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Drug Safety
Journal Prestige (SJR): 1.447
Citation Impact (citeScore): 3
Number of Followers: 169  
 
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ISSN (Print) 0114-5916 - ISSN (Online) 1179-1942
Published by Adis Homepage  [21 journals]
  • Comment on: “Patient Registries: An Underused Resource for Medicines
           Evaluation: Operational Proposals for Increasing the Use of Patient
           Registries in Regulatory Assessments”
    • PubDate: 2019-12-01
       
  • Authors’ Reply to Ravi Jandhyala’s Comment on “Patient Registries:
           An Underused Resource for Medicines Evaluation: Operational Proposals for
           Increasing the Use of Patient Registries in Regulatory Assessments”
    • PubDate: 2019-12-01
       
  • Recommendations for the Use of Social Media in Pharmacovigilance: Lessons
           from IMI WEB-RADR
    • Abstract: Abstract Over a period of 3 years, the European Union’s Innovative Medicines Initiative WEB-RADR project has explored the value of social media (i.e., information exchanged through the internet, typically via online social networks) for identifying adverse events as well as for safety signal detection. Many patients and clinicians have taken to social media to discuss their positive and negative experiences of medications, creating a source of publicly available information that has the potential to provide insights into medicinal product safety concerns. The WEB-RADR project has developed a collaborative English language workspace for visualising and analysing social media data for a number of medicinal products. Further, novel text and data mining methods for social media analysis have been developed and evaluated. From this original research, several recommendations are presented with supporting rationale and consideration of the limitations. Recommendations for further research that extend beyond the scope of the current project are also presented.
      PubDate: 2019-12-01
       
  • Measuring the Effectiveness of Safety Warnings on the Risk of Stroke in
           Older Antipsychotic Users: A Nationwide Cohort Study in Two Large
           Electronic Medical Records Databases in the United Kingdom and Italy
    • Abstract: Introduction Safety warnings relating to antipsychotic-associated stroke among older persons in the UK and Italy were issued. However, the impact of these safety warnings on stroke risk has not been measured to date. Objective The aim of this study was to measure the change in stroke incidence after two safety warnings in both the UK and Italy. Method A cohort study was conducted using electronic medical records representative of the UK (The Health Improvement Network) and Italy (Health Search—IQVIA Health LPD), containing data on 11 million and 1 million patients, respectively. After each drug safety warning, elderly antipsychotic new initiators were propensity-score matched 1:1:1 on antipsychotic initiators before any safety warning. Stroke incidence within 6 months of antipsychotic initiation, using an intention-to-treat approach, was the main outcome. Results In the UK and Italy, 6342 and 7587 elderly antipsychotic initiators were identified, respectively. A 42% stroke incidence reduction was seen in the UK after the first safety warning [42.3 (95% confidence interval (CI) 35.2–50.8) vs. 24.4 [95% CI 19.0–31.2] events per 1000 person-years (PYs)], while there was a 60% stroke incidence reduction after the second warning (16.9 [95% CI 12.2–23.4] events per 1000 PYs) compared to before the first warning. There was no significant reduction in stroke incidence in Italy. Conclusion Antipsychotic safety warnings were followed by a reduction in stroke incidence among older antipsychotic users in the UK, but not Italy.
      PubDate: 2019-12-01
       
  • Psoriasis After Exposure to Angiotensin-Converting Enzyme Inhibitors:
           French Pharmacovigilance Data and Review of the Literature
    • Abstract: Introduction Angiotensin-converting enzyme inhibitors (ACEIs) can induce or aggravate psoriasis. This risk is not specified in the Summary of Product Characteristics (SmPC) of some drugs of this class, such as captopril or enalapril. We aimed to investigate the association between psoriasis and ACEI exposure. Methods We analyzed spontaneous reports recorded in the French national Pharmacovigilance Database (FPVD) from 1985 to 31 December 2018. The association between psoriasis and ACEI exposure was assessed using the case/non-case method. We also reviewed literature reports. Results One hundred reports of psoriasis after ACEI exposure were registered in the FPVD. The reporting odds ratio (ROR) was 2.40 (95% CI 1.96–2.95). Time to onset was < 1 year in 67% of reports. Outcome was favorable in 73% of reports after ACEI discontinuation. Almost all ACEIs were concerned. In the literature, we found 21 published reports of psoriasis with ACEIs. Time to onset ranged from 1 week to 4 months. Outcome was also favorable after ACEI discontinuation in over half of the literature reports. Conclusions We found a statistically significant association between psoriasis and ACEI, which constitutes a potential safety signal. The risk of psoriasis is a class effect, time to onset is less than 1 year, and outcome is favorable after ACEI discontinuation. Psoriasis should be mentioned in the SmPCs of all ACEIs, and healthcare professionals should be informed about this risk.
      PubDate: 2019-12-01
       
