Orphan Drugs

Executive Summary

  • Orphan and ultra-orphan diseases are defined as diseases with a prevalence of ≤5 in 10,000 and ≤1 in 50,000 patients respectively
  • Most orphan diseases (~80%) are genetic rare diseases
  • 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, these tend to be reserved for ultra-orphan treatments due to the limited number of HST appraisals each year so no orphan drug has undergone an HST as of yet
  • Commissioning policies and individual funding requests (IFRs) remain a viable alternative route to market for orphan drugs under certain circumstances