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BACKGROUND: Bespoke self-report resource-use measures (RUMs) are commonly developed or adapted for each new randomised controlled trial. Consequently, RUMs lack standardisation and validation is rarely conducted. A new generic RUM, ModRUM, has been developed using a rigorous process, including consultation with health economists and patients. ModRUM includes a concise core healthcare module, designed to be included in all trials, and depth-adding questions, which can replace or be added to core questions as needed. Modules covering other sectors are under development. The aim of this study was to test the acceptability, feasibility, and criterion and construct validity of the healthcare module of ModRUM. METHODS: Patients who had a recent appointment at their GP practice were invited to complete ModRUM (core module or core module with depth questions), a characteristics form and the EQ-5D-5L. Acceptability was assessed via response rates and questionnaire completion time. Feasibility was assessed by reviewing issues observed in participants' responses and question completion rates. Construct validity was tested via hypothesis testing and known-group analyses, using Wilcoxon rank-sum and Kruskal-Wallis tests, and a generalised linear model. Criterion validity was tested by comparing ModRUM results with primary care medical records. Sensitivity, specificity, and agreement using Lin's concordance correlation coefficient (pc) were estimated. RESULTS: One hundred patients participated from five GP practices in the South-West of England. Acceptability was higher for the core module (20% versus 10% response rate). Question completion rates were high across both versions (> 90%). Some support was observed for construct validity, with results suggesting that healthcare costs differ dependent on the number of long-term conditions (p < 0.05) and are negatively associated with health-related quality of life (p < 0.01). Sensitivity was high for all questions (> 0.83), while specificity varied (0.33-0.88). There was a good level of agreement for GP contacts and costs, and prescribed medication costs (pc > 0.6). CONCLUSION: This study provided preliminary evidence of the acceptability, feasibility, and criterion and construct validity of ModRUM. Further testing is required within trials and with groups that were less well represented in this study.
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Neoplasias da Mama , Qualidade de Vida , Humanos , Feminino , Custos de Medicamentos , Medicamentos Genéricos , InglaterraRESUMO
BACKGROUND: Screening men for prostate cancer using prostate-specific antigen (PSA) testing remains controversial. We aimed to estimate the likely budgetary impact on secondary care in England and Wales to inform screening decision makers. METHODS: The Cluster randomised triAl of PSA testing for Prostate cancer study (CAP) compared a single invitation to men aged 50-69 for a PSA test with usual care (no screening). Routinely collected hospital care data were obtained for all men in CAP, and NHS reference costs were mapped to each event via Healthcare Resource Group (HRG) codes. Secondary-care costs per man per year were calculated, and cost differences (and population-level estimates) between arms were derived annually for the first five years following randomisation. RESULTS: In the first year post-randomisation, secondary-care costs averaged across all men (irrespective of a prostate cancer diagnosis) in the intervention arm (n = 189279) were £44.80 (95% confidence interval: £18.30-£71.30) higher than for men in the control arm (n = 219357). Extrapolated to a population level, the introduction of a single PSA screening invitation could lead to additional secondary care costs of £314 million. CONCLUSIONS: Introducing a single PSA screening test for men aged 50-69 across England and Wales could lead to very high initial secondary-care costs.
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Antígeno Prostático Específico , Neoplasias da Próstata , Masculino , Humanos , País de Gales , Atenção Secundária à Saúde , Programas de Rastreamento , Neoplasias da Próstata/diagnóstico , InglaterraRESUMO
Anxiety and related disorders are increasingly widespread amongst children and adolescents. Preventing mental health disorders from developing has the potential to realise long-term benefits for children and adolescents. In their paper, 'Economic evidence of preventive interventions for anxiety disorders in children and adolescents-a systematic review', Vartiainen et al. conducted a systematic review to examine economic evidence of interventions for the primary prevention of anxiety disorders in children and adolescents, under 18 years of age. Five articles were eligible for inclusion in the review, of which two were model-based economic evaluations and three conducted alongside randomised controlled trials (RCTs). All five papers used either a cost-effectiveness analysis (CEA) or cost-utility analysis (CUA) as their main analysis. Vartiainen et al. concluded that, due to the small number of studies and relatively small sample sizes, the evidence for the cost-effectiveness of anxiety prevention interventions is weak. In this commentary, the challenges of conducting economic evaluations for prevention interventions are briefly outlined and Vartiainen et al.'s findings are discussed in the context of two further reviews of economic studies, published in 2021. The first focuses on the prevention of anxiety and depression in children and young people and the second takes a broader perspective and also includes interventions for mental health promotion. Both additional reviews note the small number of published economic evaluations, and all three reviews are united in their call for economic evaluations to be conducted alongside all future mental health prevention intervention trials.
