Patient‐reported outcome measures for young people with developmental disabilities: incorporation of design features to reduce cognitive demands

Schwartz, Alan M.; Kramer, Jessica; Longo, Angela L

doi:10.1111/dmcn.13617

Cited by 16 publications

(16 citation statements)

References 26 publications

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“…The consulted HTA experts from the Dutch Health Care Institute emphasized the importance of HRQoL in relative effectiveness assessments for decision-making in the context of new therapeutics [ 71 ]. However, choosing the right instrument for (young) people with cognitive impairments, such as MLD patients, is challenging [ 72 ]. A complicating factor is that patients with MLD are often unable to complete PROMs themselves due to their young age and/or cognitive decline.…”

Section: Discussionmentioning

confidence: 99%

Modified Delphi procedure-based expert consensus on endpoints for an international disease registry for Metachromatic Leukodystrophy: The European Metachromatic Leukodystrophy initiative (MLDi)

Schoenmakers

Beerepoot

Berg

et al. 2022

Orphanet J Rare Dis

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Background Metachromatic Leukodystrophy (MLD) is a rare lysosomal disorder. Patients suffer from relentless neurological deterioration leading to premature death. Recently, new treatment modalities, including gene therapy and enzyme replacement therapy, have been developed. Those advances increase the need for high-quality research infrastructure to adequately compare treatments, execute post-marketing surveillance, and perform health technology assessments (HTA). To facilitate this, a group of MLD experts started the MLD initiative (MLDi) and initiated an academia-led European MLD registry: the MLDi. An expert-based consensus procedure, namely a modified Delphi procedure, was used to determine the data elements required to answer academic, regulatory, and HTA research questions. Results Three distinct sets of data elements were defined by the 13-member expert panel. The minimal set (n = 13) contained demographics and basic disease characteristics. The core set (n = 55) included functional status scores in terms of motor, manual, speech and eating abilities, and causal and supportive treatment characteristics. Health-related quality of life scores were included that were also deemed necessary for HTA. The optional set (n = 31) contained additional clinical aspects, such as findings at neurological examination, detailed motor function, presence of peripheral neuropathy, gall bladder involvement and micturition. Conclusion Using a modified Delphi procedure with physicians from the main expert centers, consensus was reached on a core set of data that can be collected retrospectively and prospectively. With this consensus-based approach, an important step towards harmonization was made. This unique dataset will support knowledge about the disease and facilitate regulatory requirements related to the launch of new treatments.

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Section: Discussionmentioning

confidence: 99%

Modified Delphi procedure-based expert consensus on endpoints for an international disease registry for Metachromatic Leukodystrophy: The European Metachromatic Leukodystrophy initiative (MLDi)

Schoenmakers

Beerepoot

Berg

et al. 2022

Orphanet J Rare Dis

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“…All this cognitive processing must also occur within a timeline dictated by the interviewer or survey format. Self-report measures often used in health-related research are not typically designed with cognitive accessibility in mind and, as a result, are unintelligible to people with IDD (Feldman, Bosett, Collet, & Burnham-Riosa, 2014;Loewenson, Laurell, Hogstedt, D'Ambruoso, & Shroff, 2014;Nicolaidis et al, 2015;O'Keeffe, Guerin, McEvoy, Lockhart, & Dodd, 2019;Schwartz, Kramer, & Longo, 2018). This accounts for the reliance on proxy-reporting evident in the literature.…”

Section: Current Status and Challenges In Assessing Health And Qolmentioning

confidence: 99%

“…There is not one universally accepted definition of accessibility or cognitive accessibility (Johansson, 2016;Persson, Åhman, Yngling, & Gulliksen, 2015). However, there are a key set of design principles that can promote access to people with a broad range of support needs (Schwartz et al, 2018;Tanis et al, 2012;White et al, 2015). For example, providing multiple means of representation (e.g., present content in multiple formats), multiple means of engagement (e.g., provide different ways to connect with content), and multiple means of expression (e.g., create multiple options for how to respond) are key principles of universal design for learning (Rappolt-Schlichtmann, Daley, & Rose, 2012).…”

Section: What Is Cognitive Accessibility?mentioning

confidence: 99%

State of the Field: The Need for Self‐Report Measures of Health and Quality of Life for People With Intellectual and Developmental Disabilities

Shogren

Bonardi

Cobranchi

et al. 2021

Policy Practice Intel Disabi

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Background: Collecting self-reported health and quality of life (QoL) outcomes is increasingly considered best practice, but people with intellectual and developmental disabilities (IDD) are often excluded from patient-reported outcome measures.Objective: This article provides a literature-informed overview of the state of the field of self-reporting of physical health and QoL in research with adults with IDD.Approach: We first identified and synthesized definitions of key constructs related to the self-reporting of health. Next, we summarize literature on existing and emerging practices focused on health and QoL assessment, discussing the frequent and sometimes overly broad use of proxy-respondents in the IDD field. We then highlight emerging directions focused on cognitive accessibility and universal design. Finally, we provide conclusions and recommendations for the field.Conclusions: Informed by the literature, we provide action steps to guide the field in considering how to incorporate self-reporting of health outcomes by people with IDD in research, policy, and practice.

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“…The best responses involved items that participants were able to do or were good at 25 . Congruent visual cues and using the children’s own expressions can improve the validity of PROMs 26 …”

Section: Acquiring the Relevant Proms Contentmentioning

confidence: 99%

Patient perspectives in pediatric neurology: a critical shift in the paradigm of outcome measurement

Ronen

Rosenbaum

Streiner

2021

Develop Med Child Neuro

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This review explores children’s self‐reported outcome measurements in pediatric neurology. We examine the following questions: (1) What is meant by patient‐reported health, functioning, and quality of life outcomes? (2) How can patients express whether the interventions they receive do more good than harm? (3) Why and how should pediatric neurology patients help determine the outcomes of interest? (4) What tools and recommendations are available to evaluate the outcomes of interest? Applying patients’ perspectives across the processes of evaluation of medical interventions has become an important expectation. These developments, consistent with current healthcare goals, coincide with the evolution of pediatric neurology into a sophisticated diagnostic–interventional field that aims to prolong survival, decrease impairments and symptoms, and improve patients’ well‐being – the recognized essential endpoints of interest in all medicine.

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Patient‐reported outcome measures for young people with developmental disabilities: incorporation of design features to reduce cognitive demands

Abstract: Patient-reported outcome measure (PROM) design features can reduce assessment demands related to cognitive processes. Pediatric PROMs underutilize design features that decrease cognitive demands of self-reporting.

Cited by 16 publications

References 26 publications

Modified Delphi procedure-based expert consensus on endpoints for an international disease registry for Metachromatic Leukodystrophy: The European Metachromatic Leukodystrophy initiative (MLDi)

Modified Delphi procedure-based expert consensus on endpoints for an international disease registry for Metachromatic Leukodystrophy: The European Metachromatic Leukodystrophy initiative (MLDi)

State of the Field: The Need for Self‐Report Measures of Health and Quality of Life for People With Intellectual and Developmental Disabilities

Patient perspectives in pediatric neurology: a critical shift in the paradigm of outcome measurement

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