Patient-centered benefit-risk assessment in duchenne muscular dystrophy

2017 ◽  
Vol 55 (5) ◽  
pp. 626-634 ◽  
Author(s):  
Ilene L. Hollin ◽  
Holly L. Peay ◽  
Susan D. Apkon ◽  
John F.P. Bridges
Keyword(s):  
Risk Assessment ◽  
Duchenne Muscular Dystrophy ◽  
Muscular Dystrophy ◽  
Patient Centered ◽  
Benefit Risk Assessment

Authors’ Response to Comment on “Assessing the Appropriateness of the EQ-5D for Duchenne Muscular Dystrophy: A Patient-Centered Study”

2021 ◽  
pp. 0272989X2110622
Author(s):  
Norah L. Crossnohere ◽  
Ryan Fischer ◽  
Andrew Lloyd ◽  
Lisa A. Prosser ◽  
John F. P. Bridges
Keyword(s):  
Duchenne Muscular Dystrophy ◽  
Muscular Dystrophy ◽  
Patient Centered

Assessing the Appropriateness of the EQ-5D for Duchenne Muscular Dystrophy: A Patient-Centered Study

2021 ◽  
Vol 41 (2) ◽  
pp. 209-221
Author(s):  
Norah L. Crossnohere ◽  
Ryan Fischer ◽  
Andrew Lloyd ◽  
Lisa A. Prosser ◽  
John F. P. Bridges
Keyword(s):  
Health Status ◽  
Duchenne Muscular Dystrophy ◽  
Muscular Dystrophy ◽  
The United States ◽  
Neuromuscular Disorder ◽  
Face Validity ◽  
Health State ◽  
Patient Centered ◽  
Cross Sectional ◽  
Eq Vas

Background Duchenne muscular dystrophy is a rare degenerative neuromuscular disorder with pediatric onset. Recent approvals in Duchenne have placed attention on the economic evaluation in pricing and reimbursement decisions across a range of rare conditions. We sought to assess the appropriateness of the EQ-5D, a common measure of generic health state utility, for use among patients and caregivers affected by Duchenne. Methods An international, cross-sectional sample of adults with Duchenne and caregivers reported patient health status using self- or proxy-reported EQ-5D-3L. Appropriateness was assessed across 6 domains of concern raised by stakeholders in Duchenne. These concerns were that the EQ-5D/EQ-VAS would not capture meaningful differences in health status, correlate with disease-specific measures, reflect real health status, exhibit face validity, be accurately interpreted, and be low burden. We evaluated these concerns by comparing EQ-5D index score and EQ-VAS scores to other condition-specific functional measures and open- and closed-ended questions. Results In total, 263 participants (74% response) completed the survey, 24% of whom were adult patients. Most participants lived in the United States or United Kingdom (58%). Patient age ranged from 1 to 48 y. EQ-5D index was higher in ambulatory than nonambulatory patients (0.60 v. 0.30, P < 0.001) and was negatively correlated with upper limb impairment ( r = 0.61, P < 0.001). Three-quarters of respondents agreed that EQ-5D measured real health status (74%). Most respondents interpreted EQ-VAS anchors of best and worst imaginable health as full health (61%) and death/near death (58%). Respondents indicated the EQ-5D was easy to understand (86%) and answer (71%). Conclusions Contrary to anecdotal concerns, we found support for the appropriateness of EQ-5D to assess health status in Duchenne. While other measures may be more sensitive to specific outcomes in Duchenne, there may be some value in results using the EQ-5D measure.

scholarly journals Using an Online, Modified Delphi Approach to Engage Patients and Caregivers in Determining the Patient-Centeredness of Duchenne Muscular Dystrophy Care Considerations

2019 ◽  
Vol 39 (8) ◽  
pp. 1019-1031 ◽  
Author(s):  
Dmitry Khodyakov ◽  
Sean Grant ◽  
Brian Denger ◽  
Kathi Kinnett ◽  
Ann Martin ◽  
...  
Keyword(s):  
Duchenne Muscular Dystrophy ◽  
Muscular Dystrophy ◽  
Bone Health ◽  
Guideline Development ◽  
Patient Centeredness ◽  
Patient Centered ◽  
Access To Treatment ◽  
Joint Decision ◽  
Modified Delphi ◽  
Delphi Approach

Purpose. To determine the patient-centeredness of endocrine and bone health Duchenne muscular dystrophy (DMD) care considerations using the RAND/PPMD Patient-Centeredness Method (RPM), which is a novel, online, modified-Delphi approach to engaging patients and caregivers in clinical guideline development. Methods. We solicited input on the patient-centeredness of care considerations from 28 individuals with DMD and 94 caregivers, randomly assigned to 1 of 2 mixed panels. During a 3-round online modified-Delphi process, participants rated the importance and acceptability of 19 DMD care considerations (round 1), reviewed and discussed the initial results (round 2), and revised their original ratings (round 3). Patient-centeredness was operationalized as importance and acceptability of recommendations. We considered a care consideration to be patient-centered if both panels deemed it important and acceptable. Results. Ninety-five panelists (78%) participated in this study. Of these, 88 (93%) participated in round 1, 74 (78%) in round 2, and 56 (59%) in round 3. Panelists deemed 12 care considerations to be patient-centered: 3 weight management, 3 bone health, 4 vertical growth, and 2 puberty recommendations. Seven care considerations did not meet patient-centeredness criteria. Common reasons were lack of evidence specific to DMD and concerns about insurance coverage, access to treatment, and patient safety. Conclusions. Using the RPM, Duchenne families considered most care considerations to be patient-centered. Besides being clinically appropriate, these considerations are likely to be consistent with the preferences, needs, and values of Duchenne families. While all relevant care considerations should be discussed during patient-provider encounters, those that did not meet patient-centeredness criteria in particular should be carefully considered as part of joint decision making between Duchenne families and their providers. Study Registration: HSRProj 20163126.

DUAL-COMBINATION MAINTENANCE INHALER PREFERENCES IN ASTHMA AND COPD: A PATIENT-CENTERED BENEFIT-RISK ASSESSMENT

CHEST Journal ◽  
2019 ◽  
Vol 156 (4) ◽  
pp. A9-A10
Author(s):  
Ileen Gilbert ◽  
Fernando Martinez ◽  
Tommi Tervonen ◽  
James Eudicone ◽  
Sebastian Heidenreich ◽  
...  
Keyword(s):  
Risk Assessment ◽  
Patient Centered ◽  
Benefit Risk Assessment
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