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Patient-centered benefit-risk assessment in Duchenne Muscular Dystrophy

Published on May 1, 2017in Muscle & Nerve2.39
· DOI :10.1002/mus.25411
Ilene L. Hollin9
Estimated H-index: 9
(Johns Hopkins University),
Holly Peay14
Estimated H-index: 14
(RTP: Research Triangle Park)
+ 1 AuthorsJohn F. P. Bridges27
Estimated H-index: 27
(Johns Hopkins University)
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Abstract
Objective This study quantifies caregiver and patient preferences for a therapeutic agent with demonstrated pulmonary benefits for Duchenne muscular dystrophy (DMD). Caregivers and patient differences are also explored. Methods A best-worst scaling survey (BWS) was administered to caregivers and patients. Across 9 profiles, respondents selected the best and worst attributes. Utility scores were estimated using mixed logistic regression. Results Respondents indicated greatest preference for therapies that maintain their current level of cough strength for 10 years or for 2 years. Preference scores for risks were low: 50% chance of diarrhea and 4 additional blood draws per year. Conclusion There is a strong preference for pulmonary benefit and willingness to trade off risks and burden to achieve these benefits. In exchange for maintaining cough strength for 10 years, respondents were willing to tolerate high probabilities of diarrhea and additional blood draws. This article is protected by copyright. All rights reserved.
  • References (31)
  • Citations (8)
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References31
Newest
Published on Sep 1, 2016in Value in Health5.04
Ilene L. Hollin9
Estimated H-index: 9
(Johns Hopkins University),
Caroline Young1
Estimated H-index: 1
+ 2 AuthorsHolly Peay14
Estimated H-index: 14
(RTP: Research Triangle Park)
Abstract Objectives To provide a community-engaged process to inform the design of a stated-preferences experiment. The process involved integrating patients and caregivers of people with Duchenne/Becker muscular dystrophy, advocates, clinicians, and the sponsor in conceptualizing and developing a benefit-risk survey on the basis of phase III trial results. Methods Our community-engagement process for the development of a stated-preference survey included a set of five guiding principles with a ...
Published on Dec 15, 2015in JAMA51.27
Nina L. Hunter1
Estimated H-index: 1
(CDRH: Center for Devices and Radiological Health),
Kathryn O’Callaghan2
Estimated H-index: 2
(CDRH: Center for Devices and Radiological Health),
Robert M. Califf6
Estimated H-index: 6
(FDA: Food and Drug Administration)
Published on Oct 1, 2015in Surgical Endoscopy and Other Interventional Techniques3.21
Martin P. Ho1
Estimated H-index: 1
(FDA: Food and Drug Administration),
Juan Marcos Gonzalez13
Estimated H-index: 13
(Durham University)
+ 5 AuthorsTelba Irony2
Estimated H-index: 2
(FDA: Food and Drug Administration)
Background Patients have a unique role in deciding what treatments should be available for them and regulatory agencies should take their preferences into account when making treatment approval decisions. This is the first study designed to obtain quantitative patient-preference evidence to inform regulatory approval decisions by the Food and Drug Administration Center for Devices and Radiological Health.
Ilene L. Hollin9
Estimated H-index: 9
(Johns Hopkins University),
Holly Peay14
Estimated H-index: 14
(Johns Hopkins University),
John F. P. Bridges27
Estimated H-index: 27
(Johns Hopkins University)
Background Through Patient-Focused Drug Development, the US Food and Drug Administration (FDA) documents the perspective of patients and caregivers and are currently conducting 20 public meetings on a limited number of disease areas. Parent Project Muscular Dystrophy (PPMD), an advocacy organization for Duchenne muscular dystrophy (DMD), has demonstrated a community-engaged program of preference research that would complement the FDA’s approach.
Published on May 1, 2014in Clinical Therapeutics2.94
Holly Peay14
Estimated H-index: 14
,
Ilene L. Hollin9
Estimated H-index: 9
(Johns Hopkins University)
+ 1 AuthorsJohn F. P. Bridges27
Estimated H-index: 27
(Johns Hopkins University)
Abstract Background There is growing agreement that regulators performing benefit–risk evaluations should take patients’ and caregivers’ preferences into consideration. The Patient-Focused Drug Development Initiative at the US Food and Drug Administration offers patients and caregivers an enhanced opportunity to contribute to regulatory processes by offering direct testimonials. This process may be advanced by providing scientific evidence regarding treatment preferences through engagement of a ...
