scholarly journals Pressure garment to prevent abnormal scarring after burn injury in adults and children: the PEGASUS feasibility RCT and mixed-methods study

2018 ◽  
Vol 22 (36) ◽  
pp. 1-162 ◽  
Author(s):  
Naiem Moiemen ◽  
Jonathan Mathers ◽  
Laura Jones ◽  
Jonathan Bishop ◽  
Philip Kinghorn ◽  
...  
Keyword(s):  
Mixed Methods ◽  
Health Technology Assessment ◽  
Technology Assessment ◽  
Standard Care ◽  
Pilot Trial ◽  
Health Technology ◽  
Burn Scar ◽  
Individual Interviews ◽  
Scar Management

Background Eleven million people suffer a fire-related injury worldwide every year, and 71% have significant scarring. Pressure garment therapy (PGT) is a standard part of burn scar management, but there is little evidence of its clinical effectiveness or cost-effectiveness. Objective To identify the barriers to, and the facilitators of, conducting a randomised controlled trial (RCT) of burn scar management with and without PGT and test whether or not such a trial is feasible. Design Web-based surveys, semistructured individual interviews, a pilot RCT including a health economic evaluation and embedded process evaluation. Setting UK NHS burns services. Interviews and the pilot trial were run in seven burns services. Participants Thirty NHS burns services and 245 staff provided survey responses and 15 staff participated in individual interviews. Face-to-face interviews were held with 24 adult patients and 16 parents of paediatric patients who had undergone PGT. The pilot trial recruited 88 participants (57 adults and 31 children) who were at risk of hypertrophic scarring and were considered suitable for scar management therapy. Interviews were held with 34 participants soon after recruitment, with 23 participants at 12 months and with eight staff from six sites at the end of the trial. Interventions The intervention was standard care with pressure garments. The control was standard care comprising scar management techniques involving demonstration and recommendations to undertake massage three or four times per day with moisturiser, silicone treatment, stretching and other exercises. Main outcome measures Feasibility was assessed by eligibility rates, consent rates, retention in allocated arms, adherence with treatment and follow-up and completion of outcome assessments. The outcomes from interview-based studies were core outcome domains and barriers to, and facilitators of, trial participation and delivery. Results NHS burns services treat 2845 patients per annum (1476 paediatric and 1369 adult) and use pressure garments for 6–18 months, costing £2,171,184. The majority of staff perceived a need for a RCT of PGT, but often lacked equipoise around the research question and PGT as a treatment. Strong views about the use of PGT have the potential to influence the conduct of a full-scale RCT. A range of outcome domains was identified as important via the qualitative research: perceptions of appearance, specific scar characteristics, function, pain and itch, broader psychosocial outcomes and treatment burden. The outcome tools evaluated in the pilot trial did not cover all of these domains. The planned 88 participants were recruited: the eligibility rate was 88% [95% confidence interval (CI) 83% to 92%], the consent rate was 47% (95% CI 40% to 55%). Five (6%) participants withdrew, 14 (16%) were lost to follow-up and 8 (9%) crossed over. Adherence was as in clinical practice. Completion of outcomes was high for adult patients but poorer from parents of paediatric patients, particularly for quality of life. Sections on range of movement and willingness to pay were found to be challenging and poorly completed. Limitations The Brisbane Burn Scar Impact Profile appears more suitable in terms of conceptual coverage than the outcome scales that were used in the trial but was not available at the time of the study. Conclusions A definitive RCT of PGT in burn scar management appears feasible. However, staff attitudes to the use of pressure garments may lead to biases, and the provision of training and support to sites and an ongoing assessment of trial processes are required. Future work We recommend that any future trial include an in-depth mixed-methods recruitment investigation and a process evaluation to account for this. Trial registration Current Controlled Trials ISRCTN34483199. Funding This project was funded by the National Institute for Health Research (NIHR) Health Technology Assessment programme and will be published in full in Health Technology Assessment; Vol. 22, No. 36. See the NIHR Journals Library website for further project information

scholarly journals Prophylactic levofloxacin to prevent infections in newly diagnosed symptomatic myeloma: the TEAMM RCT

