Table 1

Variables collected for each new active substance extracted from public assessment reports

VariableData pointNote on definition
Compound typeNew biological/biotechnology entityA substance isolated from animal tissues or product produced by recombinant DNA or hybridoma technology and expressed in cell lines, transgenic animals or transgenic plants for therapeutic, prophylactic or in vivo diagnostic use in humans
New chemical entityAn entity produced by chemical synthesis
Therapy areaAnatomical therapeutic chemical codeAs defined by the WHO
Approval milestone datesSponsor submission dateDefined as date of receipt of dossier by the agency
Regulatory approval dateDefined as the date of marketing authorisation; for EMA this refers to European Commission decision date
IndicationIndication submitted by the sponsorA difference in indication was defined as a restriction or expansion of the treated population.
Indication approved by the agency
Facilitated regulatory pathways to facilitate availability, review and/or approval of medicines where there is an unmet medical needExpedited review resulting in shorter review timelinesDefined as EMA ‘Accelerated Assessment’ and FDA ‘Priority Review’
Conditional review resulting in early approval based on preliminary dataDefined as EMA ‘Conditional Review’ and FDA ‘Accelerated approval’
Other non-standard pathwaysFDA Breakthrough Designation; FDA Fast Track
Orphan statusOrphan designation
  • EMA, European Medicines Agency; FDA, Food and Drug Administration.