International liver Glycogen Storage Disease PSP Engagement Summary

Contents

Gathering uncertainties

Uncertainties were gathered from responders from 58 countries using an online questionnaire (SurveyMonkey) and an email collector.

  Number %
Total respondents (across all methods) 763 100
Total patients  150 20
Total carers 370 48
Patients/carers representing GSD 0 4  
Patients/carers representing GSD Ia 212  
Patients/carers representing GSD Ib 71  
Patients/carers representing GSD III 65  
Patients/carers representing GSD IV 4  
Patients/carers representing GSD VI 9  
Patients/carers representing GSD IX 68  
Patients/carers representing GSD XI 1  
Total healthcare professionals 266 35
Of which physicians 139  
Of which nurses 5  
Of which nurses 80  
Total number of original uncertainties submitted 1388 100
Original uncertainties in scope 883 64
Original uncertainties out of scope 505 36

 

Interim prioritisation

Interim prioritisation was performed via an online questionnaire (SurveyMonkey). Responders from 58 countries answered the questionnaire.

  Number %
Total number of indicative questions (answered & unanswered) 72 100
Number of verified answered questions 0 0
Number of verified unanswered questions 72 100
Number of verified unanswered questions included in the interim prioritisation 72  
Total respondents (across all methods) 562 100
Total patients  86 15
Total carers 253 45
Patients/carers representing GSD 0 8  
Patients/carers representing GSD Ia 124  
Patients/carers representing GSD Ib 50  
Patients/carers representing GSD III 61  
Patients/carers representing GSD IV 5  
Patients/carers representing GSD VI 10  
Patients/carers representing GSD IX 45  
Patients/carers representing GSD XI 2  
Total healthcare professionals 166 30
Of which physicians 105  
Of which dieticians 11  
Of which nurses 41  
Number of questions taken to final workshop 22  

 

Final priority setting workshop

  Number %
Total participants 22 100
Total patients  5 23
Total carers 12 55
Patients/carers representing GSD 0 1  
Patients/carers representing GSD Ia 8  
Patients/carers representing GSD Ib 2  
Patients/carers representing GSD III 2  
Patients/carers representing GSD IV 0  
Patients/carers representing GSD VI 1  
Patients/carers representing GSD IX 3  
Patients/carers representing GSD XI 0  
Total healthcare professionals 11 50
Of which physicians 4  
Of which dieticians 2  
Of which nurses 4  
Of which psychologist 1  

 Notes

For the first questionnaire, we allowed responders to identify with multiple categories (e.g. patient, carer, healthcare professional), allowing for the percentages of responders to exceed 100%.

For the second prioritization questionnaire responders could choose only one option.
Additionally, of the 22 participants of the final workshop, 6 identified with 2 roles (for example carer and healthcare professional).

Furthermore, responders were allowed not to share their personal information on role in the liver GSD community. In the second prioritisation questionnaire, 10% did not answer these questions.