Published on Jan 1, 2014in PharmacoEconomics3.71
Janine Astrid van Til14
Estimated H-index: 14
(UT: University of Twente),
Maarten Joost IJzerman39
Estimated H-index: 39
(UT: University of Twente)
Published on Jun 1, 2013in Journal of Neurology, Neurosurgery, and Psychiatry8.27
V. Ricotti11
Estimated H-index: 11
(ICH: UCL Institute of Child Health),
Deborah Ridout16
Estimated H-index: 16
(ICH: UCL Institute of Child Health)
+ 4 AuthorsFrancesco Muntoni96
Estimated H-index: 96
(UCL: University College London)
Objective To assess the current use of glucocorticoids (GCs) in Duchenne muscular dystrophy in the UK, and compare the benefits and the adverse events of daily versus intermittent prednisolone regimens. Design A prospective longitudinal observational study across 17 neuromuscular centres in the UK of 360 boys aged 3–15 years with confirmed Duchenne muscular dystrophy who were treated with daily or intermittent (10 days on/10 days off) prednisolone for a mean duration of treatment of 4 years. Res...
Published on Jan 1, 2013in Value in Health5.04
F. Reed Johnson35
Estimated H-index: 35
,
Emily Lancsar21
Estimated H-index: 21
+ 6 AuthorsJohn F. P. Bridges27
Estimated H-index: 27
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 ava...
Published on Jan 1, 2013in Value in Health5.04
F. Reed Johnson35
Estimated H-index: 35
(RTP: Research Triangle Park),
Emily Lancsar21
Estimated H-index: 21
(Monash University)
+ 6 AuthorsJohn F. P. Bridges27
Estimated H-index: 27
(Johns Hopkins University)
Abstract 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 understandi...
Published on Dec 23, 2012in The Patient: Patient-Centered Outcomes Research2.67
Elizabeth T. Kinter6
Estimated H-index: 6
(Johns Hopkins University),
Thomas J. Prior3
Estimated H-index: 3
(Johns Hopkins University)
+ 1 AuthorsJohn F. P. Bridges27
Estimated H-index: 27
(Johns Hopkins University)
Background While the application of conjoint analysis and discrete-choice experiments in health are now widely accepted, a healthy debate exists around competing approaches to experimental design. There remains, however, a paucity of experimental evidence comparing competing design approaches and their impact on the application of these methods in patient-centered outcomes research.
Cited By8
Newest
Published on Dec 1, 2019in Orphanet Journal of Rare Diseases3.69
Ryan S. Paquin2
Estimated H-index: 2
(RTP: Research Triangle Park),
Ryan Fischer2
Estimated H-index: 2
+ 11 AuthorsValeria Ricotti9
Estimated H-index: 9
Purpose Several gene therapy trials for Duchenne muscular dystrophy initiated in 2018. Trial decision making is complicated by non-curative, time-limited benefits; the progressive, fatal course; and high unmet needs. Here, caregivers and patients prioritize factors influencing decision making regarding participation in early-phase gene therapy trials.
Published on 2019in Frontiers in Pharmacology3.85
C Whichello2
Estimated H-index: 2
(EUR: Erasmus University Rotterdam),
Eline van Overbeeke2
Estimated H-index: 2
(Katholieke Universiteit Leuven)
+ 7 AuthorsJ Kübler1
Estimated H-index: 1
(CSL Behring)
Objectives Patient preference information (PPI) is gaining recognition among the pharmaceutical industry, regulatory authorities, and Health Technology Assessment (HTA) bodies/payers for use in assessments and decision-making along the medical product lifecycle (MPLC). This study aimed to identify factors and situations that influence the value of patient preference studies (PPS) in decision-making along the MPLC according to different stakeholders. Methods Semi-structured interviews (n=143) wer...
