scholarly journals Feasibility and design of a trial regarding the optimal mode of delivery for preterm birth: the CASSAVA multiple methods study

2021 ◽  
Vol 25 (61) ◽  
pp. 1-102
Author(s):  
Jane E Norman ◽  
Julia Lawton ◽  
Sarah J Stock ◽  
Dimitrios Siassakos ◽  
John Norrie ◽  
...  
Keyword(s):  
Clinical Trial ◽  
Preterm Birth ◽  
Caesarean Section ◽  
Technology Assessment ◽  
Mode Of Delivery ◽  
Randomised Trial ◽  
Health Technology ◽  
Future Trial ◽  
Data Saturation ◽  
Optimal Mode

Background Around 60,000 babies are born preterm (prior to 37 weeks’ gestation) each year in the UK. There is little evidence on the optimal birth mode (vaginal or caesarean section). Objective The overall aim of the CASSAVA project was to determine if a trial to define the optimal mode of preterm birth could be carried out and, if so, determine what sort of trial could be conducted and how it could best be performed. We aimed to determine the specific groups of preterm women and babies for whom there are uncertainties about the best planned mode of birth, and if there would be willingness to recruit to, and participate in, a randomised trial to address some, but not all, of these uncertainties. This project was conducted in response to a Heath Technology Assessment programme commissioning call (17/22 ‘Mode of delivery for preterm infants’). Methods We conducted clinician and patient surveys (n = 224 and n = 379, respectively) to identify current practice and opinion, and a consensus survey and Delphi workshop (n = 76 and n = 22 participants, respectively) to inform the design of a hypothetical clinical trial. The protocol for this clinical trial/vignette was used in telephone interviews with clinicians (n = 24) and in focus groups with potential participants (n = 13). Results Planned sample size and data saturation was achieved for all groups except for focus groups with participants, as this had to be curtailed because of the COVID-19 pandemic and data saturation was not achieved. There was broad agreement from parents and health-care professionals that a trial is needed. The clinician survey demonstrated a variety of practice and opinion. The parent survey suggested that women and their families generally preferred vaginal birth at later gestations and caesarean section for preterm infants. The interactive workshop and Delphi consensus process confirmed the need for more evidence (hence the case for a trial) and provided rich information on what a future trial should entail. It was agreed that any trial should address the areas with most uncertainty, including the management of women at 26–32 weeks’ gestation, with either spontaneous preterm labour (cephalic presentation) or where preterm birth was medically indicated. Clear themes around the challenges inherent in conducting any trial emerged, including the concept of equipoise itself. Specific issues were as follows: different clinicians and participants would be in equipoise for each clinical scenario, effective conduct of the trial would require appropriate resources and expertise within the hospital conducting the trial, potential participants would welcome information on the trial well before the onset of labour and minority ethnic groups would require tailored approaches. Conclusion Given the lack of evidence and the variation of practice and opinion in this area, and having listened to clinicians and potential participants, we conclude that a trial should be conducted and the outlined challenges resolved. Future work The CASSAVA project could be used to inform the design of a randomised trial and indicates how such a trial could be carried out. Any future trial would benefit from a pilot with qualitative input and a study within a trial to inform optimal recruitment. Limitations Certainty that a trial could be conducted can be determined only when it is attempted. Trial registration Current Controlled Trials ISRCTN12295730. 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. 25, No. 61. See the NIHR Journals Library website for further project information.

Routine use of progesterone does not prevent miscarriage

BMJ ◽  
2019 ◽  
pp. l5721
Author(s):  
Rob Cook ◽  
Vaughan Thomas ◽  
Rachel Taft
Keyword(s):  
Health Technology Assessment ◽  
Early Pregnancy ◽  
Technology Assessment ◽  
Randomised Trial ◽  
Health Technology ◽  
Assessment Programme ◽  
Link Type ◽  
Health Technology Assessment Programme

The studyCoomarasamy A, Devall AJ, Cheed V, et al. A randomised trial of progesterone in women with bleeding in early pregnancy N Engl J Med 2019;380:1815-24.This project was funded by the NIHR Health Technology Assessment Programme (project number 12/167/26).To read the full NIHR Signal, go to https://discover.dc.nihr.ac.uk/content/signal-000792/miscarriage-not-prevented-by-routine-use-of-progesterone

scholarly journals E-cigarettes helped more smokers quit than nicotine replacement therapy

