Health Economic Analysis Research Articles

A time trade-off study in the UK, Canada and the US to estimate utilities associated with the treatment of haemophilia

Abstract Introduction Haemophilia is a rare bleeding disorder caused by a deficient or absent clotting factor, leading to frequent bleeding. Multiple intravenous (IV) infusions have been the standard prophylactic treatment; however, newer treatment options involve less frequent subcutaneous (SC) injections. To inform future health economic evaluations, this study applied the time trade-off (TTO) method for estimation of utilities associated with haemophilia treatment for both people with the disease and potential caregivers. Methods Using the TTO method, utilities were estimated through two online surveys distributed in the UK, Canada and the US. In survey 1 (S1), adults from the general population aged 18 years and above evaluated health states as if they were living with haemophilia themselves and were receiving treatment for the condition. In survey 2 (S2), adults from the general population with a child under the age of 15 years evaluated health states as if they were treating their child for haemophilia. The surveys assessed the following treatment aspects: frequency of treatment, treatment device and injection site reactions. Results In total, 812, 739 and 703 respondents completed S1 and 712, 594 and 527 completed S2 in the UK, Canada and the US, respectively. In both surveys, the treatment device was associated with the largest impact on utilities for both people with haemophilia and caregivers. Monthly SC injections with a prefilled pen-device were associated with a significant utility gain compared with SC injections with a syringe and IV infusions. In S1, a lower treatment frequency was preferred in all three countries, while in S2, a lower treatment frequency was preferred only in the UK. Avoiding injection site reactions was associated with a significant utility gain in both surveys, but only in the UK and Canada. Conclusions The study suggests that the administration of haemophilia treatment in particular has an impact on utilities for both people and caregivers living with the disease. Thus, less complex and time-consuming treatment devices are expected to improve health-related quality of life. This can be further modified additively by less frequent administration. These results can inform future health economic analyses of haemophilia and haemophilia treatment.

Evaluation of a complex intervention: the Latch On randomized controlled trial of multicomponent breastfeeding support for women with a raised body mass index

Abstract Background Latch On’s objective was to achieve improved breastfeeding rates in women with raised body mass indices using a multicomponent breastfeeding support intervention. Methods A hybrid type 1 implementation-effectiveness trial with mixed-methods process and health economics analyses were conducted. Data collection included stakeholder questionnaires, interviews, focus groups, fidelity data, participant and health system costs. Results The intervention was delivered with fidelity but the high breastfeeding rates at 3 months were not different between intervention and usual care. Participants receiving the minimum intervention dose were more likely to initiate breastfeeding (P = 0.045) and be breastfeeding at hospital discharge (P = 0.01) compared with participants below the threshold. Participant exit interview themes highlighted the importance of improving breastfeeding support to women, the effect of COVID-19 on the breastfeeding experience, and found that the intervention improved the experience of establishing breastfeeding. The intervention cost €157 per participant, with no other cost difference between groups. Process analysis found that follow-up breastfeeding services continued in half of sites after study completion. Conclusions This low-cost intervention resulted in a more enjoyable breastfeeding experience for participants and changed practice in some study sites. The intervention dose received may impact effectiveness, but further research is needed to provide definitive evidence of clinical and cost effectiveness.

DPYD genotype-guided dose personalisation for fluoropyrimidine-based chemotherapy prescribing in solid organ cancer patients in Australia: GeneScreen 5-FU study protocol

BackgroundFluoropyrimidine (FP) chemotherapies are commonly prescribed for upper and lower gastrointestinal, breast and head and neck malignancies. Over 16,000 people with cancer require FP chemotherapies per annum in Australia. Between 10 and 40% patients experience grade 3–4 (≥ G3) toxicities that require hospital-based management ± intensive care admission. Approximately 1% of patients die secondary to FP toxicities. Prospective screening for DPYD gene variants (encoding the key enzyme for FP catabolism) can identify patients at risk of ≥ G3 toxicity and allow for dose adjustment prior to first FP exposure. Evidence supports this as a cost-effective method of improving patient safety and reducing healthcare burden internationally; however, no Australian data confirms its feasibility on a large scale.MethodThis investigator-led, single-arm study will determine large scale feasibility of prospective DPYD genotyping, confirming patient safety and cost-effectiveness within the Australian health care system. 5000 patients aged 18 years and older with solid organ cancers requiring FP chemotherapy will be consented and genotyped prior to commencing treatment, and early toxicity (within 60 days) post-FP exposure will be determined. Toxicity data for DPYD variant carriers who have dose adjustments will be compared to the wild-type cohort and historical cohorts of carriers who did not undergo genotyping prior to FP exposure, and prospective variant carriers who do not undergo dose-adjustment. Prevalence of the four standard DPYD gene variants will be confirmed in an Australian population. Additionally, health economic analysis, implementation research via semi-structured interviews of patients and clinicians, and feasibility of UGT1A1 genotyping will be conducted.DiscussionThis study will determine the prevalence of DPYD gene variant status in Australia and its impact on FP-induced toxicity among Australians with cancer. Feasibility and cost-effectiveness for Australian health care system will be estimated to support national roll-out of prospective DPYD genotyping prior to FP administration. Additionally, feasibility will be confirmed with the intention of including UGT1A1 in future pharmacogenomic panels to aid chemotherapy prescribing.Trial registrationThis trial was registered with the Australian and New Zealand Cancer Trials Registry on 13th Dec 2023, ACTRN12623001301651.

