Suitability of Preference Methods Across the Medical Product Lifecycle: A Multicriteria Decision Analysis.

Objectives: This study aimed to understand the importance of criteria describing methods (eg, duration, costs, validity, and outcomes) according to decision makers for each decision point in the medical product lifecycle (MPLC) and to determine the suitability of a discrete choice experiment, swing weighting, probabilistic threshold technique, and best-worst scale cases 1 and 2 at each decision point in the MPLC.

Methods: Applying multicriteria decision analysis, an online survey was sent to MPLC decision makers (ie, industry, regulatory, and health technology assessment representatives). They ranked and weighted 19 methods criteria from an existing performance matrix about their respective decisions across the MPLC.

Treatment preferences for preventive interventions for rheumatoid arthritis: protocol of a mixed methods case study for the Innovative Medicines Initiative PREFER project.

Introduction: Amidst growing consensus that stakeholder decision-making during drug development should be informed by an understanding of patient preferences, the Innovative Medicines Initiative project 'Patient Preferences in Benefit-Risk Assessments during the Drug Life Cycle' (PREFER) is developing evidence-based recommendations about how and when patient preferences should be integrated into the drug life cycle. This protocol describes a PREFER clinical case study which compares two preference elicitation methodologies across several populations and provides information about benefit-risk trade-offs by those at risk of rheumatoid arthritis (RA) for preventive interventions.

Methods And Analysis: This mixed methods study will be conducted in three countries (UK, Germany, Romania) to assess preferences of (1) first-degree relatives (FDRs) of patients with RA and (2) members of the public.

Structured benefit-risk evaluation for medicinal products: review of quantitative benefit-risk assessment findings in the literature.

Unlabelled: A favorable benefit-risk profile remains an essential requirement for marketing authorization of medicinal drugs and devices. Furthermore, prior subjective, implicit and inconsistent ad hoc benefit-risk assessment methods have rightly evolved towards more systematic, explicit or "structured" approaches. Contemporary structured benefit-risk evaluation aims at providing an objective assessment of the benefit-risk profile of medicinal products and a higher transparency for decision making purposes.

Effectiveness of risk minimisation measures for valproate: A cross-sectional survey among physicians in Europe.

Purpose: This study evaluated the effectiveness of risk minimisation measures (RMMs) implemented following the 2014 referral for valproate in Europe.

Methods: Cross-sectional survey was conducted over 2-month period in 2016 among physicians who prescribed valproate in France, Germany, the United Kingdom, Spain and Sweden. The web-based questionnaire included five endpoints to evaluate physicians' knowledge on (a) prescribing valproate only for epilepsy and bipolar disorder in women if other treatments were ineffective or not tolerated; (b) ensuring supervision by experienced physicians while treating these conditions; (c) considering alternative treatments for women planning pregnancy, regular review of treatment needs and re-assessing the benefit-risk balance in women and girls reaching puberty; (d) informing patients about the risks of taking valproate during pregnancy and (e) advising women on effective contraception during their treatment.

Pre/post effectiveness evaluation of updated additional risk minimisation measures for an orphan disease: Myozyme (alglucosidase alfa) Safety Information Packet.

Background: The alglucosidase alfa (Myozyme®) Safety Information Packet ("previous SIP") was updated to improve readability and content ("updated SIP"). We compared the previous and updated SIPs.

Methods: A two-wave pre-post multicountry survey was conducted among health care professionals (HCPs) who prescribed or monitored patients on alglucosidase alfa in the largest European Union ("EU5") countries and Poland.

Comparison of text processing methods in social media-based signal detection.

Purpose: Adverse event (AE) identification in social media (SM) can be performed using various types of natural language processing (NLP) and machine learning (ML). These methods can be categorized by complexity and precision level. Co-occurrence-based ML methods are rather basic, as they identify simultaneous appearance of drugs and clinical events in a single post.

Web-Based Signal Detection Using Medical Forums Data in France: Comparative Analysis.

Background: While traditional signal detection methods in pharmacovigilance are based on spontaneous reports, the use of social media is emerging. The potential strength of Web-based data relies on their volume and real-time availability, allowing early detection of signals of disproportionate reporting (SDRs).

