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Patient-centered benefit-risk assessment in Duchenne Muscular Dystrophy

Published on May 1, 2017in Muscle & Nerve2.393
· DOI :10.1002/mus.25411
Ilene L. Hollin10
Estimated H-index: 10
(Johns Hopkins University),
Holly L. Peay15
Estimated H-index: 15
(RTP: Research Triangle Park)
+ 1 AuthorsJohn F. P. Bridges29
Estimated H-index: 29
(Johns Hopkins University)
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 (32)
  • Citations (14)
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References32
Newest
#1Ilene L. Hollin (Johns Hopkins University)H-Index: 10
#2Caroline YoungH-Index: 1
Last. Holly L. Peay (RTP: Research Triangle Park)H-Index: 15
view all 5 authors...
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 ...
20 CitationsSource
#1Nina L. Hunter (CDRH: Center for Devices and Radiological Health)H-Index: 1
#2Kathryn M. O’Callaghan (CDRH: Center for Devices and Radiological Health)H-Index: 5
Last. Robert M. Califf (FDA: Food and Drug Administration)H-Index: 8
view all 3 authors...
24 CitationsSource
#1Ilene L. Hollin (Johns Hopkins University)H-Index: 10
#2Holly L. Peay (Johns Hopkins University)H-Index: 15
Last. John F. P. Bridges (Johns Hopkins University)H-Index: 29
view all 3 authors...
26 CitationsSource
#1Martin Ho (FDA: Food and Drug Administration)H-Index: 1
#2Juan Marcos Gonzalez (Durham University)H-Index: 11
Last. Telba Irony (FDA: Food and Drug Administration)H-Index: 3
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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.
86 CitationsSource
#1Holly L. PeayH-Index: 15
#2Ilene L. Hollin (Johns Hopkins University)H-Index: 10
Last. John F. P. Bridges (Johns Hopkins University)H-Index: 29
view all 4 authors...
59 CitationsSource
#1Janine Astrid van Til (UT: University of Twente)H-Index: 15
#2Maarten Joost IJzerman (UT: University of Twente)H-Index: 40
26 CitationsSource
#1V. Ricotti (ICH: UCL Institute of Child Health)H-Index: 5
#2Deborah Ridout (ICH: UCL Institute of Child Health)H-Index: 17
Last. Francesco Muntoni (UCL: University College London)H-Index: 99
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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...
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#1F. Reed Johnson (RTP: Research Triangle Park)H-Index: 36
#2Emily Lancsar (Monash University)H-Index: 22
Last. John F. P. Bridges (Johns Hopkins University)H-Index: 29
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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...
468 CitationsSource
#1F. Reed JohnsonH-Index: 36
#2Emily LancsarH-Index: 22
Last. John F. P. BridgesH-Index: 29
view all 9 authors...
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...
377 CitationsSource
#1Elizabeth T. Kinter (Johns Hopkins University)H-Index: 7
#2Thomas J. Prior (Johns Hopkins University)H-Index: 3
Last. John F. P. Bridges (Johns Hopkins University)H-Index: 29
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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.
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#1Laurent ServaisH-Index: 21
#2Chiara S. M. Straathof (LUMC: Leiden University Medical Center)H-Index: 14
Last. Gunnar Buyse (Katholieke Universiteit Leuven)H-Index: 29
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Abstract Decline in respiratory function in patients with DMD starts during early teenage years and leads to early morbidity and mortality. Published evidence of efficacy for idebenone on respiratory function outcomes is currently limited to 12 months of follow-up time. Here we report data collected as retrospective cohort study (SYROS) from 18 DMD patients not using glucocorticoids who were treated with idebenone (900 mg/day) under Expanded Access Programs (EAPs). The objective was to assess th...
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#1Alison M. Barnard (UF: University of Florida)H-Index: 1
#2Samuel L. Riehl (UF: University of Florida)H-Index: 1
Last. Krista Vandenborne (UF: University of Florida)H-Index: 42
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Observational research benefits from inclusion of diverse cohorts. To characterize racial and ethnic diversity in observational and natural history research studies of Duchenne muscular dystrophy (DMD), highly cited and influential observational studies were identified. Fourteen United States-based articles were included. All studies cited >70% White participants with the majority having few racial minority participants. Enrollment of Black/African American individuals was particularly limited (...
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#1Eline van Overbeeke (Katholieke Universiteit Leuven)H-Index: 3
#2Rosanne Janssens (Katholieke Universiteit Leuven)H-Index: 1
Last. Isabelle Huys (Katholieke Universiteit Leuven)H-Index: 21
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Objectives: To investigate stakeholder perspectives on how patient preference studies (PPS) should be designed and conducted to allow for inclusion of patient preferences in decision-making along the medical product life cycle (MPLC), and how patient preferences can be used in such decision-making. Methods: Two literature reviews and semi-structured interviews (n=143) with healthcare stakeholders in Europe and the US were conducted; results of these informed the design of focus group guides. Eig...
1 CitationsSource
#1Rosanne Janssens (Katholieke Universiteit Leuven)H-Index: 1
#2Isabelle Huys (Katholieke Universiteit Leuven)H-Index: 21
Last. Jorien Veldwijk (EUR: Erasmus University Rotterdam)H-Index: 6
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The inclusion of patient preferences (PP) in the medical product life cycle is a topic of growing interest to stakeholders such as academics, Health Technology Assessment (HTA) bodies, reimbursement agencies, industry, patients, physicians and regulators. This review aimed to understand the potential roles, reasons for using PP and the expectations, concerns and requirements associated with PP in industry processes, regulatory benefit-risk assessment (BRA) and marketing authorization (MA), and H...
1 CitationsSource
#1C Whichello (EUR: Erasmus University Rotterdam)H-Index: 2
#2Eline van Overbeeke (Katholieke Universiteit Leuven)H-Index: 3
Last. Esther W. de Bekker-Grob (EUR: Erasmus University Rotterdam)H-Index: 24
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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...
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#1Hideo Aizaki (Hokkaido University)H-Index: 1
#2James Fogarty (UWA: University of Western Australia)H-Index: 10
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...
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#1Grazia Crescimanno (National Research Council)H-Index: 7
#2Francesca GrecoH-Index: 4
Last. Oreste Marrone (National Research Council)H-Index: 23
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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. By this questionnaire, different aspects of QoL were assessed on a scale from 0 (best) to 100 ...
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#1Ryan S. Paquin (RTP: Research Triangle Park)H-Index: 4
#2Ryan FischerH-Index: 2
Last. Holly L. Peay (RTP: Research Triangle Park)H-Index: 15
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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.
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#1Holly L. PeayH-Index: 15
#2Ryan FischerH-Index: 2
Last. Carol MansfieldH-Index: 13
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#1Rosangel CruzH-Index: 5
#2L. BelterH-Index: 1
Last. Jill JareckiH-Index: 7
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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...
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