Information retrieval for systematic reviews (SRs) should include sensitive searches in several bibliographic databases. In addition to standard databases (i.e., MEDLINE, Embase and CENTRAL), researchers might consider subject-specific ones. In the fields of nursing and midwifery, a SR would typically include CINAHL as a subject-specific database. The aim of this study was to analyze the number and relevance of references retrieved from CINAHL in six SRs on maternal care.

We conducted a retrospective analysis of six SRs (e.g., benefit of intrapartum ultrasound or one-to-one care during labor). The study type was limited to randomized controlled trials (RCTs) in all but three SRs. In all cases, MEDLINE, Embase, CENTRAL and CINAHL were searched for primary studies. Further information sources (e.g., study registries and reference lists of SRs) were also considered. The proportion of the additional number of hits and studies included from CINAHL as well as the corresponding number of participants were calculated.

Overall, the reviewers screened 12,013 references from bibliographic databases and identified forty relevant studies. CINAHL contained 2,643 (22 percent) of the references. In five out of six SRs, no additional studies were identified in CINAHL. In the remaining SR on birthing positions, the reviewers included thirteen RCTs of which one was a feasibility study with 68 participants indexed only in CINAHL. This corresponds to 0.9 percent of the women participating in all thirteen RCTs (n = 7,861). However, this study was cited in a journal article on a subsequent RCT that was identified and included via MEDLINE and ClinicalTrials.gov.

It is not necessary to search CINAHL in SRs on maternal care if standard databases and further information sources are considered. An additional study from CINAHL was included in one out of six SRs, a small feasibility study that could have been identified without CINAHL via a subsequent RCT.

Currently, the Institute for Quality and Efficiency in Health Care (IQWiG) does not restrict literature searches by language. Given limited resources, it is unclear whether the effort put into screening and translating studies published in non-English and non-German (nEnG) languages yields much new information when compared to including only English and German literature. Therefore, we aimed to analyze the impact of nEnG literature on the conclusion of IQWiG's health technology assessments (HTAs).

We checked for seventy-two IQWiG HTAs (all non-drug intervention HTAs published until August 2018 and three additional HTAs on drugs) whether they included nEnG studies. For all HTAs including at least one nEnG study, we analyzed whether the statistical significance would have changed for any endpoint without the respective nEnG study(ies). If no endpoint was impacted by a nEnG study, we classified the study as non-relevant to the HTA's conclusion and specified a reason for this.

Of seventy-two HTAs, twenty-nine (40 percent) included a total of eighty-three nEnG publications). Three HTAs were impacted by the inclusion of altogether seven Chinese publications. For one HTA on systemic therapy, five endpoints’ conclusions were changed; for the other two HTAs, the statistical significance would have changed for one endpoint each. The remaining seventy-six publications (included in sixty-nine HTAs) were judged as non-relevant to the HTA's conclusion, the most prominent reason being “meta-analysis would have had the same result without respective study” (44 percent of nEnG publications).

Only three of seventy-two HTAs (4 percent) were impacted by nEnG publications, the changes being minimal for two of these. When faced with limited time or personnel resources, searching only for English and German publications may be sufficient, especially when generalizability issues are a possible concern.

Primary biliary cholangitis (CBP) is a rare autoimmune cholestatic liver disease, inflammation and progressive destruction of small and medium-sized interlobular ducts, progressing to fibrosis, cirrhosis, and death. Currently, the Brazilian public health system (SUS) offers treatment of the symptoms of cirrhosis, and has no medication with indication for CBP.

Scientific technical opinion with systematic review (SR) of available evidence in the databases MEDLINE (Pubmed), LILACS and Cochrane Library (accessed July 2017) on ursodeoxycholic acid (AUDC). Methodological quality was evaluated with AMSTAR and Newcastle Ottawa tools. Meta-analyses were performed in Review Manager® 5.2 in the random effects model. Analysis of the budget impact calculation deterministic model, from the perspective of five years for the SUS.

