Denise Howel

Health Technology Assessment Volume: 25, Issue: 63, Published in November 2021

During independent re-analysis of the cost data for a PhD thesis, a coding error was identified in one of the sensitivity analyses of the cost-utility evaluation of the trial looking at the effect of excluding from the intervention and control costs the cost of missed school days.

Background: Loss of arm function is common after stroke. Robot-assisted training may improve arm outcomes.Objective: The objectives were to determine the clinical effectiveness and cost-effectiveness of robot-assisted training, compared with an enhanced upper limb therapy programme and with usual care.Design: This was a pragmatic, observer-blind, multicentre randomised controlled trial with embedded health economic and process evaluations.Setting: The trial was set in four NHS trial centres.Participants: Patients with moderate or severe upper limb functional limitation, between 1 week and 5 years following first stroke, were recruited.Interventions: Robot-assisted training using the Massachusetts Institute of Technology-Manus robotic gym system (InMotion commercial version, Interactive Motion Technologies, Inc., Watertown, MA, USA), an enhanced upper limb therapy programme comprising repetitive functional task practice, and usual care.Main outcome measures: The primary outcome was upper limb functional recovery ‘success’ (assessed using the Action Research Arm Test) at 3 months. Secondary outcomes at 3 and 6 months were the Action Research Arm Test results, upper limb impairment (measured using the Fugl-Meyer Assessment), activities of daily living (measured using the Barthel Activities of Daily Living Index), quality of life (measured using the Stroke Impact Scale), resource use costs and quality-adjusted life-years.Results: A total of 770 participants were randomised (robot-assisted training,n = 257; enhanced upper limb therapy,n = 259; usual care,n = 254). Upper limb functional recovery ‘success’ was achieved in the robot-assisted training [103/232 (44%)], enhanced upper limb therapy [118/234 (50%)] and usual care groups [85/203 (42%)]. These differences were not statistically significant; the adjusted odds ratios were as follows: robot-assisted training versus usual care, 1.2 (98.33% confidence interval 0.7 to 2.0); enhanced upper limb therapy versus usual care, 1.5 (98.33% confidence interval 0.9 to 2.5); and robot-assisted training versus enhanced upper limb therapy, 0.8 (98.33% confidence interval 0.5 to 1.3). The robot-assisted training group had less upper limb impairment (as measured by the Fugl-Meyer Assessment motor subscale) than the usual care group at 3 and 6 months. The enhanced upper limb therapy group had less upper limb impairment (as measured by the Fugl-Meyer Assessment motor subscale), better mobility (as measured by the Stroke Impact Scale mobility domain) and better performance in activities of daily living (as measured by the Stroke Impact Scale activities of daily living domain) than the usual care group, at 3 months. The robot-assisted training group performed less well in activities of daily living (as measured by the Stroke Impact Scale activities of daily living domain) than the enhanced upper limb therapy group at 3 months. No other differences were clinically important and statistically significant. Participants found the robot-assisted training and the enhanced upper limb therapy group programmes acceptable. Neither intervention, as provided in this trial, was cost-effective at current National Institute for Health and Care Excellence willingness-to-pay thresholds for a quality-adjusted life-year.Conclusions: Robot-assisted training did not improve upper limb function compared with usual care. Although robot-assisted training improved upper limb impairment, this did not translate into improvements in other outcomes. Enhanced upper limb therapy resulted in potentially important improvements on upper limb impairment, in performance of activities of daily living, and in mobility. Neither intervention was cost-effective.Future work: Further research is needed to find ways to translate the improvements in upper limb impairment seen with robot-assisted training into improvements in upper limb function and activities of daily living. Innovations to make rehabilitation programmes more cost-effective are required.Limitations: Pragmatic inclusion criteria led to the recruitment of some participants with little prospect of recovery. The attrition rate was higher in the usual care group than in the robot-assisted training or enhanced upper limb therapy groups, and differential attrition is a potential source of bias. Obtaining accurate information about the usual care that participants were receiving was a challenge.Trial registration: Current Controlled Trials ISRCTN69371850.Funding: This project was funded by the National Institute for Health Research (NIHR) Health Technology Assessment programme and will be published in full inHealth Technology Assessment; Vol. 24, No. 54. See the NIHR Journals Library website for further project information.

