Impact of primary health care reforms in Quebec Health Care System: a systematic literature review protocol.

INTRODUCTION: During the last decade the Quebec Public Health Care System (QPHCS) had an important transformation in primary care planning activity. The increase of the service demand together with a significant reduction of supply in primary care may be at risk of reducing access to health care services, with a negative impact on costs and health outcomes. The aims of this systematic literature review are to map and aggregate existing literature and evidence on the primary care provided in Quebec, showing the benefits and limitations associated with the health policies developed in the last two decades, and highlighting areas of improvement. METHODS AND ANALYSIS: PubMed, EMBASE, Web of Science and CINAHL will be searched for articles and government reports between January 2000 and January 2022 using a prespecified search strategy. This protocol adheres to the Preferred Reporting Items for Systematic Reviews and Meta-Analysis for Protocols and has been registered with PROSPERO. A wide range of electronic databases and grey literature sources will be systematically searched using predefined keywords. The review will include any study design, with the exclusion of protocols, with a focus on the analysis of health care policies, outcomes, costs and management of the primary health care services, published in either English or French languages. Two authors will independently screen titles, abstracts, full-text articles and select studies meeting the inclusion criteria. A customised data extraction form will be used to extract data from the included studies. Results will be presented in tabular format developed iteratively by the research team. ETHICS AND DISSEMINATION: Research ethics approval is not required as exclusively secondary data will be used. Review findings will synthesise the characteristics and the impact of the reforms of QPHCS of the last two decades. Findings will therefore be disseminated in peer-reviewed journals, conference presentations and through discussions with stakeholders. PROSPERO REGISTRATION NUMBER: CRD42023421145.

Revising the Suspected-Cancer Guidelines: Impacts on Patients' Primary Care Contacts and Costs.

OBJECTIVES: This study aimed to explore the impact of revising suspected-cancer referral guidelines on primary care contacts and costs. METHODS: Participants had incident cancer (colorectal, n = 2000; ovary, n = 763; and pancreas, n = 597) codes in the Clinical Practice Research Datalink or England cancer registry. Difference-in-differences analyses explored guideline impacts on contact days and nonzero costs between the first cancer feature and diagnosis. Participants were controls ("old National Institute for Health and Care Excellence [NICE]") or "new NICE" if their index feature was introduced during guideline revision. Model assumptions were inspected visually and by falsification tests. Sensitivity analyses reclassified participants who subsequently presented with features in the original guidelines as "old NICE." For colorectal cancer, sensitivity analysis (n = 3481) adjusted for multimorbidity burden. RESULTS: Median contact days and costs were, respectively, 4 (interquartile range [IQR] 2-7) and £117.69 (IQR £53.23-£206.65) for colorectal, 5 (IQR 3-9) and £156.92 (IQR £78.46-£272.29) for ovary, and 7 (IQR 4-13) and £230.64 (IQR £120.78-£408.34) for pancreas. Revising ovary guidelines may have decreased contact days (incidence rate ratio [IRR] 0.74; 95% confidence interval 0.55-1.00; P = .05) with unchanged costs, but parallel trends assumptions were violated. Costs decreased by 13% (equivalent to -£28.05, -£50.43 to -£5.67) after colorectal guidance revision but only in sensitivity analyses adjusting for multimorbidity. Contact days and costs remained unchanged after pancreas guidance revision. CONCLUSIONS: The main analyses of symptomatic patients suggested that prediagnosis primary care costs remained unchanged after guidance revision for pancreatic cancer. For colorectal cancer, contact days and costs decreased in analyses adjusting for multimorbidity. Revising ovarian cancer guidelines may have decreased primary care contact days but not costs, suggesting increased resource-use intensity; nevertheless, there is evidence of confounding.

Singing for people with aphasia (SPA): results of a pilot feasibility randomised controlled trial of a group singing intervention investigating acceptability and feasibility.

