Table 1

Sources used for the analysis and information investigated

SourceInformation
EuOrphan (EMA, Orphanet)

  • Active substances designated as OMP

  • ODDs with an MA

  • ODDs withdrawn with an MA

  • Dates of designation

  • Rare condition(s)

  • Orphan indication(s)

  • First and current sponsors

  • MA refusals and MAA withdrawals

  • Reasons for withdrawals or refusals

  • Clinical trials and other evidence supporting the MA

  • Possible competitors, that is, other OMPs for the same indication

Clinical trial databases (EU Clinical Trials Register and Clinicaltrials.gov)

  • Published clinical trials

  • Reasons for prematurely ended clinical trials

PubMed

  • Published clinical trials and other studies in literature

  • Efficacy and safety data

Sponsor-sourced information (company websites and pipelines, direct communications with the sponsors)

  • Sponsor type (commercial or non-commercial)

  • Stage of development of the drug

  • Reasons for failures

  • EMA, European Medicines Agency; MA, marketing authorisation; MAA, MA application; ODD, orphan drug designation; OMP, orphan medicinal product.