2019 ◽  
Vol 23 (62) ◽  
pp. 1-94 ◽  
Author(s):  
Mark T Drayson ◽  
Stella Bowcock ◽  
Tim Planche ◽  
Gulnaz Iqbal ◽  
Guy Pratt ◽  
...  
Keyword(s):  
Overall Survival ◽  
Cost Effectiveness ◽  
Health Technology Assessment ◽  
Technology Assessment ◽  
Health Technology ◽  
Newly Diagnosed ◽  
Double Blind ◽  
Double Blind Placebo ◽  
Febrile Episodes

Background Myeloma causes profound immunodeficiency and recurrent serious infections. There are approximately 5500 new UK cases of myeloma per annum, and one-quarter of patients will have a serious infection within 3 months of diagnosis. Newly diagnosed patients may benefit from antibiotic prophylaxis to prevent infection. However, the use of prophylaxis has not been established in myeloma and may be associated with health-care-associated infections (HCAIs), such as Clostridium difficile. There is a need to assess the benefits and cost-effectiveness of the use of antibacterial prophylaxis against any risks in a double-blind, placebo-controlled, randomised clinical trial. Objectives To assess the risks, benefits and cost-effectiveness of prophylactic levofloxacin in newly diagnosed symptomatic myeloma patients. Design Multicentre, randomised, double-blind, placebo-controlled trial. A central telephone randomisation service used a minimisation computer algorithm to allocate treatments in a 1 : 1 ratio. Setting A total of 93 NHS hospitals throughout England, Northern Ireland and Wales. Participants A total of 977 patients with newly diagnosed symptomatic myeloma. Intervention Patients were randomised to receive levofloxacin or placebo tablets for 12 weeks at the start of antimyeloma treatment. Treatment allocation was blinded and balanced by centre, estimated glomerular filtration rate and intention to give high-dose chemotherapy with autologous stem cell transplantation. Follow-up was at 4-week intervals up to 16 weeks, with a further follow-up at 1 year. Main outcome measures The primary outcome was to assess the number of febrile episodes (or deaths) in the first 12 weeks from randomisation. Secondary outcomes included number of deaths and infection-related deaths, days in hospital, carriage and invasive infections, response to antimyeloma treatment and its relation to infection, quality of life and overall survival within the first 12 weeks and beyond. Results In total, 977 patients were randomised (levofloxacin, n = 489; placebo, n = 488). A total of 134 (27%) events (febrile episodes, n = 119; deaths, n = 15) occurred in the placebo arm and 95 (19%) events (febrile episodes, n = 91; deaths, n = 4) occurred in the levofloxacin arm; the hazard ratio for time to first event (febrile episode or death) within the first 12 weeks was 0.66 (95% confidence interval 0.51 to 0.86; p = 0.002). Levofloxacin also reduced other infections (144 infections from 116 patients) compared with placebo (179 infections from 133 patients; p-trend of 0.06). There was no difference in new acquisitions of C. difficile, methicillin-resistant Staphylococcus aureus and extended-spectrum beta-lactamase Gram-negative organisms when assessed up to 16 weeks. Levofloxacin produced slightly higher quality-adjusted life-year gains over 16 weeks, but had associated higher costs for health resource use. With a median follow-up of 52 weeks, there was no significant difference in overall survival (p = 0.94). Limitations Short duration of prophylactic antibiotics and cost-effectiveness. Conclusions During the 12 weeks from new diagnosis, the addition of prophylactic levofloxacin to active myeloma treatment significantly reduced febrile episodes and deaths without increasing HCAIs or carriage. Future work should aim to establish the optimal duration of antibiotic prophylaxis and should involve the laboratory investigation of immunity, inflammation and disease activity on stored samples funded by the TEAMM (Tackling Early Morbidity and Mortality in Myeloma) National Institute for Health Research Efficacy and Mechanism Evaluation grant (reference number 14/24/04). Trial registration Current Controlled Trials ISRCTN51731976. Funding details This project was funded by the NIHR Health Technology Assessment programme and will be published in full in Health Technology Assessment; Vol. 23, No. 62. See the NIHR Journals Library website for further project information.

scholarly journals Stepped approach to improving sexual function after gynaecological cancer: the SAFFRON feasibility RCT

2019 ◽  
Vol 23 (6) ◽  
pp. 1-92
Author(s):  
Sue Gessler ◽  
Michael King ◽  
Alessandra Lemma ◽  
Julie Barber ◽  
Louise Jones ◽  
...  
Keyword(s):  
Sexual Function ◽  
Health Technology Assessment ◽  
Technology Assessment ◽  
Clinical Skills ◽  
Pilot Trial ◽  
Health Technology ◽  
Gynaecological Cancer ◽  
Stepped Care ◽  
Treatment Level ◽  
Psychosocial Studies