OBJECTIVES: Pilot feasibility randomised controlled trial (RCT) for the singing groups for people with aphasia (SPA) intervention to assess: (1) the acceptability and feasibility of participant recruitment, randomisation and allocation concealment; (2) retention rates; (3) variance of continuous outcome measures; (4) outcome measure completion and participant burden; (5) fidelity of intervention delivery; (6) SPA intervention costs; (7) acceptability and feasibility of trial and intervention to participants and others involved. DESIGN: A two-group, assessor-blinded, randomised controlled external pilot trial with parallel mixed methods process evaluation and economic evaluation. SETTING: Three community-based cohorts in the South-West of England. PARTICIPANTS: Eligible participants with post-stroke aphasia were randomised 1:1 to SPA or control. INTERVENTION: The manualised SPA intervention was delivered over 10 weekly singing group sessions, led by a music facilitator and assisted by an individual with post-stroke aphasia. The intervention was developed using the Information-Motivation-Behavioural skills model of behaviour change and targeted psychosocial outcomes. Control and intervention participants all received an aphasia information resource pack. OUTCOME MEASURES: Collected at baseline, 3 and 6 months post-randomisation, candidate primary outcomes were measured (well-being, quality of life and social participation) as well as additional clinical outcomes. Feasibility, acceptability and process outcomes included recruitment and retention rates, and measurement burden; and trial experiences were explored in qualitative interviews. RESULTS: Of 87 individuals screened, 42 participants were recruited and 41 randomised (SPA=20, control=21); 36 participants (SPA=17, control=19) completed 3-month follow-up, 34 (SPA=18, control=16) completed 6-month follow-up. Recruitment and retention (83%) were acceptable for a definitive RCT, and participants did not find the study requirements burdensome. High fidelity of the intervention delivery was shown by high attendance rates and facilitator adherence to the manual, and participants found SPA acceptable. Sample size estimates for a definitive RCT and primary/secondary outcomes were identified. CONCLUSIONS: The SPA pilot RCT fulfilled its objectives, and demonstrated that a definitive RCT of the intervention would be both feasible and acceptable. TRIAL REGISTRATION NUMBER: NCT03076736.

Cancer diagnostic tools to aid decision-making in primary care: mixed-methods systematic reviews and cost-effectiveness analysis.

