Primary Sjögren's Disease: a review of unmet need, outcome measures, therapeutic advances and health economic impacts. Lessons from the NEw Clinical Endpoints in primary Sjögren's Syndrome: an Interventional Trial based on stratifYing patients (NECESSITY) Innovative Health Initiative (IHI).

Primary Sjögren's disease (pSjD) is an autoimmune rheumatic disease involving exocrine glands and associated with high symptom burden (dryness, fatigue, pain), systemic features and salivary gland dysfunction. B-cell hyperactivity is common, with an increased risk of mucosa-associated lymphoid tissue lymphoma. This review describes the unmet need, scientific validity of outcome measures, optimisation of clinical trial design, therapeutic advances and how clinical improvement relates to health-related quality of life, additional quality-adjusted life years and economic benefit in pSjD.

Epidemiology of Sjögren's: A Systematic Literature Review.

Introduction: Primary Sjögren's is a multi-system autoimmune disease affecting patients' physical, mental, and emotional wellbeing. The epidemiology of Sjögren's is not well understood, and up-to-date epidemiological evidence is needed to improve knowledge and awareness of Sjögren's among patients and healthcare professionals, and to ascertain the global burden of disease. The objective of this research was to conduct a de novo systematic literature review (SLR) to identify and synthesise evidence on global epidemiology of primary Sjögren's.

Development and testing of an alternative responder definition for EULAR Sjögren's Syndrome Patient Reported Index (ESSPRI).

Objectives: Dryness, fatigue and joint/muscle pain are typically assessed in Sjögren's trials using European Alliance of Associations for Rheumatology Sjögren's Syndrome Patient Reported Index (ESSPRI). A Patient Acceptable Symptom State of <5 and a Minimal Clinically Important Improvement (MCII)/responder definition (RD) of ≥1 point or 15% on ESSPRI have previously been defined. This study explored alternative RDs to better discriminate between active treatment and placebo in trials.

Patient Experience of Sjögren's Disease and its Multifaceted Impact on Patients' Lives.

Introduction: The symptoms associated with Sjögren's disease (Sjögren's) are well-documented from the physician's perspective. However, from the patient's perspective, there is limited information on symptoms and their impact on health-related quality of life (HRQoL). This study aimed to provide an expanded understanding of patients' experience of Sjögren's and how symptoms impact HRQoL using a novel multi-method social media listening (SML) approach.

Content Validity of Sjögren's Syndrome Symptom Diary and Functional Assessment of Chronic Illness Therapy-Fatigue in Patients with Sjögren's.

Introduction: Sjögren's Syndrome Symptom Diary (SSSD) and Functional Assessment of Chronic Illness Therapy-Fatigue (FACIT-F) are patient-reported outcome (PRO) instruments assessing Sjögren's symptoms. Original SSSD items have demonstrated content validity, however qualitative evidence supporting the updated 'tiredness' item and two new supplementary items is lacking. Although well established and validated in other rheumatic diseases, there is no qualitative evidence supporting content validity of FACIT-F in Sjögren's.

Qualitative Research with Patients and Physicians to Assess Content Validity and Meaningful Change on ESSDAI and ESSPRI in Sjögren's.

Introduction: European Alliance of Associations for Rheumatology (EULAR) Sjögren's Syndrome Disease Activity Index (ESSDAI) is a clinician-reported outcome (ClinRO) instrument, assessing Sjögren's disease activity from the physician perspective. EULAR Sjögren's Syndrome Patient Reported Index (ESSPRI) is a patient-reported outcome (PRO) instrument, assessing patient-defined Sjögren's symptom severity. Both instruments are commonly used as clinical trial endpoints and have been psychometrically validated.

Patient Preferences in the Management of Wet Age-Related Macular Degeneration: A Conjoint Analysis.

Introduction: To identify patient preference drivers related to the management of wet age-related macular degeneration (wet AMD).

Methods: In this cross-sectional study, a self-explicated 'conjoint analysis' survey was administered online to eligible patients with wet AMD (receiving anti-vascular endothelial growth factor [VEGF] treatment for at least 12 months) from the USA, Canada, UK, France, Spain, Germany, Italy, Japan, Taiwan, and Australia. The survey consisted of six domains with 21 attributes, which were selected on the basis of a literature review, social media listening, and tele-interviews/discussions with patients, clinical experts, and patient groups.

