bluuebunny/crossref_metadata_2025_split huggingface dataset:
PP171 Cost And Effectiveness Of Chronic Hepatitis C Treatment In Brazil
Authors: Schwambach Karin Hepp , Farias Mareni Rocha , Neto Giacomo Balbinotto , Blatt Carine Raquel 
Published: December 2019

IntroductionWith the discovery of new direct-acting antivirals, the cure for hepatitis C appears to be a reality, but its high price and the availability of new antivirals are a major obstacle. In Brazil, treatments for hepatitis C have been available in the public health system since the 1990s, and in 2015 were made available the antivirals sofosbuvir, daclatasvir and simeprevir. The calculation of the budgetary impact of this merger estimated expenditures between 467 and 666 million Reais (USD 121 and 172 million) per year. This study aims to present and discuss the cost and effectiveness of hepatitis C treatment with direct-acting antivirals with or without alfapeginterferon and ribavirin, based on real-life data, and compare it with the world scenario.MethodsWe analyzed the treatment data and outcomes of 253 patients from a retrospective cohort performed in a Specialized Care Service, in the city of Porto Alegre. In relation to costs, the direct costs of antiviral drugs, per unit (tablet), were considered according to financial receipts from public purchases. The total cost of the medications used by each individual in each treatment and the cost per cure obtained, expressed in Sustained Viral Response (SVR), were calculated.ResultsMost patients (66.8 percent) had genotype 1 of the hepatitis virus and 92.9 percent achieved SVR. The mean total cost of treatment of patients with genotype 1 was USD 5,862.31 and USD 6,310.34 per cure; while in patients with genotype 3 the cost was USD 5,144.27 and USD 5,974.76 per cure. The cost with the most commonly used treatment regimen, sofosbuvir, daclatasvir and ribavirin was USD 5,961.25 and USD 6,536.46 per cure. These values were 30 percent lower than the values estimated at the time of drug incorporation.ConclusionsCost and effectiveness data contextualize a real-life scenario in Brazil. The evaluated treatments presented good effectiveness, but high costs.
PP185 Clinical Papers: Which Are Ongoing Studies To Assess MHealth In 2020?
Authors: Olivier Cyril , Piotto Estelle , Collignon Corinne , Galmiche Hubert , Bélorgey Chantal 
Published: December 2019

IntroductionMobile health systems (MHS) are one of the more spreading technologies in the field of medicine. However, identification of useful MHS is rather challenging. Few of them are, or could be, connected medical devices (cMD). Like other medical devices, cMD must be assessed to validate claimed benefits for reimbursement purposes. Clinical added value demonstration is a major criterion used to satisfy administrative requirements. With the increase of clinical studies that are including MHS, study registries can be used for insight into the type of evidence expected to become available in the near future.MethodsIn 2018, the French National Authority for Health (HAS) performed a review of registered MHS clinical study designs. The Clinicaltrials.gov database was consulted for all studies indexed with the terms “mHealth” and “mobile health” for the search fields “study title”, “conditions” and “interventions”.ResultsFour hundred and fifteen clinical studies were registered. Three hundred and eighty studies were interventional with most comprised of a randomized study design (75 percent). Fifteen had a crossover design. Only few observational studies (n = 35) were registered. These mainly concerned (59 percent) patient use of an app on a smartphone without any other device.ConclusionsPatterns of clinical studies were not found to significantly differ between MHS and other medical devices. Most of the clinical studies were randomized and specific criteria to assess MHS could easily be identified. However, specific methodologies for clinical development are not used in practice for cMD health technology assessment. In the absence of validated and specific methodology for clinical development, current methods that are being used in these ongoing studies will nonetheless be generating evidence for the upcoming years.
VP31 Searching Non-English Literature For HTA Reports May Be Unnecessary
Authors: Schell Lisa , Hausner Elke , Rodenhäuser Lina , Assall Oliver , Schulz Anke , Sieben Wiebke , van der Leck Kerstin , Sauerland Stefan 
Published: December 2019