Background Women affected by gynaecological cancer are often unaware of the sexual consequences of both the cancer and its treatment. Most do not receive appropriate advice or help to recover sexual function, and the effect on their sexuality may be profound, both physically and emotionally. However, several potential therapies can be effective in helping recover some sexual engagement and change self-perception around sex. A major initial challenge is informing and involving patients in an appropriate and sensitive manner, and a further issue is delivering therapies in busy gynaelogical oncology clinics. This study was conceived in response to a National Institute for Health Research (NIHR) Health Technology Assessment (HTA) call asking for proposals to improve sexual functioning in women treated for gynaecological cancer while taking into account associated issues of mood. Existing evidence-based therapies for improving sexual function after cancer treatment were adapted and placed within a ‘stepped care’ model for delivering these in the NHS setting. An assessment and treatment stepping algorithm was developed in parallel, both to assign women to a treatment level at assessment and to follow their progress session by session to advise on changing intervention level. The assessment tool was applied to all participants on the principle that the problem was sexual difficulty, not the cancer of origin. Participants Women aged > 18 years (with partners at their choice) treated for any gynaecological malignancy with surgery and/or chemotherapy and/or radiation at University College London Hospital or Bristol Gynaecological cancer centres, minimally 3 months post end of treatment, of any sexual orientation, with sexual function difficulties identified by three initial screening questions. Design A feasibility two-arm, parallel-group randomised controlled pilot trial. Setting Two NHS gynaecological cancer centres, one in London and one in Bristol. Interventions A three-level stepped care intervention. Objective To assess the feasibility of conducting a full-scale investigation of stepped therapy and indicate the potential benefits to patients and to the NHS generally. Primary outcome measures Recruitment to study, proportion of women stepping up, number of usable data points of all measures and time points over length of trial, and retention of participants to end of trial. Results Development of the intervention and accompanying algorithm was completed. The study was stopped before the recruitment stage and, hence, no randomisation, recruitment, numbers analysed, outcomes or harms were recorded. Limitations As the study did not proceed, the intervention and its accompanying algorithm have not been evaluated in practice, and the capacity of the NHS system to deliver it has not been examined. Conclusions None, as the study was halted. Future work The intervention could be studied within a clinical setting; however, the experience of the study group points to the need for psychosocial studies in medical settings to establish pragmatic and innovative mechanisms to ensure adequate resource when extending staff clinical skills and time to deliver any new intervention for the duration of the trial. Trial registration Current Controlled Trials ISRCTN12010952 and ClinicalTrials.gov NCT02458001. Funding This project was funded by the NIHR HTA programme and will be published in full in Health Technology Assessment; Vol. 23, No. 5. See the NIHR Journals Library website for further project information.

PP151 Establishing Health Technology Assessment Impact Evaluation With Stakeholder Input From Day One

2019 ◽  
Vol 35 (S1) ◽  
pp. 66-66
Author(s):  
Ruth Louise Poole ◽  
Sophie Hughes ◽  
Lauren Elston ◽  
Susan Myles
Keyword(s):  
Health Technology Assessment ◽  
Technology Assessment ◽  
Impact Evaluation ◽  
Health Technology ◽  
Support Staff ◽  
Evaluation Plan ◽  
Individual Interviews ◽  
The Core ◽  
Stakeholder Input ◽  
Written Materials

IntroductionHealth Technology Wales (HTW) is a relatively new Health Technology Assessment (HTA) agency which focuses on non-medicines. In common with other HTA organizations, it identifies and appraises a range of technologies. However, HTW is also looking beyond the publication of guidance, to assess the adoption of advice and its eventual impact.MethodsHTW commissioned development of an Evaluation Plan from independent experts (Matter of Focus). A literature review was carried out to inform an options appraisal of methods for assessing impact. The selected approach was Contribution Analysis, which estimates the counterfactual through engagement of stakeholders.ResultsWhilst it is too early to report the full impact of HTW's guidance, a number of activities have taken place to prepare for evaluation. The core HTW team developed a series of logic models to describe the anticipated impact, the mechanisms by which it would be achieved, and key assumptions. Stakeholders were consulted for insight from a range of perspectives, and to manage expectations. This was achieved through individual interviews, presentation and discussion at committee meetings, and the sharing of written materials for feedback. This information was collated to populate bespoke software (OutNav). The collection of data relating to processes, outputs and outcomes is already an ongoing routine task of researchers and support staff.ConclusionsHTW has an opportunity to build impact evaluation into its culture from the beginning. This will facilitate the future reporting of HTW's influence using a well-designed, evidence-based approach. Furthermore, this pioneering work will clearly demonstrate the value of HTA to funders, commissioners, governments, and other decision-making bodies.

