bluuebunny/crossref_metadata_2025_split huggingface dataset:
OP178 Assessing Digitally Enabled Therapies: Challenges And Opportunities
Authors: Rowark Shaun , Stephens Heather 
Published: December 2020

IntroductionIn 2017, the National Institute for Health and Care Excellence (NICE) and the National Health Service England established a program to identify Digitally Enabled Therapies (DET) that increase access to Improving Access to Psychological Therapies (IAPT) services. The aim was to determine whether DETs could improve service efficiency, and whether outcomes are at least as good as those achieved by NICE-recommended non-digital therapies.MethodsAn IAPT assessment briefing (IAB) was developed for each eligible DET. IABs included an assessment of content, technical standards, clinical effectiveness, and cost and resource impact. IABs were reviewed by the NICE IAPT expert panel to decide whether a DET is suitable for evaluation in IAPT services, needs further development, or is not suitable. Suitable DETs were evaluated for up to two years.ResultsOf 154 DETs reviewed by the program, fourteen had IAB assessments. The high dropout rate was due to ineligible products or developer withdrawal. Of the fourteen IABs, five were recommended for evaluation, one was recommended for development, and eight were not recommended.ConclusionsDETs can provide an alternative for patients who may not be able to access treatment. When establishing programs to review DETs, centers must consider the quality of the products submitted and, where necessary, make pragmatic decisions about assessment criteria.
OP179 Nationwide Electroencephalographic Screening Using Telemedicine Apps
Authors: Galvan Pedro , Rivas Ronald , Arbo Carlos , Cabrera Marta , Abente Silvia , Portillo Juan , Mazzoleni Julio , Hilario Enrique 
Published: December 2020

IntroductionDisruptive telemedicine tools can help overcome the lack of specialized care and electroencephalographic (EEG) support for diagnosing and treating nervous system disorders such as epilepsy in remote communities. However, evidence on how such cloud-based platforms could enhance data-driven health care is limited. The utility of telemedicine-based apps to achieve EEG screening of communities in rural areas of Paraguay was investigated.MethodsThis descriptive study was carried out by the Telemedicine Unit of the Ministry of Public Health in collaboration with the Department of Biomedical Engineering and Imaging of the Health Science Research Institute in Paraguay and the Basque Country University in Spain to evaluate the utility of telediagnostic apps for EEG screening. For this purpose, the results obtained by tele-EEG apps implemented in nineteen public community hospitals were analyzed to determine the utility of the apps as epidemiological surveillance tools.ResultsAmong the 10,791 remote EEG studies performed, the most common reasons for the test included epileptic seizure (44%), headache (22%), seizure disorder (8%), follow up (6%), attention deficits in children (5%), cognitive impairment (4%), cranioencephalic trauma (3%), brain death (1%), history of seizure (0.9%), abnormal movements (0.7%), and behavioral disorders (0.5%).ConclusionsThe results showed that telemedicine apps can significantly enhance nationwide EEG screening by freeing up professional time and increasing productivity, improving access and equity, and reducing costs. However, before their systematic implementation a contextualization of the apps using the regional epidemiological profile must be performed.
OP283 A Pipeline Analysis Of Advanced Therapy Medicinal Products (ATMPs) In Late-Stage Clinical Development
Authors: Woltmann James , Teague Rhiannon , Ogunbayo Dapo , Craig Dawn 
Published: December 2020