Ten SRs and three cohorts were included. There was no statistically significant difference between AUDC and placebo in outcome. Overall survival was significantly (P <0.001) higher in the AUDC group compared to that predicted by the Mayo model or placebo. Treatment with UCD showed an increase in the long-term transplant-free survival time from the fifth year of treatment, with statistically significant results for years five, eight and ten (p <0.01). There were no statistically significant differences for safety outcomes. Based on the assumptions adopted, the incremental budgetary impact with the incorporation of the AUDC into SUS would be BRL 11.77 million (EUR 2.68 million) in the first year and BRL 98.52 million (EUR 22.45 million) in the accumulated five years, considering a market share of 10 percent per year.

Despite the uncertainties in the evidence of effectiveness of the AUDC and the probably underestimated budgetary impact, AUDC was incorporated into the SUS because it is the only alternative with indication for CBP and in use for more than two decades, allowing everyone access to the medicine

The South African Pharmacoeconomic Submissions Guideline (SAPG) is currently voluntary for medicines in the private health sector but may become mandatory and more widely used under the proposed National Health Insurance system. To make recommendations on evidence generation and areas where the SAPG could be strengthened, the study compared the SAPG requirements with other African pharmacoeconomic guidelines and the National Institute for Health and Care Excellence Methods Guide (NICE MG).

The World Health Organisation, International Network of Agencies for Health Technology Assessment (INAHTA), HTA International, and the International Society for Pharmacoeconomics and Outcomes Research websites were consulted, and email requests sent to named individuals from retrieved source material. The European Network for HTA Core Model® (version 3.0) (the Model®) provided the evaluation and comparison framework, using three criteria: completely, partly or not completely requiring the same or similar information as the Model®.

Of the forty-five countries identified, only Egypt had a publicly available pharmacoeconomic guideline (Egyptian Pharmacoeconomic Guideline (EPG)). The guidelines varied considerably in their intended audience, size and content. All three guidelines’ primary focus was the cost and economic evaluation, and health problem and current use domains. Safety, organisational, ethical and legal aspects were poorly covered by the SAPG and EPG guidelines (less than thirty percent of issues in each domain completely / partly covered). The SAPG completely or partly required the same or similar information in the Model® for thirty-nine percent of total issues, the EPG thirty-three percent and the NICE MG sixty-six percent

The SAPG was not as comprehensive as the NICE MG and poorly covered some key aspects of HTAs, suggesting that the SAPG could be developed to be more informative for decision-makers. Evidence generation should focus on describing the health problem the technology is targeting and on evidence that can be synthesized into cost-effectiveness analyses.

European agencies evaluate the adverse events (AEs) of asthma drugs in studies. The impact of these evaluations on reimbursement decisions remains unclear.

Adult asthma evaluations were accessed from initial regulatory decision by the European Medicines Agency (EMA) through reimbursement evaluations. Omalizumab and reslizumab were chosen for the comparison of an older with a newer asthma drug. A timeline was then constructed to evaluate the effect of AEs on reimbursement recommendations. Evaluations from the United Kingdom (NICE) were not used because their documents are not as complete or in depth, including those from Sweden (TLV) and Germany (IQWIG).

Omalizumab was first approved as add-on therapy to improve asthma control in October 2005. Of the 6 decisions made between 2006 and 2012, safety information was found in 4 of them, all from 2006 and evaluated by either Scotland (SMC) or France (HAS). These desicions all received either a ‘Do not recommend’ or a ‘Recommend with restrictions’ decision. Reslizumab was first approved as add-on therapy for patients with severe eosinophilic asthma in August 2016. Of the 9 decisions made in 2017, safety information was found in 5 decisions evaluated by IQWiG, Germany (G-BA), HAS, or SMC, which gave them a Do not recommend, Recommend with restrictions, or Recommend decision. Of the Do not recommend decisions, both the omalizumab and reslizumab safety evaluations mentioned common AEs (worsening asthma) and less common AEs (malignant tumors). Of the Recommend with restrictions decisions, the same AEs were seen. Only reslizumab had Recommended decisions. In the safety evaluation, there were no specific AEs named.

The impact of AEs on reimbursement decisions could not be detected when comparing omalizumab and reslizumab reviews, as other factors may contribute to the decisions. Further research should be conducted to explore this issue.