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Ansiedade , Saúde Mental , Criança , Adolescente , Humanos , Análise Custo-Benefício , Transtornos de Ansiedade/terapia , Análise de Custo-EfetividadeRESUMO
OBJECTIVES: Health economics analysis plans (HEAPs) currently lack consistency, with uncertainty surrounding appropriate content. We aimed to develop a list of essential items that should be included in HEAPs for economic evaluations conducted alongside randomized trials. METHODS: A list of potential items for inclusion was developed by examining existing HEAPs. An electronic Delphi survey was conducted among professional health economists. Respondents were asked to rate potential items from 1 (least important) to 9 (most important), suggest additional items, and comment on proposed items (round 1). A second survey (round 2) was emailed to participants, including the participant's own scores from round 1 along with summary results from the whole panel; participants were asked to rerate each item. Consensus criteria for inclusion in the final list were predefined as >70% of participants rating an item 7-9 and <15% rating it 1-3 after round 2. A final item selection meeting was held to scrutinize the results and adjudicate on items lacking consensus. RESULTS: 62 participants completed round 1 of the survey. The initial list included 72 potential items; all 72 were carried forward to round 2, and no new items were added. 48 round 1 respondents (77.4%) completed round 2 and reached consensus on 53 items. At the final meeting, the expert panel (n = 9) agreed that 58 items should be included in the essential list, moved 9 items to an optional list, and dropped 5 items. CONCLUSIONS: Via expert consensus opinion, this study identified 58 items that are considered essential in a HEAP.
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Análise Custo-Benefício , Consenso , Análise Custo-Benefício/métodos , Análise Custo-Benefício/organização & administração , Técnica Delphi , Economia , Humanos , Ensaios Clínicos Controlados Aleatórios como Assunto , Inquéritos e QuestionáriosRESUMO
BACKGROUND: Self-report resource-use measures (RUMs) are often used to collect healthcare use data from participants in healthcare studies. However, RUMs are typically adapted from existing measures on a study-by-study basis, resulting in a lack of standardisation which limits comparability across studies. Psychometric testing of RUMs is rarely conducted. This paper reports on cognitive interviews with patients to test the content validity and acceptability of a new RUM (ModRUM). ModRUM is a brief, generic RUM with a core module on healthcare use and questions/modules to increase depth and breadth. METHODS: A purposeful sampling strategy with maximum variation was used to recruit patients from primary care to participate in "think-aloud" interviews with retrospective probing. Participants verbalised their thought processes as they completed ModRUM, which allowed errors (issues with completion) to be identified. The interviewer asked follow-up and probing questions to investigate errors, clarity and acceptability. Interviews were audio-recorded and transcribed verbatim. Research team members independently scored transcripts to identify errors in comprehension, recall, judgement and response. Members met to agree on final scores. Interview transcripts were analysed qualitatively using techniques of constant comparison, to identify common themes and ideas for improvement. Data collection and analysis were performed concurrently and in rounds. RESULTS: Twenty participants were interviewed between December 2019 and March 2020. Interviews were conducted in three rounds, with revisions made iteratively and in response to interview findings. Seven participants completed the core module and 13 completed the core module plus depth questions. Of 71 issues, 28 were in comprehension, 14 in retrieval, 10 in judgement, 18 in response and 1 uncategorised. Most issues (21 issues by 2 participants) were due to participants including family healthcare use. Other issues included using incorrect recall periods (5 issues) and overlooking questions leading to missing responses (9 issues). Common participant suggestions included highlighting important details and providing additional definition or examples for some terms. The length, content and layout were acceptable to most participants. CONCLUSIONS: A generic RUM is needed to increase study comparability. RUM development requires thorough testing to demonstrate and enhance validity. Cognitive interviewing has demonstrated the acceptability and content validity of ModRUM.
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Compreensão , Humanos , Psicometria , Reprodutibilidade dos Testes , Estudos Retrospectivos , Inquéritos e QuestionáriosRESUMO
BACKGROUND: There is limited evidence relating to the cost-effectiveness of treatments for localised prostate cancer. METHODS: The cost-effectiveness of active monitoring, surgery, and radiotherapy was evaluated within the Prostate Testing for Cancer and Treatment (ProtecT) randomised controlled trial from a UK NHS perspective at 10 years' median follow-up. Prostate cancer resource-use collected from hospital records and trial participants was valued using UK reference-costs. QALYs (quality-adjusted-life-years) were calculated from patient-reported EQ-5D-3L measurements. Adjusted mean costs, QALYs, and incremental cost-effectiveness ratios were calculated; cost-effectiveness acceptability curves and sensitivity analyses addressed uncertainty; subgroup analyses considered age and disease-risk. RESULTS: Adjusted mean QALYs were similar between groups: 6.89 (active monitoring), 7.09 (radiotherapy), and 6.91 (surgery). Active monitoring had lower adjusted mean costs (£5913) than radiotherapy (£7361) and surgery (£7519). Radiotherapy was the most likely (58% probability) cost-effective option at the UK NICE willingness-to-pay threshold (£20,000 per QALY). Subgroup analyses confirmed radiotherapy was cost-effective for older men and intermediate/high-risk disease groups; active monitoring was more likely to be the cost-effective option for younger men and low-risk groups. CONCLUSIONS: Longer follow-up and modelling are required to determine the most cost-effective treatment for localised prostate cancer over a man's lifetime. TRIAL REGISTRATION: Current Controlled Trials number, ISRCTN20141297: http://isrctn.org (14/10/2002); ClinicalTrials.gov number, NCT02044172: http://www.clinicaltrials.gov (23/01/2014).