IntroductionAdvanced Therapy Medicinal Products (ATMPs) are innovative biologics (gene, cells and tissue-based products) with the potential to treat diseases with significant unmet clinical need. ATMPs pose distinct regulatory, health technology assessment (HTA) and patient access challenges, hence early identification and prioritization of ATMPs is now recognized as a key concern in England. The National Institute for Health Research Innovation Observatory (NIHRIO) uses a robust methodology to identify and monitor health technologies, including ATMPs that meet the remit of key HTA stakeholders in England. This analysis provides a global overview of the current ATMPs pipeline to administer useful insights for policymakers, funders and innovators.MethodsNIHRIO's database tracks pharmaceuticals from phase I/II onwards, but this analysis focuses on late-stage development. The database (N > 12,000 records) was filtered to identify potential ATMPs using a predefined criteria based on the European Medicine's Agency's classification. Each record is categorized by stage: ‘Active’, (with an estimated three years to European licence); ‘Monitoring’ (in development with no licence date); and ‘Finished’, (output produced/discontinued and no longer tracked). Subsequently, records in ‘Active’ and ‘Monitoring’ were examined further.ResultsAnalysis identified 636 ATMPs: five percent ‘Active’, 40 percent ‘Monitoring’ and 55 percent ‘Finished’. ATMPs in the Active/Monitoring stages included: gene therapies (52%), somatic cells (43%) and tissue-engineered products (5%). Of these, 40 percent were oncological with the majority targeting hematological cancers (lymphomas). Prevalent non-oncology areas included musculoskeletal (10%) and ophthalmology (8%). Over one-third of trials were phase IIs, with almost half of all trials were based in the US.ConclusionsThe overarching findings here indicate increasing development of the ATMP pipeline towards indications with significant unmet clinical need. In oncology, the high prevalence of hematological ATMPs is largely due to recent chimeric antigen receptor T cells (CAR-T) innovation. In non-oncology areas, ATMP development is increasing due to advances in regenerative medicine. With a significant number of ATMPs projected to be licenced within three years, and many more in active late-stage trials, HTA bodies and health systems are challenged to prepare for the entry of these innovative therapies.
OP354 Cost-Effectiveness Analysis Of Different Prenatal Screening Strategies For Down Syndrome In China: Data From Shandong Province
Authors: Du Yanqiu , Shang Wenru , Huang Jiayan 
Published: December 2020

IntroductionThere are large differences between the prenatal screening strategies for Down Syndrome (DS) in different provinces in China. In Henan province there is a serological triple screening in the second trimester (STS) strategy, while in Shandong province contingent non-invasive prenatal testing (NIPT) screening strategy (NIPT delivered to older pregnant women) is used, and there is a universal NIPT screening strategy in Anhui province. Moreover, many factors varied widely in different regions, such as the proportion of older pregnant woman and the ability of people to pay. This study aimed to determine the cost-effectiveness of current strategy in Shandong compared with strategies in other provinces.MethodsA decision tree model was developed according to the screening strategies in different provinces. Four screening strategies were involved, universal STS strategy, contingent STS strategy, contingent NIPT strategy, and universal NIPT strategy. Cost-effectiveness analysis was conducted from a societal perspective in a simulated cohort of 100,000 pregnant women. The data of costs and epidemiologic parameters were collected from field surveys in Shangdong and a literature review.ResultsThe universal STS strategy, contingent STS strategy, contingent NIPT strategy, and universal NIPT strategy could prevent 17.0, 40.0, 46.2, and 53.6 DS births, respectively. There was no strategy dominated by others. The universal NIPT strategy and contingent NIPT strategy would decrease invasive procedures for prenatal diagnosis, resulting in fewer procedure-related miscarriages. The sensitivity analysis showed that the effectiveness of the screening strategy is significantly influenced by the resident's acceptance of NIPT.ConclusionsFrom the perspective of maximizing the effect, the universal NIPT strategy is the optimal strategy. But taking into account the resident's and government's ability to pay, contingent NIPT Strategy may be appropriate for the current situation in Shandong. To ensure a better cost-effective advantage in the universal NIPT strategy, the government should provide health education to increase the acceptance of NIPT while controlling the price of NIPT.
OP457 A Collaborative Horizon Scanning Alert For Disinvestment And Early Awareness
Authors: Lauvrak Vigdis , Farrah Kelly , Esmail Rosmin , Espeland Anna Lien , Hafstad Elisabet , Harboe Ingrid , Ohm Ingrid Kristine , Migliore Antonio , Topfer Leigh-Ann 
Published: December 2020