BACKGROUND: Tools based on diagnostic prediction models are available to help general practitioners diagnose cancer. It is unclear whether or not tools expedite diagnosis or affect patient quality of life and/or survival. OBJECTIVES: The objectives were to evaluate the evidence on the validation, clinical effectiveness, cost-effectiveness, and availability and use of cancer diagnostic tools in primary care. METHODS: Two systematic reviews were conducted to examine the clinical effectiveness (review 1) and the development, validation and accuracy (review 2) of diagnostic prediction models for aiding general practitioners in cancer diagnosis. Bibliographic searches were conducted on MEDLINE, MEDLINE In-Process, EMBASE, Cochrane Library and Web of Science) in May 2017, with updated searches conducted in November 2018. A decision-analytic model explored the tools' clinical effectiveness and cost-effectiveness in colorectal cancer. The model compared patient outcomes and costs between strategies that included the use of the tools and those that did not, using the NHS perspective. We surveyed 4600 general practitioners in randomly selected UK practices to determine the proportions of general practices and general practitioners with access to, and using, cancer decision support tools. Association between access to these tools and practice-level cancer diagnostic indicators was explored. RESULTS: Systematic review 1 - five studies, of different design and quality, reporting on three diagnostic tools, were included. We found no evidence that using the tools was associated with better outcomes. Systematic review 2 - 43 studies were included, reporting on prediction models, in various stages of development, for 14 cancer sites (including multiple cancers). Most studies relate to QCancer® (ClinRisk Ltd, Leeds, UK) and risk assessment tools. DECISION MODEL: In the absence of studies reporting their clinical outcomes, QCancer and risk assessment tools were evaluated against faecal immunochemical testing. A linked data approach was used, which translates diagnostic accuracy into time to diagnosis and treatment, and stage at diagnosis. Given the current lack of evidence, the model showed that the cost-effectiveness of diagnostic tools in colorectal cancer relies on demonstrating patient survival benefits. Sensitivity of faecal immunochemical testing and specificity of QCancer and risk assessment tools in a low-risk population were the key uncertain parameters. SURVEY: Practitioner- and practice-level response rates were 10.3% (476/4600) and 23.3% (227/975), respectively. Cancer decision support tools were available in 83 out of 227 practices (36.6%, 95% confidence interval 30.3% to 43.1%), and were likely to be used in 38 out of 227 practices (16.7%, 95% confidence interval 12.1% to 22.2%). The mean 2-week-wait referral rate did not differ between practices that do and practices that do not have access to QCancer or risk assessment tools (mean difference of 1.8 referrals per 100,000 referrals, 95% confidence interval -6.7 to 10.3 referrals per 100,000 referrals). LIMITATIONS: There is little good-quality evidence on the clinical effectiveness and cost-effectiveness of diagnostic tools. Many diagnostic prediction models are limited by a lack of external validation. There are limited data on current UK practice and clinical outcomes of diagnostic strategies, and there is no evidence on the quality-of-life outcomes of diagnostic results. The survey was limited by low response rates. CONCLUSION: The evidence base on the tools is limited. Research on how general practitioners interact with the tools may help to identify barriers to implementation and uptake, and the potential for clinical effectiveness. FUTURE WORK: Continued model validation is recommended, especially for risk assessment tools. Assessment of the tools' impact on time to diagnosis and treatment, stage at diagnosis, and health outcomes is also recommended, as is further work to understand how tools are used in general practitioner consultations. STUDY REGISTRATION: This study is registered as PROSPERO CRD42017068373 and CRD42017068375. FUNDING: This project was funded by the National Institute for Health Research (NIHR) Health Technology programme and will be published in full in Health Technology Assessment; Vol. 24, No. 66. See the NIHR Journals Library website for further project information.

In the UK, people with cancer tend to die sooner than people with cancer in other European countries. This may be because their cancers are caught at a later stage, perhaps after they have spread. Spotting cancer earlier in people, and testing them sooner, may extend people's lives. Researchers have developed 'diagnostic tools', which give the probability of having cancer, based on a patient's symptoms, blood test results and other information. The tools help family doctors decide who needs further testing for possible cancer, including cancers of the digestive, urinary and reproductive systems, and in the blood. We do not know how many family doctors have these tools, or how well the tools work. We systematically reviewed published studies about how these tools were developed, how good and accurate they are, and what effects their use has on patients. We found that many tools have been developed, but there is little evidence that they improve the quality or length of life. We sent surveys to family doctors all over the UK asking if they had the tools at their practice and if they used them. Based on the replies we received, we estimate that the tools are in about one in three practices. They are likely to be used in about half of the practices where they are available. For practices in England only, we looked for, but did not find, any association between using the tools and the number of urgent appointments made for cancer testing. We used a computer model to show what might happen if family doctors used the tools for patients who have symptoms of bowel cancer. In our model, if general practitioners used the tools, patients would need fewer appointments before they were referred to a specialist. This should reduce the time to diagnosis and treatment, compared with not using the tools. However, there is very little evidence as to whether or not this is indeed the case. Therefore, at the moment, we cannot say whether or not the use of such tools by general practitioners is better for patients and the NHS. More research is needed on what effect these tools have on patients, especially as to whether or not quality and length of life are improved.

The heterogeneous causal effects of neonatal care: a model of endogenous demand for multiple treatment options based on geographical access to care.

