Methods for external control groups for single arm trials or long-term uncontrolled extensions to randomized clinical trials.

Purpose: Clinical trials compare outcomes among patients receiving study treatment with comparators drawn from the same source. These internal controls are missing in single arm trials and from long-term extensions (LTE) of trials including only the treatment arm. An external control group derived from a different setting is then required to assess safety or effectiveness.

Engaging stakeholders in pharmacoepidemiology research: Current state and recommendations.

Purpose: Given current efforts to enhance patient-centered care and shared decision-making, the International Society of Pharmacoepidemiology Workgroup on Patient Engagement assessed patient and other stakeholder engagement in pharmacoepidemiology research and provides recommendations for the field.

Methods: A systematic review used MEDLINE and EMBASE to identify published literature from 2005 to 2016 addressing how stakeholders-patients, caregivers, and others-assisted researchers conducting pharmacoepidemiologic research. Three pairs of Workgroup members screened titles and abstracts to select articles for full-text review and analysis.

Qualitative analysis and reproducibility assessment of the Scalp Itch Numeric Rating Scale among patients with moderate to severe plaque psoriasis of the scalp.

Pruritus is a prevalent and bothersome symptom of scalp psoriasis. Validated scales assessing scalp itch are needed to evaluate treatment efficacy. To evaluate comprehensibility and reproducibility of the Scalp Itch Numeric Rating Scale (NRS), a novel scale being used in a phase 3 study of apremilast.

Sensitivity analysis of methods for active surveillance of acute myocardial infarction using electronic databases.

Background: The validity of conclusions from observational studies depends on decisions regarding design, analysis, data quality, and implementation. Through sensitivity analyses, we explored the impact of such decisions on balance control and risk estimates.

Methods: Using as a template the Mini-Sentinel protocol for the active surveillance of acute myocardial infarction (MI) in association with use of antidiabetic agents, we defined cohorts of new users of metformin and second-generation sulfonylureas, baseline covariates and acute MI events using three combinations of washout and baseline periods.

Choice of recall period for patient-reported outcome (PRO) measures: criteria for consideration.

Purpose: Understand the choice of recall period for PRO measures based on intended use, characteristics of the disease, treatment, and attributes of studies.

Methods: Current practice and considerations were reviewed within several disease areas (overactive bladder, menopausal hot flashes, niacin-induced flushing, osteoarthritis pain, irritable bowel symptoms, benign prostatic hyperplasia, and alopecia).

Results: Rationales were identified for using different recall periods, including event-driven (immediate), daily, up to weekly, and longer than weekly.

Recommendations for safety planning, data collection, evaluation and reporting during drug, biologic and vaccine development: a report of the safety planning, evaluation, and reporting team.

Background: The Safety Planning, Evaluation and Reporting Team (SPERT) was formed in 2006 by the Pharmaceutical Research and Manufacturers of America.

Purpose: SPERT's goal was to propose a pharmaceutical industry standard for safety planning, data collection, evaluation, and reporting, beginning with planning first-in-human studies and continuing through the planning of the post-product-approval period.

Methods: SPERT's recommendations are based on our review of relevant literature and on consensus reached in our discussions.

Acute migraine treatment with rizatriptan in real world settings - focusing on treatment strategy, effectiveness, and behavior.

Although randomized controlled trials (RCTs) are the gold standard for assessing efficacy of a drug intervention, because they are conducted in a highly selected group of patients, they do not necessarily reflect normal customary or optimized patient care. Accordingly, information from RCTs must be supplemented by outcomes research and by nonexperimental or quasi-experimental study designs. Herein, we discuss information that supplements the rigorous but sometimes rigid nature of RCTs in an effort to better understand the clinical utility of drug treatment for migraine with patient-centered outcomes in mind.

Interpreting and reporting results based on patient-reported outcomes.

This article deals with the incorporation of patient-reported outcomes (PROs) into clinical trials and focuses on issues associated with the interpretation and reporting of PRO data. The primary focus and context of this information relates to the evidentiary support and reporting for a labeling or advertising claim of a PRO benefit for a new or approved pharmaceutical product. This manuscript focuses on issues associated with assessing clinical significance and common pitfalls to avoid in presenting results related to PROs.

Analysis and interpretation of results based on patient-reported outcomes.

This article is part of a series of manuscripts dealing with the incorporation of patient-reported outcomes (PROs) into clinical trials. The issues dealt with in this manuscript concern the common pitfalls to avoid in statistical analysis and interpretation of PROs. Specifically, the questions addressed by this manuscript involve the analysis pitfalls with PRO data in clinical trials and how can they be avoided (e.