BMJ ◽  
2019 ◽  
pp. l2036 ◽  
Author(s):  
Rob Cook ◽  
Peter Davidson ◽  
Rosie Martin
Keyword(s):  
Cancer Research ◽  
Health Technology Assessment ◽  
Replacement Therapy ◽  
Nicotine Replacement Therapy ◽  
Technology Assessment ◽  
Randomised Trial ◽  
Health Technology ◽  
Nicotine Replacement ◽  
Assessment Programme ◽  
Health Technology Assessment Programme

The studyHajek P, Phillips-Waller A, Przulj D, et al. A randomised trial of e-cigarettes versus nicotine-replacement therapy. N Engl J Med 2019;380:629-37.This trial was funded by the NIHR Health Technology Assessment Programme (12/167/135) and by Cancer Research UK.To read the full NIHR Signal, go to https://discover.dc.nihr.ac.uk/content/signal-000757/e-cigarettes-helped-more-smokers-quit-than-nicotine-replacement-therapy

scholarly journals Surveillance versus ablation for incidentally diagnosed small renal tumours: the SURAB feasibility RCT

2017 ◽  
Vol 21 (81) ◽  
pp. 1-68 ◽  
Author(s):  
Naeem Soomro ◽  
Jan Lecouturier ◽  
Deborah D Stocken ◽  
Jing Shen ◽  
Ann Marie Hynes ◽  
...  
Keyword(s):  
Health Technology Assessment ◽  
Active Surveillance ◽  
Feasibility Study ◽  
Technology Assessment ◽  
Randomised Trial ◽  
Health Technology ◽  
Serious Adverse Events ◽  
Design Work ◽  
Renal Tumours ◽  
Stage 1

Background There is uncertainty around the appropriate management of small renal tumours. Treatments include partial nephrectomy, ablation and active surveillance. Objectives To explore the feasibility of a randomised trial of ablation versus active surveillance. Design Two-stage feasibility study: stage 1 – clinician survey and co-design work; and stage 2 – randomised feasibility study with qualitative and economic components. Methods Stage 1 – survey of radiologists and urologists, and development of patient information materials. Stage 2 – patients identified across eight UK centres with small renal tumours (< 4 cm) were randomised (1 : 1 ratio) to ablation or active surveillance in an unblinded manner. Randomisation was carried out by a central computer system. The primary objective was to determine willingness to participate and to randomise a target of 60 patients. The qualitative and economic data were collected separately. Results The trial was conducted across eight centres, with a site-specific period of recruitment ranging from 3 to 11 months. Of the 154 patients screened, 36 were eligible and were provided with study details. Seven agreed to be randomised and one patient was found ineligible following biopsy results. Six patients (17% of those eligible) were randomised: three patients received ablation and no serious adverse events were recorded. The 3- and 6-month data were collected for four (67%) and three (50%) out of the six patients, respectively. The qualitative substudy identified factors directly impacting on the recruitment of this trial. These included patient and clinician preferences, organisational factors (variation in clinical pathway) and standard treatment not included. The health economic questionnaire was designed and piloted; however, the sample size of recruited patients was insufficient to draw a conclusion on the feasibility of the health economics. Conclusions The trial did not meet the criteria for progression and the recruitment rate was lower than hypothesised, demonstrating that a full trial is presently not possible. The qualitative study identified factors that led to variation in recruitment across the sites. Implementation of organisational and operational measures can increase recruitment in any future trial. There was insufficient information to conduct a full economic analysis. Trial registration Current Controlled Trials ISRCTN31161700. 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. 21, No. 81. See the NIHR Journals Library website for further project information.

scholarly journals Aspects of the implementation of economic models for health technology assessment in clinical trials of medicines

2021 ◽  
pp. 45-52
Author(s):  
O. V. Shtrimaitis ◽  
V. Ye. Dobrova
Keyword(s):  
Clinical Trial ◽  
Clinical Trials ◽  
Health Technology Assessment ◽  
Technology Assessment ◽  
Pharmacoeconomic Analysis ◽  
Economic Models ◽  
Health Technology ◽  
Potential Health ◽  
Planning Stage ◽  
Effectiveness Analysis