Treatment of varicose veins is currently performed by different interventionist alternatives that include surgical, endothermal and non-thermal ablation therapies. The main guidelines recommended endovenous thermal treatment as the first choice therapy; however present side effects related to thermal energy. Non-tumescent endovenous ablation techniques such as cyanoacrylate ablation (CA) started to develop to avoid these problems. The objective of this study is to assess the effectiveness and safety of CA for saphenous vein incompetence.

A systematic review with meta-analysis was carried out. The search of scientific literature was performed in Medline, Embase, Cochrane library, CDR, WoS and Scopus databases. GRADE methodology was used to assess the quality of the evidence and Cochrane risk of bias tool to assess methodological quality of randomized control trials (RCT). Pooled risk ratio was calculated using a random effects model.

Two RCTs and one non-RCT comprising 1,077 participants were included. Additionally, 10 case series were included for safety assessment. Pooled analysis of closure rates by the two RCTs indicated there were not significant differences between CA and radiofrequency ablation (RFA) or endovenous laser ablation (EVLA). Improvements in venous clinical severity score were reported by all comparative studies without significant differences among groups. The most frequently reported adverse events were ecchymosis, phlebitis, paraesthesia, and thrombosis. The pooled analysis showed significant differences only in ecchymosis rates, with lower probability of ecchymosis in CA groups. CA treatment showed lower pain rates and shorter intervention times and recovery compared to endothermal therapies.

The effectiveness of CA devices in the treatment of varicose veins is comparable to EVLA and RFA, while the rates of adverse effects are lower. Despite the limitations of the evidence, CA may be a promising alternative to existing treatments, with the advantages of better patient comfort.

Patients can provide valuable experience on living with diseases, health-related quality of life, various therapies and relevant outcomes. Their input and perspectives can be helpful in complementing health technology assessment (HTA) processes. The European Network for HTA (EUnetHTA), funded by the European Commission, aims to further advance and standardise patient involvement processes in order to add to the quality and applicability of HTAs and to allow capability building.

Different methods for patient involvement in HTAs on non-pharmaceutical technologies were tested: Patient input templates (open questions sent to relevant patient organizations, or published on EUnetHTA website); scoping meeting with patients/patient representatives; one-on-one conversation and group conversation. Applied methods depended on the scope of the HTA and other factors like timelines of HTAs and burden of disease for patients.

Patients were included in eight of sixteen HTAs on non-pharmaceutical technologies. Applied methods were: group conversation (n = 2), scoping meeting (n = 1), patient input templates (n = 4), one-on-one conversation (n = 2,) and other approach (i.e. written feedback on scope n= 2). In some HTAs more than one method was used. Main reasons for not including patients were inability to identify suitable patients or tight timelines. Patients' feedback on health-related quality of life and outcome measures proved most useful in the scoping phase.

The different approaches were useful for complementing HTA processes. Those need to be further tested and evaluated in order to formulate deeper understanding about the impact of patient involvement on HTA. Additionally, feedback from patients that were actively involved in the HTAs should be collected to further improve the involvement methods that should serve as basis for future recommendations post 2020.

NICE (National Institute for Health and Care Excellence) makes recommendations on the public reimbursement of medicines based on their clinical- and cost-effectiveness. The recommendation is made by an Appraisal Committee (comprising a multi-disciplinary group of independent experts) as part of a technology appraisal. There are four Appraisal Committees (A,B,C,D); this research investigates whether appraisal outcomes vary by committee.

All publicly-available Final Appraisal Determinations from NICE Single Technology Appraisals (STA) were screened (01/10/2009-14/11/2018) and key data were extracted. Homogeneity in rates of acceptance or rejection across the committees was assessed using Chi-squared tests.

The Appraisal Committee was identified for 298 technologies, 56% (168/298) of which were ‘recommended’. The number of technologies assessed by each committee was similar (A:79, B:62, C:91, D:66). However, STAs conducted by Committee D were significantly less likely to receive ‘recommended’ outcomes (A:68% [54/79], B:65% [40/62], C:53% [48/91], D:39% [26/66]; p < 0.01). STAs for oncology indications had higher ’not recommended’ outcomes than those for non-oncology indications (25% vs. 9%). The lower ‘recommendation’ rates for committee D persisted across oncology (A:60%, B:83%, C:50%, D:38%; p = 0.01) and non-oncology indications (A:73%, B:53%, C:55%, D:40%; p < 0.01). However, STAs conducted by Committee D were significantly more likely to receive ‘optimized’ recommendations (A:16%, B:21%, C:33%, D: 36%; p < 0.01) and when considering the rates of ‘recommended’ and ‘optimized’ outcomes compared to ‘only in research’ and ‘not recommended’ outcomes, no significant differences were found (A:85%, B: 85%, C:86%, D:76%; p = 0.27).