Background: In the UK, most people with dementia die in the community and they often receive poorer end-of-life care than people with cancer.Objective: The overall aim of this programme was to support professionals to deliver good-quality, community-based care towards, and at, the end of life for people living with dementia and their families.Design: The Supporting Excellence in End-of-life care in Dementia (SEED) programme comprised six interlinked workstreams. Workstream 1 examined existing guidance and outcome measures using systematic reviews, identified good practice through a national e-survey and explored outcomes of end-of-life care valued by people with dementia and family carers (n = 57) using a Q-sort study. Workstream 2 explored good-quality end-of-life care in dementia from the perspectives of a range of stakeholders using qualitative methods (119 interviews, 12 focus groups and 256 observation hours). Using data from workstreams 1 and 2, workstream 3 used co-design methods with key stakeholders to develop the SEED intervention. Worksteam 4 was a pilot study of the SEED intervention with an embedded process evaluation. Using a cluster design, we assessed the feasibility and acceptability of recruitment and retention, outcome measures and our intervention. Four general practices were recruited in North East England: two were allocated to the intervention and two provided usual care. Patient recruitment was via general practitioner dementia registers. Outcome data were collected at baseline, 4, 8 and 12 months. Workstream 5 involved economic modelling studies that assessed the potential value of the SEED intervention using a contingent valuation survey of the general public (n = 1002). These data informed an economic decision model to explore how the SEED intervention might influence care. Results of the model were presented in terms of the costs and consequences (e.g. hospitalisations) and, using the contingent valuation data, a cost–benefit analysis. Workstream 6 examined commissioning of end-of-life care in dementia through a narrative review of policy and practice literature, combined with indepth interviews with a national sample of service commissioners (n = 20).Setting: The workstream 1 survey and workstream 2 included services throughout England. The workstream 1 Q-sort study and workstream 4 pilot trial took place in North East England. For workstream 4, four general practices were recruited; two received the intervention and two provided usual care.Results: Currently, dementia care and end-of-life care are commissioned separately, with commissioners receiving little formal guidance and training. Examples of good practice rely on non-recurrent funding and leadership from an interested clinician. Seven key components are required for good end-of-life care in dementia: timely planning discussions, recognising end of life and providing supportive care, co-ordinating care, effective working with primary care, managing hospitalisation, continuing care after death, and valuing staff and ongoing learning. Using co-design methods and the theory of change, the seven components were operationalised as a primary care-based, dementia nurse specialist intervention, with a care resource kit to help the dementia nurse specialist improve the knowledge of family and professional carers. The SEED intervention proved feasible and acceptable to all stakeholders, and being located in the general practice was considered beneficial. None of the outcome measures was suitable as the primary outcome for a future trial. The contingent valuation showed that the SEED intervention was valued, with a wider package of care valued more than selected features in isolation. The SEED intervention is unlikely to reduce costs, but this may be offset by the value placed on the SEED intervention by the general public.Limitations: The biggest challenge to the successful delivery and completion of this research programme was translating the ‘theoretical’ complex intervention into practice in an ever-changing policy and service landscape at national and local levels. A major limitation for a future trial is the lack of a valid and relevant primary outcome measure to evaluate the effectiveness of a complex intervention that influences outcomes for both individuals and systems.Conclusions: Although the dementia nurse specialist intervention was acceptable, feasible and integrated well with existing care, it is unlikely to reduce costs of care; however, it was highly valued by all stakeholders (professionals, people with dementia and their families) and has the potential to influence outcomes at both an individual and a systems level.Future work: There is no plan to progress to a full randomised controlled trial of the SEED intervention in its current form. In view of new National Institute for Health and Care Excellence dementia guidance, which now recommends a care co-ordinator for all people with dementia, the feasibility of providing the SEED intervention throughout the illness trajectory should be explored. Appropriate outcome measures to evaluate the effectiveness of such a complex intervention are needed urgently.Trial registration: Current Controlled Trials ISRCTN21390601.Funding: This project was funded by the National Institute for Health Research (NIHR) Programme Grants for Applied Research programme and will be published in full inProgramme Grants for Applied Research, Vol. 8, No. 8. See the NIHR Journals Library website for further project information.

