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.