Objective: This study aimed (1) to assess the consistency of SDRs detected from patients' medical forums in France compared with those detected from the traditional reporting systems and (2) to assess the ability of SDRs in identifying earlier than the traditional reporting systems.

Assessment of the Utility of Social Media for Broad-Ranging Statistical Signal Detection in Pharmacovigilance: Results from the WEB-RADR Project.

Introduction And Objective: Social media has been proposed as a possibly useful data source for pharmacovigilance signal detection. This study primarily aimed to evaluate the performance of established statistical signal detection algorithms in Twitter/Facebook for a broad range of drugs and adverse events.

Methods: Performance was assessed using a reference set by Harpaz et al.

Assessing the Risk for Peripheral Neuropathy in Patients Treated With Dronedarone Compared With That in Other Antiarrhythmics.

Purpose: There are few data on the risk for peripheral neuropathy associated with dronedarone, a newer antiarrhythmic medicine. The objective of this study was to assess whether dronedarone is potentially associated with an increased risk for peripheral neuropathy compared with other antiarrhythmics, including amiodarone, sotalol, flecainide, and propafenone.

Methods: The MarketScan database was used for identifying patients who were at least 18 years of age, had atrial fibrillation or flutter, and had not been diagnosed with peripheral neuropathy in the 180-day period prior to or on the date of the first prescription of an antiarrhythmic between July 20, 2009, and December 31, 2011.

Validation of New Signal Detection Methods for Web Query Log Data Compared to Signal Detection Algorithms Used With FAERS.

Introduction: Post-marketing drug surveillance is largely based on signals found in spontaneous reports from patients and healthcare providers. Rare adverse drug reactions and adverse events (AEs) that may develop after long-term exposure to a drug or from drug interactions may be missed. The US FDA and others have proposed that web-based data could be mined as a resource to detect latent signals associated with adverse drug reactions.

Multi-centre, multi-database studies with common protocols: lessons learnt from the IMI PROTECT project.

Purpose: To assess the impact of a variety of methodological parameters on the association between six drug classes and five key adverse events in multiple databases.

Methods: The selection of Drug-Adverse Event pairs was based on public health impact, regulatory relevance, and the possibility to study a broad range of methodological issues. Common protocols and data analytical specifications were jointly developed and independently and blindly executed in different databases in Europe with replications in the same and different databases.

The IMI PROTECT project: purpose, organizational structure, and procedures.

The Pharmacoepidemiological Research on Outcomes of Therapeutics by a European ConsorTium (PROTECT) initiative was a collaborative European project that sought to address limitations of current methods in the field of pharmacoepidemiology and pharmacovigilance. Initiated in 2009 and ending in 2015, PROTECT was part of the Innovative Medicines Initiative, a joint undertaking by the European Union and pharmaceutical industry. Thirty-five partners including academics, regulators, small and medium enterprises, and European Federation of Pharmaceuticals Industries and Associations companies contributed to PROTECT.

The risk of acute liver injury associated with the use of antibiotics--evaluating robustness of results in the pharmacoepidemiological research on outcomes of therapeutics by a European consortium (PROTECT) project.

Purpose: To examine the robustness of findings of case-control studies on the association between acute liver injury (ALI) and antibiotic use in the following different situations: (i) Replication of a protocol in different databases, with different data types, as well as replication in the same database, but performed by a different research team. (ii) Varying algorithms to identify cases, with and without manual case validation. (iii) Different exposure windows for time at risk.

Assessment of dronedarone utilization using US claims databases.

Background: A dronedarone utilization study using US MarketScan and InVision Data Mart databases was conducted to estimate the prevalence of the following: (1) dronedarone use in contraindicated patients with worsening heart failure (HF) or hospitalization for HF within 1 month before dronedarone prescription; (2) concomitant prescribing of contraindicated drugs; and (3) recommended creatinine testing after dronedarone initiation among dronedarone users.

Methods: In this retrospective cohort study, data in the MarketScan database between July 20, 2009, and December 31, 2011, and in the InVision Data Mart database between July 20, 2009, and March 31, 2012, were analyzed. The study population included patients who received ≥1 dronedarone prescription during the study period.

