Orphan Drugs

Executive Summary

  • Orphan and ultra-orphan diseases are defined as those with a prevalence of ≤5 in 10,000 and ≤1 in 50,000 patients respectively
  • Most orphan diseases (~80%) have genetic causes
  • There is no official orphan-specific HTA route conducted by NICE, and orphan drugs may be assessed under a Single Technology Appraisal (STA)
    • It is possible for orphan treatments to undergo Highly Specialised Technology (HST) appraisals, however these tend to be reserved for ultra-orphan treatments due to limited resources for HST appraisals each year; no orphan drug has yet undergone HST
  • Commissioning policies and Individual Funding Requests (IFRs) are an option for orphan drugs under certain circumstances