Understanding the patient experience and treatment benefits in patients with non-small-cell lung cancer with brain metastasis.

Background: Despite the high prevalence of brain metastases (BM) secondary to non-small-cell lung cancer (NSCLC) (NSCLC/BM), patients' experiences (symptoms and impacts) are not fully understood. This study sought to understand the patient experience with NSCLC/BM and identify a patient-reported outcome (PRO) measure fit to capture the most important NSCLC/BM symptoms and impacts.

Methods: A targeted literature review was completed; the National Comprehensive Cancer Network (NCCN)/Functional Assessment of Cancer Therapy-Brain Symptom Index, 24-item version (NFBrSI-24) was identified as a relevant measure that assessed the core symptoms and impacts associated with NSCLC/BM.

Longitudinal Trajectories of Participant- and Study Partner-Rated Cognitive Decline, in Relation to Alzheimer's Disease Biomarkers and Mood Symptoms.

Whereas discrepancies between participant- and study partner-reported cognitive concerns on the Alzheimer's disease (AD) continuum have been observed, more needs to be known regarding the longitudinal trajectories of participant- vs. study partner-reported concerns, particularly their relationship to AD biomarkers and mood symptomology. Additionally, it is unclear whether years of in-clinic data collection are needed to observe relationships with AD biomarkers, or whether more frequent, remote assessments over shorter periods of time would suffice.

Symptoms and Treatment Needs of People with Dementia-Related Psychosis: A Mixed-Methods Study of the Patient Experience.

Objectives: This study describes the person-centered experience and impact of symptoms and the treatment needs of dementia-related psychosis (DRP) from a patient and care partner perspective.

Methods: Qualitative interviews and a quantitative survey were used to collect patient experience data from persons with DRP or their care partners.

Results: Sixteen participants (1 person with DRP, 15 care partners) completed the qualitative interview; 212 participants (26 persons with DRP, 186 care partners) completed the quantitative survey.

Self-reported review of the value of esketamine in patients with treatment-resistant depression: Understanding the patient experience in the STRIVE Study.

Esketamine nasal spray (ESK) is indicated, in conjunction with an oral antidepressant (OAD), for the management of treatment-resistant depression (TRD) in adults. Select US-based patients from an open-label, long-term extension safety study of ESK (NCT02782104) participated in this study through semi-structured interviews. The study evaluated patient-reported early health changes related to emotional health, daily functioning, and social functioning in adults with TRD treated with ESK plus OAD.

A New Single-Item Sleep Quality Scale: Results of Psychometric Evaluation in Patients With Chronic Primary Insomnia and Depression.

Study Objectives: A single-item sleep quality scale (SQS) was developed as a simple and practical sleep quality assessment and psychometrically evaluated.

Methods: SQS measurement characteristics were evaluated using the Pittsburgh Sleep Quality Index (PSQI) and morning questionnaire-insomnia (MQI) according to prespecified analysis plans in separate clinical studies of patients with insomnia and depression. Patients with insomnia (n = 70) received 4 weeks' usual care with an FDA-approved hypnotic agent; patients with depression (n = 651) received 8 weeks' active or experimental therapy.

Development of the Observable Behaviors of Autism Spectrum Disorder Scale.

Background: The objective of this research was to develop a caregiver-reported clinical outcome assessment (COA) measure designed to assess observable behaviors of children, ages 4 to 12 years, with autism spectrum disorder (ASD) for supporting labeling claims of treatment benefit.

Methods: Development of the measure included a review of the literature and existing instruments, conceptual disease model development, concept elicitation focus groups, item generation, and cognitive debriefing interviews.

Results: Predominant characteristics and behaviors of ASD identified by the literature and instrument reviews included sociability, communication deficits, stereotypy, inattention and hyperactivity, irritability, anxiety, and familial impact.

A Review of Patient-Reported Outcome Labeling in the United States (2011-2015).

Background: A review of new drug approvals (NDAs) by the Food and Drug Administration (FDA) for 2006 to 2010 showed that 24.1% of new drugs had patient-reported outcome (PRO) labeling.

Objectives: To review PRO labeling for NDAs for 2011 to 2015 and to compare key findings reported previously.

Payer Perspectives on Patient-Reported Outcomes in Health Care Decision Making: Oncology Examples.

Background: Health authorities and payers increasingly recognize the importance of patient perspectives and patient-reported outcomes (PROs) in health care decision making. However, given the broad variety of PRO endpoints included in clinical programs and variations in the timing of PRO data collection and country-specific needs, the role of PRO data in reimbursement decisions requires characterization.

Objectives: To (a) determine the effect of PRO data on market access and reimbursement decisions for oncology products in multiple markets and (b) assess the effect of PRO data collected after clinical progression on payer decision making.