STAs undertaken by NICE Appraisal Committee D was associated with a significantly lower rate of ‘recommended’ outcomes but tended to an ‘optimized’ recommendation significantly more than the other committees. Further research is needed to determine if this reflects any deviation in uniform implementation of NICE methodology between Committees.

Since 2011, new pharmacological therapies in Germany are subject to an early benefit assessment (EBA) upon launch. The Institute for Quality and Efficiency in Health Care (IQWiG) usually conducts an initial assessment, followed by the Federal Joint Committee (G-BA) issuing a final resolution. If the G-BA deem a new therapy offers no additional benefit over relevant comparators, it cannot attain premium-pricing through price negotiations. This research compares G-BA and IQWiG assessment outcomes over time.

All EBA resolutions were extracted from the G-BA website alongside corresponding IQWiG assessments (01/01/2011-19/09/2018) and key information compared. For extracted outcome data, the focus was the subgroup of greatest additional benefit.

Of 261 identified EBAs with both G-BA and IQWiG assessment outcomes published, 59% (155/261) did not differ in their additional benefit. The G-BA concluded on an additional benefit where IQWiG deemed none in 13% (34/261) of cases, which was consistent pre-2015: 13% (11/87) and 2015-onwards: 13% (23/174). Conversely, IQWiG deemed an additional benefit where the G-BA concluded on none in 3% (8/261) of cases, none of which were pre-2015 (0/87) vs. 5% (8/261) for 2015-onwards. G-BA and IQWiG both agreed that additional benefit was offered but differed in its extent in 14% (37/261; in 23 cases: G-BA's rating was lower, 14 cases: G-BA's was higher) with 19% (17/87) pre-2015 vs. 8% (14/174) 2015-onwards.

The G-BA has deviated from IQWiG's initial assessment in around one-third of resolutions, with potential significant rebate negotiation consequences. The divergence in extent of additional benefit (where both agree on additional benefit) appears to be becoming less common over time. However, a slight converse time-trend appears regarding divergence on whether any additional benefit is offered, driven by increased incidence of G-BA deeming no additional benefit contrary to IQWiG. This emphasizes that companies should fully engage with the EBA consultation process post-IQWiG appraisal.

This study explores the factors (principally evidential) that predict guidance recommendations by this NICE committee. There are three main types of recommendations: Standard/normal arrangements (can be done without restriction in the NHS); Special arrangements (can be done under certain conditions); and Research only.

The following data were extracted from all published pieces of Interventional Procedure Guidance (IPGs) produced by this committee: year, IPG number, recommendations, evidence base (numbers and types of included studies, numbers of included patients etc.). All data were extracted independently by two researchers, and any disagreements clarified by consensus. Data were tabulated and descriptive statistics produced. Regression analyses will be performed using these data to identify any statistically significant predictors of recommendations.

IPG recommendations (n = 496); year range: 2003-2018. Proportion of IPGs by each recommendation: 50% Standard; 38% Special; 11% Research Only; 2% Do Not Do. Proportion of IPGs with highest level evidence (i.e. systematic review and/or RCT) by recommendation type: Standard = 64% (152/239); Special = 43% (77/180); Research Only = 48% (26/54); Do Not Do = 75% (6/8). Mean numbers of patients by recommendation type: Standard = 7,838; Special = 3,935; Research Only = 2,423. There is also a clear trend over time: Standard recommendations decrease for all IPGs from 63% in 2003-2009 to 40% in 2014-2018; and the evidence threshold for Standard recommendations increases over time from 56% based on systematic reviews and/or RCTs in 2003-2009 to 85% in 2014-2018; mean numbers of patients per Standard recommendation also increase from 2,002 to 6,098 over this period.

