Table 1

Responses of parents of participating children in the clinical trial when asked why they thought that data return was important to them, and their physicians regarding their degree of support of sponsor direct return of data to families

Parents of trial participants: why is trial data return important to them?
Personal knowledge
To be informed
It is a great benefit to be able to see how my son may have responded during the Clinical Trial in all of these areas recorded, In Hopes to see some good benefit from the medication.
We took a big risk in being in the trial. Want to know if it works and how my son paired with the other boys
It’s nice to see how things are going and not be in the dark
All data to do with how my son is managing the condition/meds is important
We would like further understanding about how the trial was going, and what difference it’s made to our child as well as the rest of the children
To understand the clinical help VBP15 provided
We would like further understanding about how the trial was going, and what difference it’s made to our child as well as the rest of the children
To see actual data of improvement and/ or progression is important. Data helps you to understand if treatment works or not.
Just to see how our son is doing. We are hopeful he is doing better because if the drug and seeing the results gives us more hope.
Physician concerns of a sponsor returning participant-level data to directly to trial participants.
Supportive	after trial is finished, data should be shared
Supportive	No comments
Supportive with reservations about timing of delivery	I agree, but it needs to be done in a thoughtful manner, properly contextualized. At the end of the trial, all data should be returned to families. However, on a week by week basis during the trial, I don’t favor providing results to individual families
Supportive, with reservations about delivery outside of the healthcare or investigative team and interpretation of data	What’s meant by ‘clinical trial data’? I don’t think getting e.g., ECG, echo or MRI data is very useful and even some of the functional or strength measurements don’t mean much to a family. It’s a nice option for a family to see clinical trial data, but it would probably be more meaningful to provide them through a healthcare professional, either a doctor or a physiotherapist.
	Has to go through PI, SI and/or site staff
	Not to disagree with this objective, but to raise the concern that the PI/treating physician for the participant could be blind-sided by the parent contacting the office and requesting an urgent discussion with the physician over an abnormal lab result. How to educate parents on labs/biomarkers/tests that are predicted to be abnormal (due to having DMD)? The poster does not go into this in any detail.
	Interpreting the data and put the individual data in the context of the study results and of a progressive disease might not be easy for all families and can create some false judgement and/or anxiety. It creates some “inequality” as proactive and well informed families are more likely to ask for the data