Video-based assessments of activities of daily living: generating real-world evidence in pediatric rare diseases.
Expert Rev Pharmacoecon Outcomes Res
; 24(6): 713-721, 2024 Jul.
Article
en En
| MEDLINE
| ID: mdl-38789406
ABSTRACT
INTRODUCTION:
Preserving function and independence to perform activities of daily living (ADL) is critical for patients and carers to manage the burden of care and improve quality of life. In children living with rare diseases, video recording ADLs offer the opportunity to collect the patients' experience in a real-life setting and accurately reflect treatment effectiveness on outcomes that matter to patients and families. AREAS COVERED We reviewed the measurement of ADL in pediatric rare diseases and the use of video to develop at-home electronic clinical outcome assessments (eCOA) by leveraging smartphone apps and artificial intelligence-based analysis. We broadly searched PubMed using Boolean combinations of the following MeSH terms 'Rare Diseases,' 'Quality of Life,' 'Activities of Daily Living,' 'Child,' 'Video Recording,' 'Outcome Assessment, Healthcare,' 'Intellectual disability,' and 'Genetic Diseases, Inborn.' Non-controlled vocabulary was used to include human pose estimation in movement analysis. EXPERT OPINION Broad uptake of video eCOA in drug development is linked to the generation of technical and clinical validation evidence to confidently assess a patient's functional abilities. Software platforms handling video data must align with quality regulations to ensure data integrity, security, and privacy. Regulatory flexibility and optimized validation processes should facilitate video eCOA to support benefit/risk drug assessment.Palabras clave
Texto completo:
1
Colección:
01-internacional
Base de datos:
MEDLINE
Asunto principal:
Calidad de Vida
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Grabación en Video
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Actividades Cotidianas
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Inteligencia Artificial
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Evaluación de Resultado en la Atención de Salud
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Enfermedades Raras
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Aplicaciones Móviles
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Teléfono Inteligente
Límite:
Child
/
Humans
Idioma:
En
Revista:
Expert Rev Pharmacoecon Outcomes Res
Asunto de la revista:
FARMACOLOGIA
Año:
2024
Tipo del documento:
Article
País de afiliación:
Reino Unido
Pais de publicación:
Reino Unido