The Potential Impact of a Single-Dose HPV Vaccination Schedule on Cervical Cancer Outcomes in Kenya: A Mathematical Modelling and Health Economic Analysis

Background: Human Papillomavirus (HPV) is the primary cause of cervical cancer. Single-dose HPV vaccination can effectively prevent high-risk HPV infection that causes cervical cancer and accelerate progress toward achieving cervical cancer elimination goals. We modelled the potential impact of adopting single-dose HPV vaccination strategies on health and economic outcomes in Kenya, where a two-dose schedule is the current standard. Methods: Using a validated compartmental transmission model of HPV and HIV in Kenya, we evaluated the costs from the payer’s perspective to vaccinate girls by age 10 with either one or two doses and increasing coverage levels (0%, 70%, 77%, 90%). Additionally, we modelled single-dose strategies supplemented with either catch-up vaccination of adolescent girls and young women or vaccination for all by age 10, funded with the first five-years of cost savings of switching from a two- to one-dose schedule. Costs and outcomes were discounted at 3% annually, and incremental cost-effectiveness ratios (ICERs) were calculated per disability-adjusted-life-year (DALY) averted. Results: All one-dose and the two-dose 90% coverage strategies were on the efficiency frontier, dominating the remaining two-dose strategies. The two-dose 90% coverage strategy had a substantially higher ICER (US$6508.80/DALY averted) than the one-dose 90% coverage (US$197.44/DALY averted). Transitioning from a two- to one-dose schedule could result in US$21.4 Million saved over the first five years, which could potentially fund 2.75 million supplemental HPV vaccinations. With this re-investment, all two-dose HPV vaccination scenarios would be dominated. The greatest DALYs were averted with the single-dose HPV vaccination schedule at 90% coverage supplemented with catch-up for 11–24-year-old girls, which had an ICER of US$78.73/DALYs averted. Conclusions: Considering the logistical and cost burdens of a two-dose schedule, a one-dose schedule for girls by age 10 would generate savings that could be leveraged for catch-up vaccination for older girls and accelerate cervical cancer elimination in Kenya.

Cost-effectiveness of hearing and vision support for residents with dementia in long-term care in Ireland: A Health Economic Analysis Plan (HEAP) for a simulation model for the SENSE-Cog Care feasibility trial

Introduction Sensory challenges exacerbate the dementia symptoms of nursing home residents. The Sense-Cog Care trial is piloting a multi-faceted intervention to investigate whether optimising and supporting hearing, vision, and sensory-friendly environments through the “sensory champion” model in Irish nursing homes can improve quality of life and dementia-related outcomes and be cost-effective (Connelly et al., 2023). A Health Economic Analysis Plan (HEAP) provides a standardised and comprehensive framework by outlining the systematic approach, methodologies, and key considerations involved in assessing the cost-effectiveness of a healthcare intervention. The HEAP presented in this paper describes the alongside-trial decision modelling approach used for preliminary estimates of cost-effectiveness and to inform the design of the final intervention. Methods A Markov model will be used to simulate residents' health progression with and without the intervention. Costs will be estimated from the Irish health and social care perspective. A key output of the planned economic analysis will be the Incremental-Cost-Effectiveness Ratio (ICER) which describes the cost over one additional unit of Quality-Adjusted Life Year (QALY). A Probabilistic Sensitivity Analysis (PSA) will be conducted in the form of a Monte Carlo simulation to examine parameter uncertainty and the probability of a sensory champion being cost-effective. Results Tables for inputs and outputs are presented, and graphical representations for uncertainty, such as a tornado plot and a Cost-Effectiveness Analysis Curve (CEAC), will be produced. The results will be interpreted in the context of the Irish cost-effectiveness thresholds of €20,000 and €45,000 per QALY. Discussion To our knowledge, this is the first study to model the cost-effectiveness of a sensory intervention for nursing home residents with dementia. We aim to develop an early-stage Bayesian model that serves as a prior for further research.

