Table 1.
Preliminary cost per QALY ICER estimates by NICE (2005) (2), illustrating the mismatch between utra-orphan drug cost and conventional cost-effectiveness benchmarks (as, e.g., adopted by NICE, from £20,000 to £30,000 per QALY gained)
| Condition | Prevalence (England) | Product | ICER (preliminary estimated cost in GBP per QALY) |
|---|---|---|---|
| M. Gaucher Type I and III | 270 | Imiglucerase (CeredaseR) | 391,200 |
| MPS Type 1 | 130 | Laronidase (AldurazymeR) | 334,900 |
| M. Fabry | 200 | Agalsidase beta (FabrazymeR) | 203,000 |
| Hemophilia B | 350 | Nonacog alpha (BeneFIXR) | 172,500 |
| M. Gaucher Type I | 270 | Miglustat (ZavescaR) | 116,800 |
ICER, incremental cost-effectiveness ratios; QALY, quality-adjusted life years; NICE, National Institute for Health and Care Excellence.