scholarly journals Application of Health Technology Assessment (HTA) to Evaluate New Laboratory Tests in a Health System: A Case Study of Bladder Cancer Testing

2020 ◽  
Vol 7 ◽  
pp. 237428952096822
Author(s):  
Erik J. Landaas ◽  
Ashley M. Eckel ◽  
Jonathan L. Wright ◽  
Geoffrey S. Baird ◽  
Ryan N. Hansen ◽  
...  
Keyword(s):  
Bladder Cancer ◽  
Pilot Study ◽  
Health Technology Assessment ◽  
Technology Assessment ◽  
Real World ◽  
Health Technology ◽  
Test System ◽  
Real World Data ◽  
World Data

We describe the methods and decision from a health technology assessment of a new molecular test for bladder cancer (Cxbladder), which was proposed for adoption to our send-out test menu by urology providers. The Cxbladder health technology assessment report contained mixed evidence; predominant concerns were related to the test’s low specificity and high cost. The low specificity indicated a high false-positive rate, which our laboratory formulary committee concluded would result in unnecessary confirmatory testing and follow-up. Our committee voted unanimously to not adopt the test system-wide for use for the initial diagnosis of bladder cancer but supported a pilot study for bladder cancer recurrence surveillance. The pilot study used real-world data from patient management in the scenario in which a patient is evaluated for possible recurrent bladder cancer after a finding of atypical cytopathology in the urine. We evaluated the type and number of follow-up tests conducted including urine cytopathology, imaging studies, repeat cystoscopy evaluation, biopsy, and repeat Cxbladder and their test results. The pilot identified ordering challenges and suggested potential use cases in which the results of Cxbladder affected a change in management. Our health technology assessment provided an objective process to efficiently review test performance and guide new test adoption. Based on our pilot, there were real-world data indicating improved clinician decision-making among select patients who underwent Cxbladder testing.

scholarly journals Enhanced motivational interviewing for reducing weight and increasing physical activity in adults with high cardiovascular risk: the MOVE IT three-arm RCT

2019 ◽  
Vol 23 (69) ◽  
pp. 1-144
Author(s):  
Khalida Ismail ◽  
Daniel Stahl ◽  
Adam Bayley ◽  
Katherine Twist ◽  
Kurtis Stewart ◽  
...  
Keyword(s):  
Physical Activity ◽  
Cost Effectiveness ◽  
Motivational Interviewing ◽  
Health Technology Assessment ◽  
Technology Assessment ◽  
Health Technology ◽  
Research Centre ◽  
Cvd Risk ◽  
Objective Evidence

