From: How to START? Four pillars to optimally begin your orphan drug development
Question | Yes/ No | More information? |
---|---|---|
Stakeholder mapping | ||
Are there patient organizations for the disease? | â–ˇYes â–ˇNo | Â |
Are there community advisory boards (CABs)? | â–ˇYes â–ˇNo | 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? | â–ˇYes â–ˇNo | Conect4Children European Joint Programme on Rare Diseases |
Have you done a landscape analysis or horizon scanning? | â–ˇYes â–ˇNo | Horizon Scanning: Landscape analysis/ Stakeholder identification and engagement |
Available information on the disease | ||
Are there Natural History (NH) Studies? | â–ˇYes â–ˇNo | Natural History studies |
Are there diagnostic tools? | â–ˇYes â–ˇNo | Companion diagnostics |
Are there patient-centered outcome measures (PCOMs) | â–ˇYes â–ˇNo | Development of Patient-Centered Outcome Measures |
Are there biomarkers? | â–ˇYes â–ˇNo | Use of biomarkers in orphan drug development |
Is there a coding for the rare disease? | â–ˇYes â–ˇNo | Coding of rare diseases: Orphanet nomenclature |
Financial Resources | ||
Did you acquire different sources of public funding? | â–ˇYes â–ˇNo | 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? | â–ˇYes â–ˇNo | Private funding |
Target Patient Value Profile | ||
Did you make a Target Patient Value Profile? | â–ˇYes â–ˇNo | Target Patient Value Profile |