Background: Looked-after children and care leavers (henceforth children in care) are young people placed under the care of local authorities, often because of a history of family abuse and/or neglect. These young people have significantly increased risk of substance use and mental health problems compared with peers. Aim: The Supporting Looked After Children and Care Leavers In Decreasing Drugs, and alcohol (SOLID) trial aimed to investigate the feasibility of a definitive randomised controlled trial comparing two behaviour change interventions to reduce risky substance use (illicit drugs and alcohol) in and improve the mental health of children in care aged 12–20 years. Methods: The study consisted of two phases: (1) a formative phase that adapted the motivational enhancement therapy and social behaviour and network therapy interventions for use with children in care and (2) a three-arm pilot randomised controlled trial (comparing motivational enhancement therapy, social behaviour and network therapy and usual care), and a linked process and economic (return on investment) evaluation. Trial feasibility was compared with prespecified STOP/GO criteria. Setting: Six local authority areas in the north-east of England. Participants: Children in care (aged 12–20 years) who screened positive for drug and/or alcohol use within the last 12 months were eligible for the trial. The formative and process evaluations included children in care, carers, social workers, and drug and alcohol workers. Outcome measures: The primary outcomes were recruitment and retention rates at 12 months’ follow-up. Baseline and 12-month follow-up questionnaires measured self-reported drug and alcohol use, mental health and health-related quality of life. The process evaluation considered acceptability and engagement with the interventions and trial procedures. Results: Formative findings (n = 65) highlighted the need for interventions to increase the emphasis on therapeutic relationships, use creative methods of engagement and support the identification of treatment goals wider than substance misuse. Within the randomised controlled trial, of 860 participants screened, 211 (24.5%) met the inclusion criteria. One hundred and twelve (53%) of the 211 eligible children were recruited and randomised. Just 15 of the 76 (20%) participants allocated to intervention attended any of the motivational enhancement therapy of social behaviour and network therapy sessions, and 60 (54%) participants completed the 12-month follow-up. The screening and recruitment of children in care required significantly more time and resource investment by researchers and children’s services than planned. The process evaluation (n = 116) demonstrated that, despite participants engaging in risky substance use, they did not often acknowledge this nor felt that they needed help. Children in care had complex, chaotic lives and children’s services departments were less research mature and extremely stretched; this, coupled with the multiple steps in the intervention pathway and study protocol, resulted in low adherence to the intervention and the trial. Conclusions: The SOLID trial demonstrated successful engagement with children in care to adapt the motivational enhancement therapy and social behaviour and network therapy interventions. However, the pilot randomised controlled trial found that a definitive trial is not feasible. The current screen, refer and treat pathway for children in care did not work. There is an urgent need to radically rethink how we deliver therapeutic services for children in care. A pragmatic evaluation design, coupled with additional research resource for children’s services, is needed to evaluate these novel models of care at scale. Trial registration: This study is registered as PROSPERO CRD42018098974 and Current Controlled Trials ISRCTN80786829. Funding: This project was funded by the National Institute for Health Research (NIHR) Public Health Research programme and will be published in full in Public Health Research; Vol. 8, No. 13. See the NIHR Journals Library website for further project information.