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Neoplasias da Próstata/terapia , Adulto , Idoso , Análise Custo-Benefício , Custos de Cuidados de Saúde , Humanos , Masculino , Pessoa de Meia-Idade , Anos de Vida Ajustados por Qualidade de VidaRESUMO
OBJECTIVES: To see if a group course delivered by rheumatology teams using cognitive-behavioural approaches, plus usual care, reduced RA fatigue impact more than usual care alone. METHODS: Multicentre, 2-year randomised controlled trial in RA adults (fatigue severity>6/10, no recent major medication changes). RAFT (Reducing Arthritis Fatigue: clinical Teams using CB approaches) comprises seven sessions, codelivered by pairs of trained rheumatology occupational therapists/nurses. Usual care was Arthritis Research UK fatigue booklet. Primary 26-week outcome fatigue impact (Bristol RA Fatigue Effect Numerical Rating Scale, BRAF-NRS 0-10). Intention-to-treat regression analysis adjusted for baseline scores and centre. RESULTS: 308/333 randomised patients completed 26 week data (156/175 RAFT, 152/158 Control). Mean baseline variables were similar. At 26 weeks, the adjusted difference between arms for fatigue impact change favoured RAFT (BRAF-NRS Effect -0.59, 95% CI -1.11 to -0.06), BRAF Multidimensional Questionnaire (MDQ) Total -3.42 (95% CI -6.44 to -0.39), Living with Fatigue -1.19 (95% CI -2.17 to -0.21), Emotional Fatigue -0.91 (95% CI -1.58 to -0.23); RA Self-Efficacy (RASE, +3.05, 95% CI 0.43 to 5.66) (14 secondary outcomes unchanged). Effects persisted at 2 years: BRAF-NRS Effect -0.49 (95% CI -0.83 to -0.14), BRAF MDQ Total -2.98 (95% CI -5.39 to -0.57), Living with Fatigue -0.93 (95% CI -1.75 to -0.10), Emotional Fatigue -0.90 (95% CI -1.44, to -0.37); BRAF-NRS Coping +0.42 (95% CI 0.08 to 0.77) (relevance of fatigue impact improvement uncertain). RAFT satisfaction: 89% scored > 8/10 vs 54% controls rating usual care booklet (p<0.0001). CONCLUSION: Multiple RA fatigue impacts can be improved for 2 years by rheumatology teams delivering a group programme using cognitive behavioural approaches. TRIAL REGISTRATION NUMBER: ISRCTN52709998.
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Artrite Reumatoide/complicações , Terapia Cognitivo-Comportamental/métodos , Fadiga/terapia , Adaptação Psicológica , Adulto , Idoso , Artrite Reumatoide/psicologia , Emoções , Fadiga/etiologia , Fadiga/psicologia , Feminino , Humanos , Masculino , Pessoa de Meia-Idade , Equipe de Assistência ao Paciente , Satisfação do Paciente , Autocuidado/métodos , Índice de Gravidade de Doença , Classe Social , Resultado do TratamentoRESUMO
BACKGROUND: Randomised controlled trials (RCTs) are generally regarded as the "gold standard" for providing quantifiable evidence around the effectiveness and cost-effectiveness of new healthcare technologies. In order to perform the economic evaluations associated with RCTs, there is a need for accessible and good quality resource-use data; for the purpose of discussion here, data that best reflect the care received. Traditionally, researchers have developed questionnaires for resource-use data collection. However, the evolution of routinely collected electronic data within care services provides new opportunities for collecting data without burdening patients or caregivers (e.g. clinicians). This paper describes the potential strengths and limitations of each data collection method and then discusses aspects for consideration before choosing which method to use. MAIN TEXT: We describe electronic data sources (large observational datasets, commissioning data, and raw data extraction) that may be suitable data sources for informing clinical trials and the current status of self-reported instruments for measuring resource-use. We assess the methodological risks and benefits, and compare the two methodologies. We focus on healthcare resource-use; however, many of the considerations have relevance to clinical questions. Patient self-report forms a pragmatic and cheap method that is largely under the control of the researcher. However, there are known issues with the validity of the data collected, loss to follow-up may be high, and questionnaires suffer from missing data. Routinely collected electronic data may be more accurate and more practical if large numbers of patients are involved. However, datasets often incur a cost and researchers are bound by the time for data approval and extraction by the data holders. CONCLUSIONS: Owing to the issues associated with electronic datasets, self-reported methods may currently be the preferred option. However, electronic hospital data are relatively more accessible, informative, standardised, and reliable. Therefore in trials where secondary care constitutes a major driver of patient care, detailed electronic data may be considered superior to self-reported methods; with the caveat of requiring data sharing agreements with third party providers and potentially time-consuming extraction periods. Self-reported methods will still be required when a 'societal' perspective (e.g. quantifying informal care) is desirable for the intended analysis.
