Utilities Associated with the Treatment of Growth Hormone Deficiency (GHD): A Time Trade-off (TTO) Study in the UK and Canada
Bibliographic record
Abstract
Susan Kirsch,1 Gary Butler,2 Lasse de Fries Jensen,3 Anna Okkels,4 Cecilie Yssing,4 Jonas Håkan-Bloch3 1Department of Pediatric Endocrinology, Hospital for Sick Children, Toronto and Oak Valley Health Hospital, Markham, Ontario, Canada; 2Department of Paediatric Endocrinology, University College London Hospitals, and UCL Great Ormond Street Institute of Child Health, London, UK; 3Novo Nordisk, Søborg, 2860, Denmark; 4EY Godkendt Revisionspartnerselskab, Frederiksberg, 2000, DenmarkCorrespondence: Lasse de Fries Jensen, Novo Nordisk, Vandtårnsvej 108-110, Søborg, 2860, Denmark, Tel +45 34482020, Email LDFJ@novonordisk.comPurpose: Growth hormone deficiency (GHD) causes decreased growth rate in children, resulting in short stature in childhood and adulthood. Daily subcutaneous injections with growth hormone (GH) have been standard treatment. Newer weekly GH formulations now exist. This study estimates utilities associated with GHD treatment for both people with the disease and caregivers by employing time trade-off (TTO) methodology.Methods: Three online surveys were conducted amongst the general population in the UK and Canada. Based on a pilot, data collection was conducted in two surveys only (Survey A and Survey B). In Survey A, adults aged ≥ 18 years evaluated health states as if they were receiving injections themselves. In Survey B, adults with a child < 15 years evaluated health states as if they were administering injections to a child. The surveys assessed device complexity, injection frequency, injection pain, needle visibility and storage possibilities.Results: 2026 and 2028 respondents completed Survey A and Survey B, respectively. Of these, 1782 respondents and 1678 respondents were valid for inclusion. Avoiding weekly injection pain was associated with a significant utility gain of 0.030 (95% CI 0.026– 0.035, p< 0.001) in Survey A and 0.044 (95% CI 0.038– 0.051, p< 0.001) in Survey B. Additionally, less complex injection devices and lower injection frequencies had a significant impact in both Survey A (0.020, 95% CI 0.016– 0.025, p< 0.001; 0.009, 95% CI 0.005– 0.014, p< 0.001) and Survey B (0.008, 95% CI 0.002– 0.014, p=0.006; 0.009, 95% CI 0.003– 0.014, p=0.003).Conclusion: Several aspects are associated with a significant impact on utilities for people with GHD and potential caregivers. Treatment options without injection pain, a time-consuming and complex injection process and daily injections are expected to result in higher health-related quality of life. These results may inform future economic evaluations and treatment choices.Keywords: caregiver, children, health-related quality of life, survey
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How this classification was reachedexpand
Full frame machine prediction
Teacher imitationNot calibrated prevalence, not ground truth. Human validation pending. The Gemma side is a direct model label for every work in the frame, read from the title-only record. The Codex side is a classifier learned from the 10,348 direct Codex labels and calibrated to design-weighted sample rates; fields without enough sample support carry no Codex call. Candidate is the union of the two sides; consensus is their intersection. These outputs are machine_predicted_unvalidated and are not human labels.
Distilled classifier scores by category (both heads)
| Category | Codex | Gemma |
|---|---|---|
| Metaresearch | 0.002 | 0.006 |
| Meta-epidemiology (narrow) | 0.000 | 0.001 |
| Meta-epidemiology (broad) | 0.001 | 0.002 |
| Bibliometrics | 0.002 | 0.008 |
| Science and technology studies | 0.002 | 0.001 |
| Scholarly communication | 0.002 | 0.001 |
| Open science | 0.002 | 0.002 |
| Research integrity | 0.001 | 0.001 |
| Insufficient payload (model declined to judge) | 0.002 | 0.000 |
Machine scores (provisional)
The two teacher heads of the student model, read on this work. A score orders the frame for review; it never asserts a category, and the validation status ships verbatim with every row.
Baseline scores from an immature model (maturity gate not passed, 7 training rounds). Scores rank; they never assert a category.
score_only:v0-immature-baseline · verbatim from the scoring run: score_only means the number may rank works, and no category label ships from itClassification
machine, unvalidatedMachine predicted; a candidate call from one source (direct Gemma or distilled Codex), not a consensus.
How this classification was reached, model by model and score by score, is at the end of the page under "How this classification was reached".