  • Prevalence and Nature of Medication Errors and Preventable Adverse Drug
           Events in Paediatric and Neonatal Intensive Care Settings: A Systematic
           Review
    • Abstract: Introduction Children admitted to paediatric and neonatal intensive care units may be at high risk from medication errors and preventable adverse drug events. Objective The objective of this systematic review was to review empirical studies examining the prevalence and nature of medication errors and preventable adverse drug events in paediatric and neonatal intensive care units. Data Sources Seven electronic databases were searched between January 2000 and March 2019. Study Selection Quantitative studies that examined medication errors/preventable adverse drug events using direct observation, medication chart review, or a mixture of methods in children ≤ 18 years of age admitted to paediatric or neonatal intensive care units were included. Data Extraction Data on study design, detection method used, rates and types of medication errors/preventable adverse drug events, and medication classes involved were extracted. Results Thirty-five unique studies were identified for inclusion. In paediatric intensive care units, the median rate of medication errors was 14.6 per 100 medication orders (interquartile range 5.7–48.8%, n = 3) and between 6.4 and 9.1 per 1000 patient-days (n = 2). In neonatal intensive care units, medication error rates ranged from 4 to 35.1 per 1000 patient-days (n = 2) and from 5.5 to 77.9 per 100 medication orders (n = 2). In both settings, prescribing and medication administration errors were found to be the most common medication errors, with dosing errors the most frequently reported error subtype. Preventable adverse drug event rates were reported in three paediatric intensive care unit studies as 2.3 per 100 patients (n = 1) and 21–29 per 1000 patient-days (n = 2). In neonatal intensive care units, preventable adverse drug event rates from three studies were 0.86 per 1000 doses (n = 1) and 0.47–14.38 per 1000 patient-days (n = 2). Anti-infective agents were commonly involved with medication errors/preventable adverse drug events in both settings. Conclusions Medication errors occur frequently in critically ill children admitted to paediatric and neonatal intensive care units and may lead to patient harm. Important targets such as dosing errors and anti-infective medications were identified to guide the development of remedial interventions.
      PubDate: 2019-12-01
       
  • The Association between Metformin Therapy and Lactic Acidosis
    • Abstract: Introduction and Objectives There is increasing evidence to suggest that therapeutic doses of metformin are unlikely to cause lactic acidosis. The aims of this research were (1) to formally evaluate the association between metformin therapy and lactic acidosis in published case reports using two causality scoring systems, (2) to determine the frequency of pre-existing independent risk factors in published metformin-associated lactic acidosis cases, (3) to investigate the association between risk factors and mortality in metformin-associated lactic acidosis cases, and (4) to explore the relationship between prescribed metformin doses, elevated metformin plasma concentrations and the development of lactic acidosis in cases with chronic renal impairment. Methods A systematic review was conducted to identify metformin-associated lactic acidosis cases. Causality was assessed using the World Health Organisation-Uppsala Monitoring Centre system and the Naranjo adverse drug reaction probability scale. Compliance to dosing guidelines was investigated for cases with chronic renal impairment as well as the association between steady-state plasma metformin concentrations prior to admission. Results We identified 559 metformin-associated lactic acidosis cases. Almost all cases reviewed (97%) presented with independent risk factors for lactic acidosis. The prescribed metformin dose exceeded published guidelines in 60% of cases in patients with impaired kidney function. Metformin steady-state plasma concentrations prior to admission were predicted to be below the proposed upper limit of the therapeutic range of 5 mg/L. Conclusions Almost all cases of metformin-associated lactic acidosis reviewed presented with independent risk factors for lactic acidosis, supporting the suggestion that metformin plays a contributory role. The prescribed metformin dose, on average, exceeded the dosing recommendations by 1000 mg/day in patients with varying degrees of renal impairment but the predicted pre-admission plasma concentrations did not exceed the therapeutic range.
      PubDate: 2019-12-01
       