Cost-utility analysis for sublingual versus intravenous edaravone in the treatment of amyotrophic lateral sclerosis

BackgroundEdaravone has been widely used in amyotrophic lateral sclerosis (ALS) treatment, and a sublingual (SL) tablet has been developed to offer a more convenient alternative for injection. We present a cost-utility analysis to comprehensively evaluate the costs and health outcomes of oral and intravenous edaravone for the treatment of ALS in Chinese medical context.MethodsCost-utility analysis of SL tablets of edaravone versus intravenous edaravone at home was performed by constructing a 20-year Markov model of ALS stage 1–4 and death. The data were extracted from the literature with model assumptions. Typical sensitivity analysis and scenario analysis for administering SL tablets at home versus intravenous tablets at the hospital were performed.ResultsIn the base case analysis, with SL tablets and intravenous injections both at home, the model estimated an additional cost of ¥12,670.04 and an additional 0.034 QALYs over 20 years (life time) of modeling analysis, and the ICER was ¥372,648.24 per QALY. However, in the scenario of intravenous administration at the hospital, SL tablet was demonstrated dominance to intravenous injection.ConclusionsUsing 3 times the GDP per capita of China in 2023 as the threshold, the SL tablet edaravone was not cost-effective in the context of home treatment for both formulationst, but was dominance to intravenous injection in hospital treatment. The results highlighted the importance of treatment context for health economic analysis.

VI-RADS followed by Photodynamic Transurethral Resection of Non-Muscle-Invasive Bladder Cancer vs White-Light Conventional and Second-resection: the 'CUT-less' Randomised Trial Protocol.

A second transurethral resection of bladder tumour (Re-TURBT) is recommended by European Association of Urology (EAU) Guidelines on non-muscle-invasive bladder cancers (NMIBCs) due to the risk of understaging and/or persistent disease following the primary resection. However, in many cases this may be unnecessary, potentially harmful, and significantly expensive constituting overtreatment. The CUT-less trial aims to combine the preoperative staging accuracy of Vesical Imaging-Reporting and Data System (VI-RADS) and the intraoperative enhanced ability of photodynamic diagnosis (PDD) to overcome the primary TURBT pitfalls thus potentially re-defining criteria for Re-TURBT indications. Single-centre, non-inferiority, phase IV, open-label, randomised controlled trial with 1:1 ratio. The primary endpoint is short-term BC recurrence between the study arms to assess whether patients preoperatively categorised as VI-RADS Score 1 and/or Score 2 (i.e., very-low and low likelihood of MIBC) could safely avoid Re-TURBT by undergoing primary PDD-TURBT. Secondary endpoints include mid- and long-term BC recurrences and progression (i-ii). Also, health-related quality of life (HRQoL) outcomes (iii) and health-economic cost-benefit analysis (iv) will be performed. All patients will undergo preoperative Multiparametric Magnetic Resonance Imaging of the bladder with VI-RADS score determination. A total of 327 patients with intermediate-/high-risk NMIBCs, candidate for Re-TURBT according to EAU Guidelines, will be enrolled over a 3-year period. Participants will be randomised (1:1 ratio) to either standard of care (SoC), comprising primary white-light (WL) TURBT followed by second WL Re-TURBT; or the Experimental arm, comprising primary PDD-TURBT and omitting Re-TURBT. Both groups will receive adjuvant intravesical therapy and surveillance according to risk-adjusted schedules. Measure of the primary outcome will be the relative proportion of BC recurrences between the SoC and Experimental arms within 4.5 months (i.e., any 'early' recurrence detected at first follow-up cystoscopy). Secondary outcomes measures will be the relative proportion of late BC recurrences and/or BC progression detected after 4.5 months follow-up. Additionally, we will compute the HRQoL variation from NMIBC questionnaires modelled over a patient lifetime horizon and the health-economic analyses including a short-term cost-benefit assessment of incremental costs per Re-TURBT avoided and a longer-term cost-utility per quality-adjusted life year gained using 2-year clinical outcomes to drive a lifetime model across the two arms of treatment. ClinicalTrial.gov identifier (ID): NCT05962541; European Union Drug Regulating Authorities Clinical Trials Database (EudraCT) ID: 2023-507307-64-00.

