Posted by Michael Wonder on 16 Jan 2020
Asfotase alfa in hypophosphatasia in children and adolescents: survival advantage for small children
15 January 2020 - Weak evidence does not allow a specific statement to be made about the extent of the added benefit; evidence about other patient groups is missing.
Asfotase alfa lost this special status when the annual turnover exceeded 50 million Euros and its added benefit was checked with an early benefit assessment of medicinal products according to AMNOG by the IQWiG.
The dossier assessment provides a clue for a non-quantifiable additional benefit for infants who started illness before the age of 6 months. The advantages and disadvantages for other patient groups remain unclear due to the lack of data.
