[HTML][HTML] Targeting the alternative complement pathway with iptacopan to treat IgA nephropathy: design and rationale of the APPLAUSE-IgAN study

DV Rizk, BH Rovin, H Zhang, N Kashihara… - Kidney international …, 2023 - Elsevier
Introduction Targeting the alternative complement pathway (AP) is an attractive therapeutic
strategy because of its role in immunoglobulin A nephropathy (IgAN) pathophysiology …

[HTML][HTML] Quantitative benefit-risk assessment in medical product decision making: a good practices report of an ISPOR task force

T Tervonen, J Veldwijk, K Payne, X Ng, B Levitan… - Value in Health, 2023 - Elsevier
Benefit-risk assessment is commonly conducted by drug and medical device developers
and regulators, to evaluate and communicate issues around benefit-risk balance of medical …

How to integrate evidence from patient preference studies into health technology assessment: a critical review and recommendations

K Marsh, E de Bekker-Grob, N Cook… - International journal of …, 2021 - cambridge.org
Health technology assessment (HTA) agencies vary in their use of quantitative patient
preference data (PP) and the extent to which they have formalized this use in their …

A discrete choice experiment on oral and injection treatment preferences among moderate‐to‐severe psoriasis patients in Japan

M Komine, H Kim, J Yi, Y Zhong, Y Sakai… - The Journal of …, 2023 - Wiley Online Library
Long‐term psoriasis (PsO) management remains challenging. With growing variation in
treatment efficacy, cost, and modes of administration, patient preferences for different …

Treatment preferences in spinal muscular atrophy: A swing weighting study for caregivers of patients with SMA types 1 and 2

A Patel, W Toro, S Bourke, Y Oluboyede, S Barbier… - Plos one, 2024 - journals.plos.org
Spinal muscular atrophy (SMA) is a genetic neuromuscular disorder characterized by
skeletal muscle weakness and atrophy. Patients with SMA types 1 and 2 develop severe …

The humanistic burden of immunoglobulin A nephropathy on patients and care-partners in the United States

J Szklarzewicz, U Floege, D Gallego, K Gibson… - Quality of Life …, 2024 - Springer
Purpose This cross-sectional survey study quantified the humanistic burden of
immunoglobulin A nephropathy (IgAN), in terms of physical and mental health-related …

Clinical and Humanistic Burden of IgA Nephropathy in Adult Patients: A Global Real-World Survey

SCW Tang, W Chen, C Aldworth, AT George… - Kidney360, 2025 - journals.lww.com
Background IgA nephropathy (IgAN) is a chronic, progressive kidney disease in which
proteinuria, reduced eGFR, pain, and fatigue are common. How symptoms interact and …

Development of a disease-specific health utility score for chronic obstructive pulmonary disease from a discrete choice experiment patient preference study

B Jones, M Ryan, NS Cook… - International Journal of …, 2024 - cambridge.org
ObjectivesWhile patient input to health technology assessment (HTA) has traditionally been
of a qualitative nature, there is increasing interest to integrate quantitative evidence from …

Humanistic and economic burden of IgA nephropathy: systematic literature reviews and narrative synthesis

KD Jhaveri, ME Bensink, M Bunke, JA Briggs… - PharmacoEconomics …, 2023 - Springer
Abstract Background Immunoglobulin A nephropathy (IgAN) is a progressive inflammatory
kidney disease requiring long-term treatment to reduce the risk of progression to kidney …

Introduction to Patient Preference Studies

B Jones, C Berlin, N Cook, S Dickinson, T Lyu… - … : Clinical Trial Analysis …, 2024 - Springer
This chapter begins with a brief introduction to PFDD (Patient-Focused Drug Development)
and the role a statistician can play in the design and analysis of Patient Preference Studies …