Transient Ischaemic attack Emergency Referral (TIER): randomised feasibility trial results

BackgroundEarly assessment of patients with suspected transient ischaemic attack (TIA) is crucial to provision of effective care, including initiation of preventive therapies and identification of stroke mimics. Many patients with...

A person-centred consultation intervention to improve shared decision-making about, and uptake of, osteoporosis medicines (iFraP): a pragmatic, parallel-group, individual randomised controlled trial protocol

Background Good quality shared decision-making (SDM) conversations involve people with, or at risk of osteoporosis and clinicians collaborating to decide, where appropriate, which evidence-based medicines best fit the person’s life, beliefs, and values. We developed the improving uptake of Fracture Prevention drug treatments (iFraP) intervention comprising a computerised Decision Support Tool (DST), clinician training package and information resources, for use in UK Fracture Liaison Service consultations. Two primary objectives to determine (1) the effect of the iFraP intervention on patient-reported ease in decision-making about osteoporosis medicines, and (2) cost-effectiveness of iFraP intervention compared to usual NHS care. Secondary objectives are to determine the iFraP intervention effect on patient reported outcome and experience measures, clinical effectiveness (osteoporosis medicine adherence), and to explore intervention acceptability, mechanisms, and processes underlying observed effects, and intervention implementation. Methods The iFraP trial is a pragmatic, parallel-group, individual randomised controlled trial in patients referred to a Fracture Liaison Service, with nested mixed methods process evaluation and health economic analysis. Participants aged ≥50 years (n=380) are randomised (1:1 ratio) to one of two arms: (1) iFraP intervention (iFraP-i) or (2) comparator usual NHS care (iFraP-u) and are followed up at 2-weeks and 3-months. The primary outcome is ease of decision-making assessed 2 weeks after the consultation using the Decisional Conflict Scale (DCS). The primary objectives will be addressed by comparing the mean DCS score in each trial arm (using analysis of covariance) for patients given an osteoporosis medicine recommendation, alongside a within-trial cost-effectiveness and value of information (VoI) analysis. Process evaluation data collection includes consultation recordings, semi-structured interviews, and DST analytics. Discussion The iFraP trial will answer important questions about the effectiveness of the new ‘iFraP’ osteoporosis DST, coupled with clinician training, on SDM and informed initiation of osteoporosis medicines. Trial registration ISRCTN 10606407, 21/11/2022 https://doi.org/10.1186/ISRCTN10606407

Health-related quality of life assessment in health economic analyses involving type 2 diabetes.

Incorporating health-related quality of life (HRQoL) measures into health economic analyses can help to provide evidence to inform decisions about how to improve patient outcomes in the most cost-effective manner. The aim of this narrative review was to assess which HRQoL instruments have been used in economic evaluations of type 2 diabetes management including in Indigenous communities. MEDLINE (Ovid), Embase (Ovid) and Cochrane were searched from inception to June 2022. Studies included patients with type 2 diabetes; economic evaluations, derived scores from direct questioning of individuals; and were in English. Records were assessed for bias using the JBI critical appraisal tools. A total of 3737 records were identified, with 22 publications meeting the criteria for inclusion. Across those 22 articles, nine HRQoL instruments had been utilised. Generic tools were most frequently used to measure HRQoL, including EQ-5D (-3 L and -5 L) (n = 10, 38%); SF-12 (n = 5, 19%); and SF-36 (n = 4, 15%). Two tools addressing the specific stressors faced by people with type 2 diabetes were utilised: Problem Areas In Diabetes tool (n = 1, 4%) and Diabetes Distress Scale (n = 1, 4%). Two publications reported whether the study population included Indigenous peoples. A wide range of HRQoL instruments are used in economic evaluations of type 2 diabetes management, with the most frequent being varying forms of the EQ-5D. Few economic evaluations noted whether Indigenous peoples were featured in the study population. More research into HRQoL in people living with type 2 diabetes is urgently needed to improve evidence on effectiveness and cost-effectiveness of interventions.