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Confiabilidade dos Dados , Conjuntos de Dados como Assunto/estatística & dados numéricos , Registros Eletrônicos de Saúde/estatística & dados numéricos , Autorrelato/estatística & dados numéricos , Inglaterra , Humanos , Ensaios Clínicos Controlados Aleatórios como AssuntoRESUMO
BACKGROUND: Resource use measurement by patient recall is characterized by inconsistent methods and a lack of validation. A validated standardized resource use measure could increase data quality, improve comparability between studies, and reduce research burden. OBJECTIVES: To identify a minimum set of core resource use items that should be included in a standardized adult instrument for UK health economic evaluation from a provider perspective. METHODS: Health economists with experience of UK-based economic evaluations were recruited to participate in an electronic Delphi survey. Respondents were asked to rate 60 resource use items (e.g., medication names) on a scale of 1 to 9 according to the importance of the item in a generic context. Items considered less important according to predefined consensus criteria were dropped and a second survey was developed. In the second round, respondents received the median score and their own score from round 1 for each item alongside summarized comments and were asked to rerate items. A final project team meeting was held to determine the recommended core set. RESULTS: Forty-five participants completed round 1. Twenty-six items were considered less important and were dropped, 34 items were retained for the second round, and no new items were added. Forty-two respondents (93.3%) completed round 2, and greater consensus was observed. After the final meeting, 10 core items were selected, with further items identified as suitable for "bolt-on" questionnaire modules. CONCLUSIONS: The consensus on 10 items considered important in a generic context suggests that a standardized instrument for core resource use items is feasible.
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Recursos em Saúde/estatística & dados numéricos , Adulto , Criança , Consenso , Análise Custo-Benefício , Custos e Análise de Custo , Técnica Delphi , Humanos , Medidas de Resultados Relatados pelo Paciente , Pacientes , Ensaios Clínicos Controlados Aleatórios como Assunto , Inquéritos e Questionários , Reino UnidoRESUMO
Importance: Prostate cancer screening remains controversial because potential mortality or quality-of-life benefits may be outweighed by harms from overdetection and overtreatment. Objective: To evaluate the effect of a single prostate-specific antigen (PSA) screening intervention and standardized diagnostic pathway on prostate cancer-specific mortality. Design, Setting, and Participants: The Cluster Randomized Trial of PSA Testing for Prostate Cancer (CAP) included 419â¯582 men aged 50 to 69 years and was conducted at 573 primary care practices across the United Kingdom. Randomization and recruitment of the practices occurred between 2001 and 2009; patient follow-up ended on March 31, 2016. Intervention: An invitation to attend a PSA testing clinic and receive a single PSA test vs standard (unscreened) practice. Main Outcomes and Measures: Primary outcome: prostate cancer-specific mortality at a median follow-up of 10 years. Prespecified secondary outcomes: diagnostic cancer stage and Gleason grade (range, 2-10; higher scores indicate a poorer prognosis) of prostate cancers identified, all-cause mortality, and an instrumental variable analysis estimating the causal effect of attending the PSA screening clinic. Results: Among 415â¯357 randomized men (mean [SD] age, 59.0 [5.6] years), 189â¯386 in the intervention group and 219â¯439 in the control group were included in the analysis (n = 408â¯825; 98%). In the intervention group, 75â¯707 (40%) attended the PSA testing clinic and 67â¯313 (36%) underwent PSA testing. Of 64â¯436 with a valid PSA test result, 6857 (11%) had a PSA level between 3 ng/mL and 19.9 ng/mL, of whom 5850 (85%) had a prostate biopsy. After a median follow-up of 10 years, 549 (0.30 per 1000 person-years) died of prostate cancer in the intervention group vs 647 (0.31 per 1000 person-years) in the control group (rate difference, -0.013 per 1000 person-years [95% CI, -0.047 to 0.022]; rate ratio [RR], 0.96 [95% CI, 0.85 to 1.08]; P = .50). The number diagnosed with prostate cancer was higher in the intervention group (n = 8054; 4.3%) than in the control group (n = 7853; 3.6%) (RR, 1.19 [95% CI, 1.14 to 1.25]; P < .001). More prostate cancer tumors with a Gleason grade of 6 or lower were identified in the intervention group (n = 3263/189â¯386 [1.7%]) than in the control group (n = 2440/219â¯439 [1.1%]) (difference per 1000 men, 6.11 [95% CI, 5.38 to 6.84]; P < .001). In the analysis of all-cause mortality, there were 25â¯459 deaths in the intervention group vs 28â¯306 deaths in the control group (RR, 0.99 [95% CI, 0.94 to 1.03]; P = .49). In the instrumental variable analysis for prostate cancer mortality, the adherence-adjusted causal RR was 0.93 (95% CI, 0.67 to 1.29; P = .66). Conclusions and Relevance: Among practices randomized to a single PSA screening intervention vs standard practice without screening, there was no significant difference in prostate cancer mortality after a median follow-up of 10 years but the detection of low-risk prostate cancer cases increased. Although longer-term follow-up is under way, the findings do not support single PSA testing for population-based screening. Trial Registration: ISRCTN Identifier: ISRCTN92187251.
