AN ASSESSMENT OF THE GLOBAL USABILITY OF QUALITY-OF-LIFE QUESTIONNAIRES IN CHILDREN ON RECOMBINANT GROWTH HORMONE THERAPY
Author(s)
Aleksandra Kapczuk, Medical Student1, Salma R. Ali, PhD1, Malika Alimussina, MPH, PhD1, Ines Alves, N/A2, Nienke Biermasz, MD, PhD3, Suet Ching Chen, MD, MRCPCH1, Ashley Gilmer, N/A4, Eleanor Grieve, PhD5, Michael Højby, MD, PhD6, Maria Koltowska-Haggstrom, MD, PhD7, Mohamad Maghnie, MD, PhD8, Aristides Maniatis, MD, FAAP9, Julia Quitmann, PhD10, Caterina Tedesco, RN11, Diana Vitali, N/A12, Bradley S Miller, MD, PhD13, Syed Faisal Ahmed, MD, FRCPCH1.
1Office for Rare Conditions, University of Glasgow, Glasgow, United Kingdom, 2ANDO Portugal, Évora, Portugal, 3Department of Medicine, Division of Endocrinology, Leiden University Medical Center, Leiden, Netherlands, 4ICOSEP, Valdosta, GA, USA, 5Health Economics and Health Technology Assessment (HEHTA), School of Health and Wellbeing, University of Glasgow, Glasgow, United Kingdom, 6Novo Nordisk A/S, Søborg, Denmark, 7Proper Medical Writing sp. z o.o., Warsaw, Poland, 8Department of Pediatrics, Endocrine Unit, IRCCS Istituto Giannina Gaslini, Genova, Italy; Department of Neuroscience, Rehabilitation, Ophthalmology, Genetics, Maternal and Child Health - DINOGMI, University of Genova, Genova, Italy, 9Rocky Mountain Pediatric Endocrinology, Centennial, CO, USA, 10University of Applied Sciences (HAW) Hamburg, Faculty of Social Work and Childhood Education, Hamburg, Germany, 11Department of Pediatrics, Endocrine Unit, IRCCS Istituto Giannina Gaslini, Genova, Italy; Department of Neuroscience, Rehabilitation, Ophthalmology, Genetics, Maternal and Child Health - DINOGMI, Genova, Italy, 12FONDAZIONE CAROL ETS rare hypothalamic and pituitary conditions, Via Di Santa Costanza, Rome, Italy, 13Division of Endocrinology, Department of Paediatrics, University of Minnesota Medical School, MHealth Fairview Masonic Children's Hospital, Minneapolis, MN, USA.
1Office for Rare Conditions, University of Glasgow, Glasgow, United Kingdom, 2ANDO Portugal, Évora, Portugal, 3Department of Medicine, Division of Endocrinology, Leiden University Medical Center, Leiden, Netherlands, 4ICOSEP, Valdosta, GA, USA, 5Health Economics and Health Technology Assessment (HEHTA), School of Health and Wellbeing, University of Glasgow, Glasgow, United Kingdom, 6Novo Nordisk A/S, Søborg, Denmark, 7Proper Medical Writing sp. z o.o., Warsaw, Poland, 8Department of Pediatrics, Endocrine Unit, IRCCS Istituto Giannina Gaslini, Genova, Italy; Department of Neuroscience, Rehabilitation, Ophthalmology, Genetics, Maternal and Child Health - DINOGMI, University of Genova, Genova, Italy, 9Rocky Mountain Pediatric Endocrinology, Centennial, CO, USA, 10University of Applied Sciences (HAW) Hamburg, Faculty of Social Work and Childhood Education, Hamburg, Germany, 11Department of Pediatrics, Endocrine Unit, IRCCS Istituto Giannina Gaslini, Genova, Italy; Department of Neuroscience, Rehabilitation, Ophthalmology, Genetics, Maternal and Child Health - DINOGMI, Genova, Italy, 12FONDAZIONE CAROL ETS rare hypothalamic and pituitary conditions, Via Di Santa Costanza, Rome, Italy, 13Division of Endocrinology, Department of Paediatrics, University of Minnesota Medical School, MHealth Fairview Masonic Children's Hospital, Minneapolis, MN, USA.