Development of a novel observer-reported outcome measure for the assessment of Respiratory Syncytial Virus (RSV) infection symptoms in pediatric clinical trials.

Background: Respiratory syncytial virus (RSV) is a seasonal infection affecting most children by 2 years of age and the leading cause of lower respiratory tract infection requiring hospitalization in infants. Novel antiviral medications are in development to improve the clinical outcomes of RSV; however, no clinical outcome assessments (COAs) for RSV have been developed in alignment with the United States Food and Drug Administration patient-reported outcome guidance to assist in the evaluation of new therapies. To address this need, an observer-reported outcome (ObsRO) measure designed to assess observable RSV symptoms was created.

Psychometric evaluation of a caregiver diary for the assessment of symptoms of respiratory syncytial virus.

Background: There are no clinical outcome assessment (COA) tools developed in accordance with Food and Drug Administration (FDA) guidance suitable for the evaluation of symptoms associated with respiratory syncytial virus (RSV) infection among infants. The Gilead RSV Caregiver Diary (GRCD) is being developed to fulfill this need; the present research evaluates the GRCD and documents its reliability, validity, and responsiveness among children < 24 months of age with acute RSV infection.

Methods: A prospective, observational study was conducted in the United States during the 2014-2015 northern hemisphere winter season.

Mortality and Causes of Death in Patients with Sporadic Inclusion Body Myositis: Survey Study Based on the Clinical Experience of Specialists in Australia, Europe and the USA.

Background: There is a paucity of data on mortality and causes of death (CoDs) in patients with sporadic inclusion body myositis (sIBM), a rare, progressive, degenerative, inflammatory myopathy that typically affects those aged over 50 years.

Objective: Based on patient records and expertise of clinical specialists, this study used questionnaires to evaluate physicians' views on clinical characteristics of sIBM that may impact on premature mortality and CoDs in these patients.

Methods: Thirteen physicians from seven countries completed two questionnaires online between December 20, 2012 and January 15, 2013.

Patient-Reported Outcomes Labeling for Products Approved by the Office of Hematology and Oncology Products of the US Food and Drug Administration (2010-2014).

Purpose: To review the use of patient-reported outcome (PRO) data in medical product labeling granted by the US Food and Drug Administration (FDA) for new molecular entities and biologic license applications by the FDA Office of Hematology and Oncology Products (OHOP) between January 2010 and December 2014, to elucidate challenges faced by OHOP for approving PRO labeling, and to understand challenges faced by drug manufacturers to include PRO end points in oncology clinical trials.

Methods: FDA Drug Approval Reports by Month were reviewed to obtain the number of new molecular entities and biologic license applications approved from 2010 to 2014. Drugs approved by the FDA OHOP during this period were selected for further review, focusing on brand and generic name; approval date; applicant; indication; PRO labeling describing treatment benefit, measures, end point status, and significant results; FDA reviewer feedback on PRO end points; and study design of registration trials.

Psychometric validation of a patient-reported measure of physical functioning in sporadic inclusion body myositis.

Introduction: To assess self-reported physical functioning in patients with sporadic inclusion body myositis (sIBM), the sIBM Physical Functioning Assessment (sIFA) was developed. This research establishes the validity, reliability, and responsiveness of the sIFA in patients with sIBM.

Methods: Data from 3 small, noninterventional, observational studies were analyzed.

Development of the sporadic inclusion body myositis physical functioning assessment.

Introduction: Sporadic inclusion body myositis (sIBM) is a progressive idiopathic inflammatory myopathy characterized by atrophy and weakness of proximal and distal muscle groups that results in a loss of independence and the need for assistive devices and supportive care. To assess treatment benefit of new therapies, a patient-reported outcome measure of physical function was developed.

Methods: The tool was rigorously developed in accordance with the United States Food and Drug Administration (FDA) patient-reported outcomes (PRO) guidance.

Patient-Reported Outcomes in Latin America: Implementation in Research and Role in Emerging HTA Systems.

Background: Patient-reported outcomes (PROs) are increasingly used to demonstrate the value of interventions and support health technology assessment (HTA).

Objective: The objective of this work was to analyze trends regarding PROs in Latin America (LatAm), highlight challenges in the application of PROs in this region, and suggest solutions.

Methods: A team of researchers with expertise in PROs conducted a nonsystematic PubMed literature search pertaining to the use of PROs in LatAm.

Overcoming Organizational Challenges of Integrating Patient-Reported Outcomes in Oncology Clinical Trials.