Higher levels evidence and numbers of patients increase the likelihood of the most positive recommendation. However, this evidence might still lack sufficient quality or certainty to answer a policy question. The evidence threshold to achieve a Standard recommendation has also increased markedly over time. As with other NICE committees, factors other than cost and perceived hierarchies of evidence clearly act as drivers of decisions.

In rare disease areas representative data are scarce. Routine sick fund claims data provide a meaningful and reliable base for the in- and outpatient treatment landscape. This real-world data (RWE) from Germany was used to describe treatment patterns for Diffuse Large B-cell Lymphoma (DLBCL), the most frequent and aggressive non-Hodgkin lymphoma type in adults.

Claims data from several sick funds of 4.8 Million insured were analyzed. Diagnosis of non-follicular Lymphoma (C83) was confirmed in 2.178 patients, DLBCL (C83.3) in 819 patients. The analysis was age- and gender-adjusted, observational period was 2014 and 2015. Treatments were analyzed for hospitalization and medication based on ATC-Code, Pharma Central Number and coded diagnoses (per ICD).

Mean age of DLBCL patients was 60.3 years, with two peaks at 50-54 and 70-74 years. Total costs for patients with DLBCL averaged 25.048 EUR versus 1.259 EUR in healthy insured. Charlson comorbidity index (CCI) of 4.58 indicates clinical relevance and severity. Comorbidities included several psychiatric diagnoses such as depression in every fifth patient. Mean 3.2 hospitalizations with average 31.5 hospital days were observed in DLBCL patients. Forty-seven percent of patients during observational time-frame did not receive oncological treatment, including relapsed / refractory patients. Only few patients received stem cell transplantation (2.6 percent) or radiation (3.9 percent). Most pharmacological treatments were Rituximab (RTX) + CHOP (57 percent), followed by RTX mono therapy (25 percent) or RTX in combination with Bendamustine (8 percent).

Despite limitations in sick fund claims analyses, these provide a reasonable database for rare diseases. They allow standard treatment pathway- and longitudinal analyses. All DLBCL patients frequently required hospitalization and generated significant costs. A high unmet medical need exists for treatments other than palliative care, especially for a tolerable and effective outpatient therapy in elderly relapsed / refractory DLBCL.

The study goal was to estimate prevalence of population in secondary prevention for Atherosclerotic Cardiovascular Disease (ASCVD) stratified by the pharmacological treatment and related outcomes using Health Information Systems (HIS).

From HIS of Marche and Umbria Regions (1.8 millions of inhabitants) which collect information related to hospitalizations, drugs prescriptions, outpatient visits and results of laboratory tests, we identified all patients aged ≤ 80 years with one or more hospitalization with DRG related to Acute Coronary Syndrome, Peripheral Artery Disease, Ischemic Stroke and Transient Ischemic Attack and discharge date between 2011 and 2014 (study period). Pharmacological treatment for each subject was defined selecting all prescriptions of Statins, Ezetimibe and Simvastatin/Ezetimibe, retrieved between the date of the last prescription in the study period and the previous 90 days. We stratified patient in no-treated, treated with low/medium intensity statins (LMS), high-dose statins (HDS) and other Lipid-Lowering Therapies (LLTs). Furthermore, for Umbria region, we selected the last blood levels test of LDL-cholesterol occurred in period 2011-2016. Starting from test date, we defined the pharmacological treatment in the previous 90 days. Subject were stratified based on LDL-C levels in target (<70) and not at-target (≥70) patients.

Population in secondary prevention for ASCVD in period 2011-2014 in Marche and Umbria was estimated in 23,043 (prevalence: 4.3 x 1,000 inhabitants), corresponding to more than 800,000 subjects in Italian population. Within treated patients: 51.3% received LMS, 38.1% HDS and 10.6% other LLTs. No-treated patients were 27.8%. LDL-C target was achieved by 34.9% of patients treated with LMS and by 46.1% of patients treated with other LLTs.

The study, based on Italian administrative databases, allowed to estimate the very high risk population in secondary prevention for ASCVD. It highlighted a relevant proportion of no-treated patients, and an high proportion of patients that did not achieve recommended LDL-C target.