Neonatal units in the UK are organised into three levels, from highest Neonatal Intensive Care Unit (NICU), to Local Neonatal Unit (LNU) to lowest Special Care Unit (SCU). We model the endogenous treatment selection of neonatal care unit of birth to estimate the average and marginal treatment effects of different neonatal designations on infant mortality, length of stay and hospital costs. We use prognostic factors, survival and hospital care use data on all preterm births in England for 2014-2015, supplemented by national reimbursement tariffs and instrumental variables of travel time from a geographic information system. The data were consistent with a model of demand for preterm birth care driven by physical access. In-hospital mortality of infants born before 32 weeks was 8.5% overall, and 1.2 (95% CI: -0.7, 3.2) percentage points lower for live births in hospitals with NICU or SCU compared to those with an LNU according to instrumental variable estimates. We find imprecise differences in average total hospital costs by unit designation, with positive unobserved selection of those with higher unexplained absolute and incremental costs into NICU. Our results suggest a limited scope for improvement in infant mortality by increasing in-utero transfers based on unit designation alone.

Economic evaluation of meningococcal vaccines: considerations for the future.

In 2018, a panel of health economics and meningococcal disease experts convened to review methodologies, frameworks, and decision-making processes for economic evaluations of vaccines, with a focus on evaluation of vaccines targeting invasive meningococcal disease (IMD). The panel discussed vaccine evaluation methods across countries; IMD prevention benefits that are well quantified using current methods, not well quantified, or missing in current cost-effectiveness methodologies; and development of recommendations for future evaluation methods. Consensus was reached on a number of points and further consideration was deemed necessary for some topics. Experts agreed that the unpredictability of IMD complicates an accurate evaluation of meningococcal vaccine benefits and that vaccine cost-effectiveness evaluations should encompass indirect benefits, both for meningococcal vaccines and vaccines in general. In addition, the panel agreed that transparency in the vaccine decision-making process is beneficial and should be implemented when possible. Further discussion is required to ascertain: how enhancing consistency of frameworks for evaluating outcomes of vaccine introduction can be improved; reviews of existing tools used to capture quality of life; how indirect costs are considered within models; and whether and how the weighting of quality-adjusted life-years (QALY), application of QALY adjustment factors, or use of altered cost-effectiveness thresholds should be used in the economic evaluation of vaccines.

Singing for people with aphasia (SPA): a protocol for a pilot randomised controlled trial of a group singing intervention to improve well-being.

INTRODUCTION: The singing for people with aphasia (SPA) intervention aims to improve quality of life and well-being for people with poststroke aphasia. A definitive randomised controlled trial (RCT) is required to assess the clinical and cost effectiveness of SPA. The purpose of this pilot study is to assess the feasibility of such a definitive trial and inform its design. METHODS AND ANALYSIS: A two-group, assessor-blinded, randomised controlled external pilot trial with parallel mixed methods process evaluation and economic evaluation. Forty-eight participants discharged from clinical speech and language therapy will be individually randomised 1:1 to SPA (10 group sessions plus a resource booklet) or control (resource booklet only). Outcome assessment at baseline, 3 and 6 months postrandomisation include: ICEpop CAPability measure for adults, Stroke and Aphasia Quality of Life, EQ-5D-5L, modified Reintegration into Normal Living Index, Communication Outcome After Stroke, Very Short Version of the Minnesota Aphasia Test, Service Receipt Inventory and Care Related Quality of Life. Feasibility, acceptability and process outcomes include recruitment and retention rates, with measurement burden and trial experiences being explored in qualitative interviews (15 participants, 2 music facilitators and 2 music champions). Analyses include: descriptive statistics, with 95% CIs where appropriate; qualitative themes; intervention fidelity from videos and session checklists; rehearsal of health economic analysis. ETHICS AND DISSEMINATION: NHS National Research Ethics Service and the Health Research Authority confirmed approval in April 2017; recruitment commenced in June 2017. Outputs will include: pilot data to inform whether to proceed to a definitive RCT and support a funding application; finalised intervention manual for multicentre replication of SPA; presentations at conferences, public involvement events; internationally recognised peer reviewed journal publications, open access sources and media releases. TRIAL REGISTRATION NUMBER: NCT03076736.