Background Motivational interviewing (MI) enhanced with behaviour change techniques (BCTs) and deployed by health trainers targeting multiple risk factors for cardiovascular disease (CVD) may be more effective than interventions targeting a single risk factor. Objectives The clinical effectiveness and cost-effectiveness of an enhanced lifestyle motivational interviewing intervention for patients at high risk of CVD in group settings versus individual settings and usual care (UC) in reducing weight and increasing physical activity (PA) were tested. Design This was a three-arm, single-blind, parallel randomised controlled trial. Setting A total of 135 general practices across all 12 South London Clinical Commissioning Groups were recruited. Participants A total of 1742 participants aged 40–74 years with a ≥ 20.0% risk of a CVD event in the following 10 years were randomised. Interventions The intervention was designed to integrate MI and cognitive–behavioural therapy (CBT), delivered by trained healthy lifestyle facilitators in 10 sessions over 1 year, in group or individual format. The control group received UC. Randomisation Simple randomisation was used with computer-generated randomisation blocks. In each block, 10 participants were randomised to the group, individual or UC arm in a 4 : 3 : 3 ratio. Researchers were blind to the allocation. Main outcome measures The primary outcomes are change in weight (kg) from baseline and change in PA (average number of steps per day over 1 week) from baseline at the 24-month follow-up, with an interim follow-up at 12 months. An economic evaluation estimates the relative cost-effectiveness of each intervention. Secondary outcomes include changes in low-density lipoprotein cholesterol and CVD risk score. Results The mean age of participants was 69.75 years (standard deviation 4.11 years), 85.5% were male and 89.4% were white. At the 24-month follow-up, the group and individual intervention arms were not more effective than UC in increasing PA [mean 70.05 steps, 95% confidence interval (CI) –288 to 147.9 steps, and mean 7.24 steps, 95% CI –224.01 to 238.5 steps, respectively] or in reducing weight (mean –0.03 kg, 95% CI –0.49 to 0.44 kg, and mean –0.42 kg, 95% CI –0.93 to 0.09 kg, respectively). At the 12-month follow-up, the group and individual intervention arms were not more effective than UC in increasing PA (mean 131.1 steps, 95% CI –85.28 to 347.48 steps, and mean 210.22 steps, 95% CI –19.46 to 439.91 steps, respectively), but there were reductions in weight for the group and individual intervention arms compared with UC (mean –0.52 kg, 95% CI –0.90 to –0.13 kg, and mean –0.55 kg, 95% CI –0.95 to –0.14 kg, respectively). The group intervention arm was not more effective than the individual intervention arm in improving outcomes at either follow-up point. The group and individual interventions were not cost-effective. Conclusions Enhanced MI, in group or individual formats, targeted at members of the general population with high CVD risk is not effective in reducing weight or increasing PA compared with UC. Future work should focus on ensuring objective evidence of high competency in BCTs, identifying those with modifiable factors for CVD risk and improving engagement of patients and primary care. Trial registration Current Controlled Trials ISRCTN84864870. Funding This project was funded by the National Institute for Health Research (NIHR) Health Technology Assessment programme and will be published in full in Health Technology Assessment; Vol. 23, No. 69. See the NIHR Journals Library website for further project information. This research was part-funded by the NIHR Biomedical Research Centre at South London and Maudsley NHS Foundation Trust and King’s College London.

scholarly journals Sexual risk reduction interventions for patients attending sexual health clinics: a mixed-methods feasibility study

2019 ◽  
Vol 23 (12) ◽  
pp. 1-122 ◽  
Author(s):  
Carina King ◽  
Carrie Llewellyn ◽  
Maryam Shahmanesh ◽  
Charles Abraham ◽  
Julia Bailey ◽  
...  
Keyword(s):  
Systematic Review ◽  
Mixed Methods ◽  
Sexual Risk ◽  
Technology Assessment ◽  
Health Technology ◽  
Intervention Package ◽  
Sexual Risk Reduction ◽  
Digital Interventions ◽  
One To One

Background Sexually transmitted infections (STIs) continue to represent a major public health challenge. There is evidence that behavioural interventions to reduce risky sexual behaviours can reduce STI rates in patients attending sexual health (SH) services. However, it is not known if these interventions are effective when implemented at scale in SH settings in England. Objectives The study (Santé) had two main objectives – (1) to develop and pilot a package of evidence-based sexual risk reduction interventions that can be delivered through SH services and (2) to assess the feasibility of conducting a randomised controlled trial (RCT) to determine effectiveness against usual care. Design The project was a multistage, mixed-methods study, with developmental and pilot RCT phases. Preparatory work included a systematic review, an analysis of national surveillance data, the development of a triage algorithm, and interviews and surveys with SH staff and patients to identify, select and adapt interventions. A pilot cluster RCT was planned for eight SH clinics; the intervention would be offered in four clinics, with qualitative and process evaluation to assess feasibility and acceptability. Four clinics acted as controls; in all clinics, participants would be consented to a 6-week follow-up STI screen. Setting SH clinics in England. Participants Young people (aged 16–25 years), and men who have sex with men. Intervention A three-part intervention package – (1) a triage tool to score patients as being at high or low risk of STI using routine data, (2) a study-designed web page with tailored SH information for all patients, regardless of risk and (3) a brief one-to-one session based on motivational interviewing for high-risk patients. Main outcome measures The three outcomes were (1) the acceptability of the intervention to patients and SH providers, (2) the feasibility of delivering the interventions within existing resources and (3) the feasibility of obtaining follow-up data on STI diagnoses (primary outcome in a full trial). Results We identified 33 relevant trials from the systematic review, including videos, peer support, digital and brief one-to-one sessions. Patients and SH providers showed preferences for one-to-one and digital interventions, and providers indicated that these intervention types could feasibly be implemented in their settings. There were no appropriate digital interventions that could be adapted in time for the pilot; therefore, we created a placeholder for the purposes of the pilot. The intervention package was piloted in two SH settings, rather than the planned four. Several barriers were found to intervention implementation, including a lack of trained staff time and clinic space. The intervention package was theoretically acceptable, but we observed poor engagement. We recruited patients from six clinics for the follow-up, rather than eight. The completion rate for follow-up was lower than anticipated (16% vs. 46%). Limitations Fewer clinics were included in the pilot than planned, limiting the ability to make strong conclusions on the feasibility of the RCT. Conclusion We were unable to conclude whether or not a definitive RCT would be feasible because of challenges in implementation of a pilot, but have laid the groundwork for future research in the area. Trial registration Current Controlled Trials ISRCTN16738765. Funding This project was funded by the National Institute for Health Research (NIHR) Health Technology Assessment programme and will be published in full in Health Technology Assessment; Vol. 23, No. 12. See the NIHR Journals Library website for further project information.