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Detecção Precoce de Câncer , Programas de Rastreamento , Antígeno Prostático Específico/sangue , Neoplasias da Próstata/diagnóstico , Distribuição por Idade , Idoso , Seguimentos , Humanos , Masculino , Pessoa de Meia-Idade , Atenção Primária à Saúde , Neoplasias da Próstata/sangue , Neoplasias da Próstata/mortalidade , Classe Social , Reino Unido/epidemiologiaRESUMO
BACKGROUND: Resource-use measurement is integral for assessing cost-effectiveness within trial-based economic evaluations. Methods for gathering resource-use data from participants are not well developed, with questionnaires typically produced for each trial and rarely validated. The healthcare module of a generic, modular resource-use measure, designed for collecting self-report resource-utilisation data, has recently been developed in the UK. The objective of this research is to identify and prioritise items for new, bolt-on modules, covering informal care, social care and personal expenses incurred due to health and care needs. METHODS: Identification and prioritisation, conducted between April and December 2021, involved a rapid review of questionnaires included in the Database of Instruments for Resource Use Measurement and economic evaluations published from 2011 to 2021 to identify candidate items, an online survey of UK-based social care professionals to identify omitted social care items and focus groups with UK-based health economists and UK-based people who access social care services either for themselves or as carers to prioritise items. RESULTS: The review identified 203 items. Over half of the 24 survey respondents reported no missing items. Five academic health economists and four people who access social care services participated in focus groups. Feedback shaped the social and informal care modules and indicated that no specific personal expenses were essential to collect in all trials. Aids/adaptations were highlighted as costly personal expenses when relevant; therefore, the personal expenses module was narrowed to aids/adaptations only. CONCLUSION: Draft informal care, social care and aids/adaptations modules were developed, ready for further testing.
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BACKGROUND: Respiratory tract infections are readily transmitted in care homes. Airborne transmission of pathogens causing respiratory tract illness is largely unmitigated. Portable high-efficiency-particulate-air (HEPA) filtration units capture microbial particles from the air, but it is unclear whether this is sufficient to reduce infections in care home residents. The Air Filtration to prevent symptomatic winter Respiratory Infections (including COVID-19) in care homes (AFRI-c) randomized controlled trial will determine whether using HEPA filtration units reduces respiratory infection episodes in care home residents. METHODS: AFRI-c is a cluster randomized controlled trial that will be delivered in residential care homes for older people in England. Ninety-one care homes will be randomised to take part for one winter period. The intervention care homes will receive HEPA filtration units for use in communal areas and private bedrooms. Normal infection control measures will continue in all care homes. Anonymised daily data on symptoms will be collected for up to 30 residents. Ten to 12 of these residents will be invited to consent to a primary care medical notes review and (in intervention homes) to having an air filter switched on in their private room. The primary outcome will be number of symptomatic winter respiratory infection episodes. Secondary outcomes include specific clinical measures of infection, number of falls / near falls, number of laboratory confirmed infections, hospitalisations, staff sickness and cost-effectiveness. A mixed methods process evaluation will assess intervention acceptability and implementation. DISCUSSION: The results of AFRI-c will provide vital information about whether portable HEPA filtration units reduce symptomatic winter respiratory infections in older care home residents. Findings about effectiveness, fidelity, acceptability and cost-effectiveness will support stakeholders to determine the use of HEPA filtration units as part of infection control policies.