S511 Direct Genetic Screening Approach in Hereditary Nonpolyposis Colorectal Cancer: A Health Economics Analysis in High-risk Colorectal Cancer Population; Perspective from Middle-Income Country

S511 Direct Genetic Screening Approach in Hereditary Nonpolyposis Colorectal Cancer: A Health Economics Analysis in High-risk Colorectal Cancer Population; Perspective from Middle-Income Country

Electronic monitoring versus manual paper-based monitoring for hand hygiene compliance: a comprehensive health economic assessment analysis

BackgroundMonitoring hand hygiene compliance (HHC) of healthcare providers (HCPs) in healthcare facilities is critical for hand hygiene (HH) promotion. However, less is known about the cost and effectiveness of different HHC monitoring tools. In this study, we aimed to compare various health economic indicators corresponding to electronic system-based monitoring (ESM) and manual paper-based monitoring (MPM) for HHC to provide evidence-based advice for HHC monitoring measures targeted selecting.MethodsA before and after study in 40 clinical departments with 4,524 healthcare providers was conducted from December 2022 to January 2023 (MPM implementation phase) and March 2023 to May 2023 (ESM implementation phase). The cost-effectiveness, cost-efficiency, the extent of the Hawthorne effect, and indirect cost-benefit of the two monitoring methods were compared.ResultsThe total cost spent on ESM for the 40 departments (17,702.92 CNY) was 4,123.76 CNY lower than that of MPM (21,826.68 CNY). The HHC of MPM (80.16%) was higher than that of ESM (69.82%) (p < 0.01). In high- and medium-risk departments, the cost-effectiveness ratio of ESM (7,977.90 CNY and 13,794.60 CNY, respectively) was lower than that of MPM (9,039.61 CNY and 14,549.05 CNY, respectively). In low-risk departments, the cost-effectiveness ratio of ESM (3,910.77 CNY) was higher than that of MPM (3,899.06 CNY). Compared with ESM, the incremental cost of MPM in all departments was 4,123.76 CNY, the incremental effectiveness was 10.34%, and the incremental cost-effectiveness ratio was 39,881.62 CNY. Between the two monitoring methods, the efficiency of ESM (48.11%) in all departments was higher than that of MPM (14.20%) (p < 0.01). The cost-efficiency ratio of MPM in all departments (155,775.56 CNY) was higher than that of ESM (36,796.76 CNY). The extent of Hawthorne effect of MPM of HHC in all departments (43.99%) was higher than that of ESM (35.69%) (p < 0.01). When ESM was used as the HHC monitoring approach, the HAI rates (1.39%) in all departments were higher than that when MPM was used (1.34%) (p = 0.562). When the payment willingness was less than 40,000 CNY, the ESM method was the better option for cost-effectiveness; When the input exceeded this threshold, the MPM method was the better option for cost-effectiveness.ConclusionsESM exhibited notable advantages over MPM in terms of cost-effectiveness, cost-efficiency, cost-benefit, and the Hawthorne effect.

Evaluating the effectiveness of simvastatin in slowing the progression of disability in secondary progressive multiple sclerosis (MS-STAT2): protocol for a multicentre, randomised controlled, double-blind, phase 3 clinical trial in the UK

IntroductionThere remains a high unmet need for disease-modifying therapies that can impact disability progression in secondary progressive multiple sclerosis (SPMS). Following positive results of the phase 2 MS-STAT study, the...

Abstract B018: UroPanc: A large prospective observational study for validation of urinary biomarker test for the earlier detection of pancreatic adenocarcinoma