  • Data-Driven Identification of Adverse Event Reporting Patterns for Japan
           in VigiBase, the WHO Global Database of Individual Case Safety Reports
    • Abstract: Introduction Adverse event reporting patterns vary between countries, reflecting differences in reporting culture, clinical practice and underlying patient populations. Japan collects about 60,000 domestic adverse event reports yearly and shares serious reports with the World Health Organization (WHO) Programme for International Drug Monitoring in VigiBase, the WHO global database of individual case safety reports. Understanding these reports in the global context can be helpful for regulators worldwide and can aid hypothesis-generation for Japanese-specific vulnerabilities to adverse drug reactions. Objective The objective of this study was to explore differences in the reporting of adverse events between Japan and other countries. Methods vigiPoint is a method for data-driven exploration in pharmacovigilance. It outlines data subsets, pinpoints key features and facilitates expert review, using odds ratios subjected to statistical shrinkage to distinguish one data subset from another. Here, we compared 260,000 Japanese reports in E2B format classified as serious and received in VigiBase between 2013 and 2018 with 2.5 million reports from the rest of the world (of which 51% are from the USA). Reporting patterns for which the 99% credibility interval of the shrunk log-odds ratios were above 0.5 or below − 0.5 were flagged as key features. The shrinkage was set to the vigiPoint default corresponding to 1% of the size of the Japanese data subset. As a sensitivity analysis, additional vigiPoint comparisons were performed between Japan and, in turn, Africa, the Americas, the Americas except the USA and Canada, Asia and Europe. Results There were higher reporting rates in Japan from physicians (83% vs. 39%) and pharmacists (17% vs. 10%). It was also more common to see reports with more than five drugs per report (22% vs. 14%) and with a single adverse event (72% vs. 45%). More than half of the Japanese reports had a vigiGrade completeness score above 0.8 compared with about one in five from the rest of the world. There were more reports than expected for patients aged 70–89 years and fewer reports for adults aged 20–59 years. Adverse events reported more often in Japan included interstitial lung disease, abnormal hepatic function, decreased platelet count, decreased neutrophil count and drug eruption. Adverse events reported less often included death, fatigue, dyspnoea, pain and headache. Drugs reported more often in Japan included prednisolone, methotrexate and peginterferon alfa-2b. Drugs reported less often included rosiglitazone and adalimumab as well as blood substitutes and perfusion solutions. The findings were generally robust to the sensitivity analysis except for the less often reported drugs, many of which were rarely reported in most countries, except in the USA. Conclusion Analysis of Japanese adverse event reporting patterns in a global context has revealed key features that may reflect possible pharmaco-ethnic vulnerabilities in the Japanese, as well as differences in adverse event reporting and clinical practice. This knowledge is essential in the global collaboration of signal detection afforded by the WHO Programme for International Drug Monitoring.
      PubDate: 2019-12-01
       
  • Comparative Effectiveness and Safety of Direct Oral Anticoagulants:
           Overview of Systematic Reviews
    • Abstract: Abstract Direct oral anticoagulants are now recommended by major guidelines as first-choice agents for both stroke prevention in non-valvular atrial fibrillation and treatment/prevention of venous thromboembolism in non-cancer patients. Although there are no published head-to-head trials comparing different direct oral anticoagulants, a growing body of evidence from indirect comparisons and observational studies is suggesting that each direct oral anticoagulant may have a specific risk profile. This review aims to (1) synthesize and critically assess the latest evidence in comparative effectiveness and safety research in the aforementioned consolidated therapeutic uses, by performing an overview of systematic reviews and (2) highlight current challenges, namely underexplored areas, where research should be directed, also considering ongoing unpublished studies. The evidence gathered so far on the risk–benefit profile of direct oral anticoagulants is appraised in the light of existing guidelines to discuss whether further implementation should be proposed.
      PubDate: 2019-12-01
       
