Table 2.
Document analysis
| Agalsidase alfa (Replagal®) and agalsidase beta (Fabrazym®) | Alglucosidase alfa (Myozyme®) | Infliximab (Remicade®) | Omalizumab (Xolair®) | Ranibizumab (Lucentis®) | |
|---|---|---|---|---|---|
| Timings of initial and re-assessment dossier | May 2007–Jun 2011 | Jul 2006–Feb 2011 | Oct 2006–Feb 2011 | May 2006–Mar 2011 | Apr 2007–Jun 2011 |
| Type of economic analysis | Cost effectiveness | Cost utility | Cost effectiveness | Cost effectiveness | Cost effectiveness |
| Comparator | BSC (current care/natural history of the disease without enzyme-replacement therapy) | Usual supportive care | Cyclosporine; colectomy with IPAA | Usual medical care | BSC and photodynamic therapy |
| Population | All adult pts in the Dutch Fabry study cohort | Children and adults with late-onset Pompe disease | Adult pts with moderate to severe UC who responded inadequately to or are intolerant to conventional therapy or to whom such therapy is contraindicated | Adults and adolescents (>12 years) with severe persistent allergic asthma | Pts with AMD |
| ICER | €3,282,252 | Children: €232,699 Adults: €2,700,000 |
Infliximab vs. cyclosporine: €23,585 (NE quadrant); infliximab vs. colectomy with IPAA: €15,057 (NE quadrant) | €39,215 NE quadrant (in initial assessment dossier); €35,257 NE quadrant (in re-assessment dossier) | Lucentis was dominant in all cases except for the MARINA trial-based comparison with BSC (€40,397/QALY) An additional ICER was estimated (using published evidence) vs. bevacizumab: €53,453/QALY, i.e. using bevacizumab instead of lucentis would lead to savings of €3,658 and a QALY loss of 0.09 |
| Was PSA undertaken? | Yes (in re-assessment dossier) | Yes (in re-assessment dossier | Yes (in re-assessment dossier) | Yes (in initial assessment and re-assessment dossier) | Yes (in re-assessment dossier) |
| Was VOI undertaken? | No | No | No | No | No |
| Additional research performed | Single-centre observational study | National prospective observational study with historic controls and data from the IPA survey were used | Retrospective chart analyses of UC pts treated with infliximab since 2003 for which two Dutch medical centres (one university hospital and one general hospital) were included | A European multi-centre pt outcomes registry (experience) was conducted for the observational collection of data. Retrospective data on the medical history and resource use over the year prior to the start of omalizumab treatment were also collected | Prospective pt registry |
AMD age-related macular degeneration, BSC best supportive care, ICER incremental cost-effectiveness ratio, IPA International Pompe Association, IPAA ileo-pouch-anal anastomosis, NE north-east, PSA probabilistic sensitivity analysis, pt(s) patient(s), QALY quality-adjusted life-year, UC ulcerative colitis, VOI value of information