IntroductionCurrently, 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).MethodsWe 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.ResultsOf 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).ConclusionsOnly 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.
VP32 Incorporation Of The Only Drug For Primary Biliary Cholangitis Brazil
Authors: Silva Thales 
Published: December 2019

IntroductionPrimary 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.MethodsScientific 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.ResultsTen 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.ConclusionsDespite 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
VP46 German Claims Data In Rare Disease HTA: Diffuse Large B-cell Lymphoma
Authors: Neubauer Aljoscha , Guthoff-Hagen Susanne , Menzler Jacob , Schwenke Carsten , Rueckert Markus , Boehnke Axel 
Published: December 2019

IntroductionIn 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.MethodsClaims 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).ResultsMean 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).ConclusionsDespite 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.
VP63 EUnetHTA Planned And Ongoing Projects Database: Usage And Challenges
Authors: Erdos Judit , Chalon Patrice 
Published: December 2019

IntroductionThe European Network for Health Technology Assessment (EUnetHTA) Planned and Ongoing Projects (POP) database allows sharing information on projects of HTA organisations participating in EUnetHTA. It enables users identifying overlaps and therefore has the potential to reduce duplication of work on similar topics. The aim of our research was to examine the usage of the database, how it helps to reduce duplication, and identify challenges to be addressed.MethodsWe conducted a systematic data collection and analysis on the topic overlaps retrospectively and complemented it with an electronic survey aimed at database users. The dataset on the overlaps was collected between June 2016 and September 2017. The survey was conducted in October 2017.ResultsDuring the data collection period, the POP database contained on average 800 projects provided by around fifty percent of the EUnetHTA partner organisations. One hundred and sixty nine identical projects could be identified from which ninety-two percent had different starting dates. Twenty-five percent of the identical projects were elaborated on by three organisations, 10 percent by four, and 6.5 percent by five organisations. Analysis of the survey showed a pattern of “wait and see”: users are informed about what other EUnetHTA partners are working on, wait for a finalised HTA or exchange the project plan, search strategies, search results, extraction tables, etc. The data collection and survey could not provide a precise number of collaborations initiated based on information gathered from the database.ConclusionsThe POP database is suitable for information sharing and has the potential to save time and resources at EUnetHTA partners. The collaboration is hindered by differences in national processes, including the timing and scope of the assessments. The impact of the POP database on the facilitation of collaboration and reduction of duplication of HTAs produced by EUnetHTA partners is yet to be strengthened.
VP71 Barriers To Access Biologic Products: A Rapid Review
Authors: Silva Roberta Borges , Farinasso Cecilia , Rego Daniela , Gomes Dalila Fernandes , de Aguiar Aurelina Aguiar , Leite Betânia , Goncalves Lenilson , Simões Luciana , Leão Camara , Sachetti Camile Giaretta 
Published: December 2019

IntroductionThe elevated costs with biologic products threaten the sustainability of health services, and, therefore, the access to these medicines in the perspectives of user, health professional, health manager and system. The entry of biosimilar products in the market could be an option to subsidize the search for solutions to those problems.MethodsWe conducted a rapid review using the databases Medline (via PubMed), EMBASE, Cochrane Library and CRD. The eligibility criteria were HTAs, systematic reviews and cross-sectional studies.ResultsLiterature search retrieved 640 registries and, after duplicate removal, screening of titles and abstracts and full text reading, nine cross-sectional studies were selected. From a user's point of view, the following barriers were identified: lack of knowledge about the medicine, distance between the place of living and the health service (especially in the rural area), long waiting periods for service, passivity in regard to treatment. From a health professional's point of view the barriers were: acceptability of the expert in regard to treatment, interchangeability and substitution, the perception of lack of data showing efficacy and safety. Finally, from the payer's (or health manager) point of view, the barriers were: high cost of medicine, problems with reimbursement and bureaucracy. We did not retrieve any barriers from the health system's perspective from the selected studies.ConclusionsThe entry of biosimilar medicines in the market can induce competition and, therefore, reduce prices of biologic treatments. It is necessary to search for potential solutions to the access barriers identified in this rapid review.
VP72 Impact Of Comparator Choice On Oncology Drugs’ Market Access
Authors: Marfin Henri , Grande Mathilde , Thuillez Christian , d'Andon Anne 
Published: December 2019