Background: There is limited evidence about the effectiveness of rehabilitation in meeting the longer-term needs of stroke patients and their carers.Objective: To determine the clinical effectiveness and cost-effectiveness of an extended stroke rehabilitation service (EXTRAS).Design: A pragmatic, observer-blind, parallel-group, multicentre randomised controlled trial with embedded health economic and process evaluations. Participants were randomised (1 : 1) to receive EXTRAS or usual care.Setting: Nineteen NHS study centres.Participants: Patients with a new stroke who received early supported discharge and their informal carers.Interventions: Five EXTRAS reviews provided by an early supported discharge team member between 1 and 18 months post early supported discharge, usually over the telephone. Reviewers assessed rehabilitation needs, with goal-setting and action-planning. Control treatment was usual care post early supported discharge.Main outcome measures: The primary outcome was performance in extended activities of daily living (Nottingham Extended Activities of Daily Living Scale) at 24 months post randomisation. Secondary outcomes at 12 and 24 months included patient mood (Hospital Anxiety and Depression Scale), health status (Oxford Handicap Scale), experience of services and adverse events. For carers, secondary outcomes included carers’ strain (Caregiver Strain Index) and experience of services. Cost-effectiveness was estimated using resource utilisation costs (adaptation of the Client Service Receipt Inventory) and quality-adjusted life-years.Results: A total of 573 patients (EXTRAS,n = 285; usual care,n = 288) with 194 carers (EXTRAS,n = 103; usual care,n = 91) were randomised. Mean 24-month Nottingham Extended Activities of Daily Living Scale scores were 40.0 (standard deviation 18.1) for EXTRAS (n = 219) and 37.2 (standard deviation 18.5) for usual care (n = 231), giving an adjusted mean difference of 1.8 (95% confidence interval –0.7 to 4.2). The mean intervention group Hospital Anxiety and Depression Scale scores were not significantly different at 12 and 24 months. The intervention did not improve patient health status or carer strain. EXTRAS patients and carers reported greater satisfaction with some aspects of care. The mean cost of resource utilisation was lower in the intervention group: –£311 (95% confidence interval –£3292 to £2787), with a 68% chance of EXTRAS being cost-saving. EXTRAS was associated with 0.07 (95% confidence interval 0.01 to 0.12) additional quality-adjusted life-years. At current conventional thresholds of willingness to pay for a quality-adjusted life-year, there is a 90% chance that EXTRAS is cost-effective.Conclusions: EXTRAS did not improve stroke survivors’ performance in extended activities of daily living but did improve their overall satisfaction with services. Given the impact on costs and quality-adjusted life-years, there is a high chance that EXTRAS could be considered cost-effective.Future work: Further research is required to identify whether or not community-based interventions can improve performance of extended activities of daily living, and to understand the improvements in health-related quality of life and costs seen by provision of intermittent longer-term specialist review.Trial registration: Current Controlled Trials ISRCTN45203373.Funding: This project was funded by the National Institute for Health Research (NIHR) Health Technology Assessment programme and will be published in full inHealth Technology Assessment; Vol. 24, No. 24. See the NIHR Journals Library website for further project information.

