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      Primary Immune Deficiency: Patients’ Preferences for Replacement Immunoglobulin Therapy

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          Abstract

          Purpose

          Immunoglobulin (Ig) replacement therapy is an important life-saving treatment modality for patients with primary antibody immune deficiency disorders (PAD). IVIG and SCIg are suitable alternatives to treat patients with PAD but vary in key ways. Existing evidence on patient preferences for Ig treatments given the complexities associated with IVIG and SCIg treatment is limited and fails to account for variations in preferences across patients. For this reason, we sought to evaluate PAD patient preferences for features of IVIG and SCIg across different patient characteristics.

          Materials and Methods

          119 PAD patients completed a discrete-choice experiment (DCE) survey. The DCE asked respondents to make choices between carefully constructed treatment alternatives described in terms of generic treatment features. Choices from the DCE were analyzed to determine the relative influence of attribute changes on treatment preferences. We used subgroup analysis to evaluate systematic variations in preferences by patients’ age, gender, time since diagnosis, and treatment experience.

          Results

          Patients were primarily concerned about the duration of treatment side effects, but preferences were heterogeneous. This was particularly true around administration features. Time since diagnosis was associated with an increase in patients’ concerns with the number of needles required per infusion. Also, patients appear to prefer the kind of therapy they are currently using which could be the result of properly aligned patient preferences or evidence of patient adaptive behavior.

          Conclusions

          Heterogeneity in preferences for Ig replacement treatments suggests that a formal shared decision making process could have an important role in improving patient care.

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          Most cited references26

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          Conjoint analysis applications in health--a checklist: a report of the ISPOR Good Research Practices for Conjoint Analysis Task Force.

          The application of conjoint analysis (including discrete-choice experiments and other multiattribute stated-preference methods) in health has increased rapidly over the past decade. A wider acceptance of these methods is limited by an absence of consensus-based methodological standards. The International Society for Pharmacoeconomics and Outcomes Research (ISPOR) Good Research Practices for Conjoint Analysis Task Force was established to identify good research practices for conjoint-analysis applications in health. The task force met regularly to identify the important steps in a conjoint analysis, to discuss good research practices for conjoint analysis, and to develop and refine the key criteria for identifying good research practices. ISPOR members contributed to this process through an extensive consultation process. A final consensus meeting was held to revise the article using these comments, and those of a number of international reviewers. Task force findings are presented as a 10-item checklist covering: 1) research question; 2) attributes and levels; 3) construction of tasks; 4) experimental design; 5) preference elicitation; 6) instrument design; 7) data-collection plan; 8) statistical analyses; 9) results and conclusions; and 10) study presentation. A primary question relating to each of the 10 items is posed, and three sub-questions examine finer issues within items. Although the checklist should not be interpreted as endorsing any specific methodological approach to conjoint analysis, it can facilitate future training activities and discussions of good research practices for the application of conjoint-analysis methods in health care studies. Copyright © 2011 International Society for Pharmacoeconomics and Outcomes Research (ISPOR). Published by Elsevier Inc. All rights reserved.
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            Constructing experimental designs for discrete-choice experiments: report of the ISPOR Conjoint Analysis Experimental Design Good Research Practices Task Force.

            Stated-preference methods are a class of evaluation techniques for studying the preferences of patients and other stakeholders. While these methods span a variety of techniques, conjoint-analysis methods-and particularly discrete-choice experiments (DCEs)-have become the most frequently applied approach in health care in recent years. Experimental design is an important stage in the development of such methods, but establishing a consensus on standards is hampered by lack of understanding of available techniques and software. This report builds on the previous ISPOR Conjoint Analysis Task Force Report: Conjoint Analysis Applications in Health-A Checklist: A Report of the ISPOR Good Research Practices for Conjoint Analysis Task Force. This report aims to assist researchers specifically in evaluating alternative approaches to experimental design, a difficult and important element of successful DCEs. While this report does not endorse any specific approach, it does provide a guide for choosing an approach that is appropriate for a particular study. In particular, it provides an overview of the role of experimental designs for the successful implementation of the DCE approach in health care studies, and it provides researchers with an introduction to constructing experimental designs on the basis of study objectives and the statistical model researchers have selected for the study. The report outlines the theoretical requirements for designs that identify choice-model preference parameters and summarizes and compares a number of available approaches for constructing experimental designs. The task-force leadership group met via bimonthly teleconferences and in person at ISPOR meetings in the United States and Europe. An international group of experimental-design experts was consulted during this process to discuss existing approaches for experimental design and to review the task force's draft reports. In addition, ISPOR members contributed to developing a consensus report by submitting written comments during the review process and oral comments during two forum presentations at the ISPOR 16th and 17th Annual International Meetings held in Baltimore (2011) and Washington, DC (2012). Copyright © 2013 International Society for Pharmacoeconomics and Outcomes Research (ISPOR). Published by Elsevier Inc. All rights reserved.
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              Statistical Methods for the Analysis of Discrete Choice Experiments: A Report of the ISPOR Conjoint Analysis Good Research Practices Task Force.