IntroductionIn France, drug assessment is performed by the Transparency Committee (TC) of the French National Authority for Health (HAS). It's based on two criteria: the clinical benefit (CB) for reimbursement recommendation and the clinical added value (CAV) serving the pricing decision. The CAV is rated on a 5-point scale, from I (major) to V (no CAV). A critical step in the CAV assessment is the identification of the clinically relevant comparators (CRC) serving the TC to recognize the appropriateness of the comparators chosen in the randomized controlled trials (RCT). The objective of this study is to investigate the comparator choice consequences on TC appraisals and pricing.MethodsA retrospective, descriptive study included all oncology indications assessed by the TC between 2015 and 2017. Based on a pre-specified grid, items on the comparators were extracted from final TC's appraisals.ResultsAmong the 135 indications included, the assessed drugs had no CRC in 20% of cases. A RCT was submitted for 89 indications (66%) whose 67 (76%) were conducted versus a CRC. A CRC was identified by the TC for 70% of the 46 indications without RCT. An important/moderate CAV (II-III) was granted when there was a RCT versus a CRC in 70% of cases, versus 50% and 43% for minor (IV) and no CAV respectively. The public price was reduced by 13.5% in average compared to the claimed price without impact of the CAV level (n = 18).ConclusionsIn oncology, comparative data assessed by the TC met its expectations (RCT versus CRC) in a majority of cases. When there is no RCT or a comparison versus a non-relevant comparator the CAV appraisal is decreased. Surprisingly this study hasn't shown any impact of this decrease on the public price. A wider analysis in different medical areas would need to be performed to better investigate these results.
Seasonality of the transmissibility of hand, foot and mouth disease: a modelling study in Xiamen City, China
Authors: Huang Zehong , Wang Mingzhai , Qiu Luxia , Wang Ning , Zhao Zeyu , Rui Jia , Wang Yao , Liu Xingchun , Hannah Mikah Ngwanguong , Zhao Benhua , Su Yanhua , Zhao Bin , Chen Tianmu 
Published: December 2019

Abstract
This study attempts to figure out the seasonality of the transmissibility of hand, foot and mouth disease (HFMD). A mathematical model was established to calculate the transmissibility based on the reported data for HFMD in Xiamen City, China from 2014 to 2018. The transmissibility was measured by effective reproduction number (Reff) in order to evaluate the seasonal characteristics of HFMD. A total of 43 659 HFMD cases were reported in Xiamen, for the period 2014 to 2018. The median of annual incidence was 221.87 per 100 000 persons (range: 167.98/100,000–283.34/100 000). The reported data had a great fitting effect with the model (R2 = 0.9212, P < 0.0001), it has been shown that there are two epidemic peaks of HFMD in Xiamen every year. Both incidence and effective reproduction number had seasonal characteristics. The peak of incidence, 1–2 months later than the effective reproduction number, occurred in Summer and Autumn, that is, June and October each year. Both the incidence and transmissibility of HFMD have obvious seasonal characteristics, and two annual epidemic peaks as well. The peak of incidence is 1–2 months later than Reff.
Phase connected X-ray light curve and He II radial velocity measurements of NGC 300 X-1
Authors: Carpano S. , Haberl F. , Crowther P. , Pollock A. 
Published: December 2019

