Fragile X syndrome clinical trials: exploring parental decision-making

C S D'Amanda; H L Peay; A C Wheeler; E Turbitt; B B Biesecker

doi:10.1111/jir.12605

Fragile X syndrome clinical trials: exploring parental decision-making

J Intellect Disabil Res. 2019 Aug;63(8):926-935. doi: 10.1111/jir.12605. Epub 2019 Feb 12.

Authors

C S D'Amanda¹, H L Peay², A C Wheeler², E Turbitt¹, B B Biesecker¹

Affiliations

¹ Social and Behavioral Research Branch, National Human Genome Research Institute, Bethesda, MD, USA.
² Center for Newborn Screening, Ethics, and Disability Studies, RTI International, Research Triangle Park, NC, USA.

Abstract

Background: The objective of this research was to understand parental proxy decision-making for drug trial participation for children with Fragile X syndrome (FXS). Specifically, we aimed to capture preferences, motivations, influencing factors and barriers related to trial involvement among trial joiners and decliners and describe ease of trial decision-making and decisional regret.

Methods: Interviews were conducted with parents from two groups: those who chose to enrol their child with FXS in a trial (N = 16; Joiners) and those who declined trial participation (N = 15; Decliners). Data were coded and interpreted through inductive content analysis.

Results: Prominent decisional factors included attitudes about medicating FXS symptoms, potential for direct benefit (primarily evaluated through the degree of match between target outcomes and child symptomatology and drug mechanism), logistical convenience and perceived risks of side effects. The ultimate motivation for participation was potential for direct benefit. None of the parents reported decisional regret, and ease of decision-making ranged from easy to difficult for our participants.

Conclusions: Therapeutic optimism was high among those who elected participation. Parents may benefit from an explanation of the rationale behind chosen outcome variables and may be more interested in trials that target or measure as an exploratory outcome the symptoms they find most concerning. Our findings reinforce the need for future trials to reduce participant inconvenience. Our results contrast with what has previously been observed in parents of children with life-threatening conditions; parents of children with FXS may be more trial risk averse and find trial decisions to be harder. Parents of children with FXS considering trials may benefit from a decisional intervention aimed at deliberating motivations and barriers.

Keywords: Fragile X syndrome; clinical trials; decisional factors; parents; proxy decision-making.

Publication types

Research Support, N.I.H., Intramural

MeSH terms

Adult
Child
Clinical Trials as Topic*
Decision Making*
Female
Fragile X Syndrome / drug therapy*
Health Knowledge, Attitudes, Practice*
Humans
Male
Motivation*
Parents*
Patient Acceptance of Health Care*
Qualitative Research

Grants and funding

ZIA HG200317-15/ImNIH/Intramural NIH HHS/United States