best interest of the service user.
"I think the whole team, if the person with the learning disability doesn't understand or won't agree, the whole team have to get together and find out, you know how we're gonna work around it." (Day services manager #1)
"I think a good consent procedure would be the key people working with the client himself or their selves, the people that are important to the client in their lives, and I think they are the best people to be able to make that decision but I don't mean doctors, consultants and people like that who only sort of see them for maybe 10 minutes, 15 minutes in a month." (Residential care manager #3)
4. Professionals
This group recognised that the area of intellectual disabilities was lacking in evidence based care that would be important for the development of treatments in this field. The professionals felt that evaluation of services was urgently needed, and in their interviews they tended to focus upon research outcomes rather than research process. As a group, they thought that it was important to disseminate research and to learn from it.
"It's a very important area for the obvious reasons that learning disabilities services have progressed so much in the last twenty to thirty years that...and again there has been very little research apart from in very key areas." (Nurse #1)
"Yeah I mean as long as it isn't...research for the sake of research, if there's a goal and it's gonna move the service on, fine, you know." (Social care professional #1)
"The main one [aim] is to do the research to see in what way we can improve the service and if possible get that published so that would disseminate the findings to other professionals." (Nurse #2)
Compared to the other stakeholders, professionals showed the greatest knowledge and understanding of the rationale for undertaking a RCT (in order to evaluate a treatment or service). Some professionals, particularly psychiatrists, were enthused about the clinical trial and considered randomisation as an essential component in increasing the reliability of the results.
"Well there's nothing to think about, randomised controlled trials are important, important to find out and...how effective the treatment that is helping people." (Psychiatrist #1)
"I quite understand that without doing it, we wouldn't know the result. It's one of those things that I believe has to be done in order to get some answers" (Nurse #1)
Other professionals agreed with the value and need for the trial but felt frustrated because their preference would have been for the service users to access such a highly regarded service as quickly as possible. The biggest problem identified was the perceived interference with referrals to the specialist service as a result of the trial which led to participants expressing dissatisfaction with the project. At times, the trial was viewed negatively because of this. However, there was an accepted six month waiting list at the time that the trial commenced.
"By randomising the cases, it's stopping us from using the service that we really badly need, because we're not just referring for the sake of referring, we've tried everything we can." (Nurse #4)
"You need a control group for comparing it against and the only fair way of doing that is to randomise it...But yeah it has just been frustrating with lots of our clients being under the control group." (Professional therapist #3)
Professionals also expressed doubt about whether service users would understand the complexities of the trial.
"I don't think a lot of our clients could ever understand the implications of something like this." (Nurse #5)
"It has always been a difficult field because you do not have a subjective view of the service user [...] I find it quite difficult to undertake research especially where the service users are...involved." (Nurse #6)
As a group they also thought that multidisciplinary consent from various sources was necessary in order for service users to take part in the trial.
"It's obviously a complex matter, however I feel that a multi-disciplinary type of community or team to offer consent in those cases, appropriate in people from professions, their family and as well as advocacy service." (Psychiatrist #1)
"We need to have a multi-disciplinary team meeting to see whether it is for the benefit or for the goodness of the client. In other words you can get the consent from the multi-disciplinary team on behalf of the clients." (Nurse #3)
Authors' contributions
AH, MK and AC conceived the study and DR wrote the protocol and carried out the data collection and analysis. SIM carried out additional literature reviews. All authors contributed to and approved submission of the final manuscript.