The aim of the work is to scientifically substantiate the basic aspects of developing a methodology for including сost-effectiveness analysis in key processes of clinical trials of medicines. Results. It is established that Ukrainian regulatory system and methodological support of clinical trials with included сost-effectiveness analysis is based on separate regulations in the field of clinical trial and health technology assessment. At the same time, there is no methodological support for the management of сost-effectiveness analysis alongside clinical trials, which would take into account the peculiarities of planning, organizing and conducting such studies. The algorithm of substantiation, development and inclusion of basic components of the analysis of economic effectiveness in the basic processes of planning and the organization of clinical trial of medicines is offered. Emphasis is placed on the need for a proper strategy for analyzing the economic efficiency of the studied medical technologies in clinical trials. The importance of proper selection of economic evaluation models (cost minimization, cost-effectiveness, cost-benefit) in accordance with the purpose of the clinical trial, the primary hypothesis and the selected primary and secondary endpoints is determined. Conclusions. It is determined that there are certain problems of methodological and organizational nature for the development of clinical trials with included сost-effectiveness analysis and economic endpoints. The solution of these tasks should begin at the planning stage of the study by implementing the basic step-by-step recommendations. Innovative treatment technologies that have potential health benefits are often investigated in clinical trials. Thus, the introduction of pharmacoeconomic analysis methods into the structure of traditional clinical trials is an additional important tool to ensure the effective functioning and development of health technology assessment. In further research it is planned to develop scientific and practical approaches to ensure the proper implementation of management processes of сost-effectiveness analysis alongside clinical trial and analysis of data on their results. Keywords: clinical trials; health technology assessment; algorithms; economic models; procedures

scholarly journals Aquatic therapy for children with Duchenne muscular dystrophy: a pilot feasibility randomised controlled trial and mixed-methods process evaluation

2017 ◽  
Vol 21 (27) ◽  
pp. 1-120 ◽  
Author(s):  
Daniel Hind ◽  
James Parkin ◽  
Victoria Whitworth ◽  
Saleema Rex ◽  
Tracey Young ◽  
...  
Keyword(s):  
Duchenne Muscular Dystrophy ◽  
Randomised Controlled Trial ◽  
Muscular Dystrophy ◽  
Health Technology Assessment ◽  
Technology Assessment ◽  
Controlled Trial ◽  
Randomised Trial ◽  
Health Technology ◽  
Aquatic Therapy ◽  
Randomised Controlled