Randomized controlled trials (RCTs) are considered the gold standard in the hierarchy of research designs for evaluating the efficacy and safety of a treatment intervention. The low external validity of RCTs and the general shortage of clinical evidence available to support the use of many medical devices have emphasized the necessity for exploring the use of real-world data (RWD) as a complementary source to RCTs data for establishing a more robust evidence base on the effectiveness of medical devices. The aim of the present project is to assess in a comprehensive way the existing sources of real world data on medical devices in Europe.

The guidelines to the mapping exercise have been outlined in a research protocol. First, all national relevant sources (e.g. website of Ministry of Health, national institutions, research bodies) are screened, both in local language and English. Second, we perform a systematic search on PubMed using a set of key words for each case study, adapted to each country setting. Finally, we seek advice from key actors in the field of the device and clinical conditions, such as manufacturers or clinicians.

Information on existing sources of RWD for each case studies are provided in a template including details on the key features of the source (e.g. data producer, data collection period, sample size, study design, geographical coverage) and the main content of the dataset, distinguishing socio-demographic information, clinical and epidemiological data, data on resource use and health outcomes. The data mapping includes all countries of the project participants, i.e. Italy, UK, Netherlands, Switzerland, Germany, Hungary, and we enlarge the scope of our mapping including other countries: Spain, France, Denmark, Finland, Sweden, Poland and Hungary as well as international databases at pan-EU level. The number of available sources of RWD and their quality vary depending on case study and across countries. For example, in the case of orthopaedics, many countries have a national registry and administrative data, such as hospital discharge, contain useful information, although not as detailed. When a registry is not available, it is often the case that more observational studies are available; this occurs for example in France.

In this work we shows the importance of RWE and map in an accurate and comprehensive way which source of RWD are currently available and to what extent they are known and used in medical, epidemiological and economic research.

The aim was to evaluate the effectiveness, safety and economic impact of long-acting insulin analogues (LAIA) compared to NPH for type 1 diabetes mellitus (DM1).

A search was performed in five electronic databases to find systematic reviews (SR) comparing at least a LAIA to NPH insulin for DM1. Budget impact analysis was performed from the perspective of Brazilian public health system (SUS), with NPH insulin as the base scenario. The costs were extracted from the Integrated System of Administration of General Services (SIASG). The market share was calculated per month, using a logarithmic function with maximum diffusion of 50% at the end of the time horizon - five years.

A total of 160 studies were identified and seven SR of low to uncertain risk of bias were selected. LAIA have shown modest clinical benefit and its effect is more prominent for the control of severe and nocturnal hypoglycaemia. Insulins glargine and detemir compared to NPH were associated with reduction in HbA1c levels between 0.16% and 0.40% and associated with lower risk of episodes of severe hypoglycemia. Insulin degludec compared to NPH showed no statistically significant difference in the reduction of HbA1c levels and in the episodes of severe hypoglycemia. The budget impact ranges from USD 210 million (detemir) to USD 670 million (degludec) over five years.

The use of LAIA as a basal insulin regimen for DM1 may benefit more patients with recurrent episodes of hypoglycemia. However, the fragility of the outcomes considered to evaluate the clinical impact of LAIA and the high budget impact with its use should be considered, and may compromise SUS sustainability. In view of these aspects, CONITEC recommended the incorporation of one of the LAIA, if the treatment is equal to or less than that of NPH insulin and according to the criteria established by a guideline.

Recruitment of participants to, and their retention in, Randomized Controlled Trials (RCTs) is a key determinant of research efficiency, but is challenging. Digital tools and media are increasingly used to reduce costs, waste and delays in the conduct and delivery of research. The aim of this UK Clinical Trials Unit (CTU) survey was to identify which digital recruitment and retention tools are being used to support RCTs, their benefits and success characteristics.

A survey was sent to all UK Clinical Research Collaboration (UKCRC)-registered CTUs with a webinar to help increase completion. A logic model and definitions of a “digital tool” were developed by iterative refinement by project team members, the Advisory Board (NIHR Research Design service, NHS Trust, NIHR Clinical Research Networks and patient input) and CTUs.