Abstract Introduction. A bleak prognosis of Pancreatic ductal adenocarcinoma (PDAC) is largely due to late diagnosis and highly accurate, non-invasive diagnostic tests are urgently needed to improve patients’ survival. Our group has previously reported on urinary biomarker panel, comprising of LYVE1, REG1B and TFF1 (1-2), which distinguished controls from PDAC stages I-II with AUC 0.936 and sensitivity/specificity (SN/SP)&amp;gt;85%. We constructed the logistic regression based algorithm for interpretation of the data, PancRISK (3), which enables the stratification of high- risk patients into those with ‘average’ or ‘elevated’ risk of developing PDAC. Recently, we showed that our panel combined with CA19-9 can detect PDAC up to 2 years before clinical diagnosis (4). Aim. Here, we report on the design and interim analysis of the UroPanc Trial (ClinicalTrials.gov NCT04449406), which aims to evaluate the accuracy of the urinary biomarker panel and the affiliated PancRISK. We plan to recruit two cohorts: the first one comprises asymptomatic individuals that have high risk of developing PDAC due to familial history and genetic syndroms - these will be recruited through the EUropean Registry Of familial PAncreatic Cancer and hereditary pancreatitis (EUROPAC) (PI: Prof W Greenhalf and team, Liverpool, UK). The second cohort comprises patients with symptoms suggestive of PDAC – such patients are recruited at gastrointestinal clinics at University College London Hospitals (UCLH, PI: Prof S Pereira and team), and The Royal London Hospital (PI: Dr P Wilson).Methods. The urinary proteins are assayed by commercially available ELISAs. Plasma CA19-9 is measured at The Doctors Laboratory in London, UK using Cobas601E, Roche platform. The statistical analysis and accuracy of the test is measured by c-statistics, SN/SP and positive/negative predictive value. The results are compared to imaging and histopathology records (where available). Human factor and budget impact analysis of the future inclusion of the urine test into the diagnostic and surveillance pathways for PDAC patients is ongoing (PI: Dr M Z Ni and the team, Imperial College London, UK). Results and conclusions. At present, 1067 patients have been recruited, and urine samples from 825 patients have been assayed. An interim analysis has shown promising results with SP of 91% and SN of 86%, and accuracy of cancer detection of 90%. Combined with the health economic analysis, we currently work towards the future implementation of this test into clinical practice, with the potential to significantly improve the current diagnostic pathway for individuals at risk of pancreatic cancer. 1. Radon, TP, et al. Clin Cancer Res, 2015; 21:3512-21. 2. Debernardi, S, et al. PLOSMed, 17(12); e1003489. 3. Blyuss, O, et al. Br J Cancer, 2020; 122:692-6. 4. Debernardi, S, et al. Int J Cancer, 2023; 152:769–80. Citation Format: Silvana Debernardi, Evelyn Kurotova, Nurshad Ali, Mariam Mahamood, Ismita Chhetri, Steve Pereira, Patrick Wilson, Bill Greenhalf, Melody Z Ni, Oleg Blyuss, Tatjana Crnogorac-Jurcevic. UroPanc: A large prospective observational study for validation of urinary biomarker test for the earlier detection of pancreatic adenocarcinoma [abstract]. In: Proceedings of the AACR Special Conference in Cancer Research: Advances in Pancreatic Cancer Research; 2024 Sep 15-18; Boston, MA. Philadelphia (PA): AACR; Cancer Res 2024;84(17 Suppl_2):Abstract nr B018.

Digital remote monitoring of chronic retinal conditions-A clinical future tool? : Remote monitoring of chronic retinal conditions

In view of the predicted increase in incidence and prevalence of chronic retinal diseases and undersupply of care in the population, telemedicine could contribute to reducing access barriers to healthcare and improving the results of treatment. A literature review on remote monitoring of chronic retinal diseases was carried out. The medical literature was searched for publications on remote monitoring of chronic retinal diseases. The results were compiled in anarrative overview. The four main topics in the literature are: validation studies, implementation strategies, acceptance/target group analyses and health economic analyses. Remote monitoring systems are based on visual function tests, imaging or patient reports and have been particularly investigated in age-related macular degeneration (AMD) and diabetic eye disease (DED). Studies indicate positive effects regarding an optimization of clinical care and afavorable safety profile but randomized controlled trials are lacking for the majority of monitoring tools. Remote monitoring could complement existing care structures for patients with chronic retinal diseases, especially AMD and DED. Promising systems are based on hyperacuity or optical coherence tomography, while patient-reported data are not commonly used; however, there is currently insufficient evidence justifying the use of remote monitoring systems in chronic retinal diseases in Europe and more research on the validation of remote monitoring systems is needed.

Linking for Impact: Enhancing Linked Data Infrastructure to Meet Clinical Trials Sector Needs

BackgroundAustralia possesses valuable population health data, yet its potential for advancing medical product development remains largely untapped. Transformative growth in the use of linked data in clinical trials will support Australia’s therapeutic development sector to be more responsive in the development of new therapeutics and the monitoring and surveillance of their safety and effectiveness. MethodsWe conducted interviews with clinical trialists and data linkage experts to understand the current state and awareness of linked data within the clinical trials sector, identify existing pain points in the linkage process, and explore opportunities for improving visibility and access for the clinical trials sector. Researchers represented all stages of the medical product development pipeline and had a diverse range of experience with linked data. ResultsInterviews revealed numerous barriers hindering the clinical trials sector from effectively using linked data, limiting its impact. Barriers included a lack of awareness, the complexity of the application and approval process, and lengthy delays in accessing data. Participants readily recognised several ways that linkage of real-world data could be applied to their work in clinical trials, with pre-recruitment data, measurements of primary and secondary endpoints, and health economic analysis being the most consistently noted use cases. ConclusionTo optimise the use of linked data to support clinical trials and medical product development in Australia, increased awareness among the therapeutic development sector regarding available real-world data and its potential is needed urgently. Lowering the barriers to accessing linked data will enhance health services and patient outcomes across Australia.