Abstract. NGC 300 X-1 and IC 10 X-1 are currently the only two robust extragalactic candidates for being Wolf-Rayet/black hole X-ray binaries, the Galactic analogue being Cyg X-3. These systems are believed to be a late product of high-mass X-ray binary evolution and direct progenitors of black hole mergers. From the analysis of Swift data, the orbital period of NGC 300 X-1 was found to be 32.8 h. We here merge the full set of existing data of NGC 300 X-1, using XMM-Newton, Chandra and Swift observations to derive a more precise value of the orbital period of 32.7932 ± 0.0029 h above a confidence level of 99.99%. This allows us to phase connect the X-ray light curve of the source with radial velocity measurements of He II lines performed in 2010. We show that, as for IC 10 X-1 and Cyg X-3, the X-ray eclipse corresponds to maximum of the blueshift of the He II lines, instead of the expected zero velocity. This indicates that for NGC 300 X-1 as well, the wind of the WR star is completely ionised by the black hole radiation and that the emission lines come from the region of the WR star that is in the shadow. We also present for the first time the light curve of two recent very long XMM-Newton observations of the source, performed on the 16th to 20th of December 2016.
Black hole high mass X-ray binary microquasars at cosmic dawn
Authors: Mirabel I. F. 
Published: December 2019

AbstractTheoretical models and observations suggest that primordial Stellar Black Holes (Pop-III-BHs) were prolifically formed in HMXBs, which are powerful relativistic jet sources of synchrotron radiation called Microquasars (MQs).Large populations of BH-HMXB-MQs at cosmic dawn produce a smooth synchrotron cosmic radio background (CRB) that could account for the excess amplitude of atomic hydrogen absorption at z∼17, recently reported by EDGES.BH-HMXB-MQs at cosmic dawn precede supernovae, neutron stars and dust. BH-HMXB-MQs promptly inject into the IGM hard X-rays and relativistic jets, which overtake the slowly expanding HII regions ionized by progenitor Pop-III stars, heating and partially ionizing the IGM over larger distance scales.BH-HMXBs are channels for the formation of Binary-Black-Holes (BBHs). The large masses of BBHs detected by gravitational waves, relative to the masses of BHs detected by X-rays, and the high rates of BBH-mergers, are consistent with high formation rates of BH-HMXBs and BBHs in the early universe.
Facilitating Respite, Communication, and Care for Children With Intellectual and Developmental Disabilities: Preliminary Evaluation of the Caregiver Pain Information Guide
Authors: Genik Lara M. , Millett Geneva E. , McMurtry C. Meghan 
Published: December 2019

Background: Pain is common in children with intellectual and developmental disabilities (I/DD) and yet is an understudied issue. Respite workers commonly care for children with I/DD but are lacking in resources to assist in pain assessment and management for this population. Without adequate knowledge in this domain, children with I/DD are at risk of their pain being underrecognized and undermanaged. A resource for caregivers was developed to address this issue, facilitating communication between parents and respite workers and better addressing the complex pain-related needs of children with I/DD. The objective of this study was to solicit initial feedback on the content, feasibility, and usability of the resource. Method/Procedure: Semistructured individual interviews and questionnaires were completed with 7 parents of children with I/DD and 6 respite workers. Results: All participants viewed the resource as important and potentially useful. Content analyses indicated that participants appreciated the comprehensiveness and format of the resource, whereas the modifiability was identified as an area for improvement. Participants believed that the resource would be useful across settings with a variety of caregivers. Three categories of considerations were identified as potential facilitators and barriers for implementation: consideration of how the resource is being completed, who is completing it, and organization-based procedures. Discussion/Conclusions: Results from this study suggest that the Caregiver Pain Information Guide is a promising resource for helping respite workers better understand pain in children with I/DD. Future work is needed on implementation and impact of this resource in community settings. Implications for Impact Statement Children with intellectual and developmental disabilities experience pain frequently and are at risk of having their pain missed by caregivers. Parents would like to share pain-related knowledge and skills specific to their children with intellectual and developmental disabilities with those who support their child in other settings. The Caregiver Pain Information Guide was created to facilitate communication between parents and caregivers of these children. The current article discusses initial feedback from parents and respite workers regarding this new tool.