A prospective observational study of treatment practice patterns in acute coronary syndrome patients undergoing percutaneous coronary intervention in Europe.

Background: The AntiPlatelet Therapy Observational Registry (APTOR) was a prospective observational study of acute coronary syndrome (ACS) patients undergoing percutaneous coronary intervention (PCI) in France, Spain, and the UK.

Aims: To evaluate patterns of ACS healthcare use, focusing on APTOR results from France.

Methods: Consecutive presenting ACS patients requiring PCI were recruited between January and August 2007.

Remission in the outpatient care of schizophrenia: 3-year results from the Schizophrenia Outpatients Health Outcomes (SOHO) study in France.

Objective: To analyse the contribution of socio-demographics, clinical profile and psychotropic treatment on remission in patients with schizophrenia.

Methods: Among 933 French outpatients recruited in the European observational Schizophrenia Outpatient Health Outcomes study (SOHO), 563 were followed-up for 3 years, had at most one missing visit, and were included in the analysis. Symptomatic remission was defined as a score of 3 (mild severity) or less on the Clinical Global Impression-Schizophrenia (CGI) overall, positive, negative and cognitive symptom scales, maintained for at least 6 months and without hospitalization.

[Evaluation of a screening programme for diabetic retinopathy (DODIA study)].

Context: In France, 43% to 63% of diabetics have an annual fundoscopy. Do the new screening tools, coupled with teletransmission of the images, allow for satisfying ophthalmological screening? It is an important matter given the foreseeable reduction in the number of French ophthalmologists in the forthcoming years.

Objectives: To measure the quality of screening for diabetic retinopathy (DR), in the framework of a network, by the provision of a retinograph by numeric camera (with teletransmission of the images and centralised interpretation), in a screening centre located in town.

Influence of the severity of stress urinary incontinence on quality of life, health care seeking, and treatment: A national cross-sectional survey.

Objectives: To determine the prevalence of stress urinary incontinence (SUI) symptoms in women and to evaluate the severity of these symptoms and resulting functional impairment with regard to urinary symptoms, quality of life (QoL) impairment, medical care seeking, and care facilities.

Methods: A national population based, cross-sectional study in France was conducted through an anonymous questionnaire that was completed by telephone. A randomized sample was extracted from the French telephone directory, December 2002 to March 2003.

Patient satisfaction with psychotropic drugs: sensitivity to change and relationship to clinical status, quality-of-life, compliance and effectiveness of treatment. Results from a nation-wide 6-month prospective study.

Objective: To see if patient satisfaction with psychotropics (PSP) could be used as a patient-oriented outcome variable in the evaluation of PSP drugs in clinical epidemiological studies, relationships between PSP, clinical status, QoL, compliance and the type of antipsychotic were analyzed. Elements of validation of PSP were also assessed.

Method: In a 6-month prospective study, 933 schizophrenic outpatients with initiation or change to their antipsychotic treatment were enrolled.

Management and relief of pain in an emergency department from the adult patients' perspective.

To estimate the prevalence of pain in adult patients attending an emergency department (ED) and to identify risk markers for insufficient pain relief, a cross-sectional survey was conducted for 16 days, 24 hours each day, in the ED of a Paris university hospital. A structured questionnaire was used to collect characteristics of pain and its management from patients. Pain intensity was evaluated both on arrival and before discharge using two scales (a numerical descriptor scale or a verbal pain intensity scale).

Pain prevalence in a French teaching hospital.

To measure the prevalence and intensity of pain in hospitalized patients and to assess the quality of pain management, an exhaustive cross-sectional study was conducted in every department in a university hospital. Patients hospitalized for 24 hours or more completed an anonymous self-report questionnaire. Among the 1,475 inpatients, 998 completed the questionnaire.

Association between first injection risk behaviors and hepatitis C seropositivity among injecting drug users.

HCV infection is rapidly acquired after drug addicts first inject drug intravenously. The risk behaviors accompanying the first intravenous substance injection are not well known. We used in 1997 a structured questionnaire to investigate the relationships between risk behaviors at the first injection and current reported HCV status.