scholarly journals Extrapolating Parametric Survival Models in Health Technology Assessment: A Simulation Study

2020 ◽  
Vol 41 (1) ◽  
pp. 37-50
Author(s):  
Daniel Gallacher ◽  
Peter Kimani ◽  
Nigel Stallard
Keyword(s):  
Health Technology Assessment ◽  
Technology Assessment ◽  
Goodness Of Fit ◽  
Information Criterion ◽  
Health Technology ◽  
Survival Models ◽  
Fit Statistics ◽  
Long Term Treatment ◽  
Parametric Survival

Extrapolations of parametric survival models fitted to censored data are routinely used in the assessment of health technologies to estimate mean survival, particularly in diseases that potentially reduce the life expectancy of patients. Akaike’s information criterion (AIC) and Bayesian information criterion (BIC) are commonly used in health technology assessment alongside an assessment of plausibility to determine which statistical model best fits the data and should be used for prediction of long-term treatment effects. We compare fit and estimates of restricted mean survival time (RMST) from 8 parametric models and contrast models preferred in terms of AIC, BIC, and log-likelihood, without considering model plausibility. We assess the methods’ suitability for selecting a parametric model through simulation of data replicating the follow-up of intervention arms for various time-to-event outcomes from 4 clinical trials. Follow-up was replicated through the consideration of recruitment duration and minimum and maximum follow-up times. Ten thousand simulations of each scenario were performed. We demonstrate that the different methods can result in disagreement over the best model and that it is inappropriate to base model selection solely on goodness-of-fit statistics without consideration of hazard behavior and plausibility of extrapolations. We show that typical trial follow-up can be unsuitable for extrapolation, resulting in unreliable estimation of multiple parameter models, and infer that selecting survival models based only on goodness-of-fit statistics is unsuitable due to the high level of uncertainty in a cost-effectiveness analysis. This article demonstrates the potential problems of overreliance on goodness-of-fit statistics when selecting a model for extrapolation. When follow-up is more mature, BIC appears superior to the other selection methods, selecting models with the most accurate and least biased estimates of RMST.

scholarly journals Comparing open and minimally invasive surgical procedures for oesophagectomy in the treatment of cancer: the ROMIO (Randomised Oesophagectomy: Minimally Invasive or Open) feasibility study and pilot trial

2016 ◽  
Vol 20 (48) ◽  
pp. 1-68 ◽  
Author(s):  
Chris Metcalfe ◽  
Kerry Avery ◽  
Richard Berrisford ◽  
Paul Barham ◽  
Sian M Noble ◽  
...  
Keyword(s):  
Minimally Invasive ◽  
Health Technology Assessment ◽  
Technology Assessment ◽  
Open Surgery ◽  
Outcome Measure ◽  
Pilot Trial ◽  
Health Technology ◽  
Definitive Trial ◽  
Post Surgery ◽  
Patient Reported