              Conjoint analysis is a stated-preference survey method that can be used to elicit responses that reveal preferences, priorities, and the relative importance of individual features associated with health care interventions or services. Conjoint analysis methods, particularly discrete choice experiments (DCEs), have been increasingly used to quantify preferences of patients, caregivers, physicians, and other stakeholders. Recent consensus-based guidance on good research practices, including two recent task force reports from the International Society for Pharmacoeconomics and Outcomes Research, has aided in improving the quality of conjoint analyses and DCEs in outcomes research. Nevertheless, uncertainty regarding good research practices for the statistical analysis of data from DCEs persists. There are multiple methods for analyzing DCE data. Understanding the characteristics and appropriate use of different analysis methods is critical to conducting a well-designed DCE study. This report will assist researchers in evaluating and selecting among alternative approaches to conducting statistical analysis of DCE data. We first present a simplistic DCE example and a simple method for using the resulting data. We then present a pedagogical example of a DCE and one of the most common approaches to analyzing data from such a question format-conditional logit. We then describe some common alternative methods for analyzing these data and the strengths and weaknesses of each alternative. We present the ESTIMATE checklist, which includes a list of questions to consider when justifying the choice of analysis method, describing the analysis, and interpreting the results.
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                Author and article information

                Contributors
                URI : https://loop.frontiersin.org/people/1137825
                Journal
                Front Immunol
                Front Immunol
                Front. Immunol.
                Frontiers in Immunology
                Frontiers Media S.A.
                1664-3224
                04 February 2022
                2022
                : 13
                : 827305
                Affiliations
                [1] 1 Department of Population Health Sciences, Duke University , Durham, NC, United States
                [2] 2 Division of Allergy & Immunology, Department of Pediatrics, Morsani College of Medicine, University of South Florida , Tampa, FL, United States
                [3] 3 Johns Hopkins All Children’s Hospital , Saint Petersburg, FL, United States
                [4] 4 Kenan-Flagler Business School, University of North Carolina at Chapel Hill , Chapel Hill, NC, United States
                [5] 5 Scientific & Medical Affairs, Global Health Economics and Outcomes Research, Grifols SSNA , Durham, NC, United States
                Author notes

                Edited by: Paul J. Maglione, Boston University, United States

                Reviewed by: Elham Hossny, Ain Shams University, Egypt; Hugo Chapdelaine, Montreal Clinical Research Institute (IRCM), Canada

                *Correspondence: Juan Marcos Gonzalez, jm.gonzalez@ 123456duke.edu

                This article was submitted to Primary Immunodeficiencies, a section of the journal Frontiers in Immunology

                Article
                10.3389/fimmu.2022.827305
                8854788
                35185918
                e19a1db3-29bd-47f5-ba27-a249200d434d
                Copyright © 2022 Gonzalez, Ballow, Fairchild and Runken

                This is an open-access article distributed under the terms of the Creative Commons Attribution License (CC BY). The use, distribution or reproduction in other forums is permitted, provided the original author(s) and the copyright owner(s) are credited and that the original publication in this journal is cited, in accordance with accepted academic practice. No use, distribution or reproduction is permitted which does not comply with these terms.

                History
                : 01 December 2021
                : 13 January 2022
                Page count
                Figures: 5, Tables: 2, Equations: 0, References: 26, Pages: 10, Words: 4717
                Funding
                Funded by: Grifols , doi 10.13039/501100016387;
                Categories
                Immunology
                Original Research

                Immunology
                primary immune deficiency disorders,immunoglobulin replacement therapy,ivig,scig,patient preferences,discrete-choice experiment

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