Parent clinical trial priorities for fragile X syndrome: a best-worst scaling.

Turbitt, E; D'Amanda, C; Hyman, S; Weber, JD; Bridges, JFP; Peay, HL; Biesecker, BB

Parent clinical trial priorities for fragile X syndrome: a best-worst scaling.

Turbitt, E

D'Amanda, C Hyman, S Weber, JD Bridges, JFP Peay, HL Biesecker, BB

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Publisher:: SPRINGERNATURE
Publication Type:: Journal Article
Citation:: Eur J Hum Genet, 2021, 29, (8), pp. 1245-1251
Issue Date:: 2021-08

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Field	Value	Language
dc.contributor.author	Turbitt, E https://orcid.org/0000-0002-6650-9702
dc.contributor.author	D'Amanda, C
dc.contributor.author	Hyman, S
dc.contributor.author	Weber, JD
dc.contributor.author	Bridges, JFP
dc.contributor.author	Peay, HL
dc.contributor.author	Biesecker, BB
dc.date.accessioned	2022-03-18T04:18:08Z
dc.date.available	2021-06-02
dc.date.available	2022-03-18T04:18:08Z
dc.date.issued	2021-08
dc.identifier.citation	Eur J Hum Genet, 2021, 29, (8), pp. 1245-1251
dc.identifier.issn	1018-4813
dc.identifier.issn	1476-5438
dc.identifier.uri	http://hdl.handle.net/10453/155335
dc.description.abstract	An expansion in the availability of clinical drug trials for genetic neurodevelopmental conditions is underway. Delineating patient priorities is key to the success of drug development and clinical trial design. There is a lack of evidence about parent decision-making in the context of clinical drug trials for genetic neurodevelopmental conditions. We assessed parents' priorities when making a decision whether to enroll their child with fragile X syndrome (FXS) in a clinical drug trial. An online survey included a best-worst scaling method for parents to prioritize motivating and discouraging factors for child enrollment. Parents were recruited through the National Fragile X Foundation and FRAXA. Sequential best-worst with conditional logit analysis was used to determine how parents prioritize motivating and discouraging factors about trial enrollment decisions. Respondents (N = 354) were largely biological mothers (83%) of an individual with FXS who ranged in age from under 5 to over 21 years. The highest motivating factor was a trial to test a drug targeting the underlying FXS mechanism (coeff = 3.28, p < 0.001), followed by the potential of the drug to help many people (coeff = 3.03, p < 0.001). Respondents rated requirement of blood draws (coeff = -3.09, p < 0.001), loss of access to the drug post trial (coeff = -3.01, p < 0.001), and drug side effects (coeff = -2.96, p < 0.001) as most discouraging. The priorities defined by parents can be incorporated into evidence-based trial design and execution to enhance the enrollment process.
dc.format	Print-Electronic
dc.language	eng
dc.publisher	SPRINGERNATURE
dc.relation.ispartof	Eur J Hum Genet
dc.relation.isbasedon	10.1038/s41431-021-00922-w
dc.rights	This is a post-peer-review, pre-copyedit version of an article published in [Eur J Hum Genet, 2021, 29, (8), pp. 1245-1251]. The final authenticated version is available online at: https://www.nature.com/articles/s41431-021-00922-w]”
dc.rights	info:eu-repo/semantics/openAccess
dc.subject	0604 Genetics, 1103 Clinical Sciences
dc.subject.classification	Genetics & Heredity
dc.title	Parent clinical trial priorities for fragile X syndrome: a best-worst scaling.
dc.type	Journal Article
utslib.citation.volume	29
utslib.location.activity	England
utslib.for	0604 Genetics
utslib.for	1103 Clinical Sciences
pubs.organisational-group	/University of Technology Sydney
pubs.organisational-group	/University of Technology Sydney/Faculty of Health
pubs.organisational-group	/University of Technology Sydney/Faculty of Health/Graduate School of Health
pubs.organisational-group	/University of Technology Sydney/Faculty of Health/Graduate School of Health/GSH.Genetic Counselling
utslib.copyright.status	open_access	*
dc.date.updated	2022-03-18T04:18:07Z
pubs.issue	8
pubs.publication-status	Published
pubs.volume	29
utslib.citation.issue	8

Abstract:

An expansion in the availability of clinical drug trials for genetic neurodevelopmental conditions is underway. Delineating patient priorities is key to the success of drug development and clinical trial design. There is a lack of evidence about parent decision-making in the context of clinical drug trials for genetic neurodevelopmental conditions. We assessed parents' priorities when making a decision whether to enroll their child with fragile X syndrome (FXS) in a clinical drug trial. An online survey included a best-worst scaling method for parents to prioritize motivating and discouraging factors for child enrollment. Parents were recruited through the National Fragile X Foundation and FRAXA. Sequential best-worst with conditional logit analysis was used to determine how parents prioritize motivating and discouraging factors about trial enrollment decisions. Respondents (N = 354) were largely biological mothers (83%) of an individual with FXS who ranged in age from under 5 to over 21 years. The highest motivating factor was a trial to test a drug targeting the underlying FXS mechanism (coeff = 3.28, p < 0.001), followed by the potential of the drug to help many people (coeff = 3.03, p < 0.001). Respondents rated requirement of blood draws (coeff = -3.09, p < 0.001), loss of access to the drug post trial (coeff = -3.01, p < 0.001), and drug side effects (coeff = -2.96, p < 0.001) as most discouraging. The priorities defined by parents can be incorporated into evidence-based trial design and execution to enhance the enrollment process.

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