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

Area of Science:

• Neuroscience
• Genetics
• Clinical Pharmacology

Background:

• Clinical drug trials for genetic neurodevelopmental conditions are expanding.
• Understanding parent priorities is crucial for successful clinical trial design and patient recruitment.
• Limited evidence exists on parental decision-making regarding trial enrollment for genetic neurodevelopmental conditions.

Purpose of the Study:

• To assess parental priorities in deciding whether to enroll their child with fragile X syndrome (FXS) in a clinical drug trial.
• To identify key motivating and discouraging factors influencing parental decisions for FXS clinical trial participation.

Main Methods:

• An online survey utilizing a best-worst scaling method was administered to parents.
• Parents were recruited via the National Fragile X Foundation and FRAXA.
• Sequential best-worst with conditional logit analysis was employed to prioritize factors.

Main Results:

• The most significant motivating factor for trial enrollment was a drug targeting the underlying FXS mechanism (coeff=3.28, p<0.001).
• The potential for a drug to benefit a large patient population was also a high motivator (coeff=3.03, p<0.001).
• The most discouraging factors included the requirement of blood draws (coeff=-3.09, p<0.001), loss of drug access post-trial (coeff=-3.01, p<0.001), and potential drug side effects (coeff=-2.96, p<0.001).

Conclusions:

• Parental priorities identified can inform the design and execution of more effective clinical trials for FXS.
• Addressing parental concerns regarding trial procedures, drug access, and safety is essential for enhancing enrollment.
• Incorporating patient and parent perspectives leads to evidence-based trial strategies.