Notes and data points for prices of orphan products

February 22, 2010., Matthew Herper, “The World’s Most Expensive Drugs,” Forbes.

Genzyme’s web page on the cost of treatment.

Some data points on Fabrazyme prices.

2004 prices for Replagal

2001 prices for Cerezyme.


A 2007 Canadian analysis of (50 mg/vial) prices for Myozyme.

Introductory Period (January to June 2007)
Country and Median Price (in Canadian dollars)
Canada $840.3100 per vial
France Not sold
Germany $840.3099 per vial
Italy $840.3099 per vial
Sweden $856.2828 per vial
Switzerland Not sold
United Kingdom $823.5171 per vial
United States $734.9248 per vial
Median $840.3099 per vial

Sources: Canada, Germany, Italy, Sweden, United Kingdom and United States: Publicly available price as per Regulations.

From Scotland:
February 9 2007. Drug Dose Cost per year (£) Alglucosidase alfa (Myozyme®)

20mg/kg by intravenous infusion every 2 weeks
10kg child: 38,333 (£)
60kg adult: 230,000 (£)
The manufacturer estimated the gross drug budget impact of alglucosidase alfa in infants at £167k in year 1 and £210k in year 5. The respective cost for late onset patients was £1.04M in year 1 and £2.6M in year 5. The model assumes 2 infants treated in year 1 and four in year 5 and for late onset disease 4 and 10 patients respectively. All eligible patients were assumed to receive treatment.

The results for the two infant groups were £244,450 and £318,283 per QALY respectively compared to current treatment. The cost per QALY for the late-onset group was £819,806. Sensitivity analysis indicated that there was variation in these estimates but only when the model horizon was set at the length of the clinical trial did the ICER fall significantly and given that the treatment is ongoing, this would not be an appropriate point at which to truncate the analysis.

The analysis was clear, concise and well conducted but given the extremely high cost for health gain, the economic case has not been demonstrated

19 July 2006, Genzyme says EU countries could take two years to agree Myozyme reimbursement, APM Health Europe.

February 16, 2009, Nick Taylor, “Myozyme becomes Lumizyme after biologics scale-up,”

Search of NIH Reporter on August 4, 2010 identifies 118 grants for Pompe disease, and 18 patents with NIH rights.