  • Effectiveness and Safety of Switching Originator and Biosimilar Epoetins
           in Patients with Chronic Kidney Disease in a Large-Scale Italian Cohort
           Study
    • Abstract: Introduction Real-world data on the comparative effectiveness and safety of switching among different epoetins (including originators and biosimilars) are limited. In light of current debate about interchangeability, prescribers, some patient groups and decision makers are calling for additional post-marketing evidence on the clinical effects of switching between originator and biosimilar epoetins in chronic kidney disease (CKD) patients. Objective The objective of this study was to evaluate the effectiveness and safety of switching versus non-switching and of switching from originator/biosimilar epoetin alpha (ESA α) to any other epoetin in CKD patients. Methods An observational, record-linkage, multi-database, retrospective cohort study was carried out in four Italian geographical areas. All subjects with at least one ESA α dispensing between 1 January 2009 and 31 December 2015 were retrieved. Switching was defined as any transition between originator/biosimilar ESA α to any other epoetin in a series of two consecutive prescriptions up to 2 years. Switchers were matched 1:1 with non-switchers by baseline propensity score and by duration of ESA α treatment. Switchers and non-switchers were followed up from switching date to a maximum of 1 year. Lack of effectiveness and safety of switching versus non-switching were evaluated through Cox regression models (hazard ratio [HR], 95% confidence interval [CI]). A direct comparison between the two switcher categories (switchers from originator/biosimilar ESA α to any other epoetin) was also performed. Results Overall, 14,400 incident users of ESA α for anaemia due to CKD (61.4% originator, 38.6% biosimilar) were available for analysis. During the follow-up, we found no differences on effectiveness (HR 1.02, 95% CI 0.79–1.31 originators; HR 1.16, 95% CI 0.75–1.79 biosimilars) and safety outcomes (HR 1.08, 95% CI 0.77–1.50 originators; HR 1.20, 95% CI 0.66–2.21 biosimilars) between switchers and non-switchers of ESA α. Cumulative probabilities of recording an adverse event, either in terms of lack of effectiveness or safety issue, were the same for two switching categories Conclusions In this large-scale Italian observational multi-database study, switching versus non-switching as well as switching from biosimilar/originator ESA α to any other epoetin in CKD patients is not associated with any effectiveness and safety outcomes.
      PubDate: 2019-12-01
       
  • The Role of Unit-Dose Child-Resistant Packaging in Unintentional Childhood
           Exposures to Buprenorphine–Naloxone Tablets
    • PubDate: 2019-11-20
       
  • Fatal Toxicity Indices for Medicine-Related Deaths in New Zealand,
           2008–2013
    • Abstract: Introduction The fatal toxicity index (FTI) is a measure for assessing the relative risks of death due to the medicines prescribed in a population. This knowledge is useful for prescribers and informs medicine safety initiatives. This study aimed to calculate FTIs for the New Zealand population using three methodologies. Methods New Zealand coronial data describing medicine-related deaths from 1 January 2008 to 31 December 2013 were retrospectively extracted from the National Coronial Information System. Three fatal toxicity indices were derived using the number of deaths attributed to each pharmaceutical as the numerator and the total defined daily doses, number of patients and number of prescriptions as denominators. Results There were 703 medicine-related deaths, of which 627 were assessed as due to one primary contributor. Median decedent age was 48 years (interquartile range 37–58), and 319 (51%) were male. Deaths were intentional in 252 cases (40%), unintentional in 284 (45%) and unknown in 91 (15%). The majority of deaths (n = 486, 78%) occurred in the community. Opioids, antidepressants, antipsychotics and hypnotic-anxiolytics caused most fatalities. While the FTIs for individual medicines varied by denominator applied, methadone and clozapine fatalities were prominent in all three indices. The antidepressants clomipramine, dosulepin and doxepin consistently returned the highest FTIs in their group. Conclusion New Zealand prescribers should be aware of the high relative risk of death associated with methadone and clozapine; that clomipramine, dosulepin and doxepin were identified as the most dangerous antidepressants; and that zopiclone carries a similar fatal risk to benzodiazepines. Varying results were found between the FTIs calculated, making comparisons, particularly between populations, difficult.
      PubDate: 2019-11-20
       
