Brian Bekker Hansen

Patient-reported outcome measures for systemic lupus erythematosus clinical trials: a review of content validity, face validity and psychometric performance

BackgroundDespite overall progress in treatment of autoimmune diseases, patients with systemic lupus erythematosus (SLE) experience many inflammatory symptoms representing an unmet medical need. This study aimed to create a conceptual model of the humanistic and economic burden of SLE, and review the patient-reported outcomes (PROs) used to measure such concepts in SLE clinical trials.MethodsA conceptual model for SLE was developed from structured review of published articles from 2007 to August 2013 identified from literature databases (MEDLINE, EMBASE, PsycINFO, EconLit) plus other sources (PROLabels, FDA/EMA websites, Clinicaltrials.gov). PROs targeting key symptoms/impacts were identified from the literature. They were reviewed in the context of available guidance and assessed for face and content validity and psychometric properties to determine appropriateness for use in SLE trials.ResultsThe conceptual model identified fatigue, pain, cognition, daily activities, emotional well-being, physical/social functioning and work productivity as key SLE concepts. Of the 68 articles reviewed, 38 reported PRO data. From these and the other sources, 15 PROs were selected for review, including SLE-specific health-related quality of life (HRQoL) measures (n = 5), work productivity (n = 1), and generic measures of fatigue (n = 3), pain (n = 2), depression (n = 2) and HRQoL (n = 2). The Functional Assessment of Chronic Illness Therapy - Fatigue Scale (FACIT-Fatigue), Brief Pain Inventory (BPI-SF) and LupusQoL demonstrated the strongest face validity, conceptual coverage and psychometric properties measuring key concepts in the conceptual model. All PROs reviewed, except for three Lupus-specific measures, lacked qualitative SLE patient involvement during development. The Hospital Anxiety and Depression Scale (HADS), Short Form [36 item] Health Survey version 2 (SF-36v2), EuroQoL 5-dimensions (EQ-5D-3L and EQ-5D-5L) and Work Productivity and Activity Impairment Questionnaire: Lupus (WPAI:Lupus) showed suitability for SLE economic models.ConclusionsBased on the identification of key symptoms and impacts of SLE using a scientifically sound conceptual model, we conclude that SLE is a condition associated with high unmet need and considerable burden to patients. This review highlights the availability and need for disease-specific and generic patient-reported measures of relevant domains of disease signs and symptoms, HRQoL and work productivity, providing useful insight for SLE clinical trial design.

Efficacy and Safety of Anti–Interleukin‐20 Monoclonal Antibody in Patients With Rheumatoid Arthritis: A Randomized Phase IIa Trial

Interleukin‐20 (IL‐20) is implicated in the pathogenesis of rheumatoid arthritis (RA). The efficacy, safety, and tolerability of NNC0109‐0012, a selective anti–IL‐20 recombinant human monoclonal antibody (mAb), were assessed in patients with active RA who had an inadequate response to methotrexate therapy.

The Economic and Health-related Impact of Crohn's Disease in the United States: Evidence from a Nationally Representative Survey.

Background:Approximately 593,000 to 780,000 people in the United States (US) have been diagnosed with Crohns disease (CD), and an additional 33,000 are diagnosed yearly. Our objective was to estimate CDs impact on medical costs, lost earnings, work and school absences, health status, and health-related quality of life (HRQOL) in the US and to compute current and forecasted national costs. Methods:We used the nationally representative Medical Expenditure Panel Survey to match 539 respondents with CD to similar respondents without any inflammatory bowel disease (IBD). We estimated annual costs, work and school absences, and self-assessed health status. HRQOL was assessed by the SF-12 Physical Component Summary and Mental Component Summary (PCS and MCS) scores. CD prevalence rates, population counts, and costs were used to forecast total national costs. Results:CD is associated with higher medical costs (

Development of a conceptual model evaluating the humanistic and economic burden of Crohn’s disease: implications for patient-reported outcomes measurement and economic evaluation

13,446 versus

Cost-Effectiveness of Insulin Degludec/Insulin Aspart Versus Biphasic Insulin Aspart in Patients with Type 2 Diabetes from a Danish Health-Care Perspective

6029) and lost earnings (

PROMs for Systemic Lupus Erythematosus

1249 versus

Key data elements for use in cost-utility modeling of biological treatments for rheumatoid arthritis

644) and is responsible for

SAT0112 Improvements in Patient-Reported Physical Function, Pain and Global Disease Activity in Patients with Rheumatoid Arthritis after Treatment with Nnc0109-0012 (Anti-IL-20 Mab) in a Phase 2A Trial

3.48 billion in total national costs (expected to increase to

Exploring the Humanistic and Economic Burden of Crohnʼs Disease: Considerations for Novel Compounds: P-68

3.72 billion in 2025). Respondents with CD were more likely to miss work (38% versus 33%) or school (64% versus 33%), less likely to report being in excellent or very good physical health (24% versus 63%), and experienced lower HRQOL measured by the Physical Component Summary (43.4 versus 48.5) and Mental Component Summary (48.6 versus 50.0) than those without IBD. Conclusions:CD is responsible for increased medical care costs and lower earnings, health status, and HRQOL. These data can serve as benchmarks when examining future CD-related costs and HRQOL.

Linguistic Validation into 20 Languages and Content Validity of the Rheumatoid Arthritis-Specific Work Productivity and Activity Impairment Questionnaire

The primary objective of this review is to develop a conceptual model for Crohn’s disease (CD) outlining the disease burden for patients, healthcare systems and wider society, as reported in the scientific literature. A search was conducted using MEDLINE, PsycINFO, EconLit, Health Economic Evaluation Database and Centre for Reviews and Dissemination databases. Patient-reported outcome (PRO) measures widely used in CD were reviewed according to the US FDA PRO Guidance for Industry. The resulting conceptual model highlights the characterization of CD by gastrointestinal disturbances, extra-intestinal and systemic symptoms. These symptoms impact physical functioning, ability to complete daily activities, emotional wellbeing, social functioning, sexual functioning and ability to work. Gaps in conceptual coverage and evidence of reliability and validity for some PRO measures were noted. Review findings also highlight the substantial direct and indirect costs associated with CD. Evidence from the literature confirms the substantial burden of CD to patients and wider society; however, future research is still needed to further understand burden from the perspective of patients and to accurately understand the economic burden of disease. Challenges with existing PRO measures also suggest the need for future research to refine or develop new measures.