OBJECTIVES: Although patient- and parent-reported outcome measures (PROMs) are widely used in paediatric recombinant human growth hormone (rhGH) trials, their use in routine clinical practice remains limited. We aimed to identify barriers and facilitators to the global implementation of PROMs for assessing quality of life (QoL) in children receiving rhGH.
METHODS: PROMs used in rhGH therapy were identified through a narrative search and consultation with an expert working group consisting of experts, patient and industry representatives assembled through the Global Registry for Novel Therapies in Rare Bone and Endocrine Conditions (https://globe-reg.net/) initiative. Six parameters—reporter perspective, language availability, administration mode, cost, age range and completion time—were evaluated to generate an integrated assessment of real-world usability.
RESULTS: Of 50 PROMs identified, 13 were excluded due to unclear methodology or development in adult populations. The remaining instruments included 26 generic and 11 condition-specific PROMs. Of these 37, 16 (43%) were available in both patient-self-report and parent-proxy formats in over 30 languages, 23(62%) were available in paper and electronic formats, and 24(65%) had a reported completion time of <15 minutes. The median lower age limit was 7years (range, 3.0,12.7). Seven (19%), all generic, were free to use, 12(32%) required a fee for clinical and research use, and for 18(49%) cost was unclear or usage-dependent. None of the condition-specific PROMs were unconditionally free. Among eight broad domains, “physical functioning and symptoms” and “emotional and mental health” were most represented condition-specific instruments. Overall, generic PROMs, including KINDL, KIDSCREEN-10 Global Index and EQ-5D, ranked higher across usability parameters than condition-specific tools.
CONCLUSIONS: Cost and accessibility remain major barriers to the routine use of condition-specific PROMs in rhGH care. Although generic and condition-specific PROMs are complementary, generic instruments currently offer the greatest potential for global implementation in research and routine clinical practice until freely available condition-specific PROMs become available for unrestricted use.
METHODS: PROMs used in rhGH therapy were identified through a narrative search and consultation with an expert working group consisting of experts, patient and industry representatives assembled through the Global Registry for Novel Therapies in Rare Bone and Endocrine Conditions (https://globe-reg.net/) initiative. Six parameters—reporter perspective, language availability, administration mode, cost, age range and completion time—were evaluated to generate an integrated assessment of real-world usability.
RESULTS: Of 50 PROMs identified, 13 were excluded due to unclear methodology or development in adult populations. The remaining instruments included 26 generic and 11 condition-specific PROMs. Of these 37, 16 (43%) were available in both patient-self-report and parent-proxy formats in over 30 languages, 23(62%) were available in paper and electronic formats, and 24(65%) had a reported completion time of <15 minutes. The median lower age limit was 7years (range, 3.0,12.7). Seven (19%), all generic, were free to use, 12(32%) required a fee for clinical and research use, and for 18(49%) cost was unclear or usage-dependent. None of the condition-specific PROMs were unconditionally free. Among eight broad domains, “physical functioning and symptoms” and “emotional and mental health” were most represented condition-specific instruments. Overall, generic PROMs, including KINDL, KIDSCREEN-10 Global Index and EQ-5D, ranked higher across usability parameters than condition-specific tools.
CONCLUSIONS: Cost and accessibility remain major barriers to the routine use of condition-specific PROMs in rhGH care. Although generic and condition-specific PROMs are complementary, generic instruments currently offer the greatest potential for global implementation in research and routine clinical practice until freely available condition-specific PROMs become available for unrestricted use.
Conference/Value in Health Info
2026-11, ISPOR Europe 2026, Vienna, Austria
Value in Health, Volume 29, Issue 12S
Code
PCR163
Topic
Patient-Centered Research
Topic Subcategory
Patient-reported Outcomes & Quality of Life Outcomes
Disease
Diabetes/Endocrine/Metabolic Disorders (including obesity), Pediatrics, Rare & Orphan Diseases