  • Prenatal Exposure to Macrolides and Risk of Congenital Malformations: A
           Meta-Analysis
    • Abstract: Introduction Macrolides are widely used during pregnancy; however, their fetal safety remains uncertain. We performed a meta-analysis to assess the relation between prenatal exposure to macrolides and occurrence of congenital malformations. Methods We searched MEDLINE, EMBASE, and other databases until June 12, 2019. We assessed the quality of the studies and checked for heterogeneity and publication bias. We performed three different analyses and compared the effect of macrolides with each of the following unexposed populations: Group 1: babies unexposed to any medicine before birth, Group 2: babies exposed to non-macrolide antibiotics/non-teratogens, and Group 3: mixed population of the first and second comparators. Results A weak association between macrolides and congenital malformation of any type was observed when macrolides were compared with the mixed population (ORgroup 3 1.06 [95% CI 1.01–1.10]). Subgroup analysis showed that this weak association is restricted to fetus exposure in the first trimester of pregnancy (OR 1.06 [95% CI 1.01–1.11]) and to cohort studies (OR 1.07 [95% CI 1.02–1.13]). Digestive system malformations were found to be slightly associated with prenatal exposure to macrolides (ORgroup 3 1.14 [95% CI 1.02–1.26]). The musculoskeletal system was also found to be potentially affected (ORgroup 2 1.21 [95% CI 1.08–1.35] and ORgroup 3 1.15 [95% CI 1.05–1.26]). European studies showed a slightly stronger association than American studies in these two comparisons. Conclusions Our study suggests a weak association between prenatal use of macrolides and congenital malformations, limited to exposure in early pregnancy, and musculoskeletal and digestive systems. In addition to studies with a larger control of confounding, risk–benefit research is needed to determine the usefulness of macrolides during pregnancy.
      PubDate: 2019-11-12
       
  • Rheumatology Common Toxicity Criteria (RCTC): An Update Reflecting
           Real-World Use
    • Abstract: Introduction The Outcome Measures in Rheumatology Clinical Trials (OMERACT) Rheumatology Common Toxicity Criteria (RCTC) version 2.0 was published in 2007 by the OMERACT Drug Safety Working Group, building on limited experience with RCTC version 1.0, to facilitate standardization of assessment (grading) and reporting of adverse events (AEs) commonly seen in rheumatic disease clinical trials (Woodworth et al. in J Rheumatol 34:1401–1414, 2007). Objectives The objectives of this study were to (1) report the real-world performance of RCTC 2.0; (2) report immediately correctable errors in RCTC 2.0, and provide a revised RCTC 2.1; and (3) begin to identify the need for a comprehensive revision of RCTC 2.0. Methods Safety data outputs for several large rheumatic/autoimmune disease clinical trials in which RCTC 2.0 was used were evaluated for accuracy of reporting and the ability to assess differences among treatments. We examined RCTC 2.0 tables for errors, as well as for omission of terms for AEs that commonly occur in more recent rheumatology clinical trials. We also considered recommendations from recent US Food and Drug Administration (FDA) and international initiatives such CDISC (Clinical Data Interchange Standards Consortium) to improve the consistency of safety data collection and interpretability of safety data analyses. Results RCTC 2.0 enabled comparisons of safety data across treatment groups, including grading. However, we discovered inaccuracies in laboratory results grading and omission of AE terms now recognized to occur in rheumatic disease clinical trials. Conclusion The RCTC 2.0 performed as intended, although some inaccuracies and omissions were found. We provide a corrected version, RCTC 2.1, and also recommend further revision of the RCTC within OMERACT guidances to include AEs that have been reported in rheumatology clinical trials since RCTC 2.0 was published. Ideally, a revised RCTC 3.0 would not only facilitate standardized assessment and reporting of AEs, but would also expand and encourage accurate comparison of the safety profiles of treatments for rheumatic/autoimmune diseases.
      PubDate: 2019-11-06
       