A total of 24/52 (46%) CTUs responded, 6 (25%) of which stated no prior use. Database screening tools (e.g. CPRD, EMIS) were the tool most widely used (45%) for recruitment and were considered very effective (67%). The most mentioned success criteria were saving GP time and reaching more patients. Social media was second (27%), but estimated effectiveness varied considerably, with only 17% stating very effective. Fewer retention tools were used, with SMS / email reminders reported most (10/15 67%), but certainty about effectiveness varied. A detailed definition on what constitutes a digital tool with examples and a logic model showing relationships between the resources, activities, outputs and outcomes for digital tools was developed.

Database screening tools are the most commonly used digital tool for recruitment, with clear success criteria and certainty about effectiveness. Our detailed definition of what constitutes a digital tool, with examples, will inform the NIHR research community about choices and help them identify potential tools to support recruitment and retention.

Recruitment of participants and their retention in randomized controlled trials (RCTs) is key for research efficiency. However, for many trials, recruiting and retaining participants meeting the eligible criteria is extremely challenging. Digital tools are increasingly being used to identify, recruit and retain participants. While these tools are being used, there is a lack of quality evidence to determine their value in trial recruitment.

The aim of the main study was to identify the benefits and characteristics of innovative digital recruitment and retention tools for more efficient conduct of RCTs. Here we report on the qualitative data collected on the characteristics of digital tools required by trialists, research participants, primary care staff, research funders and Clinical Trials Units (CTUs) to judge them useful. A purposive sampling strategy was used to identify 16 participants from five stakeholder groups. A theoretical framework was informed from results of a survey with UKCRC registered CTUs. Semi-structured interviews were conducted and analysed using an inductive approach. A content and thematic analysis was used to explore the stakeholder's viewpoint and the value of digital tools.

The content analysis revealed that ‘barriers / challenges ‘ and ‘awareness of evidence’ were the most commonly discussed areas. Three key emergent themes were present across all groups: ‘security and legitimacy of information’, ‘inclusivity’, and ‘availability of human interaction’. Other themes focused on the engagement of stakeholders in their use and adoption of digital technology to enhance the recruitment/retention process. We also noted some interesting similarities and differences between practitioner and participant groups.

The key emergent themes clearly demonstrate the use of digital technology in the recruitment and retention of participants in trials. The challenge, however, is using these existing tools without sufficient evidence to support the usefulness compared to traditional techniques. This raises important questions around the potential value for future research.

The Evelina London Children's Hospital (ELCH) is undergoing a period of growth, including a new building planned to be completed within the next five years. Due to limited space and ambitions to be a state-of-the-art hospital, Horizon Scanning (HS) was considered important to ‘future-proof’ new facilities. As the aim of HS is to identify signals of coming change, ‘scanning’ the previous five years’ trends may be beneficial to an iterative HS methodology. Thus, it was thought that capital bids could provide a range of useful information required to make procurement decisions.

King's Technology Evaluation Centre (KiTEC) provided hospital-based HTA and HS support for the expansion of a London-based paediatric hospital. KiTEC focused on imaging technology due to its large spatial requirements and high-costs and assessed all capital bids made over the previous five years. A capital bidding system is used within GSTT to allocate funding for medical equipment that costs more than GBP5000 (USD 6540.70). Information was collated for all imaging equipment bid for over the previous five years and assessed for trends in imaging modalities and purchase costs.

A total of 135 bids were made in the period 2013-2018, eight of which were by ECLH. Bids for ultrasound equipment were most common and rose over the period. Bids for CT scanners also rose, while bids for MRI scanners and x-ray technology were consistent and bids for fluoroscopy fell. The total cost of imaging bids over the interval rose steadily from GBP5.4 million to GBP6.9 million.

Due to the lifespan of imaging technology, some trends may not emerge within a five year window. While some interesting findings were made, a ten to fifteen year period may require to be scanned for a robust analysis. This methodology is best applied in an iterative fashion along with standard HS techniques.

The MedicineWise app is a free consumer health and medicine management app developed by NPS MedicineWise. With 107,000+ downloads and 78,000+ active sessions per month, the MedicineWise app's core functions include: keeping track current medicines lists, medicine dose reminders and recording health conditions, allergies, test results and other health information. Recent enhancements also enabled the app to deliver featured health- and medicine-related content to users based on their medicines and/or health conditions. The goal is to maximise the MedicineWise app's capabilities by personalizing to users’ needs and combining with health professional interventions when needed, to encourage better delivery of health and medicines information and improve medication adherence and health outcomes.