BackgroundLocalised oesophageal cancer can be curatively treated with surgery (oesophagectomy) but the procedure is complex with a risk of complications, negative effects on quality of life and a recovery period of 6–9 months. Minimal-access surgery may accelerate recovery.ObjectivesThe ROMIO (Randomised Oesophagectomy: Minimally Invasive or Open) study aimed to establish the feasibility of, and methodology for, a definitive trial comparing minimally invasive and open surgery for oesophagectomy. Objectives were to quantify the number of eligible patients in a pilot trial; develop surgical manuals as the basis for quality assurance; standardise pathological processing; establish a method to blind patients to their allocation in the first week post surgery; identify measures of postsurgical outcome of importance to patients and clinicians; and establish the main cost differences between the surgical approaches.DesignPilot parallel three-arm randomised controlled trial nested within feasibility work.SettingTwo UK NHS departments of upper gastrointestinal surgery.ParticipantsPatients aged ≥ 18 years with histopathological evidence of oesophageal or oesophagogastric junctional adenocarcinoma, squamous cell cancer or high-grade dysplasia, referred for oesophagectomy or oesophagectomy following neoadjuvant chemo(radio)therapy.InterventionsOesophagectomy, with patients randomised to open surgery, a hybrid open chest and minimally invasive abdomen or totally minimally invasive access.Main outcome measureThe primary outcome measure for the pilot trial was the number of patients recruited per month, with the main trial considered feasible if at least 2.5 patients per month were recruited.ResultsDuring 21 months of recruitment, 263 patients were assessed for eligibility; of these, 135 (51%) were found to be eligible and 104 (77%) agreed to participate, an average of five patients per month. In total, 41 patients were allocated to open surgery, 43 to the hybrid procedure and 20 to totally minimally invasive surgery. Recruitment is continuing, allowing a seamless transition into the definitive trial. Consequently, the database is unlocked at the time of writing and data presented here are for patients recruited by 31 August 2014. Random allocation achieved a good balance between the arms of the study, which, as a high proportion of patients underwent their allocated surgery (69/79, 87%), ensured a fair comparison between the interventions. Dressing patients with large bandages, covering all possible incisions, was successful in keeping patients blind while pain was assessed during the first week post surgery. Postsurgical length of stay and risk of adverse events were within the typical range for this group of patients, with one death occurring within 30 days among 76 patients. There were good completion rates for the assessment of pain at 6 days post surgery (88%) and of the patient-reported outcomes at 6 weeks post randomisation (74%).ConclusionsRapid recruitment to the pilot trial and the successful refinement of methodology indicated the feasibility of a definitive trial comparing different approaches to oesophagectomy. Although we have shown a full trial of open compared with minimally invasive oesophagectomy to be feasible, this is necessarily based on our findings from the two clinical centres that we could include in this small preliminary study.Trial registrationCurrent Controlled Trials ISRCTN59036820.FundingThis project was funded by the NIHR Health Technology Assessment programme and will be published in full inHealth Technology Assessment; Vol. 20, No. 48. See the NIHR Journals Library website for further project information.

scholarly journals Why do health technology assessment drug reimbursement recommendations differ between countries? A parallel convergent mixed methods study

2019 ◽  
Vol 15 (3) ◽  
pp. 386-402 ◽  
Author(s):  
Elena Nicod ◽  
Laia Maynou ◽  
Erica Visintin ◽  
John Cairns
Keyword(s):  
Mixed Methods ◽  
Health Technology Assessment ◽  
Conceptual Framework ◽  
Technology Assessment ◽  
Health Technology ◽  
Deliberative Process ◽  
Drug Reimbursement ◽  
Quantitative And Qualitative Research ◽  
Research Designs ◽  
Stakeholder Input

AbstractUsing quantitative and qualitative research designs, respectively, two studies investigated why countries make different health technology assessment (HTA) drug reimbursement recommendations. Building on these, the objective of this study was to (a) develop a conceptual framework integrating the factors explaining these decisions, (b) explore their relationship and (c) assess if they are congruent, complementary or discrepant. A parallel convergent mixed methods design was used. Countries included in both previous studies were selected (England, Sweden, Scotland and France). A conceptual framework that integrated and organised the factors explaining the decisions from the two studies was developed. Relationships between factors were explored and illustrated through case studies. The framework distinguishes macro-level factors from micro-level ones. Only two of the factors common to both studies were congruent, while two others reached discrepant conclusions (stakeholder input and external review of the evidence processes). The remaining factors identified within one or both studies were complementary. Bringing together these findings contributed to generating a more complete picture of why countries make different HTA recommendations. Results were mostly complementary, explaining and enhancing each other. We conclude that differences often result from a combination of factors, with an important component relating to what occurs during the deliberative process.

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