  • Medical Devices: Classification and Analysis of Faults Leading to Harms
    • Abstract: Introduction Harms from medical devices are important, but have been much less well studied than adverse drug reactions. Information provided to device users is of variable quality. Objective Our aim was to define “medical device fault” and “adverse effect of a medical device”; to establish whether medical device faults arise in design, manufacture, or use; and to consider ways of mitigating the adverse effects of medical devices. Methods We analysed 100 consecutive faults reported by the US Food and Drug Administration (FDA) and 50 faults reported by the UK Medicines and Healthcare products Regulatory Agency (MHRA), and classified faults according to the point at which they occurred. Results Nearly 70% of reported faults related to devices that entered the body. Over 70% arose at the design stage, a quarter of the faults were associated with manufacture, and less than 5% were primarily caused by faulty use. Conclusion We defined a medical device fault as an unintended failure in the design, manufacture, or use of a medical device that leads to, or has the potential to lead to, harm to the patient, and an adverse effect of a medical device as an unintended and appreciably harmful effect, caused by a medical device, which demonstrates a hazard of the device and may warrant preventive measures, or a change in the mode of use, or withdrawal of the device. Most faults that generate warnings arise from problems at the design stage, some arise at the manufacturing stage, and a few in usage. Careful assessment of the design of safety-critical devices in the light of previous problems may help to prevent repetition of errors. It would be helpful if, in addition to user manuals, manufacturers were required to produce Summaries of Device Characteristics (SDCs, “labels”) that contained a systematically presented set of information about a product.
      PubDate: 2019-11-05
       
  • Effectiveness of Risk Minimization Measures for Fentanyl Buccal Tablet
           (FENTORA) in Canada: A Mixed-Methods Evaluation Using Surveys, Medical
           Chart Records and Web Surveillance
    • Abstract: Background Fentanyl buccal tablet (FBT), a potent opioid, was approved in Canada in 2013 for breakthrough pain in opioid-tolerant adult cancer patients. Additional risk minimization measures (aRMMs), consisting of communications to patients and healthcare providers (HCPs), were implemented from November 2014 through September 2015. Objectives The aim of this study was to assess the effectiveness of FBT aRMMs as measured by prescriber knowledge, understanding, and behavior regarding key safety concerns (off-label use, use in non-opioid-tolerant patients, misuse/abuse/diversion, and drug–drug interaction) and to evaluate illicit FBT use. Methods The study included three components: (1) a knowledge and understanding (KAU) survey of FBT prescribers conducted in two waves: November 2016–February 2017 and April–September 2018; (2) a retrospective prescription study of medical records of patients treated with FBT by a subgroup of prescribers from the KAU survey; and (3) Web surveillance of illicit FBT use in Canada using the search term FENTORA (May 2014–September 2018). The aRMMs were considered effective if the lower bound of the 95% confidence interval indicated that at least 65% of respondents met or partly met the knowledge objective for each key safety concern. Results KAU survey: Of 46 eligible HCPs, 97.8% met or partly met the knowledge objective on use in breakthrough pain cancer patients, 97.8% on use in opioid-tolerant patients, 89.1% on dose and titration, 100% on abuse/addiction, and 58.7% on drug–drug interaction. Retrospective prescription study: Of 22 FBT-treated patients identified from 14 HCPs, 45.5% had cancer, 50.0% recorded a breakthrough pain indication, and 36.4% reported opioid tolerance; however, only 13.6% of patients were prescribed FBT according to the approved indication. Web surveillance: Of 932 FBT posts in Canada, only 40 (4.3%) mentioned illicit use. Conclusions The aRMMs as measured by the prescriber KAU were effective for most key safety messages; however, not all key messages of the aRMMs were stringently followed in routine practice.
      PubDate: 2019-11-05
       