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Filtros de Ar , COVID-19 , Infecções Respiratórias , Estações do Ano , Humanos , COVID-19/prevenção & controle , COVID-19/epidemiologia , COVID-19/transmissão , Inglaterra/epidemiologia , Infecções Respiratórias/prevenção & controle , Infecções Respiratórias/epidemiologia , Idoso , SARS-CoV-2/isolamento & purificação , Casas de SaúdeRESUMO
BACKGROUND: Increasing healthcare costs require evidence-based resource use allocation for which assessing costs rigorously and comparably is crucial. Harmonized cross-country costing methods for evaluating interventions from a societal perspective are lacking. This study presents the development process and content of the service costing templates developed as part of the European project PECUNIA. METHODS: The six developmental steps towards technological readiness of the templates included (1) a common conceptual costing framework and review of methodological costing issues, (2) harmonization strategy formulation, (3) proof-of-concept with expert feedback, (4) piloting, (5) validation, and (6) demonstration in six European countries. RESULTS: The PECUNIA Reference Unit Cost (RUC) Templates for service costing are three new self-completion tools to be used with secondary or primary data for top-down micro-costing or top-down gross-costing approaches. Complementary data collection and unit cost aggregation/weighting templates are available. The applications leading to the final versions including (4) piloting through calculation of 15-unit costs, (5) validation within a Health Technology Assessment framework, and (6) RUC calculations mostly based on secondary data demonstrated the templates' general feasibility, with feedback for improved usability incorporated and a supplementary user guide developed. CONCLUSION: The validated PECUNIA RUC Templates for multi-sectoral and multi-country service costing allow for harmonized RUC development while incorporating flexibility and transparency in the choice of costing approaches, data sources and magnitude of remaining heterogeneity. The templates are expected to significantly improve the quality, comparability and availability of unit costs for economic evaluations, and promote the transferability of service cost information across Europe.
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BACKGROUND: Selective serotonin reuptake inhibitors (SSRIs) are commonly prescribed to manage anxiety in adults with an autism diagnosis. However, their effectiveness and adverse effect profile in the autistic population are not well known. This trial aims to determine the effectiveness and cost-effectiveness of the SSRI sertraline in reducing symptoms of anxiety and improving quality of life in adults with a diagnosis of autism compared with placebo and to quantify any adverse effects. METHODS: STRATA is a two-parallel group, multi-centre, pragmatic, double-blind, randomised placebo-controlled trial with allocation at the level of the individual. It will be delivered through recruiting sites with autism services in 4 regional centres in the United Kingdom (UK) and 1 in Australia. Adults with an autism diagnosis and a Generalised Anxiety Disorder Assessment (GAD-7) score ≥ 10 at screening will be randomised 1:1 to either 25 mg sertraline or placebo, with subsequent flexible dose titration up to 200 mg. The primary outcome is GAD-7 scores at 16 weeks post-randomisation. Secondary outcomes include adverse effects, proportionate change in GAD-7 scores including 50% reduction, social anxiety, obsessive-compulsive symptoms, panic attacks, repetitive behaviours, meltdowns, depressive symptoms, composite depression and anxiety, functioning and disability and quality of life. Carer burden will be assessed in a linked carer sub-study. Outcome data will be collected using online/paper methods via video call, face-to-face or telephone according to participant preference at 16, 24 and 52 weeks post-randomisation, with brief safety checks and data collection at 1-2, 4, 8, 12 and 36 weeks. An economic evaluation to study the cost-effectiveness of sertraline vs placebo and a QuinteT Recruitment Intervention (QRI) to optimise recruitment and informed consent are embedded within the trial. Qualitative interviews at various times during the study will explore experiences of participating and taking the trial medication. DISCUSSION: Results from this study should help autistic adults and their clinicians make evidence-based decisions on the use of sertraline for managing anxiety in this population. TRIAL REGISTRATION: ISRCTN, ISRCTN15984604 . Registered on 08 February 2021. EudraCT 2019-004312-66. ANZCTR ACTRN12621000801819. Registered on 07 April 2021.
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Transtorno Autístico , Sertralina , Adulto , Humanos , Ansiedade/diagnóstico , Ansiedade/tratamento farmacológico , Transtornos de Ansiedade/tratamento farmacológico , Transtorno Autístico/diagnóstico , Transtorno Autístico/tratamento farmacológico , Estudos Multicêntricos como Assunto , Qualidade de Vida , Ensaios Clínicos Controlados Aleatórios como Assunto , Sertralina/efeitos adversos , Ensaios Clínicos Pragmáticos como AssuntoRESUMO
BACKGROUND: Healthcare system data (HSD) are increasingly used in clinical trials, augmenting or replacing traditional methods of collecting outcome data. This study, PRIMORANT, set out to identify, in the UK context, issues to be considered before the decision to use HSD for outcome data in a clinical trial is finalised, a methodological question prioritised by the clinical trials community. METHODS: The PRIMORANT study had three phases. First, an initial workshop was held to scope the issues faced by trialists when considering whether to use HSDs for trial outcomes. Second, a consultation exercise was undertaken with clinical trials unit (CTU) staff, trialists, methodologists, clinicians, funding panels and data providers. Third, a final discussion workshop was held, at which the results of the consultation were fed back, case studies presented, and issues considered in small breakout groups. RESULTS: Key topics included in the consultation process were the validity of outcome data, timeliness of data capture, internal pilots, data-sharing, practical issues, and decision-making. A majority of consultation respondents (n = 78, 95%) considered the development of guidance for trialists to be feasible. Guidance was developed following the discussion workshop, for the five broad areas of terminology, feasibility, internal pilots, onward data sharing, and data archiving. CONCLUSIONS: We provide guidance to inform decisions about whether or not to use HSDs for outcomes, and if so, to assist trialists in working with registries and other HSD providers to improve the design and delivery of trials.