A number of personalized medicines management service offerings were created by combining a technology solution using the MedicineWise app (including the app's core functions as well as added targeted content delivery capability) with a humanistic solution (a health professional-mediated phone-based coaching service). MedicineWise app features were developed iteratively using a human-centred design approach. Consumers were involved in the design, prototyping and testing stages before the features moved to technical-build stage.

Three use-cases will be presented to demonstrate how the MedicineWise app was used to provide personalized medicines management service offerings. These include: (1) curating relevant content and delivering push notifications to users for health conditions including asthma, rheumatoid arthritis and osteoporosis; (2) providing a triaged medication adherence support program with escalating levels of intervention for heart failure patients; and (3) collecting user-reported medication usage data and data monitoring by health professionals to provide support for heart failure patients.

The utility of MedicineWise app can be extended to provide personalized medicines management service offerings in the consumer health care space.

The CarbonCool full body suit is a portable, non-invasive core body cooling system for use by first responders and clinicians for targeted temperature management and heat stroke treatment. It uses pads made of a highly thermal conductive carbon-based cooling medium to absorb heat from the body. Our department was requested to review the effectiveness of the suit and whether it might be used in place of a Body Cooling Unit (BCU).

A rapid review was carried out on the technology. The PICO elements were: Population- Emergency department patients with exertional heat injury; Intervention- CarbonCool full body suit; Comparator- Body Cooling Unit; Outcomes- Adverse effects, clinical outcomes (survival, neurological status), physical measures (rate of cooling). The NHS Centre for Reviews & Dissemination databases, Cochrane Database of Systematic Reviews, PubMed (MEDLINE) and the US National Guidelines Clearinghouse were searched for systematic reviews, HTA reports and clinical practice guidelines. The importer was contacted to provide supporting studies for their product.

No publications were found on CarbonCool products. The importer provided the unpublished abstract of a retrospective cohort study of 124 post-cardiac arrest patients requiring targeted temperature management. The importer advised that a trial on pre-hospital heat stroke was pending. The full body suit is not intended as a replacement for body cooling units. Three clinical practice guidelines on management of heat injuries did not mention such cooling systems, but did recommend ice packs as a treatment option.

The CarbonCool Full Body Suit is not intended as a replacement for a Body Cooling Unit. No published studies were found showing effectiveness for managing exertional heat injuries. A trial on pre-hospital heat stroke was pending. Guidelines on managing heat injuries do not mention the use of the technology. It was recommended to await results of pending trials, or to use it only under research.

Good quality management (QM) and the sound application of EUnetHTA's (European network for Health Technology Assessment) well-established methodology and tools are fundamental prerequisites for reliable and trustworthy joint work. To provide ultimate support and guidance to the assessment teams of EUnetHTA – and further, to ensure a sustainable mode of work for the post-2020 period – a comprehensive web-based so called “EUnetHTA Companion Guide” is established in Joint Action 3.

The Companion Guide was created using the open source Wiki software “DokuWiki”. We divided the content into five main parts: 1. EUnetHTA's QM concept, 2. QM for rapid Relative Effectiveness Assessment (REA) Pharma, 3. QM for rapid REA Other Technologies (OT) 4. Scientific Guidance & Tools and 5. QMS-related training material. The assessment processes for rapid REA Pharma and OT have been subdivided into single process steps for which the Companion Guide provides standard operating procedures (SOPs), checklists, templates, guidelines and tools. The content of the Companion Guide is continuously subjected to evaluation by means of a structured survey with regard to the achievement of its purpose of ensuring to ensure high-quality HTA reports.

In May 2018, the Companion Guide was launched and is now available to all EUnetHTA partners. It provides central access to all components of the newly established QMquality management system for EUnetHTA. The user has access to training modules that provide information on how to use the Companion Guide. Moreover, the training material enables EUnetHTA partners to build up necessary capabilities for QMquality management, and application of methodologies and tools in the context of EUnetHTA.

The purpose of the Companion Guide is to ensure the production of high-quality HTA reports by providing ultimate support and guidance to the EUnetHTA assessment teams during their joint work. The continuous evaluation will reveal necessary revisions and the need for further developments and guidance.