Measuring Meaningful Benefit-Risk Tradeoffs to Promote Patient-Focused Drug Development in Prader-Willi Syndrome: A Discrete-Choice Experiment
| Autor: | Jui-Hua Tsai, Norah L. Crossnohere, Theresa V. Strong, John F.P. Bridges |
|---|---|
| Rok vydání: | 2021 |
| Předmět: |
Medicine (General)
medicine.medical_specialty Discrete choice experiment patient-reported outcome hyperphagia R5-920 Neurodevelopmental disorder Quality of life (healthcare) medicine Original Research Article Psychiatry patient-focused drug development business.industry Health Policy digestive oral and skin physiology Public Health Environmental and Occupational Health rare diseases nutritional and metabolic diseases Insatiable hunger medicine.disease Patient preference Drug development Patient-reported outcome Prader-Willi syndrome business patient preferences Patient centered |
| Zdroj: | MDM Policy & Practice, Vol 6 (2021) MDM Policy & Practice |
| ISSN: | 2381-4683 |
| DOI: | 10.1177/23814683211039457 |
| Popis: | Background. Prader-Willi syndrome (PWS) is a rare neurodevelopmental disorder causing quality of life impairments such as insatiable hunger (hyperphagia) and obesity. We explored caregivers’ willingness to assume treatment risk in exchange for reduced hyperphagia according to a PWS-validated observer-reported outcome measure. Methods. We partnered with PWS patient organizations to develop a discrete-choice experiment exploring caregivers’ benefit-risk tradeoffs for emerging PWS treatments. The treatment benefit was a reduction in hyperphagia (as measured by a 0-, 5-, or 10-point change on the Hyperphagia Questionnaire for Clinical Trials [HQ-CT]). Treatment risks included weight gain (none, 5%, 10%), added risk of skin rash (none, 10%, 20%), and risk of liver damage (none, 1 in 1000, 10 in 1000). Preference models were estimated using mixed logistic regression and maximum acceptable risk. We explored differences in preferences across familial caregivers of patients with and without hyperphagia. Results. Four hundred sixty-eight caregivers completed the online survey. The majority of caregivers reported that patients experienced hyperphagia (68%) and half of patients experienced obesity (52%). Caregivers of patients without hyperphagia were willing to accept greater weight gain (16.4% v. 8.1%, P = 0.004) and a higher risk of skin rash (11.7% v. 6.2% P = 0.008) as compared to caregivers of patients with hyperphagia. Caregivers of patients with hyperphagia would accept a higher risk of liver damage as compared to caregivers of patients without hyperphagia (11.9 out of 1000 v. 6.4 out of 1000, P = 0.04). Conclusions. This research demonstrates that caregivers are willing to accept risk in exchange for a five-point improvement on the HQ-CT, a smaller marginal improvement than had been previously classified as meaningful. Patient experience with hyperphagia is a modifier in how much risk caregivers will accept. |
| Databáze: | OpenAIRE |
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