Table 1.

Structure of the Questionnaire.

1) Identification of issues during registry planning     • Perception on the definition of the purpose and the objectives of the registry     • Perception on the definition of the inclusion/exclusion criteria     • Definition of the variables included in the registry     • Definition of the size, the duration, the setting and the geographical areas     • Identification of stakeholders, team building and establishment of a governance     • Data access & use of data     • Publication plan     • Development of the protocol and related documents     • Development of the project plan     • Development of risk management plans & risk management during the registry

2) Identification of issues during the operation of the registry     • Issues related to patient recruitment or retention       ° Barriers to patient recruitment/retention       ° Evaluation of success of patient recruitment strategies       ° Evaluation of success of patient retention strategies       ° Evaluation of center/physician or patient selection bias     • Issues related to data collection & quality assurance       ° Issues related to data collection       ° Identification of quality issues & timing for detection     • Issues related to budget     • Issues related to project management       ° Ownership & accountability       ° Coordination       ° Estimation of the use of resources/duration/complexity

3) Issues during data analysis     • Identification of sources of bias     • Treatment of missing data     • Appropriateness of time horizon & planned interim analysis     • Appropriateness of pre-specified analyses     • Interpretation of the results     • Identification of issues related to data access     • Identification of strengths & limitations of the registry

4) Issues during the publication of the results

5) Other issues

6) Assessment of learnings     • General learning topics     • Value of the registry organization       ° Inclusion of patients in the SAB and in the WC       ° Inclusion of clinicians in the SAB and in the WC       ° Inclusion of members from the pharmaceutical industry in the SAB and in the WC     • Pitfalls and learning opportunities emerged from the integration of research projects within the TOSCA registry     • Pitfalls and learning opportunities emerged from the integration of a Votubia® PASS within the TOSCA registry

7) Additional comments

SAB, Scientific Advisory Board; WC, Working Committee; TOSCA, TuberOus SClerosis registry to increase disease Awareness; PASS, post approval safety study.