Orphan and Ultra-Orphan Products

Executive Summary

  • Orphan and ultra-orphan diseases are defined by the European Medicines Agency (EMA) as those with a prevalence of ≤5 in 10,000 and ≤1 in 50,000 patients respectively
  • Most orphan diseases (~80%) have a genetic cause
  • There is no official orphan-specific Health Technology Assessment (HTA) route conducted by the National Institute for Health and Care Excellence (NICE)
  • Orphan products may be assessed under a Single Technology Appraisal (STA), but are poorly suited due to their limited clinical trial evidence and patient population diversity
  • Commissioning policies and Individual Funding Requests (IFRs) are an option for orphan products as defined by NICE
  • Achieving market access for orphan and ultra-orphan disease products is likely to take in excess of two years