Health Technology Assessment Volume: 24, Issue: 12, Published in March 2020

Background: Fall-related injuries are a significant cause of morbidity and mortality in people with dementia. There is presently little evidence to guide the management of such injuries, and yet there are potentially substantial benefits to be gained if the outcomes of these injuries could be improved. This study aimed to design an appropriate new health-care intervention for people with dementia following a fall and to assess the feasibility of its delivery in the UK NHS.Objectives: To determine whether or not it is possible to design an intervention to improve outcomes of falls in dementia, to investigate the feasibility and acceptability of the DIFRID (Developing an Intervention for Fall related Injuries in Dementia) intervention and to investigate the feasibility of a future randomised controlled trial and the data collection tools needed to evaluate both the effectiveness and the cost-effectiveness of the DIFRID intervention.Design: This was a mixed-methods feasibility study. A systematic review (using Cochrane methodology) and realist review [using Realist And Meta-narrative Evidence Syntheses: Evolving Standards (RAMESES) methodology] explored the existing evidence base and developed programme theories. Searches were carried out in November 2015 (updated in January 2018) for effectiveness studies and in August 2016 for economic studies. A prospective observational study identified service use via participant diary completion. Qualitative methods (semistructured interviews, focus groups and observation) were used to explore current practice, stakeholder perspectives of the health and social care needs of people with dementia following a fall, ideas for intervention and barriers to and facilitators of change. Each of the resulting data sets informed intervention development via Delphi consensus methods. Finally, a single-arm feasibility study with embedded process evaluation was conducted.Setting: This study was set in the community.Participants: The participants were (1) people with dementia presenting with falls necessitating health-care attention in each setting (primary care, the community and secondary care) at three sites and their carers, (2) professionals delivering the intervention, who were responsible for training and supervision and who were members of the intervention team, (3) professionals responsible for approaching and recruiting participants and (4) carers of participants with dementia.Interventions: This was a complex multidisciplinary therapy intervention. Physiotherapists, occupational therapists and support workers delivered up to 22 sessions of tailored activities in the home or local area of the person with dementia over a period of 12 weeks.Main outcome measures: (1) Assessment of feasibility of study procedures; (2) assessment of the acceptability, feasibility and fidelity of intervention components; and (3) assessment of the suitability and acceptability of outcome measures for people with dementia and their carers (number of falls, quality of life, fear of falling, activities of daily living, goal-setting, health-care utilisation and carer burden).Results: A multidisciplinary intervention delivered in the homes of people with dementia was designed based on qualitative work, realist review and recommendations of the consensus panel. The intervention was delivered to 11 people with dementia. The study suggested that the intervention is both feasible and acceptable to stakeholders. A number of modifications were recommended to address some of the issues arising during feasibility testing. The measurement of outcome measures was successful.Conclusions: The study has highlighted the feasibility of delivering a creative, tailored, individual approach to intervention for people with dementia following a fall. Although the intervention required greater investment of time than usual practice, many staff valued the opportunity to work more closely with people with dementia and their carers. We conclude that further research is now needed to refine this intervention in the context of a pilot randomised controlled trial.Trial registration: Current Controlled Trials ISRCTN41760734 and PROSPERO CRD42016029565.Funding: This project was funded by the National Institute for Health Research (NIHR) Health Technology Assessment programme and will be published in full inHealth Technology Assessment; Vol. 23, No. 59. See the NIHR Journals Library website for further project information.

Scalable weight loss maintenance (WLM) interventions for adults with obesity are lacking but vital for the health and economic benefits of weight loss to be fully realised. We examined the effectiveness and cost-effectiveness of a low-intensity technology-mediated behavioural intervention to support WLM in adults with obesity after clinically significant weight loss (≥5%) compared to standard lifestyle advice. The NULevel trial was an open-label randomised controlled superiority trial in 288 adults recruited April 2014 to May 2015 with weight loss of ≥5% within the previous 12 months, from a pre-weight loss BMI of ≥30 kg/m2. Participants were self-selected, and the majority self-certified previous weight loss. We used a web-based randomisation system to assign participants to either standard lifestyle advice via newsletter (control arm) or a technology-mediated low-intensity behavioural WLM programme (intervention arm). The intervention comprised a single face-to-face goal-setting meeting, self-monitoring, and remote feedback on weight, diet, and physical activity via links embedded in short message service (SMS). All participants were provided with wirelessly connected weighing scales, but only participants in the intervention arm were instructed to weigh themselves daily and told that they would receive feedback on their weight. After 12 months, we measured the primary outcome, weight (kilograms), as well as frequency of self-weighing, objective physical activity (via accelerometry), psychological variables, and cost-effectiveness. The study was powered to detect a between-group weight difference of ±2.5 kg at follow-up. Overall, 264 participants (92%) completed the trial. Mean weight gain from baseline to 12 months was 1.8 kg (95% CI 0.5–3.1) in the intervention group (n = 131) and 1.8 kg (95% CI 0.6–3.0) in the control group (n = 133). There was no evidence of an effect on weight at 12 months (difference in adjusted mean weight change from baseline: −0.07 [95% CI 1.7 to −1.9], p = 0.9). Intervention participants weighed themselves more frequently than control participants and were more physically active. Intervention participants reported greater satisfaction with weight outcomes, more planning for dietary and physical activity goals and for managing lapses, and greater confidence for healthy eating, weight loss, and WLM. Potential limitations, such as the use of connected weighing study in both trial arms, the absence of a measurement of energy intake, and the recruitment from one region of the United Kingdom, are discussed. There was no difference in the WLM of participants who received the NULevel intervention compared to participants who received standard lifestyle advice via newsletter. The intervention affected some, but not all, process-related secondary outcomes of the trial. This trial is registered with the ISRCTN registry (ISRCTN 14657176; registration date 20 March 2014).