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Atenção à Saúde , Disseminação de Informação , Humanos , Sistema de RegistrosRESUMO
BACKGROUND: Few trials have evaluated the effectiveness of psychological treatment in improving depression by the end of pregnancy. This is the first pilot randomised controlled trial (RCT) of individual cognitive behavioural therapy (CBT) looking at treating depression by the end of pregnancy. Our aim was to assess the feasibility of delivering a CBT intervention modified for antenatal depression during pregnancy. METHODS: Women in North Bristol, UK between 8-18 weeks pregnant were recruited through routine contact with midwives and randomised to receive up to 12 sessions of individual CBT in addition to usual care or to continue with usual care only. Women were eligible for randomisation if they screened positive on a 3-question depression screen used routinely by midwives and met ICD-10 criteria for depression assessed using the clinical interview schedule - revised version (CIS-R). Two CBT therapists delivered the intervention. Follow-up was at 15 and 33 weeks post-randomisation when assessments of mental health were made using measures which included the CIS-R. RESULTS: Of the 50 women assessed for the trial, 36 met ICD-10 depression criteria and were randomised: 18 to the intervention and 18 to usual care. Thirteen of the 18 (72%) women who were allocated to receive the intervention completed 9 or more sessions of CBT before the end of pregnancy. Follow-up rates at 15 and 33 weeks post-randomisation were higher in the group who received the intervention (89% vs. 72% at 15 weeks and 89% vs. 61% at 33 weeks post-randomisation). At 15 weeks post-randomisation (the end of pregnancy), there were more women in the intervention group (11/16; 68.7%) who recovered (i.e. no longer met ICD-10 criteria for depression), than those receiving only usual care (5/13; 38.5%). CONCLUSIONS: This pilot trial shows the feasibility of conducting a large RCT to assess the effectiveness of CBT for treating antenatal depression before the end of pregnancy. The intervention could be delivered during the antenatal period and there was some evidence to suggest that it could be effective. TRIAL REGISTRATION: ISRCTN44902048.
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Terapia Cognitivo-Comportamental , Depressão/terapia , Transtorno Depressivo/terapia , Complicações na Gravidez/terapia , Adulto , Feminino , Humanos , Projetos Piloto , Gravidez , Resultado do Tratamento , Adulto JovemRESUMO
BACKGROUND: Measuring objective resource-use quantities is important for generating valid cost estimates in economic evaluations. In the absence of acknowledged guidelines, measurement methods are often chosen based on practicality rather than methodological evidence. Furthermore, few resource-use measurement (RUM) instruments focus on the measurement of resource use in multiple societal sectors and their development process is rarely described. Thorn and colleagues proposed a stepwise approach to the development of RUM instruments, which has been used for developing cost questionnaires for specific trials. However, it remains unclear how this approach can be translated into practice and whether it is applicable to the development of generic self-reported RUM instruments and instruments measuring resource use in multiple sectors. This study provides a detailed description of the practical application of this stepwise approach to the development of a multi-sectoral RUM instrument developed within the ProgrammE in Costing, resource use measurement and outcome valuation for Use in multi-sectoral National and International health economic evaluAtions (PECUNIA) project. METHODS: For the development of the PECUNIA RUM, the methodological approach was based on best practice guidelines. The process included six steps, including the definition of the instrument attributes, identification of cost-driving elements in each sector, review of methodological literature and development of a harmonized cross-sectorial approach, development of questionnaire modules and their subsequent harmonization. RESULTS: The selected development approach was, overall, applicable to the development of the PECUNIA RUM. However, due to the complexity of the development of a multi-sectoral RUM instrument, additional steps such as establishing a uniform methodological basis, harmonization of questionnaire modules and involvement of a broader range of stakeholders (healthcare professionals, sector-specific experts, health economists) were needed. CONCLUSION: This is the first study that transparently describes the development process of a generic multi-sectoral RUM instrument in health economics and provides insights into the methodological aspects and overall validity of its development process.