Community-based rehabilitation training after stroke: results of a pilot randomised controlled trial (ReTrain) investigating acceptability and feasibility.

OBJECTIVES: To assess acceptability and feasibility of trial processes and the Rehabilitation Training (ReTrain) intervention including an assessment of intervention fidelity. DESIGN: A two-group, assessor-blinded, randomised controlled trial with parallel mixed methods process and economic evaluations. SETTING: Community settings across two sites in Devon. PARTICIPANTS: Eligible participants were: 18 years old or over, with a diagnosis of stroke and with self-reported mobility issues, no contraindications to physical activity, discharged from National Health Service or any other formal rehabilitation programme at least 1 month before, willing to be randomised to either control or ReTrain and attend the training venue, possessing cognitive capacity and communication ability sufficient to participate. Participants were individually randomised (1:1) via a computer-generated randomisation sequence minimised for time since stroke and level of functional disability. Only outcome assessors independent of the research team were blinded to group allocation. INTERVENTIONS: ReTrain comprised (1) an introductory one-to-one session; (2) ten, twice-weekly group classes with up to two trainers and eight clients; (3) a closing one-to-one session, followed by three drop-in sessions over the subsequent 3 months. Participants received a bespoke home-based training programme. All participants received treatment as usual. The control group received an exercise after stroke advice booklet. OUTCOME MEASURES: Candidate primary outcomes included functional mobility and physical activity. RESULTS: Forty-five participants were randomised (ReTrain=23; Control=22); data were available from 40 participants at 6 months of follow-up (ReTrain=21; Control=19) and 41 at 9 months of follow-up (ReTrain=21; Control=20). We demonstrated ability to recruit and retain participants. Participants were not burdened by the requirements of the study. We were able to calculate sample estimates for candidate primary outcomes and test procedures for process and health economic evaluations. CONCLUSIONS: All objectives were fulfilled and indicated that a definitive trial of ReTrain is feasible and acceptable. TRIAL REGISTRATION NUMBER: NCT02429180; Results.

Vaccinating Italian infants with a new multicomponent vaccine (Bexsero®) against meningococcal B disease: A cost-effectiveness analysis.

The European Medicines Agency has approved a multicomponent serogroup B meningococcal vaccine (Bexsero®) for use in individuals of 2 months of age and older. A cost-effectiveness analysis (CEA) from the societal and Italian National Health Service perspectives was performed in order to evaluate the impact of vaccinating Italian infants less than 1 y of age with Bexsero®, as opposed to non-vaccination. The analysis was carried out by means of Excel Version 2011 and the TreeAge Pro® software Version 2012. Two basal scenarios that differed in terms of disease incidence (official and estimated data to correct for underreporting) were considered. In the basal scenarios, we considered a primary vaccination cycle with 4 doses (at 2, 4, 6 and 12 months of age) and 1 booster dose at the age of 11 y, the societal perspective and no cost for death. Sensitivity analyses were carried out in which crucial variables were changed over probable ranges. In Italy, on the basis of official data on disease incidence, vaccination with Bexsero® could prevent 82.97 cases and 5.61 deaths in each birth cohort, while these figures proved to be three times higher on considering the estimated incidence. The results of the CEA showed that the Incremental Cost Effectiveness Ratio (ICER) per QALY was €109,762 in the basal scenario if official data on disease incidence are considered and €26,599 if estimated data are considered. The tornado diagram indicated that the most influential factor on ICER was the incidence of disease. The probability of sequelae, the cost of the vaccine and vaccine effectiveness also had an impact. Our results suggest that vaccinating infants in Italy with Bexsero® has the ability to significantly reduce meningococcal disease and, if the probable underestimation of disease incidence is considered, routine vaccination is advisable.