Published on Jun 1, 2019in Journal of choice modelling1.91
Hideo Aizaki1
Estimated H-index: 1
(Hokkaido University),
James Fogarty9
Estimated H-index: 9
(UWA: University of Western Australia)
Abstract Case 2 (profile case) best–worst scaling (BWS) is a question-based survey method for measuring preferences for attribute levels. Several existing R packages help to implement the construction of Case 2 BWS questions (profiles) and the discrete choice analysis of the responses to the questions. Structuring the dataset for Case 2 BWS analysis is, however, complicated: there are several model variants for the analysis, and independent variables are set according to the variants. This compl...
Published on Jun 1, 2019in Neuromuscular Disorders2.61
Grazia Crescimanno4
Estimated H-index: 4
(National Research Council),
Francesca Greco4
Estimated H-index: 4
+ 2 AuthorsOreste Marrone21
Estimated H-index: 21
(National Research Council)
Abstract The aim of this study was to evaluate quality of life (QoL) and its possible determinants in patients affected by Duchenne muscular dystrophy (DMD) in late stages of their disease, when non-invasive ventilation (NIV) is already established. Forty-eight DMD patients who were treated by NIV were enrolled. QoL was assessed by the Individualized Neuromuscular Quality of Life (INQoL) questionnaire. In addition to motor and respiratory function, dysautonomia symptoms, sleep quality, sleepines...
Published on May 1, 2019in Clinical Therapeutics2.94
Rosangel Cruz4
Estimated H-index: 4
,
Lisa Belter + 2 AuthorsJill Jarecki7
Estimated H-index: 7
Abstract Purpose Patients’ perceptions of benefit–risk are essential to informing the regulatory process and the context in which potential therapies are evaluated. To bring this critical information to regulators, Cure SMA launched a first-ever Benefit-Risk Survey for spinal muscular atrophy (SMA) to characterize decision-making and benefit–risk trade-offs in SMA associated with a potential therapy. We hypothesized that risk tolerance would be correlated with SMA type/severity and disease progr...
John F. P. Bridges27
Estimated H-index: 27
(Johns Hopkins University),
Jui-Hua Tsai1
Estimated H-index: 1
(Johns Hopkins University)
+ 3 AuthorsHolly Peay14
Estimated H-index: 14
(Johns Hopkins University)
Background Best–worst scaling methods have been used in several Duchenne and Becker muscular dystrophy (DBMD) studies to quantify patient and caregiver priorities and preferences and promote patient-focused drug development (PFDD). We sought to assess the extent to which different members of the DBMD community would accept a discrete-choice experiment (DCE) that incorporates uncertainty regarding individual-level benefit.
Published on Jan 1, 2019in Drug Discovery Today6.88
Eline van Overbeeke2
Estimated H-index: 2
(Katholieke Universiteit Leuven),
C Whichello2
Estimated H-index: 2
(EUR: Erasmus University Rotterdam)
+ 8 AuthorsEsther W. de Bekker-Grob22
Estimated H-index: 22
(EUR: Erasmus University Rotterdam)
textabstractIndustry, regulators, health technology assessment (HTA) bodies, and payers are exploring the use of patient preferences in their decision-making processes. In general, experience in conducting and assessing patient preference studies is limited. Here, we performed a systematic literature search and review to identify factors and situations influencing the value of patient preference studies, as well as applications throughout the medical product lifecyle. Factors and situations iden...
Published on Oct 23, 2018in Journal of neuromuscular diseases
Gunnar Buyse28
Estimated H-index: 28
(Katholieke Universiteit Leuven),
Christian Rummey18
Estimated H-index: 18
+ 4 AuthorsOscar H. Mayer14
Estimated H-index: 14
(Children's Hospital of Philadelphia)
Juan Marcos Gonzalez13
Estimated H-index: 13
(Duke University),
F. Reed Johnson35
Estimated H-index: 35
(Duke University)
+ 2 AuthorsHolly Peay14
Estimated H-index: 14
(RTI International)
View next paperA Community-Engaged Approach to Quantifying Caregiver Preferences for the Benefits and Risks of Emerging Therapies for Duchenne Muscular Dystrophy