IntroductionIn 2019, the Norwegian Institute for Public Health and Canadian Agency for Drugs and Technologies in Health (CADTH) received support from HTAi to produce a quarterly current awareness alert for the HTAi Disinvestment and Early Awareness Interest Group in collaboration with the HTAi Information Retrieval Interest Group. The alert focuses on methods and topical issues, and broader forecasts of potentially disruptive technologies that may be of interest to those involved in horizon scanning and disinvestment initiatives in health technology assessment (HTA).MethodsInformation specialists at both agencies developed search strategies for disinvestment and for horizon scanning in PubMed and Google. The template for the alert was based on an e-newsletter developed by the Information Retrieval Interest Group. Information specialists and researchers reviewed the monthly (PubMed) and weekly (Google) search results and selected potentially relevant publications. Additional sources were also identified through regular HTA and horizon scanning work.ResultsAlerts are posted quarterly on the HTAi Interest Group website; members receive an email notice when new alerts are available. While the revised PubMed searches are identifying relevant information, Google alerts have been disappointing, and this search may need to be revised further or dropped. When the one-year pilot project ends, in Fall 2020, interest group members will be surveyed to see if the alerts were useful, and whether they have suggestions for improving them.ConclusionsCollaborating on this alert service reduces duplication of effort between agencies, and makes new research in horizon scanning and disinvestment more accessible to colleagues in other agencies working in these areas.
OP520 Evaluating Long-Term Survival From Clinical Trials: Does Real-World Evidence Change the Paradigm?
Authors: Wang Xiaoliang , Adamson Blythe J. , Tan Katherine , Baxi Shrujal , Briggs Andrew , Ramsay Scott D. 
Published: December 2020

IntroductionBoth patient composition and medical care received in clinical trials may not be representative of clinical practice, yet health technology assessments (HTAs) commonly use extrapolation results from trials to estimate incremental benefit. Due to data limitations, external validation of trial extrapolations are uncommon. With the goal of better estimating the benefit of new therapies in practice, we compared long-term survival estimated from real-world patients who received therapy similar to the comparator arm of the OAK trial, a phase III study of patients with advanced non-small cell lung cancer (aNSCLC) who progressed following initial chemotherapy, to standard estimation approaches.MethodsWe estimated long-term survival from: (i) direct extrapolation of trial survival curves; and (ii) aNSCLC patients from the United States Flatiron Health Electronic Health Record ()-derived de-identified database diagnosed between January 2011 and August 2019 who received docetaxel monotherapy after platinum-doublet and had adequate organ function as well as functional status. Patients with unknown organ function and functional status were also included. Standard parametric extrapolations were applied and selected based on visual inspection and goodness-of-fit tests for each cohort.ResultsUsing a log-logistic model to extrapolate the trial comparator arm (N = 425), estimated lifetime mean overall survival was 19.2 months (95% confidence interval [95% CI]: 16.5–22.6), and 14.4 months (95% CI: 12.4–17.0) for the real-world cohort (N = 415). Estimated 5-year overall survival rates were 5.4 percent (95% CI: 3.9–7.3) for the trial patients, compared to 3.7 percent (95% CI: 2.6–5.0) among real-world cohort patients.ConclusionsOur results suggest that directly extrapolating observed survival for trial patients may overestimate the long-term survival compared to the experience of patients treated in routine practice. Our findings have implications for those wishing to estimate the incremental benefit for novel versus established treatments. We plan to compare our results to a generic patient cohort from national cancer registry. Further EHR-based studies utilizing real world data are needed to confirm our findings and to extend beyond this use case for other cancer types and anti-neoplastic therapies.
PP154 Funding Of Treatments For Rare Diseases In Singapore
Authors: Pearce Fiona , Lin Liang , Ng Kwong 
Published: December 2020