BackgroundDuchenne muscular dystrophy (DMD) is a rare disease that causes the progressive loss of motor abilities such as walking. Standard treatment includes physiotherapy. No trial has evaluated whether or not adding aquatic therapy (AT) to land-based therapy (LBT) exercises helps to keep muscles strong and children independent.ObjectivesTo assess the feasibility of recruiting boys with DMD to a randomised trial evaluating AT (primary objective) and to collect data from them; to assess how, and how well, the intervention and trial procedures work.DesignParallel-group, single-blind, randomised pilot trial with nested qualitative research.SettingSix paediatric neuromuscular units.ParticipantsChildren with DMD aged 7–16 years, established on corticosteroids, with a North Star Ambulatory Assessment (NSAA) score of 8–34 and able to complete a 10-m walk without aids/assistance. Exclusions: > 20% variation between baseline screens 4 weeks apart and contraindications.InterventionsParticipants were allocated on a 1 : 1 ratio to (1) optimised, manualised LBT (prescribed by specialist neuromuscular physiotherapists) or (2) the same plus manualised AT (30 minutes, twice weekly for 6 months: active assisted and/or passive stretching regime; simulated or real functional activities; submaximal exercise). Semistructured interviews with participants, parents (n = 8) and professionals (n = 8) were analysed using Framework analysis. An independent rater reviewed patient records to determine the extent to which treatment was optimised. A cost-impact analysis was performed. Quantitative and qualitative data were mixed using a triangulation exercise.Main outcome measuresFeasibility of recruiting 40 participants in 6 months, participant and therapist views on the acceptability of the intervention and research protocols, clinical outcomes including NSAA, independent assessment of treatment optimisation and intervention costs.ResultsOver 6 months, 348 children were screened – most lived too far from centres or were enrolled in other trials. Twelve (30% of target) were randomised to AT (n = 8) or control (n = 4). People in the AT (n = 8) and control (n = 2: attrition because of parental report) arms contributed outcome data. The mean change in NSAA score at 6 months was –5.5 [standard deviation (SD) 7.8] for LBT and –2.8 (SD 4.1) in the AT arm. One boy suffered pain and fatigue after AT, which resolved the same day. Physiotherapists and parents valued AT and believed that it should be delivered in community settings. The independent rater considered AT optimised for three out of eight children, with other children given programmes that were too extensive and insufficiently focused. The estimated NHS costs of 6-month service were between £1970 and £2734 per patient.LimitationsThe focus on delivery in hospitals limits generalisability.ConclusionsNeither a full-scale frequentist randomised controlled trial (RCT) recruiting in the UK alone nor a twice-weekly open-ended AT course delivered at tertiary centres is feasible. Further intervention development research is needed to identify how community-based pools can be accessed, and how families can link with each other and community physiotherapists to access tailored AT programmes guided by highly specialised physiotherapists. Bayesian RCTs may be feasible; otherwise, time series designs are recommended.Trial registrationCurrent Controlled Trials ISRCTN41002956.FundingThis 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. 21, No. 27. See the NIHR Journals Library website for further project information.

scholarly journals Postal recruitment for genetic studies of preterm birth: A feasibility study

2020 ◽  
Vol 5 ◽  
pp. 26
Author(s):  
Oonagh E. Keag ◽  
Lee Murphy ◽  
Aoibheann Bradley ◽  
Naomi Deakin ◽  
Sonia Whyte ◽  
...  
Keyword(s):  
Preterm Birth ◽  
Dna Extraction ◽  
Feasibility Study ◽  
Mode Of Delivery ◽  
Randomised Trial ◽  
Prospective Trial ◽  
Data Set ◽  
Genetic Studies ◽  
Two Samples ◽  
Pregnancy And Birth

Background: Preterm birth (PTB) represents the leading cause of neonatal death. Large-scale genetic studies are necessary to determine genetic influences on PTB risk, but prospective cohort studies are expensive and time-consuming. We investigated the feasibility of retrospective recruitment of post-partum women for efficient collection of genetic samples, with self-collected saliva for DNA extraction from themselves and their babies, alongside self-recollection of pregnancy and birth details to phenotype PTB. Methods: 708 women who had participated in the OPPTIMUM trial (a randomised trial of progesterone pessaries to prevent PTB [ISRCTN14568373]) and consented to further contact were invited to provide self-collected saliva from themselves and their babies. DNA was extracted from Oragene OG-500 (adults) and OG-575 (babies) saliva kits and the yield measured by Qubit. Samples were analysed using a panel of Taqman single nucleotide polymorphism (SNP) assays. A questionnaire designed to meet the minimum data set required for phenotyping PTB was included. Questionnaire responses were transcribed and analysed for concordance with prospective trial data using Cohen’s kappa (k). Results: Recruitment rate was 162/708 (23%) for self-collected saliva samples and 157/708 (22%) for questionnaire responses. 161 samples from the mother provided DNA with median yield 59.0µg (0.4-148.9µg). 156 samples were successfully genotyped (96.9%). 136 baby samples had a median yield 11.5µg (0.1-102.7µg); two samples failed DNA extraction. 131 baby samples (96.3%) were successfully genotyped. Concordance between self-recalled birth details and prospective birth details was excellent (k>0.75) in 4 out of 10 key fields for phenotyping PTB (mode of delivery, labour onset, ethnicity and maternal age at birth). Conclusion: This feasibility study demonstrates that self-collected DNA samples from mothers and babies were sufficient for genetic analysis but yields were variable. Self-recollection of pregnancy and birth details was inadequate for accurately phenotyping PTB, highlighting the need for alternative strategies for investigating genetic links with PTB.

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