Patients with cancer frequently experience multiple symptoms that may cause significant distress and may impair physical, emotional, and social functioning and health-related quality of life. Drug development in oncology is characterized by a high attrition rate of new compounds, faster development times encouraged by the regulatory process, studies that are often open and single-arm, and emphasis on survival-related endpoints, creating unique challenges for the inclusion of patient reported outcomes (PROs). These challenges to include PRO-related endpoints in oncology research are further exacerbated by downward pressure on budget and resources and also an overly rigorous application of the US Food and Drug Administration's PRO guidance, which can in turn prevent study teams from optimally including PROs in oncology clinical trials.

Promotion of Patient-Reported Outcome Label Claims Based on Nonprimary Endpoints.

Introduction: Patient-reported outcomes (PROs) play an increasingly pivotal role in confirmatory clinical trials with pressures to develop drug differentiation strategies. Claims based on primary endpoints that are included in the product label are likely to be fully promoted by the manufacturers; however, the extent to which manufacturers promote claims based on secondary PRO endpoints is unknown. The purpose of this review is to assess the extent of promotion of PRO label claims for 6 pharmaceutical products with nonprimary PRO endpoints.

Psychometric evaluation of the Sheehan Disability Scale in adult patients with attention-deficit/hyperactivity disorder.

Inattention and impulsivity symptoms are common among adults with attention-deficit/hyperactivity disorder (ADHD), which can lead to difficulty concentrating, restlessness, difficulty completing tasks, disorganization, impatience, and impulsiveness. Many adults with ADHD find it difficult to focus and prioritize. Resulting outcomes, such as missed deadlines and forgotten engagements, may ultimately impact the ability to function at work, school, home, or in a social environment.

Assessment of PRO label claims granted by the FDA as compared to the EMA (2006-2010).

Background: The US Food and Drug Administration (FDA) provides formal guidance for the use of patient-reported outcomes (PROs) in support of labeling claims, whereas the European Medicines Agency (EMA) offers insight in a reflection paper relating to health-related quality of life in lieu of formal guidance.

Objectives: PRO label claims granted for new molecular entities and biologic license applications from 2006 through 2010 were reviewed to evaluate consistencies and discrepancies in PRO label claims granted by the FDA and the EMA and to highlight trends in the acceptance of PRO claims across agencies.

Methods: Products approved by both the FDA and the EMA were identified.

Role of Patient-Reported Outcome Measures in the Assessment of Central Nervous System Agents.

This work aimed to provide an understanding of the current use and regulatory acceptability of patient-reported outcome (PRO) measures in labeling claims for central nervous system (CNS) agents. A subset of CNS agents was identified from all New Drug Approvals and Biologic License Applications for new drugs approved in the US from January 2006 to June 2012. Clinician-reported outcomes (ClinROs) (62%) and PROs (38%) were the most widely used primary outcome measures.

US FDA patient-reported outcome guidance: great expectations and unintended consequences.

Release of the US FDA patient-reported outcome (PRO) guidance raised expectations within the pharmaceutical industry for the use of PRO measures in support of labeling claims. The FDA developed the guidance with admirable intent, and the recommendations within this document are based on sound scientific principles. However, implementation of the guidance has been somewhat inconsistent within the Study Endpoints and Label Development (SEALD) and across the various FDA-reviewing divisions.

Potential of patient-reported outcomes as nonprimary endpoints in clinical trials.

Background: The purpose of this research was to fully explore the impact of endpoint type (primary vs. nonprimary) on decisions related to patient-reported outcome (PRO) labeling claims supported by PRO measures and to determine if nonprimary PRO endpoints are being fully optimized.This review examines the use of PROs as both primary and nonprimary endpoints in support of demonstration of treatment benefit of new molecular entities (NMEs) and biologic license applications (BLAs) in the United States in the years 2000 to 2012.

Integration of patient-reported outcomes in multiregional confirmatory clinical trials.

Introduction: The increasing complexities of conducting multiregional trials and an evolving regulatory environment contribute to unprecedented new challenges for use of patient-reported outcome measures (PROMs)(1) within clinical trials. This paper presents these challenges and potential solutions.

Methods: Real-world examples and situations are reviewed from an industry and patient-reported outcome (PRO)(2) expert position.

Proxy-reported questionnaires for young children with asthma: a structured review.

Which proxy-reported outcome measures have been developed for use with children aged 6 years and younger to assess asthma symptoms, asthma control, and asthma-specific health-related quality of life, and do these questionnaires' measurement properties support their use as end-points in clinical trials? A two-phase literature search was conducted: 1) studies describing relevant questionnaires were identified, and the questionnaires were assessed against predefined criteria; 2) studies providing information on the measurement properties of questionnaires meeting the predefined inclusion criteria were identified. Literature sources included PubMed and EMBASE databases, scientific conference proceedings, a clinical trial registry, and a quality of life instrument database. The initial search of literature databases and conference abstracts identified 631 records.