Background: Adverse effects from young people’s alcohol consumption manifest in a range of physical and psychosocial factors, including neurological issues, cognitive impairment and risk-taking behaviours. The SIPS JR-HIGH pilot trial showed alcohol screening and brief intervention (ASBI) to be acceptable to young people and schools in the north-east of England.Objectives: To conduct a two-arm, individually randomised controlled trial to evaluate the effectiveness and cost-effectiveness of ASBI for risky drinking in young people aged 14–15 years in the school setting, to monitor the fidelity of ASBI and to explore the barriers to, and facilitators of, implementation with staff, young people and parents.Design: A baseline survey with a 12-month follow-up. Interviews with 30 school staff, 21 learning mentors and nine teachers, and 33 young people and two parents.Setting: Thirty state schools in four areas of England: north-east, north-west, Kent and London.Participants: Year 10 school pupils who consented to the study (aged 14–15 years, recruited between November 2015 and June 2016), school-based staff and parents of the young people who took part in the study.Interventions: Young people who screened positively on a single alcohol screening question and consented were randomised to the intervention or control arm (blinded). The intervention was a 30-minute one-to-one structured brief intervention with a trained learning mentor and an alcohol leaflet. The control group received a healthy lifestyle leaflet (no alcohol information).Main outcome measures: The primary outcome measure was total alcohol consumed in the last 28 days. Secondary outcomes related to risky drinking, general psychological health, sexual risk-taking, energy drink consumption, age of first smoking, quality of life, quality-adjusted life-years, service utilisation and demographic information.Results: A total of 4523 young people completed the baseline survey, with 1064 screening positively (24%) and 443 being eligible to take part in the trial. Of those 443, 233 (53%) were randomised to the control arm and 210 were randomised to the intervention arm. Of the 443, 374 (84%) were successfully followed up at 12 months (intervention,n = 178; control,n = 196). The results were that the intervention showed no evidence of benefit for any alcohol-related measure when compared with the control arm. At 12 months we found a reduction from 61.9% to 43.3% using the Alcohol Use Disorders Identification Test cut-off point of 8 and cut-off point of 4 (69.0% to 60.7%). These results were not significant. A cost-effectiveness analysis showed that the average net cost saving of the brief intervention was £2865 (95% confidence interval –£11,272 to £2707) per year compared with usual practice, with the intervention showing a 76% probability of being cost saving compared with usual practice. The interview findings showed that school was an acceptable setting to carry out ASBI among staff and young people.Limitations: Recruitment of parents to take part in interviews was poor. Only 18 ASBI sessions were recorded, making it difficult to assess internal validity.Conclusions: Although the intervention was ineffective in reducing risky drinking in young people aged 14–15 years, it was well received by the young people and school staff who participated.Future work: Uniform reporting of the outcomes used for ASBI would generate more robust conclusions on the effectiveness of ASBI in the future. Pilot feasibility studies should include more than one geographical area. Future work on involving parents is needed.Trial registration: Current Controlled Trials ISRCTN45691494.Funding: This project was funded by the National Institute for Health Research (NIHR) Public Health Research programme and will be published in full inPublic Health Research; Vol. 7, No. 9. See the NIHR Journals Library website for further project information.