Validation of a questionnaire to assess niacin-induced cutaneous flushing.

Background: Niacin is currently the most effective approved agent for raising high-density lipoprotein cholesterol. However, niacin-induced cutaneous flushing (redness, warmth, tingling and/or itching) significantly limits patient acceptance. To further characterize flushing, a patient-reported Flushing Symptom Questionnaire (FSQ) was developed and validated.

Validation of the nighttime symptoms score as a clinically relevant measure of allergic rhinitis.

The aim of this study was to validate the nighttime symptoms score (NSS), which incorporates individual scores for difficulty going to sleep and nighttime awakening caused by nasal symptoms and nasal congestion on awakening, as a clinically relevant measure of allergic rhinitis (AR). Fifty-five general season AR (SAR) symptom items were generated by interviews with 14 patients with symptomatic SAR without concomitant asthma for use in an Importance Rating Questionnaire (IRQ). A second group of patients (n = 83) with symptomatic AR without asthma rated the importance of each item on the IRQ.

Montelukast effectively treats the nighttime impact of seasonal allergic rhinitis.

Background: Nighttime problems constitute a significant burden on the quality of life of patients with seasonal allergic rhinitis (SAR). The aim of this study was to evaluate the effectiveness of montelukast on nighttime AR symptoms.

Methods: In seven multicenter, double-blind, parallel-group trials, nighttime problems were assessed as the nighttime symptoms score (NSS), an average of three individual symptom scores: difficulty going to sleep, nighttime awakening, and nasal congestion on awakening (each rated 0 = none to 3 = severe).

Validation of a pediatric caregiver diary to measure symptoms of postacute respiratory syncytial virus bronchiolitis.

Acute respiratory syncytial virus (RSV)-induced bronchiolitis is often associated with continuing respiratory symptoms following hospitalization. To date, there is no validated objective measure to evaluate symptoms of RSV-induced bronchiolitis. We report on the reliability, validity, and responsiveness of the bronchiolitis caregiver diary (BCD) of symptoms and healthcare utilization associated with postacute RSV.

The effect of montelukast versus usual care on health care resource utilization in children aged 2 to 5 years with asthma.

Background: Limited clinical data are available on the long-term effects of asthma controller therapy on the utilization of health care resources in pediatric patients with asthma.

Objective: The objective of this study was to compare the effects of long-term treatment with montelukast and usual care on health care resource use in children with asthma.

Methods: Pediatric patients aged 2 to 5 years with asthma who had completed a 3-month, double-blind, double-dummy clinical trial comparing montelukast 4 mg and placebo were asked to participate in an open-label, controlled extension study comparing montelukast 4 mg and usual care.

Response to montelukast among subgroups of children aged 2 to 14 years with asthma.

Background: Determining who responds to asthma therapies, particularly leukotriene modifiers, continues to be explored.

Objective: We sought to identify patient characteristics predictive of response to montelukast.

Methods: We used data from 2 clinical trials in which children with asthma received either montelukast or placebo.

Adherence to prescribed treatment for asthma: evidence from pharmacy benefits data.

Background: Failure to use asthma controller medications as prescribed is associated with more asthma-related adverse events. Medication utilization may vary with ease of drug administration, efficacy, and tolerability as well as other factors. We hypothesized that in usual-care clinical practice settings, there would be greater adherence to oral controller than to inhaled controller asthma medications.

Treatment effectiveness of inhaled corticosteroids and leukotriene modifiers for patients with asthma: an analysis from managed care data.

We compared measures of treatment effectiveness when inhaled corticosteroids (ICSs) or leukotriene modifiers (LMs) were used as controller monotherapy for asthma. Asthma patients aged 6-55 years initiating ICS or LM monotherapy between July 1998 and June 1999 (index prescription) were identified using a managed care claims database. Asthma-related hospitalizations, emergency department (ED) visits, and use of short-acting beta-agonists and oral corticosteroids (OCSs) were assessed as proxies for treatment effectiveness.

Regulatory issues for health-related quality of life--PhRMA Health Outcomes Committee workshop, 1999.

Introduction: Health-related quality-of-life (HRQL) can be defined as the impact of disease and treatment across the physical, psychological, social and somatic domains of functioning and well-being. Health-related quality-of-life measures are included in clinical trials of drug treatment to assess the impact of therapy on the patient's functioning. HRQL guidance could allow for use of this data in drug labeling and promotion.