IntroductionA national multi-stakeholder charity fund has been established in Singapore to provide targeted support to patients with rare genetic diseases whose treatment costs remain unaffordable despite government subsidies and insurance. This presentation will provide an overview of the evaluation, price-setting, and stakeholder engagement processes established to inform the first list of drugs eligible for funding under the Rare Disease Fund (RDF).MethodsThe local prevalence of “rare” and “ultra-rare” conditions was defined in line with international rates (≤4 in 10,000 and <2 in 50,000, respectively) to facilitate an analysis of the rare disease landscape in Singapore, and to identify patients most likely to benefit from the RDF. Public healthcare institutions proposed drugs for consideration, which underwent technical evaluation and were then assessed in line with eligibility criteria by an expert clinical group and prioritized by decision makers for funding.ResultsThe number of patients with select rare diseases in Singapore was lower than global estimates contextualized to the local setting. Supporting clinical evidence, funding decisions from overseas health technology assessment agencies, reference pricing considerations, and local budget impact analyses informed the first tranche of drugs (n = 5) recommended. Extensive engagement with pharmaceutical companies was needed to negotiate fair drug prices relative to overseas countries. Additional treatments will be included in the RDF once sufficient funds are raised.ConclusionsAs the evaluation process evolves, wider considerations of disease and treatment experiences from a multi-stakeholder standpoint should be included to inform RDF listings. There is also a need to balance the sustainability of the fund in the longer term with the number of emerging treatments that may require coverage in the future.
PP164 Prescribed Medication Use, Complications, And Cost of Type 2 Diabetes Mellitus In The Real World
Authors: Chen Shengqi , Ung Carolina Oi Lam , Hu Hao 
Published: December 2020

IntroductionType 2 diabetes mellitus (T2DM) is one of the major diseases threatening the health of Chinese residents. Insufficient glycemic control results in inevitable and sometimes irreversible complications. The condition can be more complicated for patients with comorbidities. To maintain appropriate glycemic control, patients may need to add or switch to different classes of drugs as the disease progresses. The complicated drug treatment for diabetes poses a high risk for drug-drug interactions and challenges patient compliance, which is the most important factor affecting the ability to achieve glycemic control. In addition, T2DM is a life-long disease that is associated with a heavy economic burden for patients’ families and for society. Therefore, this paper aims to characterize the complexity of prescribed medications used to treat T2DM and to assess the trends in the number of complications, medications used, and costs incurred among these patients.MethodsData were retrieved from two tertiary hospitals. The population consisted of patients receiving at least one diagnosis of diabetes (International Classification of Diseases-10 codes: E11, E12, E13, and E14) between October 2007 and May 2017. Three measures were assessed, including the number of patients with different complications, concomitant drug use, and costs. Patients with a short disease duration (< 5 years) were compared with patients who had a longer disease duration (≥ 5 years).ResultsOf 31,071 patients, about half of those with a longer disease duration had at least five concomitant diseases, compared with nine percent of patients with a shorter disease duration. The maximum number of concomitant diseases was nine for both disease duration groups. Patients with longer disease duration were more likely to use multiple classes of drugs, compared with patients in the short disease duration group. The average annual medical costs for patients without concomitant disease was CNY 1,894 (USD 265).ConclusionsOverall, the results demonstrated that T2DM is a relatively complex disease. Firstly, patients have up to nine concomitant diseases and use twenty-two classes of drugs. Secondly, patients with more complications tended to use more medications. Thirdly, patients with a longer disease duration tend to have a higher number of concomitant diseases, use more classes of drugs, and have higher medical costs than patients with a shorter disease duration.
PP181 Direct Comparison Of The Effectiveness And Safety Of Apixaban, Dabigatran, Rivaroxaban, And Warfarin: Subgroup Analyses Of Medicare Beneficiaries
Authors: Yang Lanting , Brooks Maria M. , Glynn Nancy W. , Zhang Yuting , Saba Samir , Hernandez Inmaculada 
Published: December 2020