Linking for Impact: Enhancing Linked Data Infrastructure to Meet Clinical Trials Sector Needs

BackgroundAustralia possesses valuable population health data, yet its potential for advancing medical product development remains largely untapped. Transformative growth in the use of linked data in clinical trials will support Australia’s therapeutic development sector to be more responsive in the development of new therapeutics and the monitoring and surveillance of their safety and effectiveness. MethodsWe conducted interviews with clinical trialists and data linkage experts to understand the current state and awareness of linked data within the clinical trials sector, identify existing pain points in the linkage process, and explore opportunities for improving visibility and access for the clinical trials sector. Researchers represented all stages of the medical product development pipeline and had a diverse range of experience with linked data. ResultsInterviews revealed numerous barriers hindering the clinical trials sector from effectively using linked data, limiting its impact. Barriers included a lack of awareness, the complexity of the application and approval process, and lengthy delays in accessing data. Participants readily recognised several ways that linkage of real-world data could be applied to their work in clinical trials, with pre-recruitment data, measurements of primary and secondary endpoints, and health economic analysis being the most consistently noted use cases. ConclusionTo optimise the use of linked data to support clinical trials and medical product development in Australia, increased awareness among the therapeutic development sector regarding available real-world data and its potential is needed urgently. Lowering the barriers to accessing linked data will enhance health services and patient outcomes across Australia.

A Three-Day Prehabilitation Program is Cost-Effective for Preventing Pulmonary Complications after Heart Valve Surgery: A Health Economic Analysis of a Randomized Trial.

While prehabilitation (pre surgical exercise) effectively prevents postoperative pulmonary complications (PPCs), its cost-effectiveness in valve heart disease (VHD) remains unexplored. This study aims to evaluate the cost-effectiveness of a three-day prehabilitation program for reducing PPCs and improving quality adjusted life years (QALYs) in Chinese VHD patients. A cost-effectiveness analysis was conducted alongside a randomized controlled trial featuring concealed allocation, blinded evaluators, and an intention-to-treat analysis. In total, 165 patients scheduled for elective heart valve surgery at West China Hospital were randomized into intervention and control groups. The intervention group participated in a three-day prehabilitation exercise program supervised by a physiotherapist while the control group received only standard preoperative education. Postoperative hospital costs were audited through the Hospital Information System, and the EuroQol five-dimensional questionnaire was used to provide a 12-month estimation of QALY. Cost and effect differences were calculated through the bootstrapping method, with results presented in cost-effectiveness planes, alongside the associated cost-effectiveness acceptability curve (CEAC). All costs were denominated in Chinese Yuan (CNY) at an average exchange rate of 6.73 CNY per US dollar in 2022. There were no statistically significant differences in postoperative hospital costs (8484 versus 9615 CNY, 95% CI -2403 to 140) or in the estimated QALYs (0.909 versus 0.898, 95% CI -0.013 to 0.034) between the intervention and control groups. However, costs for antibiotics (339 versus 667 CNY, 95% CI -605 to -51), nursing (1021 versus 1200 CNY, 95% CI -330 to -28), and electrocardiograph monitoring (685 versus 929 CNY, 95% CI -421 to -67) were significantly lower in the intervention group than in the control group. The CEAC indicated that the prehabilitation program has a 92.6% and 93% probability of being cost-effective in preventing PPCs and improving QALYs without incurring additional costs. While the three-day prehabilitation program did not significantly improve health-related quality of life, it led to a reduction in postoperative hospital resource utilization. Furthermore, it showed a high probability of being cost-effective in both preventing PPCs and improving QALYs in Chinese patients undergoing valve surgery. This trial is registered in the Chinese Clinical Trial Registry (URL: https://www.chictr.org.cn/) with the registration identifier ChiCTR2000039671.

An out-of-court community-based programme to improve the health and well-being of young adult offenders: the Gateway RCT.