Assuntos
Pessoal de Saúde , Humanos , Europa (Continente) , Inquéritos e Questionários , Análise Custo-BenefícioRESUMO
OBJECTIVE: To assess the cost-utility and cost-effectiveness of a group-based outpatient physical therapy intervention delivered 6 weeks after primary total knee replacement (TKR) compared with usual care, alongside the Activity-Orientated Rehabilitation Following Knee Arthroplasty (ARENA) multicenter, randomized, controlled trial. METHODS: The economic analyses were performed from the perspective of the health and social care payer. We collected resource use for health and social care and productivity losses and patient outcomes for 12 months after surgery to derive costs and quality-adjusted life years (QALYs). Results were expressed in incremental cost-effectiveness ratios (ICERs), and incremental net monetary benefit statistics (INMBs) for a society willingness-to-pay (WTP) threshold of £20,000 per QALY gained, with sensitivity analyses to model specification and perspective. RESULTS: The cost of the ARENA physical therapy classes was mean ± SD £179 ± 39 per patient. Treatment in the year following surgery cost was, on average, £1,739 (95% confidence interval [95% CI] -£742, £4,221) per patient in the intervention group (n = 89), which was an additional £346 (95% CI £38, £653) per patient compared with usual care (n = 91) (£1,393 [95% CI -£780, £3,568]). QALY benefits were 0.0506 higher (95% CI 0.009, 0.09) in the intervention group, corresponding to an additional 19 days in "perfect health." The ICER for the intervention group was £6,842 per QALY gained, and the INMB was £665 (95% CI £139, £1,191), with a 92% probability of being cost-effective, and no less than 73% in all sensitivity analysis scenarios. CONCLUSION: The addition of group-based outpatient physical therapy classes to usual care improves quality of life and is a cost-effective treatment option following TKR for a society WTP threshold of £20,000 per QALY gained.
Assuntos
Artroplastia do Joelho , Humanos , Análise Custo-Benefício , Qualidade de Vida , Pacientes Ambulatoriais , Anos de Vida Ajustados por Qualidade de Vida , Modalidades de FisioterapiaRESUMO
OBJECTIVES: Patients with inflammatory arthritis report that fatigue is challenging to manage. We developed a manualised, one-to-one, cognitive-behavioural intervention, delivered by rheumatology health professionals (RHPs). The Fatigue - Reducing its Effects through individualised support Episodes in Inflammatory Arthritis (FREE-IA) study tested the feasibility of RHP training, intervention delivery and outcome collection ahead of a potential trial of clinical and cost-effectiveness. METHODS: In this single-arm feasibility study, eligible patients were ≥18 years, had a clinician-confirmed diagnosis of an inflammatory arthritis and scored ≥6/10 on the Bristol Rheumatoid Arthritis Fatigue (BRAF) Numerical Rating Scale (NRS) Fatigue Effect. Following training, RHPs delivered two to four sessions to participants. Baseline data were collected before the first session (T0) and outcomes at 6 weeks (T1) and 6 months (T2). The proposed primary outcome was fatigue impact (BRAF NRS Fatigue Effect). Secondary outcomes included fatigue severity and coping, disease impact and disability, and measures of therapeutic mechanism (self-efficacy and confidence to manage health). RESULTS: Eight RHPs at five hospitals delivered 113 sessions to 46 participants. Of a potential 138 primary and secondary outcome responses at T0, T1 and T2, there were 13 (9.4%) and 27 (19.6%) missing primary and secondary outcome responses, respectively. Results indicated improvements in all measures except disability, at either T1 or T2, or both. CONCLUSIONS: This study showed it was feasible to deliver the intervention, including training RHPs, and recruit and follow-up participants with high retention. While there was no control group, observed within-group improvements suggest potential promise of the intervention and support for a definitive trial to test effectiveness.
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Artrite Reumatoide , Fadiga , Adolescente , Adulto , Artrite Reumatoide/complicações , Artrite Reumatoide/terapia , Fadiga/etiologia , Fadiga/prevenção & controle , Estudos de Viabilidade , Humanos , Projetos de Pesquisa , Resultado do TratamentoRESUMO
BACKGROUND: While the methods for conducting health economics research in general are improving, current guidelines provide limited guidance regarding resource use measurement (RUM). Consequently, a variety of methods exists, yet there is no overview of aspects to consider when deciding on the most appropriate RUM methodology. Therefore, this study aims to (1) identify and categorize existing knowledge regarding aspects of RUM, and (2) develop a framework that provides a comprehensive overview of methodological aspects regarding RUM. METHODS: Relevant articles were identified by enrolling a search string in six databases and handsearching the DIRUM database. Included articles were descriptively reviewed and served as input for a comprehensive framework. Health economics experts were involved during the process to establish the framework's face validity. RESULTS: Forty articles were included in the scoping review. The RUM framework consists of four methodological RUM domains: 'Whom to measure', addressing whom to ask and whom to measure; 'How to measure', addressing the different approaches of measurement; 'How often to measure', addressing recall period and measurement patterns; and 'Additional considerations', which covers additional aspects that are essential for further refining the methodologies for measurement. Evidence retrieved from the scoping review was categorized according to these domains. CONCLUSION: This study clustered the aspects of RUM methodology in health economics into a comprehensive framework. The results may guide health economists in their decision making regarding the selection of appropriate RUM methods and developing instruments for RUM. Furthermore, policy makers may use these findings to review study results from an evidence-based perspective.