Meaningful clinical outcomes: perspectives of primary caregivers with lived experience of spontaneous preterm birth following spontaneous preterm labor.

Background: Patient-centered research is foundational to informing the measurement of clinical benefit in interventional studies. Understanding caregivers' lived experience of spontaneous preterm birth (sPTB) resulting from spontaneous preterm labor (sPTL) and characterizing their perspectives on the clinical outcomes of greatest importance and relevance are essential for advancing the development of interventions for the delay or prevention of sPTB.

Objective: This mixed methods study aimed to identify which clinical outcomes deriving from a (hypothetical) successful sPTL intervention are most important and relevant from the caregiver perspective to advance the therapeutic landscape for sPTB resulting from sPTL.

Designing a Patient Preference Study on Subcutaneous Medical Devices: Incorporating Health Authority Scientific Advice and Patient Perspectives.

This paper describes the planning of a patient preference study for evaluating device features for the subcutaneous (SC) delivery of high dose/large volume (e.g., > 2 ml) of drugs.

Evaluation of validity, reliability and ability to detect change for the Hand Eczema Severity Index (HECSI) and evaluation of HECSI-75 and HECSI-90 as within-patient responder definitions.

Background: The Hand Eczema Severity Index (HECSI) is a Clinician-Reported Outcome measure of the severity of hand eczema (HE).

Objectives: This study aimed to evaluate the validity, reliability and ability to detect change of the HECSI, and the HECSI-75 and HECSI-90 as responder definitions.

Methods: Analyses were performed using data from a sample of n = 258 patients with Chronic Hand Eczema (CHE) from a Phase 2b, randomised, double-blind, vehicle-controlled trial of delgocitinib cream, pooled across treatment groups.

Development and validation of a quality of life and treatment satisfaction measure in canine osteoarthritis.

Introduction: Canine osteoarthritis (OA) causes pain and mobility impairment. This can reduce dog quality of life (QoL), owner QoL and owners' satisfaction with, and adherence to, treatments. No existing canine OA-specific instrument assesses all three impacts.

Qualitative concept elicitation and cognitive debriefing interviews of symptoms, impacts and selected customized PROMIS Short Forms: a study in patients with axial spondyloarthritis.

Background: Sleep disturbance, pain, and fatigue are key symptoms/impacts of axial spondyloarthritis (axSpA). Three customized Patient-Reported Outcomes Measurement Information System (PROMIS) Short Forms (Sleep Disturbance, Pain Interference, and Fatigue) have been proposed for use in axSpA to assess these key disease concepts. This study was designed to further understand the patient experience of axSpA and evaluate the content validity of the three customized PROMIS Short Forms to support their use in axSpA clinical trials.

Comparison of Literature Review, Social Media Listening, and Qualitative Interview Research Methods in Generating Patient-Reported Symptom and Functional Impact Concepts of Presbyopia.

Introduction: To compare the insights obtained about the experience of individuals with presbyopia (age-related impaired near vision) across three different sources of qualitative data: a structured targeted literature review, a social media listening (SML) review, and qualitative concept elicitation (CE) interviews with individuals with presbyopia and healthcare professionals (HCPs). The number of concepts identified, depth of data, cost and time implications, and value of the patient insights generated were explored and compared for each method.

Methods: Keyword searches in bibliographic databases and review of abstracts identified 120 relevant publications; in-depth targeted literature review of the qualitative studies identified key symptoms/functioning concepts.

Interleukin-1 Trap Rilonacept Improved Health-Related Quality of Life and Sleep in Patients With Recurrent Pericarditis: Results From the Phase 3 Clinical Trial RHAPSODY.

Background Recurrent pericarditis is characterized by painful flares and inflammation, which negatively impact health-related quality of life. RHAPSODY (rilonacept inhibition of interleukin-1 alpha and beta for recurrent pericarditis: a pivotal symptomatology and outcomes study) evaluated the efficacy and safety of rilonacept (IL-1α and -β cytokine trap) in recurrent pericarditis. A secondary analysis of these data evaluated the patient-reported outcome questionnaire score change during the trial.

Patient, Family Member and Physician Perspectives and Experiences with AML Treatment Decision-Making.

Introduction: Treatment decisions in older adults with acute myeloid leukemia (AML) are challenging, particularly for those who are not candidates for intensive chemotherapy (IC), and the trade-offs patients, their families and physicians consider when choosing a treatment option are not well understood. This qualitative research explored the value of extending survival and the treatment decision-making process from a multi-stakeholder perspective.

Methods: Overall, 28 patients with AML (≥ 65 years old, unsuitable for IC), 25 of their relatives and 10 independent physicians from the US, UK and Canada took part in one-on-one, 60-minute qualitative interviews.

Health-related quality of life in patients with recurrent pericarditis: results from a phase 2 study of rilonacept.

Background: Impact of recurrent pericarditis (RP) on patient health-related quality of life (HRQoL) was evaluated through qualitative patient interviews and as an exploratory endpoint in a Phase 2 trial evaluating the efficacy and safety of rilonacept (IL-1α/IL-1β cytokine trap) to treat RP.

Methods: Qualitative interviews were conducted with ten adults with RP to understand symptoms and HRQoL impacts, and the 10-item Patient-Reported Outcomes Measurement Information System Global Health (PROMIS GH) v1.2 was evaluated to determine questionnaire coverage of patient experience.

Patient-reported outcome measures in presbyopia: a literature review.

Presbyopia is the age-related loss of near-distance focusing ability. The aim of this study was to identify patient-reported outcome measures (PROMs) used in clinical trials and quality-of-life studies conducted in individuals with presbyopia and to assess their suitability for use in individuals with phakic presbyopia. Literature searches were performed in Medline and Embase up until October 2017.