Young adults represent a third of the United Kingdom prison population and are at risk of poor health outcomes, including drug and alcohol misuse, self-harm and suicide. Court diversion interventions aim to reduce the negative consequences of criminal sanctions and address the root causes of offending. However, evidence of their effectiveness has not yet been established. The Gateway programme, issued as a conditional caution, aimed to improve the life chances of young adults committing low-level offences. Participants agreed not to reoffend during the 16-week caution and, following a needs assessment, received individual support from a Gateway navigator and attended two workshops encouraging analysis of own behaviour and its consequences. To evaluate the effectiveness and cost-effectiveness of Gateway in relation to health and well-being of participants compared to usual process (court summons or a different conditional caution). Pragmatic, multisite, parallel-group, superiority randomised controlled trial with two 6-month internal pilots and a target sample size of 334. Randomisation between Gateway and usual process was on a 1 : 1 basis. Four Hampshire Constabulary sites recruited 18- to 24-year-old residents of Hampshire and Isle of Wight who were questioned for an eligible low-level offence. Semistructured interviews were also held with a sample of Gateway programme participants, staff and police study recruiters. Primary outcome was the Warwick-Edinburgh Mental Wellbeing Scale score at 12 months. Secondary outcomes included health status, alcohol and drug use, recidivism and resource use. Recruitment commenced in October 2019 and the trial stopped in April 2021. A total of 191 participants were recruited, with 109 randomised to Gateway and 82 to usual process. Due to an initial overestimation of potentially eligible young people and low retention rates, recruitment targets were adjusted, and a range of mitigating measures introduced. Although recruitment broadly met study progression criteria [35/50 (70%) Pilot 1: 64/74 (86%) Pilot 2], retention was low throughout (overall: data collected at week 4 was 50%: at week 16 it was 50%: 1-year 37%). Low retention was multifactorial, with one of the main barriers being difficulties contacting participants. It was therefore not possible to complete the randomised controlled trial or the health economics analyses. Qualitative interviews held with 58 individuals yielded rare insights into the benefits and limitations of this type of intervention, as well as barriers and facilitators in relation to recruitment in this setting. Despite close collaboration with the police to address recruitment and consent issues, expansion of the inclusion criteria and recruitment area and introducing other measures, the researchers were unable to collect sufficient data within an acceptable timeframe. The Gateway study was a unique endeavour to gather evidence for a potentially life-changing intervention for an underserved population. The experience gained indicates that randomised controlled trials of interventions, with a health-related outcome, are possible in this setting but point towards the need for conservative recruitment and retention estimates in this target population. Other study designs should be considered. The qualitative evaluation provided a range of valuable lessons for those seeking to design similar interventions or conduct research in similar settings. This study is registered as ISRCTN11888938. This award was funded by the National Institute for Health and Care Research (NIHR) Public Health Research programme (NIHR award ref: 16/122/20) and is published in full in Public Health Research; Vol. 12, No. 7. See the NIHR Funding and Awards website for further award information.

45 Optimising the management of chronic ischemic heart disease by training general practitioners to deliver very brief advice on physical activity (OptiCor)

Abstract Purpose The German treatment guideline “chronic ischemic/coronary heart disease (IHD)” recommends that general practitioners (GPs) deliver advice on physical activity (PA) to IHD patients. This recommendation is inadequately implemented, often due to GP’s insufficient specific training. International guidelines recommend training GPs in how to deliver PA advice effectively and efficiently. Evidence is lacking on how such training should be designed to match the recipients’ needs, and whether it improves the frequency and quality of advice. With OptiCor we aim to systematically develop and evaluate such a training for GPs to optimise the routine care of IHD patients. Project OptiCor has started in 2022. It comprises three phases over five years. The methodology matches the Medical Research Council framework recommendations for complex interventions. GPs and patients are substantially involved throughout the entire project. OptiCor (funded by the German Ministry of Education and Research) will be presented internationally for the first time. Phase 1 (needs analysis/development)–completed: A nationwide, face-to-face household survey was used to collect data on the receipt of GP-delivered PA advice in people with IHD. Focus group discussions and problem-centred interviews were conducted with GPs and patients with IHD to explore attitudes and motivation towards, experiences with, and barriers and facilitators of PA advice implementation or reception, respectively. The findings are now being used to inform the theory-based training development. Phase 2 (pilot): A pragmatic cluster randomised controlled trial (cRCT) on the effectiveness of the GP training on delivered PA advice during routine care of IHD patients will be pilot tested in summer 2024. Phase 3 (evaluation): A full pragmatic cRCT will be conducted from 2025 onwards (primary endpoint: patient-reported proportions of GP-delivered PA advice). Health economic and process-related analyses will facilitate a potential future broad dissemination and health economic evaluation of the training. Conclusions If our training successfully enhances the proportions and quality of delivered PA advice to IHD patients, it represents a low-threshold strategy for a broad implementation in general practice. The training could extend existing projects on PA referral/prescription schemes, and it could be transferred to the management of other diseases or to other healthcare settings.