Table 1 START Checklist

Are there patient organizations for the disease?

□Yes

□No

Are there community advisory boards (CABs)?

□Yes

□No

Community advisory boards

Are there clinical stakeholder networks?

□Yes

□No

Engagement with established research networks

The NIH rare diseases clinical research networks

Japan Agency for Medical Research and Development (AMED) – Initiative on Rare and Undiagnosed Diseases

Are there general development support platforms and infrastructures?

□Yes

□No

Conect4Children

European Joint Programme on Rare Diseases

Have you done a landscape analysis or horizon scanning?

□Yes

□No

Horizon Scanning: Landscape analysis/ Stakeholder identification and engagement

Are there Natural History (NH) Studies?

□Yes

□No

Natural History studies

Are there diagnostic tools?

□Yes

□No

Companion diagnostics

Are there patient-centered outcome measures (PCOMs)

□Yes

□No

Development of Patient-Centered Outcome Measures

Are there biomarkers?

□Yes

□No

Use of biomarkers in orphan drug development

Is there a coding for the rare disease?

□Yes

□No

Coding of rare diseases: Orphanet nomenclature

Did you acquire different sources of public funding?

□Yes

□No

European Commission funded programs and resources

European Joint Program on Rare Diseases

NIH funded programs and resources

AMED funded programs and resources

Did you acquire different sources of private funding?

□Yes

□No

Private funding

Did you make a Target Patient Value Profile?

□Yes

□No

Target Patient Value Profile