IntroductionNo studies have directly compared the effectiveness and safety of direct oral anticoagulants (DOACs) and warfarin in patients with atrial fibrillation (AF), with or without a history of ischemic stroke and transient ischemic attack (TIA). This is important for two reasons: first, previous research reports important differences between DOACs and warfarin across other patient subgroups, and second, patients with previous stroke or TIA have a high risk of recurrent stroke.MethodsUsing 2012–2014 Medicare claims data, we identified patients newly diagnosed with AF in 2013–014 who started taking apixaban, dabigatran, rivaroxaban, or warfarin. We categorized the patients according to whether they had a history of stroke or TIA. We constructed Cox proportional hazard models that included indicator variables for treatment groups, a history of stroke or TIA, and the interaction between them, and controlled for demographic and clinical characteristics.ResultsThe hazard ratio (HR) for stroke with dabigatran, compared with warfarin, was 0.64 (95% confidence interval [CI]: 0.48–0.85) for patients with a history of stroke or TIA and 0.94 (95% CI: 0.75–1.16) for patients without a history of stroke or TIA (p-value for interaction = 0.034). In patients with previous stroke or TIA, the risk of stroke was lower with dabigatran (HR 0.64, 95% CI: 0.48–0.85) and rivaroxaban (HR 0.70, 95%CI: 0.56–0.87), compared with apixaban, but there was no difference for patients in the other subgroup.ConclusionsDOACs were generally more effective than warfarin for preventing stroke. The superiority of dabigatran was more pronounced in patients with a history of stroke or TIA. The comparative effectiveness of DOACs differed substantially between patients with and without a history of stroke or TIA; specifically, apixaban was less effective in patients with a history of stroke or TIA. Our results reinforce the need to tailor anticoagulation to patient characteristics and to support the investigation of the underlying mechanisms associated with DOACs.
PP185 Oral Health Status And Food Consumption Patterns In Selected Primary School Children
Authors: Islam Nabhira Aftabi Binte , Haque Mahmudul 
Published: December 2020

IntroductionNutrition is critical to the oral health of the individual. From gestation through to end of life, nutrition influences the integrity and function of the dentition and supporting oral structures and has a direct effect on health in general. According to the World Health Organization, diet has an important role in the prevention of oral diseases such as dental caries, dental erosion, defects in oral development, diseases of the oral mucosa, and periodontal disease.MethodsA study was conducted to assess the oral health status and food consumption patterns of students attending the Rotary School and College at Mirpur-14, Dhaka on November 2018. Consent was provided by the school headmaster and guardians. A purposive sample of seventy students was taken. A semi-structured questionnaire and checklist was developed in the English and Bengali languages. Data were presented in simple frequency tables.ResultsAmong the respondents the following eating habits were reported:
(i)Thirty-one percent drank milk and twenty-eight percent consumed chocolate four to six times per week;(ii)Forty-three percent consumed fast food least seven times per week; and(iii)Forty-two percent ate vegetables and thirty-four percent ate fruits one to three times per week.In terms of oral hygiene practices, sixty-one percent of respondents used toothpaste and thirty-nine percent used tooth powder. Seventy-eight percent of respondents brushed their teeth once a day and twenty-four percent brushed twice daily. The mean number of decayed, missing (due to caries), and filled permanent teeth was 1.47.ConclusionsThe relationship between oral health, dietary practices, nutritional status, and general health is complex, with many interrelated factors. To help children develop healthy eating patterns from an early age, it is important that the food and eating patterns to which they are exposed, both inside and outside the home, promote positive attitudes to good nutrition.
PP193 Research On The Correlation Mechanism Between The Psychological Contract Of Physicians In Public Hospitals And Their Medical Professionalism
Authors: Lyu Chao , Zhou Ping 
Published: December 2020

IntroductionGuiding and motivating doctors' attitudes and behaviors in the public welfare of public hospitals is the key to promoting medical reform. This study explored the correlation between the hospital responsibilities perceived by physicians and their medical professionalism practices in Chinese public hospitals, and the moderating effects of variables such as sex, hospital level, working period, department, and reason for career choice on the correlation model.MethodsA pre-developed, reliable, and valid scale for the physicians’ psychological contract was developed. Survey data from 123 public hospitals in three provinces in the east, central, and west areas of China were analyzed. The authors constructed and tested the correlation mechanism model and then analyzed the effects of particular variables.ResultsThe fulfillment of hospital ideological responsibility has a significant positive effect on physicians' medical professionalism practices, with occupational satisfaction mediating the effect. In addition, hospital transactional, developmental, and relational responsibility can improve physicians’ medical professionalism practices through fairness perception and work pleasure. Hospital level, working period, department, and reason for career choice had a moderating effect on the correlation mechanism model, with the effects varying between different paths.ConclusionsThe fulfillment of hospital responsibilities can have a positive effect on physicians’ practices of medical professionalism, especially in the domain of ideological responsibility where the largest and most direct impact was observed. Furthermore, the results suggested that hospital administrators need to consider the differences between various types of physicians in order to improve the effects of guidance and motivation in these health professionals.