  • Acknowledgement to Referees
    • PubDate: 2019-11-04
       
  • Development and First Use of the Patient’s Qualitative Assessment of
           Treatment (PQAT) Questionnaire in Type 2 Diabetes Mellitus to Explore
           Individualised Benefit–Harm of Drugs Received During Clinical Studies
    • Abstract: Introduction Individualised benefit–harm assessments can help identify patient-perceived benefits and harms of a treatment, and associated trade-offs that may influence patients’ willingness to use a treatment. This research presents the first use of a patient-reported outcome measure designed to assess patient-perceived benefits and disadvantages of drugs received during clinical studies. Methods The Patient’s Qualitative Assessment of Treatment (PQAT) was developed in English and cognitively tested with US (n = 4) and Canadian (n = 3) patients with type 1 and type 2 diabetes mellitus (T2DM). The revised version of the PQAT comprises three qualitative open-ended questions focused on the benefits and disadvantages of treatment and reasons why patients would choose to continue/discontinue treatment. A final quantitative question asks patients to evaluate the balance between benefits and disadvantages using a 7-point scale. The revised version of the questionnaire was administered as an exploratory endpoint in a phase II clinical trial for a new injectable treatment for T2DM. Qualitative data were analysed using thematic analysis, and relationships between qualitative and quantitative data were identified. Results Patient-reported benefits of treatment administered during the clinical trial included clinical markers of efficacy and subjective markers. Disadvantages reported by patients were mainly related to drug adverse effects or to the mode of administration. Of the 57 patients completing the PQAT, 70.2% reported being willing to continue treatment, with 59.6% reporting that the benefits outweighed the disadvantages. The reported benefits of feeling better and improved energy levels were more likely to be associated with a more positive ratio (70% and 71.4%, respectively), while the disadvantages of fatigue, headaches, and stomach pain were associated with a negative ratio and patients not being willing to continue the treatment. Conclusions The PQAT is a unique patient-reported outcome tool designed to aid understanding patients’ real experience of benefits and disadvantages of a treatment. It combines the richness of qualitative data with quantitative data—information valuable for various stakeholders to make well-informed treatment decisions. Trial Registration ClinicalTrials.gov identifier: NCT02973321.
      PubDate: 2019-11-02
       
  • Correction to: Patient Registries: An Underused Resource for Medicines
           Evaluation
    • Abstract: The fourth sentence under the heading “1.1 Use of Patient Registries for Supporting Regulatory Assessments” in “1 Introduction” section should read as below.
      PubDate: 2019-11-01
       
  • Identifying the DEAD: Development and Validation of a Patient-Level Model
           to Predict Death Status in Population-Level Claims Data
    • Abstract: Introduction US claims data contain medical data on large heterogeneous populations and are excellent sources for medical research. Some claims data do not contain complete death records, limiting their use for mortality or mortality-related studies. A model to predict whether a patient died at the end of the follow-up time (referred to as the end of observation) is needed to enable mortality-related studies. Objective The objective of this study was to develop a patient-level model to predict whether the end of observation was due to death in US claims data. Methods We used a claims dataset with full death records, Optum© De-Identified Clinformatics® Data-Mart-Database—Date of Death mapped to the Observational Medical Outcome Partnership common data model, to develop a model that classifies the end of observations into death or non-death. A regularized logistic regression was trained using 88,514 predictors (recorded within the prior 365 or 30 days) and externally validated by applying the model to three US claims datasets. Results Approximately 25 in 1000 end of observations in Optum are due to death. The Discriminating End of observation into Alive and Dead (DEAD) model obtained an area under the receiver operating characteristic curve of 0.986. When defining death as a predicted risk of > 0.5, only 2% of the end of observations were predicted to be due to death and the model obtained a sensitivity of 62% and a positive predictive value of 74.8%. The external validation showed the model was transportable, with area under the receiver operating characteristic curves ranging between 0.951 and 0.995 across the US claims databases. Conclusions US claims data often lack complete death records. The DEAD model can be used to impute death at various sensitivity, specificity, or positive predictive values depending on the use of the model. The DEAD model can be readily applied to any observational healthcare database mapped to the Observational Medical Outcome Partnership common data model and is available from https://github.com/OHDSI/StudyProtocolSandbox/tree/master/DeadModel.
      PubDate: 2019-11-01
       
 
 
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