Reliability of a Novel Hematological Malignancy Specific Patient-Reported Outcome Measure: HM-PRO.

Background: Patients' experience of symptoms often goes undetected during consultation in an outpatient clinic, and the use of a patient-reported outcome measure (PRO) in such a setting could be useful to aid treatment decision-making. A new PRO measure, the HM-PRO (Hematological Malignancy Specific Patient-Reported Outcome Measure) has been recently developed to evaluate hematological malignancy (HM) patients' health-related quality of life (HRQoL) and their symptom experience in daily clinical practice as well as in research. The objectives of the study were to assess: the internal consistency of the scores for Part A (impact) and its four domains (physical behavior; social well-being; emotional behavior; and eating and drinking habits) and Part B (signs and symptoms); and the test-retest reliability of the individual items of the newly developed hematological malignancy specific composite measure, the HM-PRO.

Quality-of-life issues and symptoms reported by patients living with haematological malignancy: a qualitative study.

Background: Our aim was to identify health-related quality-of-life (HRQoL) issues and symptoms in patients with haematological malignancies (HMs) and develop a conceptual framework to reflect the inter-relation between them.

Methods: A total of 129 patients with HMs were interviewed in a UK multicentre qualitative study. All interviews were audio recorded, transcribed and analysed using NVivo-11.

Hematological Malignancy Specific Patient-Reported Outcome Measure (HM-PRO): Construct Validity Study.

Background: Validity is the ability of an instrument to measure what it claims to measure. It means the degree to which the empirical evidence supports the trustworthiness of interpretations based on the calculated scores. The hematological malignancy (HM) specific patient reported outcome measure (HM-PRO), is a newly developed instrument for use in daily clinical practice as well as in research.

Development of a Novel Hematological Malignancy Specific Patient-Reported Outcome Measure (HM-PRO): Content Validity.

Background: The quality of life of patients at all stages of hematological malignancy is greatly affected by the disease and its treatment. There is a wide range of health-related quality of life (HRQoL) issues important to these patients. Any new instrument developed to measure HRQoL of such patients should be content valid, i.

Paper and electronic versions of HM-PRO, a novel patient-reported outcome measure for hematology: an equivalence study.

To determine measurement equivalence of paper and electronic application of the hematological malignancy-patient-reported outcome (HM-PRO), a specific measure for the evaluation of patient-reported outcomes in HMs. Following International Society of Pharmacoeconomics and Outcomes Research ePRO Good Research Practice Task Force guidelines, a total of 193 adult patients with different HMs were recruited into a multicenter prospective study. The paper and the electronic version of the instrument were completed in the outpatient clinics in a randomized crossover design with a 30 min time interval to minimize the learning effect.

Haematological malignancy: Are we measuring what is important to patients? A systematic review of quality-of-life instruments.

The wide range of health-related quality-of-life (HRQoL) instruments used in haematology makes it challenging for haematologists and other care team members in practice to select, use and understand the scoring system and finally interpret the results. The main objectives of this study were to: (a) provide a comprehensive list of quality-of-life issues important to patients suffering from haematological malignancies, identified through the literature; (b) provide a list of health-related quality-of-life (HRQoL) instruments used in haematological malignancies in both daily clinical practice and research; and (c) evaluate the relevance and comprehensibility of the identified instruments in haematological malignancies. Systematic literature review of two databases, followed by addition of articles by manual searching, was carried out.

Economic analysis of paliperidone long-acting injectable for chronic schizophrenia in Portugal.

Objective: Patients with chronic schizophrenia suffer a huge burden, as do their families/caregivers. Treating schizophrenia is costly for health systems. The European Medicines Agency has approved paliperidone palmitate (PP-LAI; Xeplion), an atypical antipsychotic depot; however, its pharmacoeconomic profile in Portugal is unknown.

Pharmacoeconomics of long-acting atypical antipsychotics for acutely relapsed chronic schizophrenia in Finland.

Background: Atypical long-acting injectable (LAI) antipsychotics are increasingly available for treating chronic schizophrenia in patients chronically non-adherent to prescribed regimens. Few economic studies have compared these products.

Purpose: To determine the cost-effectiveness of aripiprazole (ARI-LAI), paliperidone (PP-LAI), olanzapine (OLZ-LAI), and risperidone (RIS-LAI) in patients with chronic schizophrenia in Finland.